Biomedtracker Pharma intelligence | HEALTHCARE CONFERENCE: CONFERENCE REVIEW Healthcare Conference:JP Morgan Healthcare Conference Conference: Review Review

Biomedtracker August 2016 / 2 Pharma intelligence |

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SUMMARY The 34th annual JP Morgan Healthcare Conference was held in San Francisco, CA from January 11-14, 2016. A comprehensive list of events and catalysts that were announced or updated at the conference is included in this report. Additionally, below are some key points from the conference’s company presentations.

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KEY POINTS – CONFERENCE REVIEW

 Medivation (MDVN) disclosed a new SREBP inhibitor, MDV4463, which has reached clinical development. The SREBP inhibitor is still being evaluated for safety but could eventually be developed for NASH, hyperlipidemia, diabetes, obesity, and metabolic syndrome. MDVN also suggested that its PD-1 inhibitor pidilizumab (which was acquired from Curetech) is likely not primarily a PD-1 inhibitor. Its MOA for efficacy is in fact not fully understood by the company, although the company is fully pursuing DLBCL as its fastest path towards regulatory approval. Finally, look forward to continued evaluation of Medivation’s lead drug, Xtandi (enzalutamide), as it expands its potential therapeutic use including data from PLATO evaluating treatment through rising PSA levels in 2H2016.

 Biomarin (BMRN) reported numerically positive, albeit preliminary, data from an uncontrolled Phase II trial of reveglucosidase alfa in late-onset Pompe's disease. The company needs to meet with regulators to discuss what will be needed for Phase III/registration.

 Ionis (IONS) presented a new analysis of data from its trial of nusinersen in infants with SMA, showing markedly improved ventilation-free survival compared to a matched natural history cohort. Officials noted the number of patients over 2 years of age with nusinersen, with a couple at least 3 years old, was unprecedented. Officials also pointed to improvements in children with SMA, with 3 non-ambulatory patients who were able to walk with treatment. Phase III trials are underway, with results expected early 2017, but officials noted they are in discussions with regulators on bringing the drug to market as quickly as possible for infants.

 Astellas (4503:JP) presented new data from its Japanese lung cancer study of ASP8273, its entrant into the next generation of EGFR inhibitors. ASP8273 trails Tagrisso (AZN), which has already been FDA approved, and rociletinib (CLVS), for which an NDA has already been filed.

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 For their cardiovascular franchise, Amgen (AMGN) reiterated important catalysts for Repatha in the second half of 2016, which should demonstrate that its LDL-c lowering does improve atherosclerosis and CV events: top-line data from both the cardiovascular outcomes study and the IVUS coronary imaging study. Officials view the latter as a complementary differentiating factor. Competitors REGN/SNY, in turn, are expecting interim analyses in Praluent's cardiovascular outcomes study in 2016, with a futility analysis at 50% of events and, in H2, a futility/overwhelming efficacy analysis at 75% of events. If those are negative, the final results are expected in H2 2017. o AMGN's presentation had Rx data showing Repatha and Praluent are currently running neck and neck, but with only about 400 total Rxs per week each. AMGN officials hope payor utilization management criteria will change with positive results from the outcomes study. o AMGN officials dismissed reports of neurotoxicity with Repatha, saying the recent source was just an anonymous post on Café Pharma. They acknowledged theoretical concerns with the novel mechanism, but pointed out factors that could mitigate cholesterol being too low, as well as the fact that Repatha does not cross the blood-brain barrier. o In heart failure, AMGN officials were excited about the prospects for omecamtiv, but still need to go through several hurdles for a decision on Phase III.

 Amicus (FOLD) had new, patient-level data on migalastat's impact on diarrhea in Fabry disease, showing more clearly the larger separation at higher levels of baseline symptoms, though the number of placebo patients in the higher baseline group was small. The company expects to submit answers to the day-150 EMA questions shortly, with a CHMP opinion continued to be expected early 2016. They still need to meet with the FDA later this quarter to determine on a regulatory pathway (due to mixed data). For their epidermolysis bullosa drug, Zorblisa, acquired with Scioderm, the company noted the Phase III was powered for a difference on target wound healing of ~17%. They just barely saw that degree of difference in the small Phase IIb in the ITT population (19%), but there was a substantially larger difference in the evaluable population (41%). They are also selecting patients with larger and more chronic wounds to improve the chances for a positive study. The Phase III trial is approximately half enrolled, and officials extended the estimate for top-line data to H2 2016.

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 Enanta (ENTA) announced nomination of FXR agonist EDP-305 as their developmental candidate for NASH (nonalcoholic steatohepatitis) and PBC (primary biliary cirrhosis). The Company is currently on track to initiate clinical trials in 2016. In preclinical data presented, EDP-305 was more potent than obeticholic acid (OCA, the active ingredient of INT-747) on FXR activation, and in the STAM mouse model, there were suggestions it may have an advantage on hepatocyte ballooning and the NAFLD activity score at a similar dose.

 Esperion (ESPR) reiterated the FDA has never told them they will need a CV outcomes trial completed before approval of ETC-1002—just that it must be well underway-- but said they will only be able to disclose Phase III plans in Q2 2016, after discussions with the agency (they are submitting the design of the CVOT this quarter). Their focus is on statin intolerant patients, for which no drugs have previously been formally approved.

 Clovis (CLVS) gave reassurance that their next-generation EGFR inhibitor, rociletinib, would both gain approval this year and be able to compete with Tagrisso (AZN), which beat it to market late last year. Officials did suggest that an ODAC is a distinct possibility, perhaps in April, but were not sure what questions the FDA would have given the rather straight-forward nature of the single-arm study. They also suggested that both Tagrisso and rociletinib would be viewed relatively comparable on efficacy in the relevant US population, and competition with thus likely come down to the different safety profiles (namely rash vs hyperglycemia).

 Kite (KITE) presented new data from its NCI study of KTE-C19 showing consistency of PR/CRs in the total population, aggressive NHLs, and with its new cell manufacturing process. Notably, the durability of response has remained impressive for this refractory population. Officials also discussed in depth the neurotoxicity associated with CAR-T therapies, and overall, they appear of only modest concern since they typically self-resolve in a few days. Next for Kite are interim data from the pivotal ZUMA study and the initiation of clinical development for its T-cell receptor therapies in 2016.

 Officials from beleaguered Aegerion (AEGR) acknowledged that the launch of the less expensive PCSK9 inhibitors has reduced the number of patients on Juxtapid and it was still difficult to tell what the ultimate impact would be (they estimated PCSK9 inhibitors accounted for half of a roughly 20% drop over 2015). They are trying to understand the cycle time for patients who may not be able to meet goals on PCSK9 inhibitors and resume Juxtapid. They also plan to start a Juxtapid-PCSK9 inhibitor combination study in Q2 and review the size of their customer facing organization.

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CONTENTS Acorda Therapeutics, Inc. (ACOR) ...... 9 Actelion Pharmaceuticals Ltd. (ALIOF) ...... 9 Aegerion Pharmaceuticals, Inc. (AEGR) ...... 11 Aimmune Therapeutics, Inc. (AIMT) ...... 12 Alexion Pharmaceuticals, Inc. (ALXN) ...... 13 Alkermes plc (ALKS) ...... 13 Amicus Therapeutics, Inc. (FOLD) ...... 14 Array BioPharma, Inc. (ARRY) ...... 16 Astellas Pharma, Inc...... 18 Bayer AG (BAYRY) ...... 19 BioCryst Pharmaceuticals, Inc. (BCRX) ...... 20 BioMarin Pharmaceutical Inc. (BMRN) ...... 21 Chimerix, Inc. (CMRX) ...... 26 China Biologic Products, Inc. (CBPO) ...... 28 Clovis Oncology, Inc. (CLVS) ...... 28 Coherus BioSciences, Inc. (CHRS) ...... 36 Dynavax Technologies Corporation (DVAX) ...... 39 Eagle Pharmaceuticals, Inc. (EGRX)...... 41 Eli Lilly & Company (LLY) ...... 42 Emergent BioSolutions, Inc. (EBS) ...... 43 Enanta Pharmaceuticals, Inc. (ENTA) ...... 44 Endo International plc (ENDP) ...... 46 Endologix, Inc. (ELGX) ...... 46 Envisia Therapeutics ...... 47 Esperion Therapeutics, Inc. (ESPR) ...... 49 Genmab ...... 51 Gilead Sciences, Inc. (GILD) ...... 52 GlaxoSmithKline plc (GSK) ...... 54 Glenmark Pharmaceuticals Limited ...... 55

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Global Blood Therapeutics Inc. (GBT) ...... 56 Halozyme Therapeutics, Inc. (HALO) ...... 57 Hanmi Pharmaceutical Co., Ltd...... 61 HeartWare International, Inc. (HTWR) ...... 63 Ignyta, Inc. (RXDX) ...... 65 ImmunoGen, Inc. (IMGN) ...... 66 Incyte Corporation (INCY) ...... 67 Indivior plc ...... 72 Intrexon Corporation (XON) ...... 73 Ionis Pharmaceuticals, Inc. (IONS) ...... 75 Ironwood Pharmaceuticals, Inc. (IRWD) ...... 76 Jazz Pharmaceuticals plc (JAZZ) ...... 77 Luminex Corporation (LMNX) ...... 78 MannKind Corporation (MNKD) ...... 78 Medgenics Inc. (MDGN) ...... 79 Medivation, Inc. (MDVN) ...... 79 Merck & Co., Inc. (MRK) ...... 80 Merrimack Pharmaceuticals, Inc. (MACK) ...... 81 Mesoblast Limited (MESO) ...... 82 Moderna Therapeutics, Inc...... 82 Momenta Pharmaceuticals (MNTA) ...... 86 NeuroDerm, Ltd. (NDRM) ...... 88 OncoMed Pharmaceuticals, Inc. (OMED) ...... 88 Ophthotech Corp. (OPHT)...... 89 Opko Health (OPK) ...... 90 OptiNose AS ...... 92 ProNAi Therapeutics, Inc. (DNAI) ...... 92 Radius Health, Inc. (RDUS) ...... 93 Regeneron Pharmaceuticals, Inc. (REGN) ...... 94 REVA Medical Inc...... 95 Rigel Pharmaceuticals, Inc. (RIGL) ...... 96

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Roche Holding AG (RHHBF) ...... 96 SAGE Therapeutics (SAGE) ...... 97 Sangamo Biosciences, Inc. (SGMO) ...... 98 Seattle Genetics, Inc. (SGEN) ...... 99 Spark Therapeutics, LLC (ONCE) ...... 101 St. Jude Medical, Inc. (STJ) ...... 103 T2 Biosystems, Inc. (TTOO) ...... 103 Taiwan Liposome Company, Ltd...... 104 TG Therapeutics, Inc. (TGTX) ...... 107 True North Therapeutics, Inc...... 108 United Therapeutics Corporation (UTHR) ...... 109 Veracyte, Inc. (VCYT) ...... 110 List of New BioMedTracker Catalysts…………………………………………………………………………………..112 List of Updated BioMedTracker Catalyts………………………………………………..…………………………...124 List of MedDeviceTracker Catalysts……………………………………………………………………………………..144

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ACORDA THERAPEUTICS, INC. (ACOR) Ampyra for Ischemic Stroke

Event Date: 01/11/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase I - QD Formulation Market Group: Neurology

Lead Company: Acorda Therapeutics, Inc. (ACOR) Alkermes (ALKS) Biogen (BIIB) Partner Companies: UCB (UCB:BB) DRI Capital Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 55% (3% Above Avg.) Average Approval: 52%

Analysis: Acorda announced that three prototypes of its QD formulation of dalfampridine is in Phase I PK studies. Data from this study is expected in the first quarter of 2016. Source: J.P. Morgan Healthcare Conference 01/11/2016 (ACOR, Slide 20)

ACTELION PHARMACEUTICALS LTD. (ALIOF) Opsumit for Pulmonary Arterial Hypertension (PAH)

Event Date: 12/31/2015 Event Type: Trial Data - Top-Line Results (Clinical Analysis)

Trial Name: Phase II - MELODY-1 Market Group: Cardiovascular

Lead Company: Actelion Pharmaceuticals Ltd. (ALIOF)

Partner Companies: Nippon Shinyaku (4516:JP) Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Actelion announced that at the end of 2015, the initial results of the Phase II MELODY

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study with macitentan became available.

Context The company will now fully evaluate the data and, together with the results of the current Phase II study with a new endothelin receptor antagonist (ERA), make a decision on the future development strategy of Actelion's ERAs beyond PAH.

Design MELODY was a prospective, multicenter, double-blind, randomized, placebo- controlled, parallel-group, 12-week study to evaluate the safety and tolerability of macitentan in subjects with combined pre- and post-capillary pulmonary hypertension (CpcPH) due to left ventricular dysfunction.

Results In the exploratory Phase II study, macitentan was generally well tolerated in this Group 2 pulmonary hypertension patient population with heart failure. In addition, encouraging hemodynamic effects were observed. Source: J.P. Morgan Healthcare Conference 01/11/2016 (ALIOF, Press Release)

Opsumit for Brain Cancer (malignant glioma; AA and GBM)

Event Date: 01/11/2016 Event Type: Progress Update - Suspension (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Actelion Pharmaceuticals Ltd. (ALIOF)

Partner Companies: Nippon Shinyaku (4516:JP) Phase: Suspended Change to Likelihood of Approval: -6% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Actelion announced that despite encouraging preclinical data, results from the Phase I/Ib open-label studies in patients with recurring glioblastoma and newly diagnosed glioblastoma disappointingly do not support further investigation of Opsumit in this indication. Dose escalation had reached 300 mg per day, without any significant tolerability issues, before the decision was made to stop the investigation. Source: J.P. Morgan Healthcare Conference 01/11/2016 (ALIOF, Press Release)

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AEGERION PHARMACEUTICALS, INC. (AEGR) Juxtapid for Dyslipidemia / Hypercholesterolemia

Event Date: 01/08/2016 Event Type: Regulatory - J-NDA Filing (Japan) (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Aegerion Pharmaceuticals, Inc. (AEGR) Bristol-Myers Squibb (BMY) Partner Companies: UPenn Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Aegerion reported that a Japanese filing for marketing approval of Juxtapid for the treatment of HoFH was submitted on January 8, 2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (AEGR, Slide 10)

Juxtapid for Dyslipidemia / Hypercholesterolemia

Event Date: 01/14/2016 Regulatory - Approval (Emerging Markets) (Clinical Event Type: Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Aegerion Pharmaceuticals, Inc. (AEGR) Bristol-Myers Squibb (BMY) Partner Companies: UPenn Phase: Approved Change to Likelihood of 0% Approval: Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Aegerion reported that Juxtapid has been approved for the treatment of HoFH in Korea.

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Source: J.P. Morgan Healthcare Conference 01/14/2016 (AEGR, Slide 14) AIMMUNE THERAPEUTICS, INC. (AIMT) AR101 for Food Allergies

Event Date: 01/11/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase III - PALISADE Market Group: Allergy

Lead Company: Aimmune Therapeutics, Inc. (AIMT) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 38% Likelihood of Approval: 63% (1% Above Avg.) Average Approval: 62%

Analysis: Aimmune Therapeutics announced that it has enrolled the first patient in the pivotal Phase III PALISADE trial of its lead product candidate, AR101 for the treatment of peanut allergy.

Phase III PALISADE (PEANUT ALLERGY ORAL IMMUNOTHERAPY STUDY OF AR101 FOR DESENSITIZATION IN CHILDREN AND ADULTS) Study This Phase III is an international, randomized 3:1, double-blind, placebo-controlled, Phase III trial of the efficacy and safety of AR101 in a characterized desensitization (CODIT) approach in patients with peanut allergy. Aimmune expects PALISADE to enroll approximately 500 peanut-allergic patients 4-55 years of age at more than 60 clinical sites in the United States, Canada, and nine countries in the European Union. Aimmune expects to complete enrollment in PALISADE in the second half of 2016 and complete the trial in the second half of 2017.

PALISADE will evaluate the safety and efficacy of AR101 for desensitizing peanut- allergic patients to a level sufficient to reliably protect them from allergic reactions upon accidental exposures to peanut. Under the PALISADE protocol, patients will undergo a dose escalation period of approximately 22 weeks to reach the maintenance dose of 300 mg per day, then continue with daily maintenance at 300 mg per day for approximately six months. At the end of the maintenance period, patients will undergo an exit double-blind, placebo-controlled food challenge (DBPCFC).

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PALISADE’s primary endpoint is toleration of a cumulative amount of 1,043 mg of peanut protein in the exit DBPCFC. PALISADE’s inclusion criteria allow for baseline toleration of a cumulative amount not exceeding 44 mg of peanut protein in an entry DBPCFC. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/14/2016 (AIMT) ALEXION PHARMACEUTICALS, INC. (ALXN) Kanuma for Lysosomal Acid Lipase Deficiency

Event Date: 01/12/2016 Event Type: Progress Update - Product Launch (U.S.) (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: Alexion Pharmaceuticals, Inc. (ALXN) Partner Companies: N/A Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Alexion announced that Kanuma has been launched in the U.S. Source: J.P. Morgan Healthcare Conference 01/12/2016 (ALXN, Slide 20)

ALKERMES PLC (ALKS) ALKS 3831 for Schizophrenia

Event Date: 01/12/2016 Event Type: Regulatory - Meeting with FDA (Clinical Analysis) Trial Name: N/A Market Group: Psychiatry

Lead Company: Alkermes plc (ALKS) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0%

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Likelihood of Approval: 17% (5% Above Avg.) Average Approval: 12%

Analysis: Alkermes announced that they completed an End-of-Phase II meeting with the FDA to discuss ALKS 3831 for the treatment of schizophrenia. Source: J.P. Morgan Healthcare Conference 01/12/2016 (ALKS, Slide 36)

AMICUS THERAPEUTICS, INC. (FOLD)

Zorblisa for Epidermolysis Bullosa

Event Date: 12/31/2015 Event Type: Regulatory - Rolling NDA/BLA Initiated (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: Amicus Therapeutics, Inc. (FOLD) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 62% (Same As Avg.) Average Approval: 62%

Analysis: Amicus reported that the Company's rolling NDA for Zorblisa for the treatment of epidermolysis bullosa was initiated in the fourth quarter of 2015. Source: J.P. Morgan Healthcare Conference 01/12/2016 (FOLD, Slide 13)

Zorblisa for Epidermolysis Bullosa

Event Date: 01/10/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase IIb - SD-003 Market Group: Metabolic

Lead Company: Amicus Therapeutics, Inc. (FOLD) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 2%

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Likelihood of Approval: 64% (2% Above Avg.) Average Approval: 62% Placebo Treatment Treatment Placebo Treatment Treatment Treatment Description Placebo SD-101 3% SD-101 6% Placebo SD-101 3% SD-101 6% Evaluable Evaluable Evaluable ITT ITT ITT Patients Patients Patients Patients Patients Patients Number of Evaluable Patients 17 16 12 N/A N/A N/A Target Wound Healing at 41.000 % 38.000 % 67.000 % 41.00 % 38.000 % 53.000 % Month 1 (Endpoint=Primary) Target Wound Healing at 41.000 % 44.000 % 82.000 % 41.00 % 44.000 % 60.000 % Month 2 (P= 0.0400) Median Time to Wound 91.000 86.000 30.000 N/A N/A N/A Closure (Endpoint=Secondary) Days Days Days Analysis: Amicus presented results for the Phase IIb study of SD-101 for epidermolysis bullosa (EB).

Previous data from this study were last seen in September 2014.

Context  42/44 patients have entered the optional extension study (SD-004)  SD-101 6% concentration selected for Phase III study based on Phase IIb dose response  Wound closure at Month 2 (versus Month 1) is optimal time to measure primary endpoint. Endpoint is accepted by FDA and EU regulators  Defined approval pathway with Phase III (Study 005) study design based on EMA and FDA feedback  Top-line data for Study 005 expected 2H 2016  Rolling NDA initiated 4Q15

Design 48 EB patients (age ≥ 6 months)* - 1:1:1 Randomization - Daily Topical Application

*Initial Disease Severity: Mean target lesion size (cm2) 14.0 (range 5-39); mean lesionalBSA: 19.4% (range 0.4-48%); mean wound age (days): 182 (range 21-1,639)

EB Subtypes enrolled: Simplex (n=11), Recessive Dystrophic (n=29), and Junctional (n=8)

Endpoints Primary Efficacy Endpoint - Target Wound Healing at Month 1 - Baseline wound: Chronic (≥ 21 days),size 5-50 cm2

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Results  Placebo response minimized by analyzing subgroup of patients with wounds ≥ 10 cm2 o Complete target wound closure SD-101 6% - 50% (n= 4) vs. Placebo - 12.5% (n=8) at Month 2  Phase IIb results used to calculate appropriate sample size in Phase III study o p ≤ 0.05 if treatment difference ~17% or greater  Wound closure at Month 2 (versus Month 1) is optimal time to measure primary endpoint Most Common Adverse Events Treatment-emergent adverse events (TEAE) generally similar across treatment groups. No deaths and no severe TEAEs. No serious adverse events reported in SD- 101 6% group.

Comment The ITT analysis for the high dose only showed a modest improvement in patients with complete target wound closure at 2 months (19% difference), but the evaluable patient analysis had a much larger difference (41%). The company noted at the JPM conference that the Phase III study is powered for a 17% difference, and also is including baseline entry criteria that will hopefully improve the chances for success (more chronic and larger wounds).

We should note, though, that the Phase IIb study was relatively small (only 45 patients) and there was no evidence of a dose response. Time to wound closure was more consistent between the ITT and evaluable populations, but again, there was no dose response.

As a result, we are limiting our increase in likelihood of approval to 2%. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/12/2016 (FOLD) Dermatology Summit 01/10/2016 (Company Presentation)

ARRAY BIOPHARMA, INC. (ARRY) Binimetinib for Melanoma

Event Date: 01/13/2016 Event Type: Trial Announcement (Clinical Analysis)

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Trial Name: Phase II - SECOMBIT Market Group: Oncology

Lead Company: Array BioPharma, Inc. (ARRY)

Partner Companies: Pierre Fabre Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 39% (4% Above Avg.) Average Approval: 35%

Analysis: Array BioPharma announced the SECOMBIT trial, evaluating the sequential use of MEK+RAF and PD1+CTLA4 in BRAF+ melanoma, being conducted as a multicenter international cooperative group study.

The trial will randomize ~230 patients to receive encorafenib/binimetinib or nivolumab/ipilimumab combination until progression followed by treatment with the alternate combination regimen. A third arm will evaluate induction encorafenib/binimetinib treatment (2-cycles) followed by nivolumab/ipilimumab, and retreatment with encorafenib/binimetinib on progression. Source: J.P. Morgan Healthcare Conference 01/13/2016 (ARRY, Slide 12)

ARRY-797 for Congestive Heart Failure (CHF)

Event Date: 01/13/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase II - LMNA-Related Dilated Cardiomyopathy Market Group: Cardiovascular

Lead Company: Array BioPharma, Inc. (ARRY) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 13% (Same As Avg.) Average Approval: 13%

Analysis: Array announced that the Phase II trial of ARRY-797 for LMNA-related dilated cardiomyopathy is ongoing. Data is expected to be presented in the near future.

Array also stated that compared with historical benchmarks in the 6 minute walk test, the study's primary endpoint, the data emerging is "encouraging." ARRY-797

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secondary endpoint measures also suggest activity (NT-proBNP, patient reported outcomes). Source: J.P. Morgan Healthcare Conference 01/13/2016 (ARRY, Slide 30-31, 33)

ASTELLAS PHARMA, INC. Gilteritinib for Acute Myelogenous Leukemia (AML)

Event Date: 01/12/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase I/II - CHRYSALIS Market Group: Oncology

Lead Company: Astellas Pharma, Inc. (4503:JP) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 37% (2% Above Avg.) Average Approval: 35%

Analysis: Astellas reported new data for Gilteritinib (ASP2215) from the Phase I/II trial in FLT3+ AML patients at the 34th annual JP Morgan Healthcare conference.

Results from this study were last seen in December 2015.

Design Patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) were studied in an open-label safety, tolerability, and PK trial. Response rate, overall survival, event free survival, and leukemia free survival were also evaluated.

Results Across all FLT3+ subjects treated with ASP2215 great or equal to 80mg, the median duration of response was 111 days (8-383 range). Duration of response was calculated from the first observed response of PR or better.

Median time to best response was 32 days (26-364 range). Time to best response was only evaluated for subjects who achieved best response of PR or better. Median overall survival was 218 days (12-430 range). Source: J.P. Morgan Healthcare Conference 01/12/2016 (Astellas, Slide 8)

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ASP8273 for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/12/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase I/II - EGFRmut (Japan) Market Group: Oncology

Lead Company: Astellas Pharma, Inc. (4503:JP) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10% Treatment Treatment Description ASP8273 300 mg Number of Evaluable Patients 70 Overall Response Rate (ORR) 64.000 % Analysis: Astellas reported new data for ASP8273 from the Phase I/II trial in T790M+ NSCLC patients in Japan at the 34th annual JP Morgan Healthcare conference.

Results from this study were last seen in February 2015.

Design Patients with non-small cell lung cancer (NSCLC) with EGFR activating mutations previously treated with EGFR-TKIs were studied in an open-label, dose escalation safety, tolerability, MTD, and RP2D trial.

Results Subjects with evaluable data (n=70) treated with 300mg ASP8273 showed at least stable disease in target lesions. Preliminary ORR was 64% (n=45/70), including both confirmed and unconfirmed responses. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Astellas, Slide 9) BAYER AG (BAYRY) Finerenone for Congestive Heart Failure (CHF)

Event Date: 01/13/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

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Biomedtracker August 2016 / 20 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Trial Name: Phase III - FINESSE-HF Market Group: Cardiovascular

Lead Company: Bayer AG (BAYRY) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 34% Likelihood of Approval: 49% (2% Above Avg.) Average Approval: 47%

Analysis: Bayer announced that Phase III trials of finerenone for diabetic kidney disease and chronic heart failure have been initiated. Source: J.P. Morgan Healthcare Conference 01/13/2016 (BAYRY, Slide 21) BIOCRYST PHARMACEUTICALS, INC. (BCRX) BCX7353 for Hereditary Angioedema (HAE)

Event Date: 01/13/2016 Event Type: Trial Announcement (Clinical Analysis)

Trial Name: Phase II - APeX-1 Market Group: Autoimmune/ immunology

Lead Company: BioCryst Pharmaceuticals, Inc. (BCRX) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 14% (Same As Avg.) Average Approval: 14%

Analysis: BioCryst announced the study design for a Phase II proof of concept study of BCX7353 for the treatment of hereditary angioedema (HAE). The study is planned to start in the first quarter of 2016 following regulatory review and IRB approvals, with results expected in mid-2016.

Phase II APeX-1 Study APeX-1 is a Phase II randomized, placebo-controlled, blinded, oral, once daily, 4-week cohort study of BCX7353 in HAE patients. The study is dose-ranging, with three doses of BCX7353:  Lowest dose not less than 100 mg  Highest dose not greater than 350 mg

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The primary endpoint of the study is mean acute angioedema attack rate. Source: J.P. Morgan Healthcare Conference 01/13/2016 (BCRX, Slides 19 and 23)

Avoralstat for Hereditary Angioedema (HAE)

Event Date: 01/13/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase III - OPuS-4 (Long-Term Safety) Market Group: Autoimmune/ immunology

Lead Company: BioCryst Pharmaceuticals, Inc. (BCRX) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 66% (7% Above Avg.) Average Approval: 59%

Analysis: BioCryst announced the initiation of a long-term safety study of avoralstat for the treatment of hereditary angioedema (HAE) entitled OPuS-4. Results from the study are expected in 2017.

Phase III OPuS-4 Study OPuS-4 is a long-term safety study of avoralstat. The goals of the study are to evaluate 6 to 12 month dosing with avoralstat and satisfy filing requirements. Source: J.P. Morgan Healthcare Conference 01/13/2016 (BCRX, Slide 13)

BIOMARIN PHARMACEUTICAL INC. (BMRN) Kuvan for Phenylketonuria (PKU)

Event Date: 01/11/2016 Event Type: Patent - Extension (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: BioMarin Pharmaceutical Inc. (BMRN) Merck KGaA (MKGAY) Partner Companies: Daiichi Sankyo (DSKYF) Phase: Approved

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Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: BioMarin announced that the EU patent protection for Kuvan has been extended 4 additional years through 2024. Source: J.P. Morgan Healthcare Conference 01/11/2016 (BMRN, Slide 30)

BMN-701 for Pompe Disease

Event Date: 01/11/2016 Event Type: Trial Data - Top-Line Results (Clinical Analysis)

Trial Name: Phase II - INSPIRE (Switchover Study) Market Group: Metabolic

Lead Company: BioMarin Pharmaceutical Inc. (BMRN) Partner Companies: N/A Phase: II Change to Likelihood of Approval: -37% Likelihood of Approval: 29% (5% Above Avg.) Average Approval: 24% Treatment Treatment Treatment Treatment Description Reveglucosidase Alfa Reveglucosidase Alfa Reveglucosidase Alfa Baseline Week 24 Baseline to Week 24 Mean MIP 50.000 % 52.100 % 2.200 % (Endpoint=Primary) Mean MEP 38.900 % 42.000 % 3.100 % (Endpoint=Secondary) Mean 6MWT 345.800 m 371.900 m 26.100 m (Endpoint=Secondary)

Mean FVCupright 60.700 % 56.900 % -3.700 % (Endpoint=Secondary) Analysis: BioMarin announced interim results from INSPIRE, a Phase II trial for reveglucosidase alfa being studied for the treatment of late-onset Pompe disease (LOPD).

Context BioMarin will present these data at an upcoming medical meeting.

Design The INSPIRE clinical trial is a Phase II single-arm, open-label, switchover study of

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reveglucosidase alfa in patients with late-onset Pompe disease (LOPD) who have been receiving treatment with recombinant human acid alpha glucosidase (rhGAA) for 48 weeks or longer. This study was changed from a Phase II/III to a Phase II study to allow use of drug employing a new purification process, which could be used in an anticipated Phase III registration-enabling trial. All patients in the study have been transferred to the new material, and all future patients will be treated with the new material.

Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive reveglucosidase alfa 20 mg/kg by IV infusion every other week. The change in value in primary endpoint, Maximum Inspiratory Pressure (MIP), and secondary endpoint, Maximum Expiratory Pressure (MEP), Forced Vital Capacity (FVC) Upright and Six-Minute Walk Test (6MWT) will be measured as the difference between the Baseline value and the Week 24 value within each individual subject. The study has a 24-week treatment period followed by an extension period of up to 240 weeks.

The interim efficacy and safety analysis is based on 24 patients who previously had been on treatment with the enzyme replacement therapy, alglucosidase alfa, and were switched to reveglucosidase alfa. Baseline characteristics can be seen in the link below.

Endpoints The primary endpoint of the study is the change in value (from baseline to Week 24) of Maximum Inspiratory Pressure (MIP). Secondary endpoints include change in value (from baseline to Week 24) of Maximum Expiratory Pressure (MEP), Forced Vital Capacity (FVC) Upright and Six-Minute Walk Test (6MWT).

Results Investigators indicated that, while on treatment with alglucosidase alfa, the majority of the patient population were considered to have worsening of their Pompe disease over the last 12 months. At week 24, the 18 patients on treatment with reveglucosidase alfa and who completed the study demonstrated respiratory muscle improvements with a mean increase of 2.2 points from baseline in percent predicted Maximal Inspiratory Pressure (MIP) and a mean increase of 3.1 points from baseline in percent predicted Maximal Expiratory Pressure (MEP). Patients completing the study also experienced a mean improvement of 26.1 meters in 6 Minute Walk Test (6MWT).

In the 14 patients who met eligibility at both screening and baseline and completed

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the study, a mean increase of 3.8 points from baseline in percent predicted MIP also was observed. The 18 patients completing the study showed a mean decrease of 3.7 points from baseline in percent predicted Forced Vital Capacity (FVC), but were considered relatively unchanged from screening at -0.7 points in percent predicted.

Results from patients with baseline and week 24 results can be seen in the table above.

Most Common Adverse Events Six patients discontinued treatment early, and two patients experienced serious adverse events. Hypoglycemia was generally similar in frequency and severity as in the previous study, and the overall pattern of safety was otherwise consistent with experience using other enzyme replacement therapy.

Comment Interim II data for a Phase II switch study from approved Myozyme to reveglucosidase alfa, in patients with late-onset Pompe disease, showed positive numerical improvements in several measures, though the trial was uncontrolled, so the data is tentative.

The interim analysis was done and the study changed from Phase II/III to Phase II, because of a new purification process, which is being used for subsequent treatment of these and future patients (like the anticipated Phase III study). Patients in the current study had been on Myozyme an average of 5.5 years, and about 61% of patients had overall worsening in their disease status in the past 12 months, albeit with 52.2% only having minimal worsening.

While preliminary, maximal inspiratory pressure (MIP) improved 2.2% (absolute percent predicted, 3.8% in the per protocol analysis) and maximal expiratory pressure (MEP) 3.1%. 6MWT also improved 26.1 m. The company also noted that MIP in untreated patients typically declines by 3.2% annually, though without a control in such a small trial, it is difficult to draw conclusions based on such comparisons.

On the negative side, FVC declined by 3.7%, but the presentation noted the change from screening was only -0.7%, so there could have been some chance variation (it would be useful to know the screening values for the other parameters to see how much variation they had, as well).

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Overall, while preliminary, the data are encouraging and support further testing. We are increasing our likelihood of approval another 1%. The next step is for the company to meet with regulatory agencies and see what is required for Phase III. They will give an update at their R&D day in April 2016. Source: Press Release 01/11/2016 (BMRN) Sagient Analysis J.P. Morgan Healthcare Conference 01/11/2016 (BMRN, Slides 15-19)

BMN 111 for Achondroplasia

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase II - 202 (Pediatric) Market Group: Endocrine

Lead Company: BioMarin Pharmaceutical Inc. (BMRN) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 25% (1% Above Avg.) Average Approval: 24%

Analysis: BioMarin announced that the fourth cohort in the Phase II study of BMN 111 for the treatment of achondroplasia is enrolled at the 30 µg/kg/QD dose. All 15 µg/kg dose subjects are currently in the 18 month extension. Source: J.P. Morgan Healthcare Conference 01/11/2016 (BMRN, Slide 35)

BMN-270 for Hemophilia A

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase I/II - PoC Market Group: Hematology

Lead Company: BioMarin Pharmaceutical Inc. (BMRN) St. Jude Partner Companies: UCL Phase: II

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Change to Likelihood of Approval: 0% Likelihood of Approval: 30% (Same As Avg.) Average Approval: 30%

Analysis: BioMarin announced that safety data support treating patients at the third dose level in the Phase I/II study of BMN 270 for the treatment of hemophilia A. Source: J.P. Morgan Healthcare Conference 01/11/2016 (BMRN, Slide 38)

Kyndrisa for Muscular Dystrophy

Event Date: 01/11/2016 Event Type: Regulatory - Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: BioMarin Pharmaceutical Inc. (BMRN) Partner Companies: N/A Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 79% (10% Below Avg.) Average Approval: 89%

Analysis: BioMarin provided updated information regarding regulatory filings of Kyndrisa for the treatment of Duchenne Muscular Dystrophy (exon 51). The Company continues to pursue FDA approval in this indication, after they were notified in December 2015 that an extension would be necessary in order to reach a decision. A decision is now anticipated in January 2016.

Regarding European filings, BioMarin anticipates filing day 120 questions to the EMA in March 2016. In addition, a CHMP opinion regarding Kyndrisa in this indication is expected in the second quarter of 2016, putting expected approval roughly in the second half of 2016. Source: J.P. Morgan Healthcare Conference 01/11/2016 (BMRN, Slide 11)

CHIMERIX, INC. (CMRX) CMX8521 for Norovirus

Event Date: 01/12/2016

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Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Chimerix, Inc. (CMRX) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Chimerix announced an update to its Norovirus development program at the JPM Conference. CMX8521 is a nucleoside with in vitro activity against mouse and human norovirus. A 7-day non-GLP toxicology/toxicokinetic study has been completed in-life with no clinical or gross post-mortem signs of toxicity. Clinical pathology and TK results are pending. There has been no off-target pharmacology observed in vitro when screened against a panel of 87 receptors, transporters and enzymes associated with adverse pharmacology. Source: J.P. Morgan Healthcare Conference 01/12/2016 (CMRX, Slide 28)

Brincidofovir (IV) for Cytomegalovirus (CMV) Infection (Antiviral)

Event Date: 01/12/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Chimerix, Inc. (CMRX) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Chimerix announced that the Company is developing an intravenous formulation of brincidofovir. A 28-day IND-enabling preclinical study is currently ongoing.

Chimerix anticipates a FTIH study for brincidofovir (IV) will begin in the second half of 2016. Source: J.P. Morgan Healthcare Conference 01/12/2016 (CMRX, Slide 14)

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CHINA BIOLOGIC PRODUCTS, INC. (CBPO) Human fibrinogen (CBPO) for Hemostasis

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Hematology

Lead Company: China Biologic Products, Inc. (CBPO) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: China Biologic Products currently lists its human fibrinogen program in Phase III development in its pipeline. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CBPO, Slide 18)

CLOVIS ONCOLOGY, INC. (CLVS) Lucitanib for Breast Cancer

Event Date: 01/13/2016 Event Type: Trial Data (Clinical Analysis)

Trial Name: Phase II - Advanced BC (FGFR1-amp; 11q-amp) (Clovis; US) Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS) Servier Partner Companies: Advenchen Laboratories SIMM Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 15% (5% Above Avg.) Average Approval: 10%

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Biomedtracker August 2016 / 29 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Analysis: Clovis announced an overview of lucitanib at the 34th annual JP Morgan Healthcare Conference.

Context There is an unclear regulatory path as monotherapy, and future development is focused on combination studies. Enrollment (n=160) completion is expected the first quarter of 2016 for the Phase II trial of lucitanib for breast cancer. Design The Phase II trial of lucitanib for advanced breast cancer, with no limit on previous therapies, randomizes patients to 10mg or 15mg. The 10mg dose was selected as the recommended dose, and this arm is still enrolling. The 15mg arm is closing.

Results Positive PFS data is emerging from the ongoing U.S. breast cancer study in patients with median of four prior therapies. There is an unclear regulatory path as monotherapy, and future development is focused on combination studies. The emerging profile suggests VEGF inhibition drives risk:benefit. There was no additional benefit in FGFR1-amp patients.

Most Common Adverse Events No new safety signals were observed. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 20-21)

Lucitanib for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/13/2016 Trial Announcement - Trial/Enrollment Suspension (Clinical Event Type: Analysis)

Trial Name: Phase II - FGFR1-amplified Squamous NSCLC Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS) Servier Partner Companies: Advenchen Laboratories SIMM Phase: II Change to Likelihood of 0% Approval: Likelihood of Approval: 15% (5% Above Avg.)

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Average Approval: 10%

Analysis: Clovis announced that they are discontinuing the Phase II lung cancer study for lucitanib as it has been a very difficult study to enroll. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 21)

Lucitanib for Ovarian Cancer

Event Date: 01/13/2016 Event Type: Trial Announcement (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS) Servier Partner Companies: Advenchen Laboratories SIMM Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Clovis announced that there is an opportunity for an intra-portfolio lucitanib/rucaparib combination in ovarian cancer. The company stated a Phase Ib study is planned to initiate the second half of 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 22)

Lucitanib for Hepatocellular (Liver) Cancer (HCC) (including secondary metastases)

Event Date: 01/13/2016 Event Type: Trial Announcement (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS) Servier Partner Companies: Advenchen Laboratories SIMM

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Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Clovis announced that there was synergy with lucitanib and mTOR inhibitors in pre- clinical models in hepatocellular cancer. The company stated a Phase Ib study is planned to initiate the second half of 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 22)

Rociletinib for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/13/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase I/II - TIGER-X (US) Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Celgene (CELG) Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 90% (8% Above Avg.) Average Approval: 82% Treatment Treatment Treatment Treatment Description Rociletinib 500mg Rociletinib 625mg Rociletinib 625mg T790M-Positive T790M-Positive T790M-Negative Number of Patients 79 170 24 Confirmed Overall Response 28.000 % 34.000 % 29.000 % Rate (ORR) (Endpoint=Primary) Analysis: Clovis Oncology gave an overview of clinical data for rociletinib for the treatment of non-small cell lung cancer (NSCLC) at the 34th annual JP Morgan Healthcare Conference.

Results from this study were last seen in September 2015.

Context The 625mg BID dose is the likely go-forward dose.

Results The T790M-positive patients had a confirmed ORR of 34% at the 625mg BID dose

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(n=170) and a confirmed ORR of 28% at the 500mg BID dose (n=79). Median duration of response was ~9 months for both doses. The T790M-negative patients had a confirmed ORR of 29% at the 625mg BID dose (n=24). Median duration of response was ~7 months. The magnitude of benefit compares with SoC chemotherapy. Most Common Adverse Events Notable AEs include hyperglycemia and QTc prolongation. QTc was readily managed and few arrhythmic events were observed. Safety profiles were similar between doses. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 5)

Rociletinib for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: Phase II/III - TIGER-1 (vs. Erlotinib) , Phase III - TIGER-3 Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Celgene (CELG) Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 90% (8% Above Avg.) Average Approval: 82%

Analysis: Clovis announced its rociletinib clinical priorities in mEGFR NSCLC. They plan to complete the TIGER-3 monotherapy confirmatory study vs. chemotherapy in both T790M-positive and -negative cohorts. In addition, the focus now will be the on best combination therapies in first and later line patients with immuno-oncology agents, including inhibitors of PD1 and PDL-1, bevacizumab, and cetuximab.

Clovis is also deprioritizing certain monotherapy programs:  Discontinuing TIGER-1 monotherapy study in treatment-naïve patients  Front line treatment paradigm evolving to combination therapies o Tarceva/Avastin combination with 13.8 month PFS (Stahel, ECC, Sept 2015)  Evaluating best front-line combination  Delaying TIGER-J2 Phase II study in Japanese patients; enrolling Asian patients in other ongoing studies Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 7)

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Rociletinib for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/13/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase Ib/II - w/Atezolizumab Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Celgene (CELG) Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 90% (8% Above Avg.) Average Approval: 82%

Analysis: Clovis announced that the Phase Ib/II study of the safety and efficacy of rociletinib (CO-1686) administered in combination with atezolizumab in patients with activating EGFR mutation-positive (EGFRm) advanced or metastatic non-small cell lung cancer (NSCLC) has begun clinical activities this week, starting with the first clinical site. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 8)

Rociletinib for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/13/2016 Event Type: Regulatory - Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Celgene (CELG) Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 90% (8% Above Avg.) Average Approval: 82%

Analysis: Clovis announced that an Oncologic Drugs Advisory Committee (ODAC) meeting remains a possibility ahead of the June 28, 2016, PDUFA date for rociletinib for the treatment of advanced, EGFR-mutant, T790M-positive non-small cell lung cancer. A potential time when the ODAC currently has no meeting agenda may be from April 11-13, 2016. Any discussion may be related to the FDA requesting additional clinical

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data for use in the efficacy analysis for both the 500mg and 625mg BID dose patient groups for rociletinib. Source: www.fda.gov (2016 Advisory Committee Tentative Meetings) J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 4)

Rucaparib for Prostate Cancer

Event Date: 01/13/2016 Event Type: Trial Announcement (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Pfizer (PFE) Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Clovis announced that they are expanding Rucaparib into prostate cancer, with a registration study to initiate in the third quarter of 2016.

Two trials total are planned to initiate in the second half of 2016:  Clovis-sponsored Phase III trial in metastatic castrate-resistant BRCA mutant (inclusive of germline and somatic) prostate cancer patients  STRAT-STAMPEDE study in newly-diagnosed castrate-sensitive de novo metastatic tBRCAmut and BRCA-like prostate cancer patients The proposed design for the CRPC registration trial includes metastatic prostate adenocarcinoma, castration resistant, Germline or Somatic BRCA mutant, progressive disease patients with prior Enzalutamide or Abiraterone treatment. They will receive Rucaparib 600 mg po BID or a control. Key study endpoints will be rPFS, OS, ORR, PSA kinetics, and patient reports outcomes. Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 10, 16-17)

Rucaparib for Ovarian Cancer

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Event Date: 01/13/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II - ARIEL2 Market Group: Oncology

Lead Company: Clovis Oncology, Inc. (CLVS)

Partner Companies: Pfizer (PFE) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 41% (6% Above Avg.) Average Approval: 35% Treatment Treatment Treatment Treatment Description Rucaparib Rucaparib Rucaparib tBRCAmut BRCAwt BRCAwt (tBRCA-like) (Biomarker Negative) Number of Patients 40 77 68 Confirmed Overall Response Rate 75.000 % 30.000 % 10.000 % (ORR) (Endpoint=Primary) Complete Response 15.000 % N/A N/A Median Duration of Response 9.500 Months 9.500 Months 5.500 Months (Endpoint=Secondary) Median PFS 12.800 Months 5.700 Months 5.300 Months (Endpoint=Secondary) Analysis: Clovis Oncology gave an update of clinical data for rucaparib for the treatment of ovarian cancer at the 34th annual JP Morgan Healthcare Conference.

Results from this study were last seen in September 2015.

Context The initial NDA for tBRCAmut patients with advanced disease plans to start by a rolling submission to initiate in the first quarter of 2016 and to complete by the second quarter of 2016.

A supplemental NDA for maintenance indication in tBRCAmut and BRCA-like populations is to follow, based on ARIEL 3, for which enrollment is expected to complete in the first quarter of 2016 with data approximately 12 months later.

Clovis plans on initiating the ARIEL4 confirmatory study in mid 2016, which includes tBRCAmut and BRCA-like patients, as well as initiating multiple combination studies in ovarian cancer during 2016.

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Results Part 1 of the ARIEL 2 study showed tumor tissue BRCAmut confirmed ORR was 75%. 6 patients achieved a CR (15%). Median duration of response was 9.5 months. Median number of prior therapies was 2. Other results are represented in the table above.

Overall confirmed response and duration of response in patients with tBRCAmut advanced ovarian cancer who received 3 or more prior lines of chemotherapy was 61% and 12.9 months, respectively. Complete response was 13% and partial response was 48%. Rucaparib data was from 23 patients from ARIEL2 part 1 and study 010; data cutoff dates are consistent with ECC ESMO presentations in September 2015. All responses are confirmed responses per RECIST 1.1 tumor response criteria.

Most Common Adverse Events Grade 3/4 treatment-related AEs observed in >15% of patients:  In ARIEL2, anemia/decreased hemoglobin (19%)  In Study 10, fatigue/asthenia (21%) and anemia (26%)  Readily managed with dose modifications Source: J.P. Morgan Healthcare Conference 01/13/2016 (CLVS, Slide 11-15) COHERUS BIOSCIENCES, INC. (CHRS) Biosimilar Ranibizumab (Coherus) for Wet Age-Related Macular Degeneration (Wet AMD) (Ophthalmology)

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Ophthalmology

Lead Company: Coherus BioSciences, Inc. (CHRS) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Coherus announced that CHS-3351 is in preclinical development as a biosimilar Lucentis with an IND to be filed in 2016.

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Source: J.P. Morgan Healthcare Conference 01/11/2016 (CHRS, Slide 28)

Biosimilar Bevacizumab (Coherus) for Cancer

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Coherus BioSciences, Inc. (CHRS) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Coherus announced that CHS-5217 is in preclinical development as a biosimilar Avastin for oncology. Source: J.P. Morgan Healthcare Conference 01/11/2016 (CHRS, Slide 28)

Biosimilar Etanercept (Coherus) for Rheumatoid Arthritis (RA)

Event Date: 01/11/2016 Event Type: Trial Data - Top-Line Results (Clinical Analysis)

Trial Name: Phase III - vs. Enbrel Market Group: Autoimmune/ immunology

Lead Company: Coherus BioSciences, Inc. (CHRS) Daiichi Sankyo (DSKYF) Partner Companies: Baxalta (BXLT) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 59% (Same As Avg.) Average Approval: 59% Difference Between Treatment and Comparator Treatment Description CHS-0214 vs. Enbrel ACR20 At 24 Weeks N/A (Endpoint=Primary) (P< 0.0500)

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Analysis: Coherus BioSciences and Baxalta announced that CHS-0214, a proposed biosimilar of Enbrel (etanercept), met its primary endpoint in a confirmatory, double-blind, randomized, controlled, two-part clinical study. Context This rheumatoid arthritis study is the second of two, large, Phase III confirmatory trials intended for inclusion in global marketing applications for CHS-0214. Results for the first Phase III study in patients with chronic plaque psoriasis were released in November 2015 and showed that this first study also met its primary endpoints. Coherus plans to advance this product to registration in various territories in 2016.

Design This ongoing study is evaluating the efficacy and safety of CHS-0214 compared to Enbrel in patients with moderate-to-severe rheumatoid arthritis that is inadequately controlled with methotrexate alone. The study continues as planned until Week 52.

Endpoints The primary efficacy endpoint was the proportion of subjects achieving ACR20 (20% improvement according to the American College of Rheumatology criteria) at Week 24.

Results The primary endpoint was within the pre-specified margins for demonstrating equivalence of CHS-0214 compared to Enbrel.

Most Common Adverse Events There were no clinically meaningful differences in the safety and immunogenicity profiles of the two products.

Comment The top-line information for this etanercept biosimilar was positive, noting that efficacy was within prespecified margins and there were no clinically meaningful differences in safety or immunogenicity, but no numerical details were given. The company gave similar top line results for the biosimilar in psoriasis late last year.

There have not yet been any biosimilars approved in the US for rheumatoid arthritis, but there are several under review, including biosimilar infliximab (Celltrion and Pfizer), another etanercept biosimilar from Sandoz, and an adalimumab biosimilar from Amgen. Those will help reveal if there are any particular issues the FDA is raising for this class of products. In Europe, the biosimilar infliximab has been approved and last year the CHMP recommended approval of a biosimilar etanercept (Benepali from

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Samsung Bioepis).

An MAA filing is expected in the second half of 2016. In the US, the company said if any patent developments occur such that the market would be available to them, they would be able to launch into that (this will likely depend on how the patent dispute between Sandoz and Amgen turns out, since Amgen had two "submarine" patents expiring in 2028 and 2029). Source: Press Release 01/11/2016 (CHRS) Press Release 01/11/2016 (BXLT) Sagient Analysis J.P. Morgan Healthcare Conference 01/11/2016 (CHRS)

DYNAVAX TECHNOLOGIES CORPORATION (DVAX) Heplisav for Hepatitis B Prevention (Vaccines, Antiviral)

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Event Date: 01/14/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase III - HBV-23 Market Group: Infectious disease

Lead Company: Dynavax Technologies Corporation (DVAX)

Partner Companies: Pfizer (PFE) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 66% (5% Above Avg.) Average Approval: 61% Comparator Treatment Comparator All Treatment Treatment Description Engerix-B Heplisav-B Engerix-B Heplisav-B HBV-23 Trial HBV-23 Trial Entire Phase III Entire Phase III Program Program Number of Patients 2781 5587 4200 10038 Patients w/Medically-Attended 46.200 % 46.000 % N/A N/A Adverse Events Patients w/Serious Adverse Events 5.300 % 6.200 % N/A N/A Deaths 0.300 % 0.400 % N/A N/A Patients w/Granulomatosis 0.000 % 0.000 % N/A N/A w/Polyangiitis Patients w/Tolosa Hunt Syndrome 0.000 % 0.000 % N/A N/A Patients w/Thrombotic Events 0.300 % 0.200 % N/A N/A Patients w/Adverse Events of Special 11 Patients 22 Patients N/A N/A Interest (AESI) Patients w/Pre-Existing AESI 10 Patients 8 Patients N/A N/A Patients w/Diagnosis Not 0 Patients 6 Patients N/A N/A Confirmed/Secondary AESI Patients w/New Onset AESI 1 Patients 8 Patients 7 Patients 18 Patients Patients w/Bell's Palsy 1 Patients 5 Patients 2 Patients 7 Patients Patients w/AESI (not Bell's Palsy) 0 Patients 3 Patients 5 Patients 11 Patients Analysis: Dynavax provided updated safety results from its Phase III HBV-23 study of Heplisav- B at the 34th Annual J.P. Morgan Conference on January 14, 2016. Data from this study were last seen in January 2016.

Endpoints The primary safety endpoint was to evaluate the overall safety of HEPLISAV-B with respect to clinically significant adverse events. Secondary endpoint was to identify frequency of granulomatosis with polyangiitis and Tolosa Hunt syndromes as well as thrombotic adverse events.

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Results For results, see table above.

Comment These updated safety results show an imbalance in Bell's palsy in the HBV-23 trial with incidences of 0.09% and 0.04% in the Heplisav-B and Engerix-B arms, respectively. However, when the data from all safety trials were analyzed, the imbalance was smaller and statistically insignificant with incidences of 0.07% and 0.05%, respectively. We do not think that this small imbalance will affect the LOA and so we are leaving it unchanged at 5% above average. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/14/2016 (DVAX, Slide 6-9)

EAGLE PHARMACEUTICALS, INC. (EGRX) Ryanodex for Neurology - Other

Event Date: 01/11/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II - Exertional Heatstroke (Saudi Arabia) Market Group: Neurology

Lead Company: Eagle Pharmaceuticals, Inc. (EGRX) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 18% (1% Above Avg.) Average Approval: 17% Comparator Treatment Treatment Description Standard Of Care Ryanodex + (SOC) SOC Number of Patients 17 17 Mean Cooling Time to Normal Temperature in All 70.400 min 55.100 min Patients Mean Cooling Time to Normal Temperature in Patients 68.000 min 50.600 min Who Resolve from Glasgow Analysis: Eagle Pharmaceuticals presented updated results from the Phase II Saudi Arabia trial of Ryanodex for exertional heat stroke at the 34th annual J.P. Morgan Healthcare Conference on January 11, 2016.

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Results from this study were last seen in December 2015.

Results Ryanodex with current standard of care (body cooling methods) showed evidence of increased effectiveness in treating exertional heat stroke than just the standard of care alone. These cooling methods compared in the standard of care include cold water immersion, cold water mist, ice pack application, and supportive measures. In these updated results, patients in Group A (Ryanodex with Standard of Care) reached a desired cooling outcome faster than patients in Group B (SOC only).

In the measure for all patients, Group A patients were able to reach a normal body temperature of 101 degrees Fahrenheit from 106 degrees Fahrenheit within a mean time of 55.1 minutes versus the Group B mean time of 70.4 minutes. In the measure of patients who resolve from Glasgow, Group A patients were able to reach a normal body temperature of 101 degrees Fahrenheit from 106 degrees Fahrenheit within a mean time of 50.6 minutes versus the Group B mean time of 68.0 minutes. Source: J.P. Morgan Healthcare Conference 01/11/2016 (EGRX, Slide 15)

ELI LILLY & COMPANY (LLY) LY3012207 for Sarcoma

Event Date: 01/12/2016 Event Type: Regulatory - Rolling NDA/BLA Initiated (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Eli Lilly & Company (LLY)

Partner Companies: Bristol-Myers Squibb (BMY) Phase: NDA/BLA Change to Likelihood of Approval: 47% Likelihood of Approval: 82% (Same As Avg.) Average Approval: 82%

Analysis: Eli Lilly announced that the Company has begun U.S. rolling regulatory submission for olaratumab for the treatment of soft tissue sarcoma in late 2015. Source: J.P. Morgan Healthcare Conference 01/12/2016 (LLY, Slide 11)

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EMERGENT BIOSOLUTIONS, INC. (EBS) VAX161 for Pandemic Influenza Vaccines

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Emergent BioSolutions, Inc. (EBS)

Partner Companies: VaxInnate Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 19% (Same As Avg.) Average Approval: 19%

Analysis: Emergent Biosolutions continues to list VAX161C, a pandamic influenza vaccine, in Phase I clinical studies. Source: J.P. Morgan Healthcare Conference 01/11/2016 Company 8-K 01/11/2016 (EBS, Slide 13)

Ixinity for Hemophilia B

Event Date: 12/31/2015 Event Type: Progress Update - Product Launch (U.S.) (Clinical Analysis) Trial Name: N/A Market Group: Hematology

Lead Company: Emergent BioSolutions, Inc. (EBS)

Partner Companies: Ipsen (IPSEY) Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Emergent Biosolutions announced that the Company has launched Ixinity for the treatment of hemophilia B in 2015. Source: J.P. Morgan Healthcare Conference 01/11/2016 Company 8-K 01/11/2016 (EBS, Slide 5)

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Average Approval: N/A

Analysis: Enanta announced that the Company is currently studying a core inhibitor for the treatment of hepatitis B virus, and a non-fusion inhibitor for the treatment of RSV. Both compounds are in preclinical development, and the Company expects to advance one of the two product candidates to Phase I clinical studies in 2017. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/13/2016 (ENTA, Slide 5 and 32)

EDP-305 for Non-alcoholic Steatohepatitis (NASH)

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Enanta Pharmaceuticals, Inc. (ENTA) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Enanta announced the nomination of EDP-305 as the developmental candidate for the study of NASH and PBC. The Company is currently on tract to initiate clinical trials in 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (ENTA, Slide 28)

EDP-305 for Primary Biliary Cirrhosis (PBC) and Hepatic Fibrosis

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: N/A Market Group: Autoimmune/ immunology

Lead Company: Enanta Pharmaceuticals, Inc. (ENTA) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.)

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Average Approval: N/A

Analysis: Enanta announced that the Company is currently studying a core inhibitor for the treatment of hepatitis B virus, and a non-fusion inhibitor for the treatment of RSV. Both compounds are in preclinical development, and the Company expects to advance one of the two product candidates to Phase I clinical studies in 2017. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/13/2016 (ENTA, Slide 5 and 32)

EDP-305 for Non-alcoholic Steatohepatitis (NASH)

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Enanta Pharmaceuticals, Inc. (ENTA) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Enanta announced the nomination of EDP-305 as the developmental candidate for the study of NASH and PBC. The Company is currently on tract to initiate clinical trials in 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (ENTA, Slide 28)

EDP-305 for Primary Biliary Cirrhosis (PBC) and Hepatic Fibrosis

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: N/A Market Group: Autoimmune/ immunology

Lead Company: Enanta Pharmaceuticals, Inc. (ENTA) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.)

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Average Approval: N/A

Analysis: Enanta announced the nomination of EDP-305 as the developmental candidate for the study of NASH and PBC. The Company is currently on tract to initiate clinical trials in 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (ENTA, Slide 28)

ENDO INTERNATIONAL PLC (ENDP) Xiaflex for Cellulite

Event Date: 12/31/2015 Event Type: Regulatory - Meeting with FDA (Clinical Analysis) Trial Name: N/A Market Group: Dermatology

Lead Company: Endo International plc (ENDP) Actelion (ALIOF) Swedish Orphan Biovitrum (SOBI:SS) Partner Companies: Asahi Kasei Corporation (3407:JP) BioSpecifics Technologies (BSTC) Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 26% (2% Above Avg.) Average Approval: 24%

Analysis: Endo announced that they held a meeting with the FDA in December 2015 for Xiaflex for cellulite. A Phase IIb clinical trial initiation is expected in the near-term. In addition, there has been increasing partnership interest and discussions are ongoing. Source: J.P. Morgan Healthcare Conference 01/11/2016 (ENDP, Slide 7)

ENDOLOGIX, INC. (ELGX) Nellix Endovascular Aneurysm Sealing System for Aortic Aneurysm

Event Date: 01/12/2016 Event Type: Regulatory - PMA Modular Filing (Clinical Analysis)

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Trial Name: N/A Market Group: Cardiovascular

Lead Company: Endologix, Inc. (ELGX) Partner Companies: N/A Phase: PMA Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Endologix announced that it has completed the submission of PMA modules 1 and 3. The company is currently working toward modular 2, which is expected in February, and modular 4, which is expected in the March or April time frame. Source: J.P. Morgan Healthcare Conference 01/12/2016 (ELGX; Slide 9)

ENVISIA THERAPEUTICS ENV905 for Ocular Inflammation (Ophthalmology)

Event Date: 01/12/2016 Event Type: Trial Data - Preclinical Results (Clinical Analysis)

Trial Name: Preclinical Studies Market Group: Ophthalmology

Lead Company: Envisia Therapeutics Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Envisia announced preclinical results of ENV905 for the treatment of post-operative inflammation.

Context The Company plans to file an IND for ENV905 in the second half of 2016.

Design ENV905 was tested in a rabbit model of inflammation, compared to daily topical QID Durezol and placebo.

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Results ENV905 is efficacious for more than 3 weeks in a rabbit model of inflammation. Single dose of ENV905 is equivalent or superior to daily topical QID Durezol in vivo. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Envisia, Slide 16)

ENV1105 for Diabetic Macular Edema (Ophthalmology)

Event Date: 01/12/2016 Event Type: Trial Data - Preclinical Results (Clinical Analysis)

Trial Name: Preclinical Studies Market Group: Ophthalmology

Lead Company: Envisia Therapeutics Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Envisia presented preclinical data for ENV1105 for the treatment of diabetic macular edema.

Design ENV1105 was studied in a rabbit model versus Ozurdex.

Results ENV1105 showed 6 to 12 months of duration in the study. PRINT dexamethasone formulation achieves therapeutic concentrations in the rabbit vitreous/retina for more than 3 months, compare with roughly 1 month for Ozurdex. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Envisia, Slide 19)

ENV1305 for Wet Age-Related Macular Degeneration (Wet AMD) (Ophthalmology)

Event Date: 01/12/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Ophthalmology

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Lead Company: Envisia Therapeutics Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Envisia lists ENV1305 in preclinical development for the treatment of wet age-related macular degeneration (AMD). The Company anticipates elevating ENV1305 into preclinical development in 2016. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Envisia, Slides 6 and 23)

ESPERION THERAPEUTICS, INC. (ESPR) ETC-1002 for Dyslipidemia / Hypercholesterolemia

Event Date: 01/12/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase II - 035 - w/High Dose Statins Market Group: Cardiovascular

Lead Company: Esperion Therapeutics, Inc. (ESPR) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 14% (1% Above Avg.) Average Approval: 13%

Analysis: Esperion Therapeutics announced initiation of its Phase II pharmacokinetics/pharmacodynamics (PK/PD) study (ETC-1002-035) of bempedoic acid in patients treated with atorvastatin 80 mg, the most commonly prescribed high- dose statin. The Company expects to announce top-line results from the study by mid-year.

In February 2016, the Company also intends to initiate a Phase I clinical pharmacology study of bempedoic acid (ETC-1002-037), which will be an open label, drug-drug interaction study to assess the safety and tolerability of bempedoic acid at steady-state, as well as the PK of single doses of the highest-doses of the most commonly prescribed statins: atorvastatin 80 mg, rosuvastatin 40 mg, simvastatin 40

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mg, and pravastatin 80 mg. The Company expects to announce top-line results from the study by mid-year.

Phase II w/High Dose Statins Study ETC-1002-035 is a Phase II randomized, double-blind, parallel group study evaluating 60 patients on stable atorvastatin 80 mg per day. All patients in the study will receive 80 mg of atorvastatin for four weeks. Patients will then be randomized to receive either 180 mg of bempedoic acid, or placebo, for four weeks. The study will enroll patients at approximately 20 centers across the U.S. The primary objectives of the study are to assess the LDL-C lowering efficacy of bempedoic acid versus placebo on a background of atorvastatin 80 mg, as well as multiple-dose plasma PK of atorvastatin 80 mg alone and in combination with bempedoic acid. Secondary objectives include assessing the effect of bempedoic acid on lipid and cardiometabolic biomarkers, including high-sensitivity C-reactive protein; characterizing the tolerability and safety of bempedoic acid; and evaluating the steady-state plasma PK of bempedoic acid. Source: Press Release 01/12/2016 (ESPR) J.P. Morgan Healthcare Conference 01/13/2016 (ESPR)

ETC-1002 for Dyslipidemia / Hypercholesterolemia

Event Date: 01/13/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase III - ETC-1002-040 Market Group: Cardiovascular

Lead Company: Esperion Therapeutics, Inc. (ESPR) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 34% Likelihood of Approval: 48% (1% Above Avg.) Average Approval: 47%

Analysis: Esperion Therapeutics announced the start of a global Phase III long-term safety and tolerability study (ETC-1002-040) of bempedoic acid in patients with hyperlipidemia whose LDL-C is not adequately controlled with low- and moderate-dose statins. This study will enable the Company to understand the 52 week safety profile of bempedoic acid, and top-line results are expected in the fourth quarter of 2017.

This study marks the launch of the Phase III clinical program — known as Cholesterol

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Lowering via ETC-1002, an ACL-inhibiting Regimen (CLEAR) — which will be focused on the development of bempedoic acid for statin intolerant patients with uncontrolled LDL-C levels. The Company will provide details of the full Phase III global development strategy in the second quarter of 2016. Separately, the Company anticipates formalizing and communicating the design of the planned cardiovascular outcomes trial in the second quarter of 2016.

Phase III ETC-1002-040 Study ETC-1002-040 is a Phase III randomized, multicenter, double-blind, placebo- controlled study evaluating 180 mg of bempedoic acid versus placebo in 900 patients with hyperlipidemia at high cardiovascular disease risk and whose LDL-C is not adequately controlled with maximally tolerated lipid-modifying therapy. The study will enroll patients at approximately 125 sites in the U.S., Canada and the European Union. The primary objective is to assess safety and tolerability of patients treated with bempedoic acid for 52 weeks. Secondary objectives include assessing the effects of bempedoic acid on other lipid and cardiometabolic risk markers, including LDL-C and high-sensitivity C-reactive protein. Source: Press Release 01/13/2016 J.P. Morgan Healthcare Conference 01/13/2016 (ESPR) GENMAB HuMax-Inflam for Solid Tumors

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Cormorant Pharmaceuticals Bristol-Myers Squibb (BMY) Partner Companies: Genmab (GEN:DC) Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6%

Analysis: Genmab currently lists HuMax-IL8 in Phase I clinical development for the treatment of metastatic solid tumors. Source: J.P. Morgan Healthcare Conference 01/14/2016 (Genmab, Slide 5 and 8)

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Teprotumumab for Diabetic Macular Edema (Ophthalmology)

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Ophthalmology

Lead Company: River Vision Development Corp. Chugai (4519:JP) Partner Companies: Roche (RHHBF) Genmab (GEN:DC) Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 21% (Same As Avg.) Average Approval: 21%

Analysis: Genmab currently lists Teprotumumab in Phase I clinical development for the treatment of diabetic macular edema. Source: J.P. Morgan Healthcare Conference 01/14/2016 (Genmab, Slide 5)

GILEAD SCIENCES, INC. (GILD) GS-9674 for Non-alcoholic Steatohepatitis (NASH)

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Gilead Sciences, Inc. (GILD) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 14% Likelihood of Approval: 14% (Same As Avg.) Average Approval: 14%

Analysis: Gilead lists GS-9674 in Phase I development for the treatment of non-alcoholic steatohepatitis (NASH).

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HealthcareSource: J.P. Morgan Healthcare Conference: Conference 01/11/2016 Conference (GILD, Slide 28) Review

TAF/emtricitabine/rilpivirine for HIV / AIDS

Event Date: 07/31/2015 Event Type: Regulatory - MAA Submission (Europe) (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Gilead Sciences, Inc. (GILD)

Partner Companies: Johnson & Johnson (JNJ) Phase: NDA/BLA Change to Likelihood of Approval: 0% Likelihood of Approval: 88% (Same As Avg.) Average Approval: 88%

Analysis: Gilead announced that the EU MAA for R/F/TAF was submitted to the European Medicines Agency (EMA) in July 2015. Source: J.P. Morgan Healthcare Conference 01/11/2016 (GILD, Slide 11)

Harvoni for Hepatitis C (HCV) (Antiviral)

Event Date: 01/11/2016 Progress Update - Product Launch (Japan) (Clinical Event Type: Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Gilead Sciences, Inc. (GILD) Partner Companies: N/A Phase: Approved Change to Likelihood of 0% Approval: Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Gilead announced that Harvoni has been approved and launched in Japan. Source: J.P. Morgan Healthcare Conference 01/11/2016 (GILD, Slide 3)

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Healthcare Conference: Conference Review

GLAXOSMITHKLINE PLC (GSK) IONIS-TTRrx for Transthyretin-related Hereditary Amyloidosis (Familial Amyloid Polyneuropathy)

Event Date: 01/11/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II/III - NEURO-TTR (Ext.) Market Group: Metabolic

Lead Company: GlaxoSmithKline plc (GSK)

Partner Companies: Ionis Pharmaceuticals, Inc. (IONS) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 62% (Same As Avg.) Average Approval: 62% Treatment Treatment Description IONIS-TTRRx Number of Patients 38 Mean Max TTR Reduction 76.000 % Max TTR Reduction 92.000 % Analysis: Isis announced updated data from an ongoing open-label extension (OLE) study of IONIS-TTRRx in patients with familial amyloid polyneuropathy (FAP) at the 2016 J.P. Morgan Conference.

Results from this study were last seen in April 2015.

Context Updated results from the Phase III portion of this trial are expected mid-2016. Results from the Phase III NEURO-TTR trial are expected in the first half of 2017.

Results As of October 2015, the mean max TTR reduction from baseline seen in 38 patients was 76% while the max TTR reduction seen was 92%. Source: J.P. Morgan Healthcare Conference 01/11/2016 (IONS, Slide 20)

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IONIS-TTRrx for Transthyretin-related Hereditary Amyloidosis (Familial Amyloid Polyneuropathy)

Event Date: 12/31/2015 Trial Announcement - Patient Enrollment Completed (Clinical Event Type: Analysis)

Trial Name: Phase II/III - NEURO-TTR (Ext.) Market Group: Metabolic

Lead Company: GlaxoSmithKline plc (GSK)

Partner Companies: Ionis Pharmaceuticals, Inc. (IONS) Phase: III Change to Likelihood of 0% Approval: Likelihood of Approval: 62% (Same As Avg.) Average Approval: 62%

Analysis: Ionis announced that patient enrollment was completed at the end of 2015 for the Phase III NEURO-TTR study of IONIS-TTRrx for the treatment of familial amyloid polyneuropathy. Results from the study are expected in the first half of 2017. Source: J.P. Morgan Healthcare Conference 01/11/2016 (IONS, Slide 24)

GLENMARK PHARMACEUTICALS LIMITED GBR 830 for Atopic Dermatitis (Eczema)

Event Date: 01/12/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase II Market Group: Allergy

Lead Company: Glenmark Pharmaceuticals Limited (GNP:IN) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 8% Likelihood of Approval: 24% (Same As Avg.) Average Approval: 24%

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Analysis: Glenmark reported that a Phase IIa trial of GBR 830 has been initiated in the US with atopic dermatitis as the primary indication. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Glenmark, Slide 10)

GBR 1302 for Cancer

Event Date: 01/12/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Glenmark Pharmaceuticals Limited (GNP:IN) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 6% Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6%

Analysis: Glenmark reported that it has initiated a Phase I trial of GBR-1302 in Germany. GBR- 1302 is listed in the pipeline with HER2-positive cancers are the primary indications. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Glenmark, Slide 10)

GLOBAL BLOOD THERAPEUTICS INC. (GBT) Oral Kallikrein Inhibitor (GBT) for Hereditary Angioedema (HAE)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Autoimmune/ immunology

Lead Company: Global Blood Therapeutics Inc. (GBT) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Global Blood Therapeutics (GBT) is currently conducting preclinical research to

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identify and develop an oral therapy for hereditary angioedema (HAE). The oral therapeutic could potently and selectively inhibit plasma kallikrein for the treatment of HAE.

GBT intends to initiate a Phase I clinical trial of the HAE candidate in the second half of 2016. Source: Company Website (GBT) J.P. Morgan Healthcare Conference 01/13/2016 (GBT)

HALOZYME THERAPEUTICS, INC. (HALO) PEGPH20 for Pancreatic Cancer

Event Date: 01/12/2016 Trial Announcement - Patient Enrollment Completed (Clinical Event Type: Analysis)

Trial Name: Phase II - HALO-202 (w/Abraxane + Gemcitabine) Market Group: Oncology

Lead Company: Halozyme Therapeutics, Inc. (HALO)

Partner Companies: Nektar (NKTR) Phase: II Change to Likelihood of 0% Approval: Likelihood of Approval: 13% (3% Above Avg.) Average Approval: 10%

Analysis: Halozyme Therapeutics provided key program updates and its Annual financial guidance at the 34th annual JP Morgan Healthcare Conference. Halozyme announced that they achieved target enrollment in Stage 2 of Halozyme Study 202 of investigational new drug PEGPH20 in metastatic pancreatic ductal adenocarcinoma patients. Halozyme has enrolled approximately 120 patients through the end of 2015.

The company remains blinded to the efficacy results and projects presentation of mature progression-free survival data in the event driven study in the fourth quarter of 2016. 45 total sites (US only) enrolled 146 patients in Stage 1, which was shown in the interim data that has been released. 114 patients was the target enrollment for Stage 2.

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Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/12/2016 (HALO, Slide 9)

PEGPH20 for Pancreatic Cancer

Event Date: 01/12/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II - HALO-202 (w/Abraxane + Gemcitabine) Market Group: Oncology

Lead Company: Halozyme Therapeutics, Inc. (HALO)

Partner Companies: Nektar (NKTR) Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 13% (3% Above Avg.) Average Approval: 10%

Analysis: Halozyme Therapeutics reported a continued reduction in the rate of thromboembolic (TE) events in the PEGPH20 treatment arm in Stage 2 of Study 202.

Results from this study were last seen in May 2015.

Design Study 202 (Halo 109-202) is a Phase II multi-center, randomized clinical trial evaluating investigational new drug PEGPH20 as a first-line therapy for potential treatment of patients with metastatic pancreatic cancer.

Endpoints The primary outcome of the trial is to measure improvement in progression-free survival in patients receiving investigational new drug PEGPH20 in combination with gemcitabine and ABRAXANE (nab-paclitaxel) compared to gemcitabine and ABRAXANE alone. A second primary endpoint will assess the TE event rate in the PEGPH20 treatment arm. Secondary endpoints also include objective response rate and overall survival.

Results With patient data through December 15, 2015, Halozyme reported a TE event rate in the PEGPH20 arm of 12 percent (9 out of 73 patients) compared to the previously reported 42 percent (31 out of 74 patients) in Stage 1 of the study. Halozyme amended the study protocol in 2014, including the addition of prophylactic

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administration of low molecular weight heparin (enoxaparin) in both treatment arms based on a reported potential imbalance of TE events in the study.

The TE event rate in both stages and arms of the study are: Enoxaparin PEGPH20 + ABRAXANE + Prophylaxis Dose ABRAXANE + Gemcitabine Gemcitabine Stage 1 (through N/A 42% (n=74) 25% (n=61) Dec. 5, 2014) Stage 2 (through 40 mg/day; or 40 28% (n=18) 29% (n=7) Dec. 15, 2015) mg/day increased to 1 mg/kg/day Stage 2 (through Started on 1 7% (n=55) 4% (n=27) Dec. 15, 2015) mg/kg/day Stage 2 (through TOTAL – Stage 2 12% (n=73) 9% (n=34) Dec. 15, 2015)

Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/12/2016 (HALO, Slide 12)

PEGPH20 for Pancreatic Cancer

Event Date: 01/12/2016 Event Type: Trial Announcement (Clinical Analysis)

Trial Name: Phase III - HALO-301 Market Group: Oncology

Lead Company: Halozyme Therapeutics, Inc. (HALO)

Partner Companies: Nektar (NKTR) Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 13% (3% Above Avg.) Average Approval: 10%

Analysis: Halozyme Therapeutics announced that they remain on schedule to initiate a Phase III HALO-301 study at the end of March 2016 for PEGPH20 in Stage IV metastatic pancreatic ductal adenocarcinoma high-HA patients.

Phase III Study The company provided an update on the Phase III trial design, selecting approximately 200 sites in 20 countries concentrated in North America, Europe, South America and Asia Pacific. The protocol and statistical design have been

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reviewed by the majority of participating countries, including the U.S. and multiple E.U. member states through the voluntary harmonization procedure (VHP). Target enrollment is 420 patients.

The trial will be a randomized 2:1 double blind, placebo-controlled, global study. The trial is powered for two primary endpoints, progression-free survival (PFS) and overall survival. Statistical powering to support PFS is based on achieving a hazard ratio of 0.59. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/12/2016 (HALO, Slide 13)

PEGPH20 Companion Diagnostic for Pancreatic Cancer

Event Date: 01/12/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Roche Holding AG (RHHBF)

Partner Companies: Halozyme (HALO) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Halozyme announced they are advancing development of the companion diagnostic test to prospectively identify patients with high levels of hyaluronan, or HA and expect a U.S. IDE submission in February 2016.

Halozyme has partnered with Ventana to develop the companion diagnostic and announced the methodology and scoring algorithm have been finalized. Based on the cutpoint for the Ventana diagnostic, Halozyme now expects approximately 35 to 40 percent of metastatic pancreatic cancer patients to have high-HA tumors, similar to the previously reported interim results from Stage 1 of its Phase II study using the Halozyme prototype assay. Analysis of biopsy samples from patients in Stage 1 of Study 202 with the new diagnostic show a PFS benefit in the PEGPH20 arm with a hazard ratio of 0.48. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/12/2016 (HALO, Slide 15-16)

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HANMI PHARMACEUTICAL CO., LTD. HCP1105 for Dyslipidemia / Hypercholesterolemia

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HCP1105 under Phase III development for the treatment of complex dyslipidemia. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9) HCP1302 for Hypertension (Systemic)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HCP1302 under Phase III development for hypertension/erectile dysfunction. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9)

HCP1405 for Osteoporosis / Osteopenia

Event Date: 01/13/2016

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Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HCP1405 under Phase I development for osteoporosis. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9)

HCP1401 for Hypertension (Systemic)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HCP1401 under Phase III development for hypertension. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9) HIP1402 for Benign Prostatic Hyperplasia (BPH)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Urology

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S.

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Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HIP1402 under Phase III development for the treatment of prostatism. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9)

HCP1202 for Chronic Obstructive Pulmonary Disease (COPD)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Respiratory

Lead Company: Hanmi Pharmaceutical Co., Ltd. (128940:KS) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Hanmi lists HCP1202 under Phase I development for the treatment of COPD. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Hanmi, Slide 9)

HEARTWARE INTERNATIONAL, INC. (HTWR) HeartWare Ventricular Assist System for Congestive Heart Failure (CHF)

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Development - HVAD LATERAL Market Group: Cardiovascular

Lead Company: HeartWare International, Inc. (HTWR) Partner Companies: N/A Phase: Approved Change to Likelihood of Approval: 0%

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Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Heartware announced it has reached 75% enrollment in the HVAD LATERAL study and expects to complete enrollment in Q1 2016. Source: J.P. Morgan Healthcare Conference 01/11/2016 (HTWR; Slide 16)

MVAD System for Congestive Heart Failure (CHF)

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis)

Trial Name: Development - MVAdvantage (Europe & Australia) Market Group: Cardiovascular

Lead Company: HeartWare International, Inc. (HTWR) Partner Companies: N/A Phase: IDE Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Heartware announced that it is has completed the alterations to the controller and software updates. The company said it is now working to identify the cause of pump thrombus events that were observed in December 2015 following the pause in the trial due to an issue with the controller.

The company believes that the power and efficiency in the device is causing some patients to experience sustained suction, which appears to lead to thrombus. The company is looking into the qPulse algorithm as a potential cause of the suction problem and is investigating potential solutions including a suction alarm algorithm.

The company has set in place the following steps to review with investigators in order to submit a solution to regulators to continue the study:  Turn off qPulse on all patients, monitor to confirm suction reduction  Improve suction alarm to identify events sooner The company said it is also evaluating other algorithms that could reduce the pumps sensitivity, assessing more moderate pulsatility modes, and incorporating an external pump designer to aid in the MVAD evaluation.

The company said it hopes to resume enrollment in the current study, however it

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noted that design enhancements may require the company to begin a new trial.

The company expects that all efforts in resolving the MVAD issues may take several months to complete, and the timetable for regulatory filings and restarting clinical implants cannot be precisely projected at this time. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (HTWR; Slide 19)

IGNYTA, INC. (RXDX) Taladegib for Skin Cancer - Basal Cell Carcinoma (BCC)

Event Date: 01/14/2016 Event Type: Trial Data - Top-Line Results (Clinical Analysis)

Trial Name: Phase I - Dose-Escalation Market Group: Oncology

Lead Company: Ignyta, Inc. (RXDX)

Partner Companies: Eli Lilly (LLY) Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6% Treatment Treatment All Treatment Treatment Treatment Treatment Treatment Taladegib Taladegib Taladegib Taladegib Taladegib Taladegib Description Prior No Prior All Patients Prior HHI HHI Naive, HHI Naive, mBCC Hedgehog Hedgehog IaBCC Therapy Therapy Number of Patients 31 16 47 N/A N/A N/A Complete Response 2 Patients 3 Patients 5 Patients 2 Patients 2 Patients 1 Patients Partial Response 9 Patients 8 Patients 17 Patients 9 Patients 6 Patients 2 Patients Stable Disease 17 Patients 4 Patients 21 Patients 17 2 Patients 2 Patients Patients Progressive Disease 1 Patients 0 Patients 1 Patients 1 Patients 0 Patients 0 Patients Response Rate 37.900 % 68.800 % 46.800 % 38.000 % 73.000 % 60.000 % Clinical Benefit Rate 90.300 % 100.000 % 93.600 % N/A N/A N/A Median Duration of N/A N/A 10.200 N/A N/A N/A Response Months

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Analysis: Ignyta announced results from a Phase I study of Taladegib in patients with basal cell carcinoma at the 34th annual J.P. Morgan Healthcare Conference on January 14, 2016.

Results For results, see table above.

Most Common Adverse Events Treatment-emergent adverse events occurred in >10% of patients on taladegib, irrespective of causality. Dose-limiting toxicities were noted in four among twenty- five patients in the dose-escalation phase:  100 mg (N=1), grade 3 hyponatremia  400 mg (N=1), grade 3 vomiting  600 mg (N=2), grade 3 maculo-papular rash and grade 1 anorexia; grade 2 confusion and nausea  The maximum tolerated dose was 400 mg Source: J.P. Morgan Healthcare Conference 01/14/2016 (RXDX, Slide 25-29)

IMMUNOGEN, INC. (IMGN) Mirvetuximab soravtansine for Ovarian Cancer

Event Date: 01/12/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase I - FOLR1-Positive Market Group: Oncology

Lead Company: ImmunoGen, Inc. (IMGN) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 8% (2% Above Avg.) Average Approval: 6%

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Treatment Treatment Treatment Treatment Description Mirvetuximab Soravtansine Mirvetuximab Soravtansine Mirvetuximab Soravtansine All Patients High and Medium FRa Patients on Treatment >6 Expression Months Number of Patients 20 16 7 Confirmed Objective 7 Patients 7 Patients 6 Patients Response Analysis: ImmunoGen reported updated results on their Phase I trial of Mirvetuximab Soravtansine for ovarian cancer at the 34th annual J.P. Morgan Healthcare Conference on January 12, 2016.

Results were previously seen in November 2015.

Context ImmunoGen reported that clinical data from the 40-patient cohort from the Phase I Mirvetuximab Soravtansince trial for ovarian cancer will be presented at a medical meeting in the second quarter of 2016.

These findings informed inclusion criteria for FORWARD I.

Results The results show that most patients had tumor shrinkage. Of all the patients, 35% (7/20) had a confirmed objective response and 6 of 7 patients had a confirmed reponse on treatment 6 months or longer.

Objective reponse rates for all patients with high or medium FRa expression was 44% (7/16).

Most Common Adverse Events The most common (>25% patients) side effects were diarrhea, blurred vision, vomiting, fatigue, nausea, and headaches. The majority of these effects were grade 1 or 2 and were seen to be manageable or reversible. Source: Press Release 01/10/2016 J.P. Morgan Healthcare Conference 01/12/2016 (IMGN, Slides 7-9)

INCYTE CORPORATION (INCY) Epacadostat for Solid Tumors

Event Date: 01/11/2016

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Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase I/II - KEYNOTE-037 (w/Pembrolizumab) Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10%

Analysis: Recruitment has begun into the eight tumor-specific cohorts of ECHO-202, the Phase II trial of epacadostat in combination with pembrolizumab. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 14)

Epacadostat for Solid Tumors

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase I/II - ECHO-204 Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10%

Analysis: Incyte is conducting three additional Phase I/II clinical trials of epacadostat in combination with immune checkpoint inhibitors. ECHO-204, in combination with Bristol-Myers Squibb’s PD-1 inhibitor, Opdivo (nivolumab), has opened seven tumor specific cohorts. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 14)

Epacadostat for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/11/2016

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Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase I - ECHO-110 Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6%

Analysis: Incyte is conducting three additional Phase I/II clinical trials of epacadostat in combination with immune checkpoint inhibitors. ECHO-110, a trial in combination with Roche/Genentech’s investigational PD-L1 inhibitor, atezolizumab, in patients with NSCLC is ongoing. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 14)

Jakafi for Pancreatic Cancer

Event Date: 01/11/2016 Trial Announcement - Patient Enrollment Completed (Clinical Event Type: Analysis)

Trial Name: Phase III - JANUS 1 (SPA) Market Group: Oncology

Lead Company: Incyte Corporation (INCY)

Partner Companies: Novartis (NVS) Phase: III Change to Likelihood of 0% Approval: Likelihood of Approval: 37% (2% Above Avg.) Average Approval: 35%

Analysis: The Phase III JANUS 1 trial of ruxolitinib, a JAK1 / JAK2 inhibitor, in second line metastatic pancreatic cancer is now fully recruited, with results expected in 2016. The primary endpoint of JANUS 1 is overall survival.

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Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 11)

INCB52793 for Hematologic Cancer

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase Ia/Ib - Advanced Malignancies Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10%

Analysis: INCB52793 is in a dose escalation study in patients with advanced malignancies. INCB52793 has shown synergistic efficacy in combination with standard of care in preclinical models of multiple myeloma. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 11)

INCB40093 for Non-Hodgkin's Lymphoma (NHL)

Event Date: 01/11/2016 Trial Announcement - Trial/Enrollment Suspension (Clinical Event Type: Analysis) Trial Name: Phase I - w/INCB39110 , Phase II - Study 201 Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: Suspended Change to Likelihood of -10% Approval: Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Incyte’s PI3Kδ inhibitor clinical development program is focused on INCB50465,

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based on the molecule’s potential lack of hepatotoxicity and improved potency compared to approved PI3Kδ inhibitors and compared to Incyte’s PI3Kδ inhibitor INCB40093. A Phase I/I trial of INCB50465, both as monotherapy and in combination with INCB39110, is already underway in multiple B-cell malignancies. Clinical activities of INCB40093 are being closed. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY)

INCB50465 for Hematologic Cancer

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase I - w/INCB039110 Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10%

Analysis: Incyte’s PI3Kδ inhibitor clinical development program is focused on INCB50465, based on the molecule’s potential lack of hepatotoxicity and improved potency compared to approved PI3Kδ inhibitors and compared to Incyte’s PI3Kδ inhibitor INCB40093. A Phase I/II trial of INCB50465, both as monotherapy and in combination with INCB39110, is already underway in multiple B-cell malignancies. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 18)

INCB59872 for Solid Tumors

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Incyte Corporation (INCY) Partner Companies: N/A Phase: Preclinical

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Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Two new compounds are expected to enter Incyte’s clinical development portfolio in the first half of 2016. INCB59872, a potent and selective LSD1 inhibitor, is expected to enter clinical trials in patients with advanced malignancies in the coming months. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 17)

INCAGN1876 for Cancer

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Agenus Inc. (AGEN)

Partner Companies: Incyte (INCY) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Two new compounds are expected to enter Incyte’s clinical development portfolio in the first half of 2016. INCAGN1876, an anti-GITR agonist antibody that is part of the ongoing discovery alliance with Agenus, is expected to enter clinical trials for the treatment of patients with advanced cancer during the first half of 2016. Incyte’s alliance with Agenus has recently been expanded to include a total of 7 therapeutic targets, with options for additional expansion. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (INCY, Slide 16)

INDIVIOR PLC RBP-6300 for Drug Addiction

Event Date: 09/30/2015

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 72 Biomedtracker August 2016 / 73 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase I - RB-EU-14-0001 Market Group: Neurology

Lead Company: Indivior plc (INDV:LN) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 17% (Same As Avg.) Average Approval: 17%

Analysis: Indivior announced that they enrolled and dosed the first patients in a PK study (RB- EU-14-0001) of RBP-6300 in September 2015. Source: J.P. Morgan Healthcare Conference 01/13/2016 (INDV, Slide 15)

RBP-7000 for Schizophrenia

Event Date: 01/13/2016 Trial Announcement - Trial/Enrollment Reinitiated (Clinical Event Type: Analysis)

Trial Name: Phase III - RB-US-13-0005 (Long-Term Safety) Market Group: Psychiatry

Lead Company: Indivior plc (INDV:LN) Partner Companies: N/A Phase: III Change to Likelihood of 0% Approval: Likelihood of Approval: 56% (5% Above Avg.) Average Approval: 51%

Analysis: Indivior announced that enrollment is currently on-going in a Phase III long-term safety study of RBP-7000 for the treatment of schizophrenia. Source: J.P. Morgan Healthcare Conference 01/13/2016 (INDV, Slide 16) INTREXON CORPORATION (XON)

Ocular Disease Program (Intrexon/Sun) for Wet Age-Related Macular Degeneration (Wet AMD) (Ophthalmology)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 73 Biomedtracker August 2016 / 74 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase I - RB-EU-14-0001 Market Group: Neurology

Lead Company: Indivior plc (INDV:LN) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 17% (Same As Avg.) Average Approval: 17%

Analysis: Indivior announced that they enrolled and dosed the first patients in a PK study (RB- EU-14-0001) of RBP-6300 in September 2015. Source: J.P. Morgan Healthcare Conference 01/13/2016 (INDV, Slide 15)

RBP-7000 for Schizophrenia

Event Date: 01/13/2016 Trial Announcement - Trial/Enrollment Reinitiated (Clinical Event Type: Analysis)

Trial Name: Phase III - RB-US-13-0005 (Long-Term Safety) Market Group: Psychiatry

Lead Company: Indivior plc (INDV:LN) Partner Companies: N/A Phase: III Change to Likelihood of 0% Approval: Likelihood of Approval: 56% (5% Above Avg.) Average Approval: 51%

Analysis: Indivior announced that enrollment is currently on-going in a Phase III long-term safety study of RBP-7000 for the treatment of schizophrenia. Source: J.P. Morgan Healthcare Conference 01/13/2016 (INDV, Slide 16) INTREXON CORPORATION (XON)

Ocular Disease Program (Intrexon/Sun) for Wet Age-Related Macular Degeneration (Wet AMD) (Ophthalmology)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 73 Biomedtracker August 2016 / 75 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

IONIS PHARMACEUTICALS, INC. (IONS) IONIS-SMNRx for Spinal Muscular Atrophy

Event Date: 12/31/2015 Trial Announcement - Patient Enrollment Completed (Clinical Event Type: Analysis)

Trial Name: Phase III - CHERISH Market Group: Metabolic

Lead Company: Ionis Pharmaceuticals, Inc. (IONS)

Partner Companies: Biogen (BIIB) Phase: III Change to Likelihood of 0% Approval: Likelihood of Approval: 67% (5% Above Avg.) Average Approval: 62%

Analysis: Ionis Pharmaceuticals announced that it has earned a milestone payment of $2.15 million from Biogen for completing the target enrollment in the Phase III CHERISH study of IONIS-SMNRx for spinal muscular atrophy at the end of 2015. Top-line results from the study are expect in the first half of 2017.

Phase III CHERISH Study CHERISH, a Phase III study of nusinersen, is a randomized, double-blind, sham- procedure controlled fifteen month study in approximately 117 children who are non-ambulatory with SMA between the ages of 2 to 12. The study will evaluate the efficacy and safety of 12 mg doses of nusinersen with a primary endpoint of a change in the Hammersmith Functional Motor Scale-Expanded (HFMSE), a validated method to measure changes in muscle function in patients with SMA. Additional efficacy endpoints are also included in the study. Source: Press Release 01/12/2016 J.P. Morgan Healthcare Conference 01/11/2016 (IONS, Slide 10)

IONIS-SMNRx for Spinal Muscular Atrophy

Event Date: 01/11/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II - Infantile-Onset SMA Market Group: Metabolic

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 75 Biomedtracker August 2016 / 76 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Lead Company: Ionis Pharmaceuticals, Inc. (IONS)

Partner Companies: Biogen (BIIB) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 67% (5% Above Avg.) Average Approval: 62% Treatment Treatment Description Nusinersen Number of Patients 17 Mean Change in CHOP-INTEND Score 22.700 points Mean Increase in HFMSE Score 4.400 Analysis: Ionis Pharmaceuticals announced update results from the Phase II trial of nusinersen for the treatment of infantile onset spinal muscular atrophy at the 2016 J.P Morgan Conference.

Results from this study were last seen in June 2015.

Results A 22.7 point mean change in the CHOP-INTEND score at 21 months was observed. Infants are continuing to achieve new developmental milestones. All infants continuing in the study are older than 2 years old. The mean increase in the HFMSE score is 4.4 and there were observed increases in other measures of muscle function.

Most Common Adverse Events In 2015, no patients required permanent ventilation and there were no deaths. The LP injection procedure in infants and children with SMA has been well tolerated. Nusinersen has been well tolerated with no safety concerns to date. There have been no drug-related SAEs, adverse changes on neurological exams, or clinically significant changes in CSF safety labs. All AEs observed have been mostly mild in severity. Source: Press Release 01/11/2016 (IONS) J.P. Morgan Healthcare Conference 01/11/2016 (IONS, Slide 13-15)

IRONWOOD PHARMACEUTICALS, INC. (IRWD) Linzess for Irritable Bowel Syndrome (IBS)

Event Date: 01/11/2016 Regulatory - Filing for Approval (Emerging Markets) (Clinical Event Type: Analysis) Trial Name: N/A

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 76 Biomedtracker August 2016 / 77 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Market Group: Gastroenterology (non inflammatory bowel disease)

Lead Company: Ironwood Pharmaceuticals, Inc. (IRWD) AstraZeneca (AZN) Partner Companies: Allergan (AGN) Astellas (4503:JP) Phase: Approved Change to Likelihood of 0% Approval: Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Ironwood announced that Linzess is in review for approval for IBS-C by the China Food and Drug Administration. Source: J.P. Morgan Healthcare Conference 01/11/2016 (IRWD, Slide 25)

JAZZ PHARMACEUTICALS PLC (JAZZ) JZP-416 for Acute Lymphocytic Leukemia (ALL)

Event Date: 01/11/2016 Event Type: Progress Update - Development Review (Clinical Analysis)

Trial Name: Phase II - Children and Young Adults (ALL/LBL) Market Group: Oncology

Lead Company: Jazz Pharmaceuticals plc (JAZZ)

Partner Companies: Alizé Pharma Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 5% (5% Below Avg.) Average Approval: 10%

Analysis: Based on the outcome of the analyses, Jazz has decided that the pivotal Phase II clinical trial of JZP-416 for the treatment of acute lymphoblastic leukemia has been terminated. Source: J.P. Morgan Healthcare Conference 01/11/2016 (JAZZ, Slide 25)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 77 Biomedtracker August 2016 / 78 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

LUMINEX CORPORATION (LMNX) ARIES HSV 1&2 Assay for Herpes Simplex Virus (HSV) (Antiviral)

Event Date: 11/07/2015 Event Type: Progress Update - Product Launch (U.S.) (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Luminex Corporation (LMNX) Partner Companies: N/A Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Luminex stated that the ARIES HSV 1&2 Assay was launched at the Association for Molecular Pathology (AMP) 2015 meeting which was held November 5-7. Source: J.P. Morgan Healthcare Conference 01/14/2016 (LMNX, Slide 4)

MANNKIND CORPORATION (MNKD) Treprostinil (MannKind) for Pulmonary Arterial Hypertension (PAH)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: MannKind Corporation (MNKD) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: MannKind announced that they are pursuing development of an inhaled version of Treprostinil using their proprietary Technosphere technology. They have currently tested three formulations and anticipate conducting IND enabling studies throughout 2016, with the aim of submitting an IND in the fourth quarter of 2016.

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 78 Biomedtracker August 2016 / 79 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNKD, Slide 10-11)

MEDGENICS INC. (MDGN) NFC-1 for Attention Deficit Hyperactivity Disorder (ADHD)

Event Date: 12/31/2015 Event Type: Regulatory - IND Filing (Clinical Analysis)

Trial Name: Phase I/II - 22q11.2 Deletion Syndrome Market Group: Psychiatry

Lead Company: Medgenics Inc. (MDGN) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 7% (Same As Avg.) Average Approval: 7%

Analysis: Medgenics announced that the Company submitted an Investigational New Drug (IND) application for a Phase I/II trial of NFC-1 for 22q11.2 Deletion Syndrome in the fourth quarter of 2015. Source: J.P. Morgan Healthcare Conference 01/11/2016 (MDGN, Slide 28)

MEDIVATION, INC. (MDVN) MDV4463 for Undisclosed

Event Date: 01/11/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Medivation, Inc. (MDVN) Partner Companies: N/A Phase: I Change to Likelihood of Approval: N/A Likelihood of Approval: 16% (Same As Avg.)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 79 Biomedtracker August 2016 / 80 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Average Approval: 16%

Analysis: Medivation announced that it has advanced MDV4463, a novel small molecule inhibitor of the sterol regulatory element binding pathway (SREBP) pathway, into a Phase I study in healthy volunteers.

Preclinical studies demonstrated that MDV4463 lowered triglycerides, cholesterol, glucose, insulin and weight in animals. MDV4463 also reduced fat in the liver in animal models of non-alcoholic hepatic steatosis (NASH). Potential indications being considered include NASH, hyperlipidemia, diabetes, obesity, metabolic syndrome. Source: J.P. Morgan Healthcare Conference 01/11/2016 (MDVN, Slide 41)

MERCK & CO., INC. (MRK) Keytruda for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 12/31/2015 Event Type: Regulatory - sNDA/sBLA Filing (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Merck & Co., Inc. (MRK) Partner Companies: N/A Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Merck announced they have submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration for KEYTRUDA in second line NSCLC based on findings from KEYNOTE-010 at the end of 2015. Source: J.P. Morgan Healthcare Conference 01/11/2016 (MRK)

TDO/IDO Inhibitors for Cancer

Event Date: 01/11/2016 Event Type: Partnership - Acquisition Closed (Clinical Analysis) Trial Name: N/A Market Group: Oncology

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 80 Biomedtracker August 2016 / 81 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Lead Company: Merck & Co., Inc. (MRK) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Merck announced that it has acquired IOmet, a privately-held UK-based drug discovery company focused on the development of innovative medicines for the treatment of cancer, with a particular emphasis on the fields of cancer immunotherapy and cancer metabolism.

Under terms of the agreement, Merck, through a subsidiary, will acquire IOmet, including its comprehensive pre-clinical pipeline of IDO (indoleamine-2,3- dioxygenase 1), TDO (tryptophan-2,3-dioxygenase), and dual-acting IDO/TDO inhibitors. Based on the transaction, IOmet will become a wholly owned subsidiary of Merck. Financial terms of the acquisition were not disclosed. Source: Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016 (MRK)

MERRIMACK PHARMACEUTICALS, INC. (MACK) Onivyde for Pancreatic Cancer

Event Date: 10/26/2015 Event Type: Progress Update - Product Launch (U.S.) (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Merrimack Pharmaceuticals, Inc. (MACK) PharmaEngine Partner Companies: Baxalta (BXLT) Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Merrimack announced that Onivyde for pancreatic cancer was launched in the

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 81 Biomedtracker August 2016 / 82 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

United States on October 26, 2015. Source: J.P. Morgan Healthcare Conference 01/12/2016 (MACK)

MESOBLAST LIMITED (MESO) MPC-06-ID for Chronic Pain

Event Date: 01/13/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase III - MSB-DR003 Market Group: Neurology

Lead Company: Mesoblast Limited (MESO) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 52% (Same As Avg.) Average Approval: 52%

Analysis: Mesoblast reported that the Phase III trial is currently enrolling about 330 patients. The primary efficacy endpoint is a composite of > 50% pain improvement (VAS) and > 15 point functional improvement (ODI) over 12 and 24 months. An interim analysis for efficacy will be performed during the fourth quarter of 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MESO, Slide 19)

MODERNA THERAPEUTICS, INC. MT-004 for Cancer

Event Date: 01/11/2016 Event Type: Partnership - Licensing Deal (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Moderna Therapeutics, Inc.

Partner Companies: AstraZeneca (AZN) Phase: Preclinical Change to Likelihood of Approval: 0%

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 82 Biomedtracker August 2016 / 83 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: AstraZeneca, along with its global biologics research and development arm, MedImmune, and Moderna Therapeutics announced a new collaboration to discover, co-develop and co-commercialize messenger RNA (mRNA) therapeutic candidates for the treatment of a range of cancers. The collaboration is in addition to the agreement announced by the companies in 2013 to develop mRNA Therapeutics for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology. The collaboration will combine MedImmune’s protein engineering and cancer biology expertise with Moderna’s mRNA platform.

Under the terms of the new agreement, AstraZeneca and Moderna have agreed to collaborate on two specific immuno-oncology programs, based on promising pre- clinical data, including pharmacology in tumor models. Moderna will fund and be responsible for discovery and preclinical development of product candidates, with the aim of delivering one Investigational New Drug (IND) application-ready molecule for each of the two programs. Moderna’s efforts will be led by its oncology-focused venture, Onkaido. AstraZeneca will be responsible for early clinical development, led by MedImmune, and Moderna and AstraZeneca will share the costs of late-stage clinical development. The two companies will co-commercialize resulting products in the US under a 50:50 profit sharing arrangement. AstraZeneca will lead ex-US commercialization efforts, with Moderna receiving tiered royalties up to substantial double digits on ex-US sales. Source: Press Release 01/11/2016 Press Release 01/11/2016 (AZN) J.P. Morgan Healthcare Conference 01/11/2016

mRNA 1440 for Infectious Disease

Event Date: 01/11/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase I - Europe Market Group: Infectious disease

Lead Company: Moderna Therapeutics, Inc. Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 83 Biomedtracker August 2016 / 84 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Average Approval: N/A

Analysis: Moderna Therapeutics announced that it initiated a Phase I study of mRNA 1440 in Europe in 2015. 12 healthy volunteers have already been dosed in this study. Source: J.P. Morgan Healthcare Conference 01/11/2016 (Press Release)

mRNA 1851 for Infectious Disease

Event Date: 01/11/2016 Event Type: Regulatory - IND Filing (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: Moderna Therapeutics, Inc. Partner Companies: N/A Phase: IND Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Moderna announced it has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for mRNA 1851, its second clinical program, with a Phase I study anticipated to begin in the first quarter of 2016. Source: J.P. Morgan Healthcare Conference 01/11/2016 (Press Release)

mRNA 1388 for Biodefense

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: Moderna Therapeutics, Inc. Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Moderna announced that they have extended an existing grant from the Defense

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 84 Biomedtracker August 2016 / 85 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Advanced Research Projects Agency (DARPA) to finance preclinical toxicology studies and the Phase I study of mRNA 1388 expected to begin in 2016. This extension builds on the original grant announced in 2013 to develop antibody-producing drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats. Source: J.P. Morgan Healthcare Conference 01/11/2016 (Press Release)

mRNA 1566 for Undisclosed

Event Date: 01/11/2016 Partnership - Amendment/Restructuring (Clinical Event Type: Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Moderna Therapeutics, Inc. Partner Companies: N/A Phase: Preclinical Change to Likelihood of 0% Approval: Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Moderna Therapeutics announced that Merck has licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates, as part of the ongoing collaboration between the companies to discover and develop vaccines and passive immunity treatments against viral diseases using modified messenger RNA (mRNA). The inclusion of this new program, which was not part of the original collaboration agreement, follows the rapid progress made in the first year of the collaboration. Moderna’s contribution is led by Valera, its infectious disease-focused venture. mRNA 1566 is slated to enter the clinic is 2016.

Under the terms of the amendment, Moderna has licensed a vaccine program against an undisclosed viral target, including mRNA 1566 and a set of related novel vaccine candidates to Merck. Moderna will receive an upfront payment for this program and will be eligible to receive development, regulatory and commercial milestone payments related to the new target, as well as tiered royalties on net sales of resulting products. Consistent with the existing collaboration, Merck will lead the development of the candidate and commercialization of any products resulting from the agreement, while Moderna will design and synthesize the mRNA vaccine

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 85 Biomedtracker August 2016 / 86 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

candidates directed against selected targets. As part of the initial three-year research agreement announced in January 2015, Merck made an upfront cash payment of $50 million and a $50 million equity investment to utilize granted licenses to commercialize five product candidates. Source: Press Release 01/11/2016 Press Release 01/11/2016 J.P. Morgan Healthcare Conference 01/11/2016

MOMENTA PHARMACEUTICALS (MNTA) M740 for Undisclosed

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Mylan Inc. (MYL)

Partner Companies: Momenta Pharmaceuticals (MNTA) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Momenta reported that the M740 program is undergoing process development. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNTA, Slide 10)

M615 for Undisclosed

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Mylan Inc. (MYL)

Partner Companies: Momenta Pharmaceuticals (MNTA) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 86 Biomedtracker August 2016 / 87 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Average Approval: N/A

Analysis: Momenta reported that the M615 program is undergoing process development. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNTA, Slide 10)

M706 for Undisclosed

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Mylan Inc. (MYL)

Partner Companies: Momenta Pharmaceuticals (MNTA) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Momenta reported that the M706 program is undergoing process development. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNTA, Slide 10)

M710 for Undisclosed

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Mylan Inc. (MYL)

Partner Companies: Momenta Pharmaceuticals (MNTA) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Momenta reported that the M710 program is undergoing process development. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNTA, Slide 10)

M730 for Undisclosed

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 87 Biomedtracker August 2016 / 88 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Not Specified

Lead Company: Mylan Inc. (MYL)

Partner Companies: Momenta Pharmaceuticals (MNTA) Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Momenta reported that the M730 program is undergoing process development. Source: J.P. Morgan Healthcare Conference 01/13/2016 (MNTA, Slide 10)

NEURODERM, LTD. (NDRM) ND0612H for Parkinson's Disease (PD)

Event Date: 01/14/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase I - Study 005 (EU PK) Market Group: Neurology

Lead Company: NeuroDerm, Ltd. (NDRM) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 22% (5% Above Avg.) Average Approval: 17%

Analysis: NeuroDerm announced that they have initiated a Phase I PK equivalence study of ND0612H versus Duodopa in Europe. They expect the results to read out in the second quarter of 2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (NDRM, Slide 32)

ONCOMED PHARMACEUTICALS, INC. (OMED) Demcizumab for Ovarian Cancer QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 88 Biomedtracker August 2016 / 89 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Event Date: 12/31/2015 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase Ib/II - SIERRA Market Group: Oncology

Lead Company: OncoMed Pharmaceuticals, Inc. (OMED) Celgene (CELG) Partner Companies: GlaxoSmithKline (GSK) Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10%

Analysis: OncoMed Pharmaceuticals announced that the Phase Ib/II SIERRA study of demcizumab for ovarian cancer completed the Phase Ib portion in the second half of 2015. Source: J.P. Morgan Healthcare Conference 01/14/2016 (OMED, Slide 30)

OPHTHOTECH CORP. (OPHT) Fovista for Wet Age-Related Macular Degeneration (Wet AMD) (Ophthalmology)

Event Date: 01/11/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: Phase III - OPH1004 (w/Avastin or Eylea) Market Group: Ophthalmology

Lead Company: Ophthotech Corp. (OPHT) Nektar (NKTR) Novartis (NVS) Partner Companies: Novo Nordisk (NVO) Astellas (4503:JP) Archemix Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 56% (5% Above Avg.) Average Approval: 51%

Analysis: Ophthotech reports that the Phase III OPH1004 trial of Fovista in combination with

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 89 Biomedtracker August 2016 / 90 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

either Eylea or Avastin continues to enroll patients on track. Source: J.P. Morgan Healthcare Conference 01/11/2016 (OPHT, Slide 44)

Zimura for Dry Age-Related Macular Degeneration (Dry AMD) (Ophthalmology)

Event Date: 01/11/2016 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase II/III - Geographic Atrophy Market Group: Ophthalmology

Lead Company: Ophthotech Corp. (OPHT)

Partner Companies: Archemix Phase: III Change to Likelihood of Approval: 27% Likelihood of Approval: 51% (Same As Avg.) Average Approval: 51%

Analysis: Ophthotech has initiated a Phase II/III geographic atrophy study for Zimura. The study is estimated to last from 18 to 24 months. The primary analysis will be the mean change in best corrected visual acuity and the main secondary analysis will be the mean rate of change in geographic atrophy. Source: J.P. Morgan Healthcare Conference 01/11/2016 (OPHT, Slide 48)

OPKO HEALTH (OPK) MOD-6030 for Obesity

Event Date: 01/12/2016 Event Type: Progress Update - Suspension (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: Opko Health (OPK) Partner Companies: N/A Phase: Suspended Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 90 Biomedtracker August 2016 / 91 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Analysis: Opko is moving forward with MOD-6031 and expects initiate a Phase I study in 1Q 2016. As such, we are suspending MOD-6030. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/12/2016 (OPK, Slide 22)

MOD-6030 for Diabetes Mellitus, Type II

Event Date: 01/12/2016 Event Type: Progress Update - Suspension (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Opko Health (OPK) Partner Companies: N/A Phase: Suspended Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Opko is moving forward with MOD-6031 and expects initiate a Phase I study in 1Q 2016. As such, we are suspending MOD-6030. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/12/2016 (OPK, Slide 22)

CTAP201 for Hyperparathyroidism (Secondary)

Event Date: 01/12/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Endocrine

Lead Company: Opko Health (OPK) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 24% (Same As Avg.) Average Approval: 24%

Analysis: Opko continues to list CTAP201 in Phase II development for mild-to-moderate

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 91 Biomedtracker August 2016 / 92 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

secondary hyperparathyroidism (SHPT) in CKD stage 5 patients. Source: J.P. Morgan Healthcare Conference 01/12/2016 (OPK, Slide 10)

OPTINOSE AS OPN-375 for Nasal Polyposis

Event Date: 11/30/2015 Event Type: Regulatory - Meeting with FDA (Clinical Analysis) Trial Name: N/A Market Group: Allergy

Lead Company: OptiNose AS Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 64% (2% Above Avg.) Average Approval: 62%

Analysis: OptiNose announced that a Pre-NDA meeting with the FDA occurred in November 2015 regarding OPN-375 for CRS with nasal polyps. In addition, they have completed groundwork for 2016 scale up of manufacturing (drug and device). Source: J.P. Morgan Healthcare Conference 01/11/2016 (OptiNose, Slide 6)

PRONAI THERAPEUTICS, INC. (DNAI) PNT2258 for Diffuse Large B-Cell Lymphoma (DLBCL) - NHL

Event Date: 01/14/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase II - 02 Market Group: Oncology

Lead Company: ProNAi Therapeutics, Inc. (DNAI)

Partner Companies: Marina (MRNA) Phase: II

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 92 Biomedtracker August 2016 / 93 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Change to Likelihood of Approval: 0% Likelihood of Approval: 10% (Same As Avg.) Average Approval: 10% Treatment Treatment Treatment Treatment Treatment Description PNT2258 PNT2258 PNT2258 PNT2258 CLL/SLL MCL FL DLBCL Number of Evaluable Patients 2 2 5 4 Complete Response N/A N/A 1 Patients 3 Patients Partial Response N/A N/A 1 Patients 1 Patients Stable Disease 1 Patients 1 Patients 3 Patients N/A Progressive Disease 1 Patients 1 Patients N/A N/A Analysis: ProNAi Therapeutics reported long-term results from its Phase II pilot trial of PNT2258 in relapsed/refractory NHL.

Results from this study were last seen in December 2014.

Design This trial enrolled 13 patients with relapsed/refractory lymphoma across three centers. PNT2258 was administered as a single agent IV as induction followed by dosing until disease progression.

Results Results are tabled above. One patient had significant tumor disruption and decrease in SUV uptake after six cycles of single-agent PNT2258. There was a complete metabolic response by a blinded read of the FDG PET-CT scan using the Lugano criteria. This patient maintained clinical benefit for 10 months.

Most Common Adverse Events There were no drug-related discontinuations from treatment. The majority of adverse events were grade 1/2 requiring, at most, supportive care measures. Hematologic and metabolic adverse events most often occurred in subjects with pre- disposing conditions (e.g. transfusion dependent anemia, thrombocytopenia). No tumor lysis events and no evidence of significant metabolic adverse events even in subjects with high-volume disease that achieved durable clinical responses to treatment. Source: J.P. Morgan Healthcare Conference 01/14/2016 (DNAI, Slide 13-17)

RADIUS HEALTH, INC. (RDUS) RAD1901 for Menopause (including Hormone Replacement Therapy [HRT])

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 93 Biomedtracker August 2016 / 94 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Event Date: 12/31/2015 Event Type: Trial Announcement - Initiation (Clinical Analysis)

Trial Name: Phase IIb - Vasomotor Symptoms Market Group: Endocrine

Lead Company: Radius Health, Inc. (RDUS) Partner Companies: N/A Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 24% (Same As Avg.) Average Approval: 24%

Analysis: Radius announced that the Phase IIb study of RAD1901 in vasomotor symptoms was initiated in December 2015. Source: J.P. Morgan Healthcare Conference 01/11/2016 (RDUS, Slide 4)

REGENERON PHARMACEUTICALS, INC. (REGN) Praluent for Dyslipidemia / Hypercholesterolemia

Event Date: 01/13/2016 Progress Update - Product Launch (Europe) (Clinical Event Type: Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Regeneron Pharmaceuticals, Inc. (REGN)

Partner Companies: Sanofi (SNY) Phase: Approved Change to Likelihood of 0% Approval: Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: Regeneron announced that Praluent has been made available in Germany, UK, and the Nordic countries. Source: J.P. Morgan Healthcare Conference 01/13/2016 (REGN, Slide 10)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 94 Biomedtracker August 2016 / 95 Pharma intelligence | JP Morgan Healthcare Conference: Review

Healthcare Conference: Conference Review

Fasinumab for Chronic Pain

Event Date: 01/13/2016 Event Type: Regulatory - Meeting with FDA (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: Regeneron Pharmaceuticals, Inc. (REGN) Sanofi (SNY) Partner Companies: Mitsubishi Tanabe (4508:JP) Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 45% (7% Below Avg.) Average Approval: 52%

Analysis: Regeneron announced that the partial hold on Fasinumab has been lifted. Based on discussions with the FDA, Phase III trials (>16 weeks) are expected to begin in the first half of 2016. Source: J.P. Morgan Healthcare Conference 01/13/2016 (REGN, Slide 24)

REVA MEDICAL INC. Fantom Bioresorbable Scaffold for Coronary Artery Disease

Event Date: 01/14/2016 Event Type: Trial Announcement - Trial Progressing (Clinical Analysis)

Trial Name: CE Mark Study - FANTOM II (EU) Market Group: Cardiovascular

Lead Company: REVA Medical Inc. (RVA:ASX) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: REVA Medical provided an enrollment update for the FANTOM II Study. Cohort A

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 95

Biomedtracker August 2016 / 96 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

(110 patients for CE Mark application data set) has currently enrolled 117 patients and Cohort B (additional cohort; planned 110 patients) has currently enrolled 70 patients. Source: J.P. Morgan Healthcare Conference 01/14/2016 (RVA:ASX; Slide 16)

RIGEL PHARMACEUTICALS, INC. (RIGL) IRAK Inhibitor Program for Acute Myelogenous Leukemia (AML)

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Rigel Pharmaceuticals, Inc. (RIGL) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Rigel announced that they are developing an IRAK inhibitor for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The Company plans to file an IND and progress the drug into clinical testing in 2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (RIGL, Slide 20)

ROCHE HOLDING AG (RHHBF) Atezolizumab for Non-Small Cell Lung Cancer (NSCLC)

Event Date: 01/12/2016 Event Type: Regulatory - Rolling NDA/BLA Initiated (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Roche Holding AG (RHHBF)

Partner Companies: Chugai (4519:JP) Phase: NDA/BLA Change to Likelihood of Approval: 47% Likelihood of Approval: 87% (5% Above Avg.)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 96 Biomedtracker August 2016 / 97 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Average Approval: 82%

Analysis: Roche announced that it has initiated a rolling NDA for the approval of Atezolizumab for the treatment of NSCLC in the US. Roche is seeking for approval based on the Phase III FIR, BIRCH and POPLAR studies of Atezolizumab monotherapy. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Roche, Slide 21)

Atezolizumab for Bladder Cancer

Event Date: 01/12/2016 Event Type: Regulatory - Rolling NDA/BLA Initiated (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Roche Holding AG (RHHBF)

Partner Companies: Chugai (4519:JP) Phase: NDA/BLA Change to Likelihood of Approval: 47% Likelihood of Approval: 92% (10% Above Avg.) Average Approval: 82%

Analysis: Roche announced that it has initiated a rolling NDA for the approval of Atezolizumab for the treatment of bladder cancer in the US. Roche is seeking for approval based on the Phase III IMvigor210 study of Atezolizumab monotherapy in patients who are treatment-naive and ineligible for platinum-containing therapy. Source: J.P. Morgan Healthcare Conference 01/12/2016 (Roche, Slide 21)

SAGE THERAPEUTICS (SAGE) SAGE-105 for Neurology - Other

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: SAGE Therapeutics (SAGE) Partner Companies: N/A Phase: Preclinical

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 97 Biomedtracker August 2016 / 98 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: SAGE-105 is currently listed in preclinical development in SAGE Therapeutic's pipeline for a GABA related disorder. Source: J.P. Morgan Healthcare Conference 01/11/2016 (SAGE, Slide 7)

SAGE-324 for Neurology - Other

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: SAGE Therapeutics (SAGE) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: SAGE-324 is currently listed in preclinical development in SAGE Therapeutic's pipeline for a GABA related disorder. Source: J.P. Morgan Healthcare Conference 01/11/2016 (SAGE, Slide 7)

SANGAMO BIOSCIENCES, INC. (SGMO) SB-318 for Mucopolysaccharidosis I (MPS I; Hurler Syndrome)

Event Date: 12/31/2015 Event Type: Regulatory - IND Filing (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: Sangamo Biosciences, Inc. (SGMO) Partner Companies: N/A Phase: IND Change to Likelihood of Approval: 0%

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 98 Biomedtracker August 2016 / 99 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Sangamo filed an IND application for SB-318 for the treatment of Hurler Syndrome (MPS I) at the end of 2015. Source: J.P. Morgan Healthcare Conference 01/12/2016 (SGMO, Slide 18)

SB-LSD4 for Fabry's Disease

Event Date: 01/12/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Metabolic

Lead Company: Sangamo Biosciences, Inc. (SGMO) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Sangamo currently lists SB-LSD4 for the treatment of Fabry disease in preclinical development in its pipeline. Sangamo plans to file an IND for SB-LSD4 in the second half of 2016. Source: J.P. Morgan Healthcare Conference 01/12/2016 (SGMO, Slide 18)

SEATTLE GENETICS, INC. (SGEN) SGN-LIV1A for Breast Cancer

Event Date: 01/11/2016 Trial Announcement - Protocol Amendment (Clinical Event Type: Analysis)

Trial Name: Phase I - Safety (LIV-1 Positive) Market Group: Oncology

Lead Company: Seattle Genetics, Inc. (SGEN) Partner Companies: N/A Phase: I

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 99 Biomedtracker August 2016 / 100 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Change to Likelihood of 0% Approval: Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6%

Analysis: Seattle Genetics announced that the Company recently expanded the Phase I study of SGN-LIV1A to evaluate the HER2+ cohort of SGN-LIV1A in combination with Herceptin. Source: J.P. Morgan Healthcare Conference 01/11/2016 (SGEN, Slide 20)

Adcetris for Diffuse Large B-Cell Lymphoma (DLBCL) - NHL

Event Date: 01/11/2016 Event Type: Trial Data - Top-Line Results (Clinical Analysis)

Trial Name: Phase II - Rituxan/Bendamustine +/- Adcetris Market Group: Oncology

Lead Company: Seattle Genetics, Inc. (SGEN)

Partner Companies: Takeda (4502:JP) Phase: II Change to Likelihood of Approval: 0% Likelihood of Approval: 11% (1% Above Avg.) Average Approval: 10% Treatment Treatment Description Adcentris Number of Evaluable Patients 44 ORR 44.000 % CR Rate 19.000 % Median PFS 4.000 Months Analysis: Seattle Genetics announced interim results from the Phase II study of Rituxan/Bendamustine +/- Adcentris at the J.P. Morgan Healthcare Conference.

Context The Phase II study is ongoing.

Design The Phase II study is an ongoing randomized study of Rituxan and Bendamustine +/- Adcentris.

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 100 Biomedtracker August 2016 / 101 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Results The following results were reported (n=48):  ORR: 40%  CR rate: 19%  Median PFS: 4 months Source: J.P. Morgan Healthcare Conference 01/11/2016 (SGEN, Slide 12)

SEA-CD40 for Solid Tumors

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Seattle Genetics, Inc. (SGEN) Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 6% (Same As Avg.) Average Approval: 6%

Analysis: Seattle Genetics currently lists SEA-CD40, for the treatment of relapsed tumors, in Phase I clinical development. Source: J.P. Morgan Healthcare Conference 01/11/2016 (SGEN, Slide 5)

SPARK THERAPEUTICS, LLC (ONCE) SPK-RPE65 for Leber's Congenital Amaurosis (Ophthalmology)

Event Date: 01/11/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase III - 301 - LCA2 Market Group: Ophthalmology

Lead Company: Spark Therapeutics, LLC (ONCE) Partner Companies: N/A Phase: III Change to Likelihood of Approval: 0% Likelihood of Approval: 64% (13% Above Avg.) Average Approval: 51%

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 101 Biomedtracker August 2016 / 102 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Placebo Treatment Difference Between Treatment and Placebo Treatment Description Control Group SPK-RPE65 SPK-RPE65 vs. Controsl (Subjects that Developed (Subjects that Developed (Intervention Subjects) Cataracts that are Cataracts that are Removed) Removed) Mean Improvement of Letters N/A N/A N/A Averaged Across Both Eyes (P= 0.0470) (Lange scale) Average Improvement of 1.600 letters 10.600 letters N/A Letters Across Both Eyes (P= 0.0070) Analysis: Spark Therapeutics announced updated results from the Phase III 301 study of SPK- RPE65 at the 2016 J.P. Morgan Healthcare Conference.

Results from this study were previously seen in November 2015.

Results The following results were reported:  Using the Lange scale, intervention subjects demonstrated a 9 letter average improvement vs. 1.6 for controls (p=0.047).  In a subgroup analysis recommended by the Company's DSMB where subjects that developed cataracts (n=3) are removed, average improvement was 10.6 letters vs. 1.6 for controls (p=0.007).  In accordance with the SAP, VA did not acheive statistical significance (0.17). Source: J.P. Morgan Healthcare Conference 01/11/2016 Company 8-K 01/11/2016 (ONCE, Slide 12)

SPK-FVIII for Hemophilia A

Event Date: 01/11/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Hematology

Lead Company: Spark Therapeutics, LLC (ONCE) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 102 Biomedtracker August 2016 / 103 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Analysis: Spark Therapeutics currently lists SPK-FVIII in preclinical development for the treatment of hemophilia A. The Company expects to file an IND for SPK-FVIII in the second half of 2016 with initial efficacy data in the first half of 2017. Source: J.P. Morgan Healthcare Conference 01/11/2016 Company 8-K 01/11/2016 (ONCE, Slide 19)

ST. JUDE MEDICAL, INC. (STJ) Portico TAVI System for Cardiac Valve Surgery

Event Date: 01/13/2016 Progress Update - Product Launch (Europe) (Clinical Event Type: Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: St. Jude Medical, Inc. (STJ) Partner Companies: N/A Phase: IDE Change to Likelihood of 0% Approval: Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: St. Jude announced that it has now launched all 4 sizes of Portico (23, 25, 27, and 29 mm) in Europe. Source: J.P. Morgan Healthcare Conference 01/13/2016 (STJ)

T2 BIOSYSTEMS, INC. (TTOO) T2HemoStat for Hemostasis

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Hematology

Lead Company: T2 Biosystems, Inc. (TTOO) Partner Companies: N/A

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 103 Biomedtracker August 2016 / 104 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Phase: Development Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: T2 Biosystems announced that they are developing T2HemoStat for hemostasis. The panel is designed to rapidly assess the risk of bleeding and clotting at the point of care from a finger stick of blood. The company plans to start an FDA clinical study in mid-2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (TTOO)

T2Bacteria for Sepsis and Septic Shock

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Infectious disease

Lead Company: T2 Biosystems, Inc. (TTOO) Partner Companies: N/A Phase: Development Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: T2 Biosystems is developing T2Bacteria, a diagnostic test designed to detect the bacterial pathogens associated with sepsis that often are not effectively treated by first-line antibiotics. Enrollment in a pivotal trial started in Q4 2015 and the company expects FDA clearance by early 2017. Source: J.P. Morgan Healthcare Conference 01/14/2016 (TTOO, Slide 21)

TAIWAN LIPOSOME COMPANY, LTD. TLC178 for Cutaneous T-Cell Lymphoma (CTCL) - NHL

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 104 Biomedtracker August 2016 / 105 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Taiwan Liposome currently lists TLC178 in preclinical development for CTCL. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slides 8, 11) TLC198 for Anesthesia

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Preclinical Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Taiwan Liposome currently lists TLC198 in preclinical development as a local anesthetic. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slide 11)

Lipotecan for Hepatocellular (Liver) Cancer (HCC) (including secondary metastases)

Event Date: 01/13/2016 Event Type: Progress Update - Development Review (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: -6% Likelihood of Approval: 0% (Same As Avg.)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 105 Biomedtracker August 2016 / 106 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Average Approval: N/A

Analysis: Taiwan Liposome outlined its development plan for TLC388. The Company intends to pursue TLC388 for rectal cancer, HCC and other indications in Taiwan and China.

Given that US development was not included, we are moving the phase to Development Outside US. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slide 22)

Lipotecan for Renal Cell Cancer (RCC)

Event Date: 01/13/2016 Event Type: Progress Update - Suspension (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Suspended Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Taiwan Liposome currently lists TLC388 in Phase II development for hepatocellular carcinoma and rectal cancer in Taiwan and China. Given that renal cancer is not in the pipeline, we are suspending this drug in this indication. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slide 11, 22)

Lipotecan for Prostate Cancer

Event Date: 01/13/2016 Event Type: Progress Update - Suspension (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Suspended

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 106 Biomedtracker August 2016 / 107 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Taiwan Liposome currently lists TLC388 in Phase II development for hepatocellular carcinoma and rectal cancer in Taiwan and China. Given that prostate cancer is not in the pipeline, we are suspending this drug in this indication. Source: Sagient Analysis J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slide 11, 22)

Lipotecan for Colorectal Cancer (CRC)

Event Date: 01/13/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Oncology

Lead Company: Taiwan Liposome Company, Ltd. (4152:TWO) Partner Companies: N/A Phase: Development Outside U.S. Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Taiwan Liposome outlined its development plan for TLC388. The Company intends to pursue TLC388 for rectal cancer, HCC and other indications in Taiwan and China.

TLC388 is currently listed in Phase II development for rectal cancer. Source: J.P. Morgan Healthcare Conference 01/13/2016 (Taiwan Liposome, Slide 11, 22)

TG THERAPEUTICS, INC. (TGTX) Ublituximab for Multiple Sclerosis (MS)

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Neurology

Lead Company: TG Therapeutics, Inc. (TGTX)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 107 Biomedtracker August 2016 / 108 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

LFB Group Partner Companies: Ildong Pharmaceutical Phase: II Change to Likelihood of Approval: N/A Likelihood of Approval: 17% (Same As Avg.) Average Approval: 17%

Analysis: TG Therapeutics announced that anti-CD20 monoclonal antibodies have demonstrated activity in multiple sclerosis (MS). As such, the Company believes that TG-1101 has the potential for a best-in-class product profile in MS. TG Therapeutics expects to initiate a Phase I/II study of TG-1101 in MS in the first half of 2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (TGTX, Slide 23)

TRUE NORTH THERAPEUTICS, INC. TNT009 for Complement Deficiencies / Abnormalities

Event Date: 01/12/2016 Event Type: Trial Data - Updated Results (Clinical Analysis)

Trial Name: Phase Ia/Ib - TNT009-01 Market Group: Autoimmune/ immunology

Lead Company: True North Therapeutics, Inc. Partner Companies: N/A Phase: I Change to Likelihood of Approval: 0% Likelihood of Approval: 14% (Same As Avg.) Average Approval: 14%

Analysis: True North Therapeutics announced more results at the 34th annual JP Morgan Healthcare Conference from their Phase Ia/Ib study of TNT009.

Initial results were released earlier in January 2016.

Context Top-line Phase Ib data are expected in mid-2016.

Design

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 108 Biomedtracker August 2016 / 109 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

The Phase Ia trial in NHVs is completed. The single ascending dose (0.3, 1, 3, 10, 30, 60, and 100 mg/kg IV) and multiple ascending dose (30 and 60 mg/kg IV weekly for four weeks) portions have been completed for NHVs.

Results POM demonstrated via blood-based PD assay. Additional results can be seen in the presentation slides.

Most Common Adverse Events No relevant AEs reported to date. Source: J.P. Morgan Healthcare Conference 01/12/2016 (True North, Slides 7-10)

UNITED THERAPEUTICS CORPORATION (UTHR) Remodulin (injection) for Pulmonary Arterial Hypertension (PAH)

Event Date: 12/31/2015 Event Type: Regulatory - sNDA/sBLA Filing (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: United Therapeutics Corporation (UTHR) GlaxoSmithKline (GSK) Medtronic (MDT) Mochida Partner Companies: Pfizer (PFE) Lee's Pharmaceutical (950:HK) Ascendis (ASND) DEKA Phase: Approved Change to Likelihood of Approval: 0% Likelihood of Approval: 100% (Same As Avg.) Average Approval: 100%

Analysis: United Therapeutics (UTHR) noted that in December 2015, Medtronic had answered questions from the FDA regarding their Synchromed II implantable pump for Remodulin, and has a PMA expected in March. As a result, UTHR filed an SNDA for Remodulin in December, with a 10-month time frame, though UTHR is working to accelerate that.

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 109 Biomedtracker August 2016 / 110 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Source: J.P. Morgan Healthcare Conference 01/11/2016 (UTHR)

Synchromed for Pulmonary Arterial Hypertension (PAH)

Event Date: 01/11/2016 Event Type: Regulatory - Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Cardiovascular

Lead Company: Medtronic, plc (MDT)

Partner Companies: Alnylam (ALNY) Phase: PMA Change to Likelihood of Approval: 0% Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: United Therapeutics, developer of Remodulin, announced at the 2016 J.P. Morgan Healthcare Conference that Medtronic responded to FDA questions regarding the Synchromed pump in December and now expects a PMA approval in March. Source: J.P. Morgan Healthcare Conference 01/11/2016 (UTHR)

VERACYTE, INC. (VCYT) Idiopathic Pulmonary Fibrosis Test for Idiopathic Pulmonary Fibrosis (IPF)

Event Date: 01/14/2016 Event Type: Progress Update (Clinical Analysis) Trial Name: N/A Market Group: Respiratory

Lead Company: Veracyte, Inc. (VCYT) Partner Companies: N/A Phase: Laboratory Developed Test (LDT) Change to Likelihood of Approval: N/A Likelihood of Approval: 0% (Same As Avg.) Average Approval: N/A

Analysis: Veracyte announced that it is leveraging its technology used in the Percepta

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 110 Biomedtracker August 2016 / 111 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review

Bronchial Genomic Classifier to develop a diagnostic solution for patients who are at risk for idiopathic pulmonary fibrosis that doesn't require diagnostic surgery. The company plans to launch the product by the end of 2016. Source: J.P. Morgan Healthcare Conference 01/14/2016 (VCYT; Slide 22)

QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 111 Biomedtracker August 2016 / 112 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Abbvie announced that mid-stage results from their Phase II Trial Data - Top-Line 01/13/2016-12/31/2016 AbbVie Inc. ABBV ABT-122 Psoriatic Arthritis (PA) II Phase II - Top-Line Results trial of ABT-122 for the treatment of psoriatic arthritis (PsA) 118306 Results will be available in 2016.

Abbvie expects data from the expansion arm of the second- line GBM study of ABT-414 to be available in 2016. If the Brain Cancer (malignant glioma; Trial Data - Top-Line 01/13/2016-06/30/2016 AbbVie Inc. ABBV ABT-414 II Phase II - Top-Line Results data is positive the Company will then target a regulatory 118300 AA and GBM) Results submission in the second half of 2016. As such we await top- line results in the first half of 2016.

Abbvie expects data from the expansion arm of the second- Brain Cancer (malignant glioma; Regulatory - NDA/BLA line GBM study of ABT-414 to be available in 2016. If the 07/01/2016-12/31/2016 AbbVie Inc. ABBV ABT-414 II NDA Filing - 2nd Line GBM 118301 AA and GBM) Filing data is positive the Company will then target a regulatory submission in the second half of 2016.

Abbvie announced that beginning in the second half of 2016 Trial Data - Top-Line 07/01/2016-12/31/2016 AbbVie Inc. ABBV ABT-493 Hepatitis C (HCV) (Antiviral) III Phase III - Top-Line Results data from the Phase III trials for their next-gen HCV portfolio 118308 Results will become available. Abbvie announced that beginning in the second half of 2016 Trial Data - Top-Line 07/01/2016-12/31/2016 AbbVie Inc. ABBV ABT-530 Hepatitis C (HCV) (Antiviral) III Phase III - Top-Line Results data from the Phase III trials for their next-gen HCV portfolio 118310 Results will become available. Diffuse Large B-Cell Lymphoma Trial Data - Top-Line Abbvie expects data from the Phase II study of Venetoclax in 01/13/2016-12/31/2016 AbbVie Inc. ABBV Venetoclax I/II Phase II - Top-Line Results 118298 (DLBCL) - NHL Results DLBCL to be available in 2016. Indolent Non-Hodgkin's Trial Data - Top-Line Abbvie expects data from the Phase II study of Venetoclax in 01/13/2016-12/31/2016 AbbVie Inc. ABBV Venetoclax II Phase II - Top-Line Results 118297 Lymphoma - NHL Results iNHL to be available in 2016. Acadia announced that the Company plans on initiating a Acadia Pharmaceuticals, Trial Announcement - Phase II Agitation - Study to Now-06/30/2016 ACAD Nuplazid Alzheimer's Disease (AD) II Phase II Alzheimer's Disease agitation study for Nuplazid in 118210 Inc. Initiation Start the first half of 2016. Acadia Pharmaceuticals expects to report results from its Acadia Pharmaceuticals, Trial Data - Top-Line Phase II 019 Study - Topline 07/01/2016-12/31/2016 ACAD Nuplazid Alzheimer's Disease (AD) II Phase II study of Nuplazid in Alzheimer's Disease in the 118211 Inc. Results Results second half of 2016.

Trial Data - Top-Line Phase I QD Formulation - Top- Acorda expects to have data from its Phase I QD Formulation Now-03/31/2016 Acorda Therapeutics, Inc. ACOR Ampyra Ischemic Stroke III 117993 Results Line Results studies of dalfampridine in the first quarter of 2016.

Trial Data - Top-Line Acorda expects to present data from its Phase I study of CVT- Now-03/31/2016 Acorda Therapeutics, Inc. ACOR CVT-427 Migraine and Other Headaches I Phase I - Top-Line Results 117995 Results 427 at a medical meeting in the first quarter of 2016.

Trial Announcement - Phase I MS-1033 - Trial Acorda expects to complete the Phase I study of rHIgM22 in 01/01/2017-06/30/2017 Acorda Therapeutics, Inc. ACOR rHIgM22 Multiple Sclerosis (MS) I 117996 Trial Completion Completion patients with acute MS relapses in the first half of 2017.

Actelion Pharmaceuticals Trial Data - Top-Line Actelion expects to have Phase Ib results for Lucerastat for Now-12/31/2016 ALIOF Lucerastat Fabry's Disease I Phase Ib - Top-Line Results 118166 Ltd. Results the treatment of Fabry disease in 2016.

Actelion Pharmaceuticals Pulmonary Arterial Trial Data - Top-Line Phase II MERIT - Top-Line Actelion expects to have results from its Phase II MERIT Now-12/31/2016 ALIOF Opsumit Approved 118164 Ltd. Hypertension (PAH) Results Results study of Opsumit in 2016.

Actelion Pharmaceuticals Hemorrhagic stroke / Trial Data - Top-Line Phase II REVERSE - Top-Line Actelion expects to have Phase II results for Clazosentan in Now-12/31/2016 ALIOF Pivlaz II 118165 Ltd. Intracerebral Hemorrhage (ICH) Results Results 2016.

Actelion Pharmaceuticals Progress Update - Actelion plans to provide an update on its Phase III multiple Now-12/31/2016 ALIOF Ponesimod Multiple Sclerosis (MS) III Development Update 118167 Ltd. Development Review sclerosis program of Ponesimod in 2016. GlaxoSmithKline announced that they expect to initiate a Non-Small Cell Lung Cancer Trial Announcement - clinical study of NY-ESO-1 for the treatment of non-small cell 01/12/2016-12/31/2016 Adaptimmune Ltd. ADAP NY-ESO-1(C259) I/II Trial to Start 118240 (NSCLC) Initiation lung cancer (NSCLC) in 2016 or 2017. We await an update through 2016. GlaxoSmithKline announced that they expect to initiate a Trial Announcement - clinical proof of concept (POC) study of NY-ESO-1 for the 01/12/2016-12/31/2016 Adaptimmune Ltd. ADAP NY-ESO-1(C259) Sarcoma I/II POC Study to Start 118237 Initiation treatment of sarcoma in 2016 or 2017. We await an update through 2016. Aduro announced that the Company intends to finalize its Progress Update - 07/01/2016-12/31/2016 Aduro Biotech ADRO CRS-207 Pancreatic Cancer IIb Phase III Plan Finalized Phase III trial design for CRS-207 in pancreatic cancer in the 118490 Development Review second half of 2016. Aegerion Dyslipidemia / Trial Announcement - PCSK9 Combination Study to Aegerion expects to initiate a Juxtapid and PCSK9i 04/01/2016-06/30/2016 AEGR Juxtapid Approved 118464 Pharmaceuticals, Inc. Hypercholesterolemia Initiation Start combination study in the second quarter of 2016. Aegerion is pursuing the pro drug development of Juxtapid Aegerion Dyslipidemia / as a second generation form to reduce GI side-effects. 10/01/2016-12/31/2016 AEGR Juxtapid Approved Trial Data - Other Preclinical Results 118467 Pharmaceuticals, Inc. Hypercholesterolemia Pharmacokinetic data in animals are anticipated in the fourth quarter of 2016.

Aegerion reported that a Japanese filing for marketing approval of Juxtapid for the treatment of HoFH was Aegerion Dyslipidemia / Regulatory - Approval submitted on January 8 2016. Based on an internal analysis 10/01/2016-04/30/2017 AEGR Juxtapid Approved Approval Decision (Japan) 118451 Pharmaceuticals, Inc. Hypercholesterolemia Decision (Japan) of the Japanese approval procedure we estimate an approval decision for this drug for this indication will be granted in approximately 9-15 months.

Aegerion reported that it plans to meet with US regulatory Aegerion Regulatory - Meeting with authorities to discuss expanding the label for Myalept to 01/14/2016-06/30/2016 AEGR Myalept Lipodystrophy Approved Meeting w/FDA 118469 Pharmaceuticals, Inc. FDA include the treatment of severe lipodystrophy. We expect an update in the first half of 2016.

Alder Biopharmaceuticals, Alder expects to submit an IND filing for ALD1613 for the Now-12/31/2016 ALDR ALD1613 Cushing's Syndrome Preclinical Regulatory - IND Filing IND Filing 118203 Inc. treatment of Cushing's syndrome in 2016.

Alder Biopharmaceuticals, Trial Data - Updated Phase IIb Chronic Migraines - Alder expects 24-week data for the Phase IIb study of 04/01/2016-09/30/2016 ALDR ALD403 Migraine and Other Headaches III 118192 Inc. Results Updated Results ALD403 for chronic migraine in mid-2016.

Alder seeks to identify a strategic partner for its Alder Biopharmaceuticals, 01/12/2016-12/31/2016 ALDR Clazakizumab Psoriatic Arthritis (PA) IIb Partnership - New Partnership - New clazakizumab program. We await and update through the 118202 Inc. end of 2016.

Alexion Pharmaceuticals, Paroxysmal Nocturnal Regulatory - NDA/BLA Alexion is targeting approval for ALXN1210 for the treatment 01/01/2017-12/31/2017 ALXN ALXN1210 II NDA Filing 118191 Inc. Hemoglobinuria (PNH) Filing of PNH in 2018. As such we expect a NDA filing in 2017.

ALXN 1540 one of the mRNA rare disease programs from the Alexion Pharmaceuticals, Trial Announcement - Company’s collaboration with Moderna is expected to enter Now-12/31/2016 ALXN ALXN1540 Crigler-Najjar syndrome Preclinical Clinical Trials to Start 118208 Inc. Initiation the clinic in 2016 in patients with Crigler-Najjar Syndrome (CN-1). Alkermes expects to initiate a Phase I study of ALKS 7119 Trial Data - Top-Line with an initial focus on agitation in Alzheimer's disease (AD) 01/12/2016-12/31/2016 Alkermes plc ALKS ALKS 7119 Alzheimer's Disease (AD) Preclinical Phase I - Top-Line Results 118256 Results in the first quarter of 2016. Data from this trial is expected in 2016. Alkermes announced that they plan to advance RDB 1450 for Trial Data - Top-Line 01/12/2016-12/31/2016 Alkermes plc ALKS RDB 1450 Cancer IND Phase I - Top-Line Results the treatment of cancer into clinical trials and have early 118252 Results data in 2016. Alnylam plans to initiate a Phase III trial of ALN-AS1 an Alnylam Pharmaceuticals, Trial Announcement - investigational RNAi therapeutic targeting aminolevulinic 01/01/2017-12/31/2017 ALNY ALN-AS1 Porphyria I Phase III - Trial to Start 117963 Inc. Initiation acid synthase-1 (ALAS1) for the treatment of acute hepatic porphyrias in 2017. Alnylam plans to Start a Phase I trial for ALN-HBV an Alnylam Pharmaceuticals, Hepatitis B (HBV) Treatment Trial Announcement - investigational RNAi therapeutic targeting the hepatitis B 04/01/2016-09/30/2016 ALNY ALN-HBV Preclinical Phase I - Trial to Start 117975 Inc. (Antiviral) Initiation viral (HBV) genome for the treatment of chronic HBV infection in mid-2016.

Biomedtracker August 2016 / 113 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Transthyretin Amyloid (ATTR) Alnylam plans to start a Phase I study of ALN-TTRsc02 an Alnylam Pharmaceuticals, Trial Announcement - 04/01/2016-09/30/2016 ALNY ALN-TTRsc02 Cardiomyopathy (Familial Preclinical Phase I - Trial to Start investigational RNAi therapeutic targeting TTR for the 117954 Inc. Initiation Amyloidotic Cardiomyopathy) treatment of all forms of ATTR amyloidosis in mid-2016.

Alnylam plans to present additional data from the ongoing Alnylam Pharmaceuticals, Hemophilia A and B - General Trial Data - Updated Phase I trial of fitusiran an investigational RNAi therapeutic 04/01/2016-09/30/2016 ALNY Fitusiran I Phase I - 001 - Updated Results 117960 Inc. Clotting Products Results targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD) in mid-2016.

Transthyretin-related Alnylam announced that the Company will present 24- Alnylam Pharmaceuticals, Trial Data - Updated 04/01/2016-09/30/2016 ALNY Patisiran Hereditary Amyloidosis (Familial III Phase II - OLE - 24-Month Data month data from the Phase II open-label extension (OLE) 117950 Inc. Results Amyloid Polyneuropathy) study with patisiran in mid-2016.

Alnylam expects to be in a position to report data from the Phase III APOLLO trial of patisiran for the treatment of TTR- Transthyretin-related Alnylam Pharmaceuticals, Regulatory - MAA mediated amyloidosis (ATTR amyloidosis) in patients with 09/01/2017-12/31/2017 ALNY Patisiran Hereditary Amyloidosis (Familial III MAA Filing 117946 Inc. Submission (Europe) familial amyloidotic polyneuropathy (FAP) in 2017 and to Amyloid Polyneuropathy) submit a New Drug Application (NDA) and Marketing Authorisation Application (MAA) for patisiran in late 2017.

Alnylam plans to continue enrollment in the Phase III Transthyretin Amyloid (ATTR) Alnylam Pharmaceuticals, Trial Data - Top-Line Phase III - ENDEAVOR - Top- ENDEAVOR study. Based on ongoing enrollment the 01/01/2018-12/31/2018 ALNY Revusiran Cardiomyopathy (Familial III 117951 Inc. Results Line Results Company is guiding that it expects to report data from the Amyloidotic Cardiomyopathy) ENDEAVOUR trial in 2018.

Transthyretin Amyloid (ATTR) Alnylam Pharmaceuticals, Trial Data - Updated Alnylam plans to present 12-month data from the Phase II 04/01/2016-09/30/2016 ALNY Revusiran Cardiomyopathy (Familial III Phase II - OLE - 12-Month Data 117952 Inc. Results OLE study with revusiran in mid-2016. Amyloidotic Cardiomyopathy)

AMAG announced that Velo plans to complete preclinical AMAG Pharmaceuticals, Trial Announcement - Severe Pre-Eclampsia - Clinical 01/13/2016-12/31/2016 AMAG Digoxin Immune Fab Eclampsia/Pre-Eclampsia II/III work and initiate a clinical program of Digoxin Immune Fab 118324 Inc. Initiation Program to Start for the treatment of severe pre-eclampsia in 2016.

AMAG announced that they expect to launch the single-dose vial of Makena for the treatment of preterm labor within 3 AMAG Pharmaceuticals, Progress Update - Product Launch (US) - Single- 03/23/2016-04/30/2016 AMAG Makena Preterm Labor Approved or 4 weeks of FDA approval. As we expect an approval 118333 Inc. Product Launch (U.S.) Dose Vial decision by March 23 2016 we await an update between March 23 2016 and the end of April 2016.

AMAG Pharmaceuticals, Trial Announcement - AMAG announced that they plan to initiate a Makena PK 01/13/2016-12/31/2016 AMAG Makena Preterm Labor Approved PK Study - Trial to Start 118323 Inc. Initiation study for the treatment of preterm labor in 2016.

AMAG Pharmaceuticals, Regulatory - sNDA/sBLA AMAG announced that they plan to file a sNDA for a Makena 01/01/2017-03/31/2017 AMAG Makena Preterm Labor Approved sNDA Filing - Auto-Injector 118321 Inc. Filing subcutaneous auto-injector in the first quarter of 2017.

Trial Data - Updated Phase III ENDEAVOR - Updated Amgen expects results for the Kyprolis ENDEAVOR study in 09/01/2016-12/31/2016 Amgen, Inc. AMGN Kyprolis Multiple Myeloma (MM) Approved 118169 Results Results later 2016.

Amicus expects to publish results from the Phase IIb study of Trial Data - Updated Phase IIb SD-003 - Updated 01/12/2016-04/30/2016 Amicus Therapeutics, Inc. FOLD Zorblisa Epidermolysis Bullosa III Zorblisa for EB at dermatology conferences in the coming 118255 Results Results months. We expect an update through early 2016.

Trial Data - Updated Phase III STEADY (Study 003) - Antares expects 52-week safety data from the Phase III QST- Now-03/31/2016 Antares Pharma, Inc. ATRS Vibex QST Hypogonadism III 118496 Results Updated Safety Data 13-003 study of Vibex QST during the first quarter of 2016.

Braeburn announced that the Company anticipates to launch Progress Update - 01/13/2016-03/31/2016 Apple Tree Partners Probuphine Drug Addiction NDA Product Launch - U.S. Probuphine 6 months buprenorphine implant in the first 118381 Product Launch quarter of 2016. Arena reported that they expect top-line APD371 Phase I Arena Pharmaceuticals, Trial Data - Top-Line Phase Ib MAD - Top-Line Now-03/31/2016 ARNA APD371 Pain Indications I MAD data in healthy volunteers for pain management in the 118508 Inc. Results Results first quarter of 2016.

Trial Announcement - ARMO plans to initiate registration enabling Phase II/III 01/12/2016-12/31/2016 ARMO Biosciences AM0010 Solid Tumors I Phase II/II - Study to Start 118258 Initiation studies for AM0010 in multiple solid tumors in 2016.

Array BioPharma expects to initiate a Phase III global Trial Announcement - 01/13/2016-12/31/2016 Array BioPharma, Inc. ARRY Binimetinib Colorectal Cancer (CRC) I Registration Study to Start registration trial for binimetinib in BRAF-mutant metastatic 118425 Initiation colorectal cancer in 2016. A three arms prospective randomized Phase II study to evaluate the best sequential approach with combo Trial Announcement - Phase II SECOMBIT Trial to immunotherapy (Ipilimumab/Nivolumab) and combo target 02/01/2016-02/29/2016 Array BioPharma, Inc. ARRY Binimetinib Melanoma III 118427 Initiation Start therapy (LGX818/MEK162) in patients with metastatic melanoma and BRAF mutation is expected to start in February 2016. Trial Announcement - Array BioPharma announced that the Phase III MILO study of Phase III Study - Patient 01/13/2016-12/31/2016 Array BioPharma, Inc. ARRY Binimetinib Ovarian Cancer III Patient Enrollment binimetinib on ovarian cancer is expected to complete 118419 Enrollment Complete Completed enrollment in 2016.

Medivation announced that they anticipate the publication Trial Data - Published Phase II STRIVE - Published 01/11/2016-03/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Prostate Cancer Approved of data from the Phase II STRIVE trial of MDV3100 for 118041 Results Results prostate cancer in the first quarter of 2016.

Medivation announced that they anticipate the publication Trial Data - Published Phase II TERRAIN - Published 01/11/2016-03/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Prostate Cancer Approved of data from the Phase II TERRAIN trial of MDV3100 for 118044 Results Results prostate cancer in the first quarter of 2016.

Medivation announced that they anticipate initiating a Phase Trial Announcement - Phase III mHSPC - Study to 01/11/2016-06/30/2016 Astellas Pharma, Inc. 4503:JP Xtandi Prostate Cancer Approved III study of Xtandi for metastatic hormone sensitive prostate 118047 Initiation Start cancer (mHSPC) in the first half of 2016.

Medivation announced that top-line results from the Phase Trial Data - Top-Line Phase IV - PLATO Top-Line 07/01/2016-12/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Prostate Cancer Approved IV PLATO study may be released as early as the second half 118082 Results Results of 2016.

An Investigational New Drug (IND) application for CMVpoly- Atara Biotherapeutics, Brain Cancer (malignant glioma; 01/01/2017-03/31/2017 ATRA CMVpoly-CTL Preclinical Regulatory - IND Filing IND Filing CTL for the initial condition of glioblastoma multiforme 117978 Inc. AA and GBM) (GBM) is expected in the first quarter of 2017.

Atara Biotherapeutics, Trial Announcement - Pivotal Trial (EBV-PTLD after Atara expects to initiate a pivotal trial of EBV-CTL in 10/01/2016-12/31/2016 ATRA EBV-CTL Hematologic Cancer II 117964 Inc. Initiation HCT) to Start refractory EBV-PTLD patients after HCT by the end of 2016.

Atara Biotherapeutics, Trial Announcement - Pivotal Trial (EBV after SOT) to Atara expects to initiate a pivotal trial of EBV-CTL in 10/01/2016-12/31/2016 ATRA EBV-CTL Hematologic Cancer II 117965 Inc. Initiation Start refractory EBV patients after SOT by the end of 2016. An Investigational New Drug (IND) application for EBVpoly- Atara Biotherapeutics, CTL for the initial conditions of nasopharyngeal cancer (NPC) 01/01/2017-03/31/2017 ATRA EBVpoly-CTL Head and Neck Cancer Preclinical Regulatory - IND Filing IND Filing (NPC) 117974 Inc. and multiple sclerosis is expected in the first quarter of 2017. An Investigational New Drug (IND) application for EBVpoly- Atara Biotherapeutics, CTL for the initial conditions of nasopharyngeal cancer (NPC) 01/01/2017-03/31/2017 ATRA EBVpoly-CTL Multiple Sclerosis (MS) Preclinical Regulatory - IND Filing IND Filing (MS) 117973 Inc. and multiple sclerosis is expected in the first quarter of 2017.

Biomedtracker August 2016 / 114 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link aTyr plans to initiate a clinical program in rare pulmonary iMod.Fc Discovery Trial Announcement - diseases with an immune component (RPIC) in patients with 01/01/2017-12/31/2017 aTyr Pharma LIFE Pulmonary Fibrosis Preclinical Phase Ib/II - Trial to Start 118326 Program Initiation interstitial lung disease (ILD). In 2017 the Company expects to initiate a Phase Ib/II study with iMod.Fc.

BioCryst announced that data from a solid-dose PK study of BioCryst Pharmaceuticals, Trial Data - Updated Phase I Healthy Volunteers 04/01/2016-09/30/2016 BCRX Avoralstat Hereditary Angioedema (HAE) III avoralstat for the treatment of hereditary angioedema (HAE) 118307 Inc. Results (PK) - Updated Results are expected in mid-2016.

BioCryst announced that they plan to conduct an end-of- BioCryst Pharmaceuticals, Regulatory - Meeting with 04/01/2016-09/30/2016 BCRX Avoralstat Hereditary Angioedema (HAE) III End of Phase II Meeting w/FDA Phase II meeting with the FDA regarding avoralstat for the 118313 Inc. FDA treatment of hereditary angioedema (HAE) in mid-2016.

BioCryst announced that they plan to initiate a study entitled BioCryst Pharmaceuticals, Trial Announcement - 09/01/2016-12/31/2016 BCRX Avoralstat Hereditary Angioedema (HAE) III Phase III OPuS-3 - Trial to Start OPuS-3 of avoralstat for the treatment of hereditary 118309 Inc. Initiation angioedema (HAE) at the end of 2016. BioCryst announced the initiation of a long-term safety study BioCryst Pharmaceuticals, Trial Data - Top-Line Phase III OPuS-4 - Top-Line of avoralstat for the treatment of hereditary angioedema 01/01/2017-12/31/2017 BCRX Avoralstat Hereditary Angioedema (HAE) III 118311 Inc. Results Results (HAE) entitled OPuS-4. Results from the study are expected in 2017.

BioCryst announced that the Phase II dose-ranging study of BioCryst Pharmaceuticals, Trial Announcement - Phase II APeX-1 - Trial to 04/01/2016-09/30/2016 BCRX BCX7353 Hereditary Angioedema (HAE) I BCX7353 for the treatment of hereditary angioedema (HAE) 118315 Inc. Trial Completion Complete is expected to complete in mid-2016.

BioCryst announced that results from the Phase II dose- BioCryst Pharmaceuticals, Trial Data - Top-Line Phase II APeX-1 - Top-Line 04/01/2016-09/30/2016 BCRX BCX7353 Hereditary Angioedema (HAE) I ranging study of BCX7353 for the treatment of hereditary 118299 Inc. Results Results angioedema (HAE) are expected in mid-2016.

BioCryst announced that a pivotal trial for Japanese filing of BioCryst Pharmaceuticals, Trial Announcement - Phase III Confirmatory Japan BCX7353 for the treatment of hereditary angioedema (HAE) 09/01/2016-12/31/2016 BCRX BCX7353 Hereditary Angioedema (HAE) I 118304 Inc. Initiation Trial to Start is expected to start at the end of 2016. This study will proceed under the sakigake process.

BioCryst announced that data from a pivotal trial for BioCryst Pharmaceuticals, Trial Data - Top-Line Phase III Confirmatory Japan 04/01/2017-09/30/2017 BCRX BCX7353 Hereditary Angioedema (HAE) I Japanese filing of BCX7353 for the treatment of hereditary 118305 Inc. Results Trial - Top-Line Results angioedema (HAE) are expected in mid-2017.

Trial Announcement - Biogen announced that they anticipate initiating a Phase IIb 01/11/2016-12/31/2016 Biogen, Inc. BIIB Raxatrigine Sciatica II Phase IIb - Trial to Start 118070 Initiation trial of raxatrigine for sciatica in 2016. Progress Update - Biogen anticipates launching Zinbryta for relapsing MS in the 01/11/2016-06/30/2016 Biogen, Inc. BIIB Zinbryta Multiple Sclerosis (MS) BLA U.S. Product Launch 118066 Product Launch U.S. in the first half of 2016.

BioMarin announced that the Company plans to initiate a BioMarin Pharmaceutical Trial Announcement - Trial to Start - Younger than 5 younger than 5 years study of BMN 111 for the treatment of 01/11/2016-06/30/2016 BMRN BMN 111 Achondroplasia II 118020 Inc. Initiation Years achondroplasia to explore earlier treatment options. We await an update in the first half of 2016.

BioMarin announced that based on interactions with the Scientific Advice Working Party and the FDA the Company BioMarin Pharmaceutical Neuronal Ceroid Lipofuscinosis Regulatory - NDA/BLA 04/01/2016-09/30/2016 BMRN BMN 190 I/II BLA Filing anticipates BLA and MAA submissions for BMN 190 for the 118008 Inc. (NCL) Filing treatment of CLN2 disorder in mid-year 2016 with potential launch in 2017. BioMarin announced that based on interactions with the Scientific Advice Working Party and the FDA the Company BioMarin Pharmaceutical Neuronal Ceroid Lipofuscinosis Regulatory - MAA 04/01/2016-09/30/2016 BMRN BMN 190 I/II MAA Filing anticipates BLA and MAA submissions for BMN 190 for the 118009 Inc. (NCL) Submission (Europe) treatment of CLN2 disorder in mid-year 2016 with potential launch in 2017. BioMarin announced that they will provide a program BioMarin Pharmaceutical Progress Update - 05/01/2016-05/31/2016 BMRN BMN-270 Hemophilia A I/II Program Update update for BMN 270 for the treatment of hemophilia A in 118033 Inc. Development Review May 2016.

Trial Announcement - BioMarin announced that enrollment is expected to BioMarin Pharmaceutical Phase I/II - Enrollment 11/15/2016-12/31/2016 BMRN BMN-270 Hemophilia A I/II Patient Enrollment complete in the Phase I/II study of BMN 270 for the 118038 Inc. Complete Completed treatment of hemophilia A late in the fourth quarter of 2016.

BioMarin announced that they plan to meeting with health BioMarin Pharmaceutical Regulatory - Meeting with authorities (FDA/EMA) to discuss findings and the most 01/11/2016-06/30/2016 BMRN BMN-701 Pompe Disease II Meeting with FDA/EMA 117743 Inc. FDA efficient path forward for BMN-701 for the treatment of Pompe disease. We await an update in the first half of 2016.

BioMarin announced that they will provide an update on the BioMarin Pharmaceutical Progress Update - 04/01/2016-04/30/2016 BMRN BMN-701 Pompe Disease II Development Review next step for BMN-701 for the treatment of Pompe disease 118042 Inc. Development Review at their R&D Day in April 2016.

BioMarin announced that the Company will submit day 120 BioMarin Pharmaceutical Regulatory - Progress Submission of Day 120 03/01/2016-03/31/2016 BMRN Kyndrisa Muscular Dystrophy NDA questions regarding Kyndrisa for the treatment of Duchenne 118028 Inc. Update Questions Muscular Dystrophy (exon 51) in March 2016.

BioMarin anticipates filing a BLA for BMN 165 for the BioMarin Pharmaceutical Regulatory - NDA/BLA 07/01/2016-12/31/2016 BMRN PEG-PAL Phenylketonuria (PKU) III BLA Filing treatment of phenylketonuria (PKU) in the second half of 118015 Inc. Filing 2016.

bluebird bio announced that it plans to give a data update for LentiGlobin for beta-thalassemia and sickle cell disease at 12/03/2016-12/06/2016 bluebird bio BLUE LentiGlobin Anemia I/II Trial Data - Other Clinical Results at ASH 118266 the 2016 American Society of Hematology Annual Meeting which will take place from December 3-6 2016.

bluebird bio announced that they plan to pursue conditional approval in Europe on the basis of data from the ongoing Regulatory - MAA 01/12/2016-12/31/2016 bluebird bio BLUE LentiGlobin Anemia I/II MAA Submission Northstar (HGB-204) and HGB-205 studies as part of the 118268 Submission (Europe) adapative licensing pilot. We await an update in the time frame above.

bluebird bio announced that it plans to give a data update for LentiGlobin for beta-thalassemia and sickle cell disease at 12/03/2016-12/06/2016 bluebird bio BLUE LentiGlobin Sickle Cell Anemia I Trial Data - Other Clinical Results at ASH 118265 the 2016 American Society of Hematology Annual Meeting which will take place from December 3-6 2016.

Eli Lilly expects an external data readout for the Phase IIIb Boehringer Ingelheim Trial Data - Top-Line Phase IIIb MARLINA - Topline 01/12/2016-12/31/2016 Tradjenta Diabetes Mellitus, Type II Approved MARLINA study of Linagliptin for the treatment of type 2 118275 GmbH Results Results diabetes in 2016.

Five Prime announced that they expect to expand FPA008 Bristol-Myers Squibb Pigmented Villonodular Trial Announcement - Phase I/II Study 002 - Phase II 01/13/2016-06/30/2016 BMY FPA008 I/II into a Phase II portion for the treatment of pigmented 118291 Company Synovitis (PVNS) Initiation Portion to Start villonodular synovitis (PVNS) in the first half of 2016. Five Prime announced that they expect to complete Phase Ia Bristol-Myers Squibb Trial Announcement - Phase Ia/b Study 003 - Phase Ia 04/01/2016-09/30/2016 BMY FPA008 Solid Tumors I dose escalation testing FPA008 in 8 tumor settings in mid- 118295 Company Other Dose Escalation Complete 2016. Five Prime announced that they expect to complete Phase Ia Bristol-Myers Squibb Trial Announcement - Phase Ia/b Study 003 - Phase Ib 04/01/2016-09/30/2016 BMY FPA008 Solid Tumors I dose escalation testing FPA008 in 8 tumor settings and 118293 Company Initiation Portion to Start expand to Phase Ib in mid-2016.

Biomedtracker August 2016 / 115 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Trial Data - Top-Line Phase II - CheckMate-275 - Top- 01/12/2016-12/31/2016 BMY Opdivo Bladder Cancer II from the Phase II CheckMate-275 study of Opdivo for second 118179 Company Results Line Results line bladder cancer will be available in 2016.

Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Brain Cancer (malignant glioma; Trial Data - Top-Line Phase III - CheckMate-143 - 01/12/2016-12/31/2016 BMY Opdivo III from the Phase II CheckMate-143 study of Opdivo for 118181 Company AA and GBM) Results Top-Line Results recurrent glioblastoma will be available in 2016.

Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Trial Data - Top-Line Phase III - CheckMate-141 - 01/12/2016-12/31/2016 BMY Opdivo Head and Neck Cancer III from the Phase II CheckMate-141 study of Opdivo in second 118176 Company Results Top-Line Results line head and neck cancer will be available in 2016.

Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Trial Data - Top-Line Phase II - CheckMate-205 - Top- 01/12/2016-12/31/2016 BMY Opdivo Hodgkin's Lymphoma II from the Phase II CheckMate-205 study of Opdivo for second 118175 Company Results Line Results line Hodgkin lymphoma will be available in 2016.

Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Non-Hodgkin's Lymphoma Trial Data - Top-Line Phase II - CheckMate-139 - Top- from the Phase II CheckMate-139 study of Opdivo for 01/12/2016-12/31/2016 BMY Opdivo II 118177 Company (NHL) Results Line Results relapsed/refractory diffuse large B-cell lymphoma will be available in 2016.

Bristol-Myers Squibb anticipates that registrational data Bristol-Myers Squibb Non-Small Cell Lung Cancer Trial Data - Top-Line Phase III - CheckMate-026 - 01/12/2016-12/31/2016 BMY Opdivo Approved from the Phase II CheckMate-026 study of Opdivo in first- 118182 Company (NSCLC) Results Top-Line Results line PD-L1 expressers with NSCLC will be available in 2016.

BTG announced that they plan to launch Vistogard for the Progress Update - 01/13/2016-06/30/2016 BTG plc BTC:LN Vistogard Drug Toxicity Approved Product Launch (U.S.) treatment of drug toxicity in the United States in the first 118345 Product Launch (U.S.) half of 2016. Trial Data - Updated Phase II/III tnAcity - Updated Celgene expects to have data from its Phase II/III tnAcity 01/01/2018-12/31/2018 Celgene Corporation CELG Abraxane Breast Cancer Approved 117739 Results Results study of Abraxane in 2018. Regulatory - Supplemental European Filing - Celgene expects to file for approval of Abraxane for the Now-12/31/2016 Celgene Corporation CELG Abraxane Breast Cancer Approved Supplemental Filing 117747 Early-Stage treatment of early-stage breast cancer in Europe in 2016. (Europe)

Celgene expects to have data from the Phase III cooperative Trial Data - Top-Line Phase III ETNA - Top-Line Now-12/31/2016 Celgene Corporation CELG Abraxane Breast Cancer Approved group ETNA trial with ABRAXANE as neoadjuvant therapy in 117769 Results Results HER2-negative high-risk breast cancer in 2016.

Trial Data - Top-Line Phase II/III tnAcity - Top-Line Celgene expects to have data from the Phase II portion of its Now-12/31/2016 Celgene Corporation CELG Abraxane Breast Cancer Approved 117757 Results Results Phase II/III tnAcity study in 2016. Non-Small Cell Lung Cancer Trial Data - Top-Line Phase III Abound - Top-Line Celgene expects to have data from its Phase III abound study 01/01/2017-12/31/2017 Celgene Corporation CELG Abraxane Approved 117738 (NSCLC) Results Results of Abraxane in 2017. Trial Data - Top-Line Phase III APACT - Top-Line Celgene expects to have data from its Phase III APACT study 01/01/2017-12/31/2017 Celgene Corporation CELG Abraxane Pancreatic Cancer Approved 117737 Results Results of Abraxane in 2017. Trial Announcement - Phase III APACT - Patient Celgene expects to complete enrollment of its Phase III Now-12/31/2016 Celgene Corporation CELG Abraxane Pancreatic Cancer Approved Patient Enrollment 117760 Enrollment Completed APACT study of Abraxane in 2016. Completed

Chronic Lymphocytic Leukemia Trial Announcement - Celgene plans to initiate a Phase III trial with CC-122 in NHL Now-12/31/2016 Celgene Corporation CELG CC-122 (CLL)/Small Cell Lymphocytic II Phase III to Start 117749 Initiation in 2016. Lymphoma (SLL) - NHL

Chronic Lymphocytic Leukemia Trial Data - Top-Line Celgene expects to have Phase II data of CC-122 in NHL in Now-12/31/2016 Celgene Corporation CELG CC-122 (CLL)/Small Cell Lymphocytic II Phase II - Top-Line Results 117754 Results 2016. Lymphoma (SLL) - NHL

Systemic Lupus Erythematosus Trial Data - Top-Line Celgene expects to have Phase II data on CC-220 for the Now-12/31/2016 Celgene Corporation CELG CC-220 II Phase II - Top-Line Results 117758 (SLE) Results treatment of SLE in 2016.

Trial Data - Top-Line Phase I Endoscopic Outcome - Celgene expects to have endoscopic data for GED-0301 in 01/01/2017-12/31/2017 Celgene Corporation CELG GED 0301 Crohn's Disease III 117740 Results Top-Line Results 2017.

Trial Announcement - Celgene plans to initiate a second Phase III study of GED- Now-12/31/2016 Celgene Corporation CELG GED 0301 Crohn's Disease III Second Phase III Trial to Start 117752 Initiation 0301 in adults in 2016. Trial Announcement - Celgene expects to complete enrollment in its Phase II trial Non-Small Cell Lung Cancer Phase II w/MK-3475 - Patient Now-12/31/2016 Celgene Corporation CELG Oral Azacitidine II Patient Enrollment with CC-486 in combination with pembrolizumab in NSCLC in 117931 (NSCLC) Enrollment Completed Completed 2016. Trial Announcement - Celgene plans to initiate a Phase III trial with Otezla in atopic Now-12/31/2016 Celgene Corporation CELG Otezla Atopic Dermatitis (Eczema) II Phase III to Start 117750 Initiation dermatitis in 2016.

Trial Data - Top-Line Phase III RELIEF - Top-Line Celgene expects to have top-line results from its Phase III 01/01/2017-12/31/2017 Celgene Corporation CELG Otezla Behçet Syndrome III 117728 Results Results study of Otezla for Behcet Syndrome in 2017.

Trial Announcement - Phase III RELIEF - Patient Celgene expects to complete enrollment of its Phase III Now-12/31/2016 Celgene Corporation CELG Otezla Behçet Syndrome III Patient Enrollment 117761 Enrollment Completed RELIEF study of Otezla in 2016. Completed Trial Data - Top-Line Phase II Japan - Top-Line Celgene expects to have data from its PSOR-011 trial with Now-12/31/2016 Celgene Corporation CELG Otezla Psoriasis Approved 117934 Results Results Otezla in Japanese psoriasis patients in 2016. Trial Data - Updated Phase IIIB PSA-006 - Updated Celgene plans to have data from its Phase III PSA-006 study Now-12/31/2016 Celgene Corporation CELG Otezla Psoriatic Arthritis (PA) Approved 117753 Results Results of Otezla in 2016. Trial Announcement - Phase II - Patient Enrollment Celgene expects to complete enrollment in a Phase II trial Now-12/31/2016 Celgene Corporation CELG Otezla Ulcerative Colitis (UC) II Patient Enrollment 117936 Completed with Otezla in ulcerative colitis in 2016. Completed Trial Announcement - Phase II STEPSTONE - Patient Celgene expects to complete enrollment in the Phase II Now-12/31/2016 Celgene Corporation CELG Ozanimod Crohn's Disease II Patient Enrollment 117939 Enrollment Completed STEPSTONE trial with ozanimod in Crohn's disease in 2016. Completed

Celgene expects to submit renal impairment data for label Regulatory - sNDA/sBLA sNDA Filng - Renal Impairment Now-12/31/2016 Celgene Corporation CELG Pomalyst Multiple Myeloma (MM) Approved update for Pomalyst/Imnovid in relapsed/refractory multiple 117765 Filing Label Update myeloma in the US and Europe in 2016.

Regulatory - Supplemental European Filing - Celgene expects to submit renal impairment data for label Now-12/31/2016 Celgene Corporation CELG Pomalyst Multiple Myeloma (MM) Approved Supplemental Filing Renal Impairment Label update for Pomalyst/Imnovid in relapsed/refractory multiple 117767 (Europe) Update myeloma in the US and Europe in 2016.

Diffuse Large B-Cell Lymphoma Trial Data - Top-Line Phase III ROBUST - Top-Line Celgene expects to have data from its Phase III ROBUST 01/01/2018-12/31/2018 Celgene Corporation CELG Revlimid III 117734 (DLBCL) - NHL Results Results study of Revlimid in 2018. Diffuse Large B-Cell Lymphoma Trial Data - Top-Line Phase III REMARC - Top-Line Celgene expects to have data from its Phase III REMARC 04/01/2016-09/30/2016 Celgene Corporation CELG Revlimid III 117732 (DLBCL) - NHL Results Results study of Revlimid in mid-2016. Indolent Non-Hodgkin's Trial Data - Top-Line Phase III MAGNIFY - Top-Line Celgene expects to have data from its Phase III MAGNIFY 01/01/2018-12/31/2018 Celgene Corporation CELG Revlimid III 117735 Lymphoma - NHL Results Results study of Revlimid in 2018.

Indolent Non-Hodgkin's Trial Data - Top-Line Phase III AUGMENT - Top-Line Celgene expects to have data from its Phase III AUGMENT 07/01/2017-12/31/2017 Celgene Corporation CELG Revlimid III 117733 Lymphoma - NHL Results Results study of Revlimid in the second half of 2017.

Trial Announcement - Indolent Non-Hodgkin's Phase III AUGMENT - Patient Celgene expects to complete enrollment of its Phase III Now-12/31/2016 Celgene Corporation CELG Revlimid III Patient Enrollment 117759 Lymphoma - NHL Enrollment Completed AUGMENT study of Revlimid in 2016. Completed Regulatory - sNDA/sBLA Celgene plans to submit Revlimid for approval in the US and Now-12/31/2016 Celgene Corporation CELG Revlimid Multiple Myeloma (MM) Approved sNDA Filing - post-ASCT 117741 Filing EU for maintenance post-ASCT in 2016. Regulatory - Supplemental European Filing - Celgene plans to submit Revlimid for approval in the US and Now-12/31/2016 Celgene Corporation CELG Revlimid Multiple Myeloma (MM) Approved Supplemental Filing 117742 post-ASCT EU for maintenance post-ASCT in 2016. (Europe) Trial Announcement - Celgene plans to initiate a Phase III trial with RPC-4046 in Now-12/31/2016 Celgene Corporation CELG RPC4046 Esophagitis II Phase III to Start 117751 Initiation patients with esophagitis in 2016.

Biomedtracker August 2016 / 116 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Cempra announced the Company expects to complete the Chronic Obstructive Pulmonary Trial Announcement - 10/01/2016-12/31/2016 Cempra, Inc. CEMP Solithromycin II Phase II - Trial Completed Phase II study of Solithromycin for the treatment of COPD by 118418 Disease (COPD) Trial Completion the end of 2016.

ChemoCentryx plans to report initial progression free Trial Data - Updated 07/01/2016-12/31/2016 ChemoCentryx, Inc. CCXI CCX872 Pancreatic Cancer I Phase Ib - PFS Data survival data from the Phase Ib clinical trial of CCX872 in 118460 Results patients with pancreatic cancer in the second half of 2016.

Chimerix announced that the company will review data from the AdVise study with Regulatory agencies to determine a Regulatory - Meeting with 01/12/2016-09/30/2016 Chimerix, Inc. CMRX Brincidofovir Antiviral - Other Treatments III Meeting with FDA path for approval. The Company aims to determine its 118171 FDA development plan by the summer of 2016. As such we await a meeting with the FDA in the time frame above.

Chimerix plans to meet with the U.S. Food and Drug Cytomegalovirus (CMV) Regulatory - Meeting with Administration (FDA) and European Medicines Agency (EMA) 01/12/2016-12/31/2016 Chimerix, Inc. CMRX Brincidofovir III Meeting with FDA 118172 Infection (Antiviral) FDA in 2016 to define regulatory paths for CMV prevention and/or treatment with brincidofovir.

Chimerix plans to meet with the U.S. Food and Drug Regulatory - Meeting with Cytomegalovirus (CMV) Administration (FDA) and European Medicines Agency (EMA) 01/12/2016-12/31/2016 Chimerix, Inc. CMRX Brincidofovir III European Medicines Meeting with EMA 118173 Infection (Antiviral) in 2016 to define regulatory paths for CMV prevention Agency and/or treatment with brincidofovir.

Chimerix announced that the Company is developing an intravenous formulation of brincidofovir. A 28-day IND- Cytomegalovirus (CMV) Trial Announcement - 07/01/2016-12/31/2016 Chimerix, Inc. CMRX Brincidofovir (IV) Preclinical FTIH Study to Start enabling preclinical study is currently ongoing. Chimerix 118254 Infection (Antiviral) Initiation anticipates a FTIH study for brincidofovir (IV) will begin in the second half of 2016.

Trial Announcement - Phase II Pediatric - Trial Circassia reports that the Cat-SPIRE pediatric safety study is 07/01/2016-12/31/2016 Circassia Ltd. LSE: CIR Cat-SPIRE Allergic Rhinitis III 117967 Trial Completion Completed on track to complete in the second half of 2015.

Regulatory - NDA/BLA Circassia expects to submit an approval filing for Cat SPIRE in 07/01/2016-12/31/2016 Circassia Ltd. LSE: CIR Cat-SPIRE Allergic Rhinitis III NDA Filing 117992 Filing the second half of 2016. Trial Data - Top-Line Phase IIb Follow-up - Top-Line Circassia expects a Phase IIb follow-up study of Ragweed Now-06/30/2016 Circassia Ltd. LSE: CIR Ragweed-SPIRE Allergic Rhinitis IIb 117976 Results Results SPIRE to report in the first half of 2016.

Clovis announced an overview of lucitanib at the 34th annual JP Morgan Healthcare Conference. There is an unclear Trial Announcement - Phase II Study - Patient regulatory path as monotherapy and future development is 01/13/2016-03/31/2016 Clovis Oncology, Inc. CLVS Lucitanib Breast Cancer II Patient Enrollment 118405 Enrollment Complete focused on combination studies. Enrollment (n=160) Completed completion is expected the first quarter of 2016 for the Phase II trial of lucitanib for breast cancer.

Clovis announced that there was synergy with lucitanib and Hepatocellular (Liver) Cancer Trial Announcement - mTOR inhibitors in pre-clinical models in hepatocellular 07/01/2016-12/31/2016 Clovis Oncology, Inc. CLVS Lucitanib (HCC) (including secondary Preclinical Phase Ib Study to Start 118407 Initiation cancer. The company stated a Phase Ib study is planned to metastases) initiate the second half of 2016.

Clovis announced that there is an opportunity for an intra- Trial Announcement - portfolio lucitanib/rucaparib combination in ovarian cancer. 07/01/2016-12/31/2016 Clovis Oncology, Inc. CLVS Lucitanib Ovarian Cancer Preclinical Phase Ib Study to Start 118406 Initiation The company stated a Phase Ib study is planned to initiate the second half of 2016. Clovis announced that two additional anti-PD(L)-1 pembrolizumab trials will be initiating in the first half of 2016 Non-Small Cell Lung Cancer Trial Announcement - Phase Ib/II w/Pembrolizumab 01/13/2016-06/30/2016 Clovis Oncology, Inc. CLVS Rociletinib NDA one Phase Ib/II in the U.S. in EGFRm NSCLC 2nd line + 118367 (NSCLC) Initiation Trials to Start T790M+ and - and the other a Phase II in Asia in EGFRm NSCLC 2ndline + T790M+ and -.

Trial Announcement - Clovis plans to initiate the RIO trial in triple-negative or Now-12/31/2016 Clovis Oncology, Inc. CLVS Rucaparib Breast Cancer II RIO Study to Start 118399 Initiation gBRCA breast cancer patients with rucaparib in 2016.

Clovis announced that results from Part 2 of the Phase II Trial Data - Updated Phase II ARIEL2 Part 2 - Results 06/03/2016-06/07/2016 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III ARIEL2 study of rucaparib will be presented at the 2016 118396 Results at ASCO ASCO meeting.

Clovis is planning a U.S. NDA submission in the advanced tumor tissue BRCA mutant (germline and somatic mutations) ovarian cancer population. The NDA will include platinum Regulatory - Rolling 04/01/2016-06/30/2016 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III Rolling NDA Completion sensitive and platinum resistant patients. The initial NDA for 118389 NDA/BLA Completion tBRCAmut patients with advanced disease plans to start by a rolling submission to initiate in the first quarter of 2016 and to complete by the second quarter of 2016.

Regulatory - MAA Clovis is planning a MAA submission for rucaparib in ovarian 07/01/2016-09/30/2016 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III MAA Filing 118369 Submission (Europe) cancer in the third quarter of 2016. Clovis plans on initiating the ARIEL4 confirmatory study in Trial Announcement - ARIEL4 Confirmatory Study to mid 2016 which includes tBRCAmut and BRCA-like patients 04/01/2016-09/30/2016 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III 118391 Initiation Start as well as initiating multiple combination studies in ovarian cancer during 2016.

Clovis announced that a supplemental NDA for the rucaparib Regulatory - sNDA/sBLA sNDA Filing - tBRCAmut and 04/01/2017-09/30/2017 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III maintenance indication in tBRCAmut and BRCA-like 118390 Filing BRCA-like populations is to follow based on ARIEL 3 in mid-2017.

Clovis announced that they are expanding Rucaparib into Trial Announcement - 07/01/2016-09/30/2016 Clovis Oncology, Inc. CLVS Rucaparib Prostate Cancer Preclinical Registration Study to Start prostate cancer with a registration study to initiate in the 118371 Initiation third quarter of 2016.

Biosimilar Adalimumab Trial Data - Top-Line Phase III PsOsim - Top-Line Coherus expects to have Phase III study results for CHS-1420 04/01/2016-09/30/2016 Coherus BioSciences, Inc. CHRS Psoriasis III 117982 (Coherus) Results Data in mid-2016.

Biosimilar Pegfilgrastim Trial Data - Top-Line Phase I - PK/PD Follow-On Top- The PK/PD follow-on study readouts for CHS-1701 are 04/01/2016-06/30/2016 Coherus BioSciences, Inc. CHRS Neutropenia / Leukopenia I 117969 (Coherus) Results Line Results expected in the second quarter of 2016.

Wet Age-Related Macular Coherus announced that CHS-3351 is in preclinical Biosimilar Ranibizumab 01/11/2016-12/31/2016 Coherus BioSciences, Inc. CHRS Degeneration (Wet AMD) Preclinical Regulatory - IND Filing IND Filing development as a biosimilar Lucentis with an IND to be filed 117991 (Coherus) (Ophthalmology) in 2016.

Trial Data - Top-Line Coherus expects results from the Phase II study of CHS-131 05/01/2016-06/30/2016 Coherus BioSciences, Inc. CHRS CHS-131 Multiple Sclerosis (MS) IIb Phase IIb - Top-Line Results 117988 Results for multiple sclerosis in the May-June 2016 timeframe.

Dynavax expects a Phase II study of pembrolizumab alone or Dynavax Technologies Trial Announcement - Phase II w/Pembrolizumab to pembrolizumab in combination with SD-101 in patients with 07/01/2016-09/30/2016 DVAX SD-101 (Dynavax) Hematologic Cancer I/II 118506 Corporation Initiation Start metastatic prostate cancer at UCSF to start in the third quarter of 2016. Dynavax plans to initiate an additional study of SD-101 in Dynavax Technologies Trial Announcement - Additional Combination Trial 10/01/2016-12/31/2016 DVAX SD-101 (Dynavax) Hematologic Cancer I/II combination with pembrolizumab in an additional tumor 118503 Corporation Initiation w/Pembrolizumab to Start type in the fourth quarter of 2016. Dynavax plans to initiate a combination study of SD-101 with Dynavax Technologies Trial Announcement - Combination Trial 10/01/2016-12/31/2016 DVAX SD-101 (Dynavax) Hematologic Cancer I/II an undisclosed investigational checkpoint inhibitor in the 118504 Corporation Initiation w/Checkpoint Inhibitor to Start fourth quarter of 2016.

Biomedtracker August 2016 / 117 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Dynavax expects to complete part 1 of its Phase I/II study of Dynavax Technologies Trial Announcement - Phase I/II w/Pembrolizumab - 07/01/2016-09/30/2016 DVAX SD-101 (Dynavax) Melanoma I/II SD-101 in combination with pembrolizumab and initiate Part 118501 Corporation Other Part 2 to Start 2 expansion in the third quarter of 2016.

Eli Lilly expects data readout for a Phase II study of Trial Data - Top-Line 01/12/2016-12/31/2016 Eli Lilly & Company LLY Abemaciclib Breast Cancer III Phase II - Topline Results Abemaciclib as a single agent for the treatment of breast 118270 Results cancer in 2016. Eli Lilly expects an external data readout for the Phase III RA- Trial Data - Top-Line Phase III RA BEYOND - Topline 01/12/2016-12/31/2016 Eli Lilly & Company LLY Baricitinib Rheumatoid Arthritis (RA) III BEYOND (long-term extension) study of baricitinib for the 118271 Results Results treatment of rheumatoid arthritis.

Regulatory - J-NDA Filing Eli Lilly announced plans for a Japanese regulatory filing for 01/12/2016-12/31/2016 Eli Lilly & Company LLY Baricitinib Rheumatoid Arthritis (RA) III Japanese NDA Filing 118277 (Japan) Baricitinib for the treatment of rheumatoid arthritis in 2016.

Eli Lilly announced the potential initiation of a Phase III study Trial Announcement - Now-12/31/2016 Eli Lilly & Company LLY Ixekizumab Ankylosing Spondylitis Suspended Phase III - Trial to Start of Ixekizumab for the treatment of axial spondyloarthritis in 118269 Initiation 2016. Eli Lilly expects an internal data readout for the Company's Trial Data - Top-Line Phase III IXORA S - Topline 01/12/2016-12/31/2016 Eli Lilly & Company LLY Ixekizumab Psoriasis BLA Phase III IXORA-S study of Ixekizumab for the treatment of 118274 Results Results psoriasis in 2016. Eli Lilly expects an internal data read out for its Phase III Trial Data - Top-Line Phase III SPIRIT P2 - Topline 01/12/2016-12/31/2016 Eli Lilly & Company LLY Ixekizumab Psoriatic Arthritis (PA) III SPIRIT P2 study of Ixekizumab for the treatment of psoriatic 118273 Results Results arthritis in 2016. Eli Lilly announced that the Company has begun a U.S. rolling regulatory submission for olaratumab for the treatment of Regulatory - Rolling Now-06/30/2016 Eli Lilly & Company LLY LY3012207 Sarcoma III Rolling NDA Completed soft tissue sarcoma in late 2015. We await on the 118262 NDA/BLA Completion completion of the rolling NDA submission through the first half of 2016.

Enatna announced that the Company is currently studying a core inhibitor for the treatment of hepatitis B virus and a Enanta Pharmaceuticals, Hepatitis B (HBV) Treatment Trial Announcement - non-fusion inhibitor for the treatment of RSV. Both 01/01/2017-12/31/2017 ENTA Core Inhibitor Program Preclinical Clinical Trials to Start 118356 Inc. (Antiviral) Initiation compounds are in preclinical development and the Company expects to advance one of the two product candidates to Phase I clinical studies in 2017.

Enanta announced plans to initiate clinical studies for EDP- Enanta Pharmaceuticals, Primary Biliary Cirrhosis (PBC) Trial Announcement - 01/13/2016-12/31/2016 ENTA EDP-305 Preclinical Clinical Trials to Start 305 for the treatment of primary biliary cirrhosis and hepatic 118353 Inc. and Hepatic Fibrosis Initiation fibrosis in 2016.

Enatna announced that the Company is currently studying a core inhibitor for the treatment of hepatitis B virus and a Enanta Pharmaceuticals, Non-Fusion Inhibitor Respiratory Syncytial Virus Trial Announcement - non-fusion inhibitor for the treatment of RSV. Both 01/01/2017-12/31/2017 ENTA Preclinical Clinical Trials to Start 118359 Inc. Program (RSV) Initiation compounds are in preclinical development and the Company expects to advance one of the two product candidates to Phase I clinical studies in 2017.

Endo announced that they have a meeting with the FDA Regulatory - Meeting with scheduled for January 19 2016 for Xiaflex for Dupuytren's 1/19/2016 Endo International plc ENDP Xiaflex Dupuytren's Contracture Approved Meeting With FDA 118084 FDA nodules. A registration trial initiation is expected in mid- 2016. Endo announced that they have a meeting with the FDA Trial Announcement - Registration Study to Start - scheduled for January 19 2016 for Xiaflex for Dupuytren's 04/01/2016-09/30/2016 Endo International plc ENDP Xiaflex Dupuytren's Contracture Approved 118086 Initiation Nodules nodules. A registration trial initiation is expected in mid- 2016.

Endo expects to meet with the FDA in the first quarter of Frozen Shoulder Syndrome Regulatory - Meeting with 01/11/2016-03/31/2016 Endo International plc ENDP Xiaflex II Meeting With FDA 2016 to discuss Xiaflex for adhesive capsulitis. Trial initiation 118081 (Adhesive Capsulitis) FDA is anticipated after that discussion.

Diabetic Macular Edema Trial Announcement - Envisia expects to initiate a Phase II study of ENV1105 for 01/01/2017-04/30/2017 Envisia Therapeutics ENV1105 Preclinical Phase II - Trial to Start 118197 (Ophthalmology) Initiation the treatment of diabetic macular edema in early 2017.

Diabetic Macular Edema Trial Announcement - Envisia expects to initiate a Phase III study of ENV1105 for 09/01/2017-12/31/2017 Envisia Therapeutics ENV1105 Preclinical Phase III - Trial to Start 118198 (Ophthalmology) Initiation the treatment of diabetic macular edema at the end of 2017.

Diabetic Macular Edema Trial Data - Top-Line Envisia expects Phase II results of ENV1105 for the 01/01/2017-12/31/2017 Envisia Therapeutics ENV1105 Preclinical Phase II - Top-Line Results 118205 (Ophthalmology) Results treatment of diabetic macular edema in 2017. Wet Age-Related Macular Envisia anticipates elevating ENV1305 for the treatment of Trial Announcement - Preclinical Study - Trials to 01/12/2016-12/31/2016 Envisia Therapeutics ENV1305 Degeneration (Wet AMD) Preclinical wet age-related macular degeneration (AMD) into preclinical 118222 Initiation Start (Ophthalmology) development in 2016.

Envisia announced that they expect to initiate a Phase IIb/III Glaucoma / Ocular Trial Announcement - 09/01/2016-09/30/2017 Envisia Therapeutics ENV515 II Phase IIb/III - Trial to Start study of ENV515 for the treatment of glaucoma in late 2016 118201 Hypertension (Ophthalmology) Initiation through approximately the third quarter of 2017.

Envisia announced that they expect to initiate a Phase III Glaucoma / Ocular Trial Announcement - 09/01/2017-12/31/2017 Envisia Therapeutics ENV515 II Phase III - Trial to Start study of ENV515 for the treatment of glaucoma at the end of 118199 Hypertension (Ophthalmology) Initiation 2017. Envisia announced that they plan to file an IND for ENV905 Ocular Inflammation 07/01/2016-12/31/2016 Envisia Therapeutics ENV905 Preclinical Regulatory - IND Filing IND Filing for the treatment of post-operative inflammation in the 118168 (Ophthalmology) second half of 2016. Envisia expects to initiate a Phase II study of ENV905 in Ocular Inflammation Trial Announcement - 09/01/2016-06/30/2017 Envisia Therapeutics ENV905 Preclinical Phase II - Trial to Start patients with post-cataract inflammation in late 2016 118195 (Ophthalmology) Initiation through approximately mid-year 2017.

Ocular Inflammation Trial Announcement - Envisia expects to initiate a Phase III study of ENV905 in 09/01/2017-12/31/2017 Envisia Therapeutics ENV905 Preclinical Phase III - Trial to Start 118196 (Ophthalmology) Initiation patients with post-cataract inflammation at the end of 2017.

Ocular Inflammation Trial Data - Top-Line Envisia expects Phase II results of ENV905 for the treatment 01/01/2017-12/31/2017 Envisia Therapeutics ENV905 Preclinical Phase II - Top-Line Results 118206 (Ophthalmology) Results of post-operative inflammation in 2017.

Progress Update - Esperion Therapeutics anticipates formalizing and Esperion Therapeutics, Dyslipidemia / Progress Update - 04/01/2016-06/30/2016 ESPR ETC-1002 III Cardiovascular Outcomes Trial communicating the design of the planned cardiovascular 118282 Inc. Hypercholesterolemia Development Review Update outcomes trial in the second quarter of 2016.

Genmab announced plans to file an IND in 2016. We await Now-06/30/2016 Genmab A/S GEN:DC HuMax-AXL ADC Solid Tumors Preclinical Regulatory - IND Filing IND Filing 118473 an update in the time frame above. Gilead announced that they plan to initiate a study of F/TAF Emtricitabine/tenofovir Trial Announcement - 01/11/2016-12/31/2016 Gilead Sciences, Inc. GILD HIV / AIDS NDA PrEP Study to Start for the treatment of pre-exposure prophylaxis (PrEP) in 118003 alafenamide Initiation 2016. Gilead announced that a Phase III study evaluating filgotinib Trial Announcement - for the treatment of rheumatoid arthritis (RA) and Crohn's 01/11/2016-06/30/2016 Gilead Sciences, Inc. GILD Filgotinib Crohn's Disease II Phase III - Trial to Start 117999 Initiation disease is expected to initiate in 2016. We await an update in the first half of 2016. Gilead announced that top-line data from Phase III studies Sofosbuvir/velpatasvir/GS- Trial Data - Top-Line Phase III POLARIS-1 - Top-Line 01/11/2016-12/31/2016 Gilead Sciences, Inc. GILD Hepatitis C (HCV) (Antiviral) III evaluating SOF/VEL/GS-9857 in HCV-infected patients are 117997 9857 FDC Results Results expected in 2016. Gilead announced that top-line data from Phase III studies Sofosbuvir/velpatasvir/GS- Trial Data - Top-Line Phase III POLARIS-2 - Top-Line 01/11/2016-12/31/2016 Gilead Sciences, Inc. GILD Hepatitis C (HCV) (Antiviral) III evaluating SOF/VEL/GS-9857 in HCV-infected patients are 117998 9857 FDC Results Results expected in 2016.

GlaxoSmithKline announced that they plan to submit Systemic Lupus Erythematosus Regulatory - NDA/BLA 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK Benlysta SC III BLA Filing regulatory filings for Benlysta SC for the treatment of 118223 (SLE) Filing systemic lupus erythematosus (SLE) in 2016.

Biomedtracker August 2016 / 118 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link GlaxoSmithKline announced that they plan to submit Systemic Lupus Erythematosus Regulatory - MAA 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK Benlysta SC III MAA Filing regulatory filings for Benlysta SC for the treatment of 118224 (SLE) Submission (Europe) systemic lupus erythematosus (SLE) in 2016.

GlaxoSmithKline announced that they expect to start Phase Trial Announcement - 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK-1265744 LAP HIV / AIDS IIb Phase III PrEP - Studies to Start III studies of cabotegravir for the treatment of pre-exposure 118228 Initiation prophylaxis (PrEP) in 2016.

Trial Announcement - GlaxoSmithKline announced that they plan to start a Phase 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK1278863 Anemia II Phase III - Trial to Start 118226 Initiation III study of daprodustat for the treatment of anemia in 2016.

GlaxoSmithKline announced that they expect to have the Trial Data - Top-Line Phase I - Recommended Phase recommended Phase II dose of GSK-2816126 in 2016. As 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK-2816126 Cancer I 118267 Results II Dose (RP2D) such we expect results from the Phase I study of GSK- 2816126 within that same time frame.

GlaxoSmithKline announced that they expect to have the Trial Data - Top-Line Phase I - Recommended Phase recommended Phase II dose of GSK-2879552 in 2016. As 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK-2879552 Small Cell Lung Cancer (SCLC) I 118242 Results II Dose (RP2D) such we expect results from the Phase I study of GSK- 2879552 within that same time frame.

Trial Announcement - GlaxoSmithKline announced that they plan to start a 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK525762 Solid Tumors I Combination Study to Start 118243 Initiation combination study with GSK525762 in 2016.

Trial Data - Top-Line GlaxoSmithKline announced that they plan to release single 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK GSK525762 Solid Tumors I Single Agent Efficacy Data 118244 Results agent efficacy data of GSK525762 across indications in 2016.

Ionis announced that it expects results from the Phase III Transthyretin-related Trial Data - Updated portion of the ongoing open-label extension (OLE) study of 04/01/2016-09/30/2016 GlaxoSmithKline plc GSK IONIS-TTRrx Hereditary Amyloidosis (Familial II/III Phase II/III - Updated Results 118100 Results IONIS-TTRRx in patients with familial amyloid Amyloid Polyneuropathy) polyneuropathy (FAP) in mid-2016.

Progress Update - GlaxoSmithKline announced that they expect to launch 01/12/2016-12/31/2016 GlaxoSmithKline plc GSK Nucala Asthma Approved Product Launch (Europe) 118229 Product Launch (Europe) Nucala for the treatment of asthma in Europe in 2016.

Glenmark reported that it plans to file an Investigational Glenmark 01/12/2016-09/30/2016 GNP:IN GBR 1342 Cancer Preclinical Regulatory - IND Filing IND Filing New Drug (IND) application to initiate a Phase I study of GBR 118260 Pharmaceuticals Limited 1342 in the US in the next 9 months.

Global Blood Therapeutics Oral Kallikrein Inhibitor Trial Announcement - Global Blood Therapeutics intends to initiate a Phase I 07/01/2016-12/31/2016 GBT Hereditary Angioedema (HAE) Preclinical Phase I to Start 118423 Inc. (GBT) Initiation clinical trial of the HAE candidate in the second half of 2016.

Halozyme Therapeutics provided key program updates and its Annual financial guidance at the 34th annual JP Morgan Healthcare Conference. Halozyme announced that they achieved target enrollment in Stage 2 of Halozyme Study 202 of investigational new drug PEGPH20 in metastatic Halozyme Therapeutics, Trial Data - Top-Line Phase II 202 Stage 2 - Top-Line 10/01/2016-12/31/2016 HALO PEGPH20 Pancreatic Cancer II pancreatic ductal adenocarcinoma patients. Halozyme has 118272 Inc. Results Results enrolled approximately 120 patients through the end of 2015. The company remains blinded to the efficacy results and projects presentation of mature progression-free survival data in the event driven study in the fourth quarter of 2016.

Trial Announcement - Horizon reports that the Phase III STEADFAST trial of Phase III STEADFAST - Patient 04/01/2016-09/30/2016 Horizon Pharma plc HZNP Actimmune Friedreich's Ataxia III Patient Enrollment Actimmune for Friedreich's Ataxia is set to complete 118374 Enrollment Completion Completed enrollment by mid-year 2016. Horizon reports that the Phase III STEADFAST trial of Trial Data - Top-Line Phase III STEADFAST - Top-Line 12/01/2016-12/31/2016 Horizon Pharma plc HZNP Actimmune Friedreich's Ataxia III Actimmune for Friedreich's Ataxia will have data available in 118377 Results Results December 2016. Horizon expects to file a supplemental New Drug Application Regulatory - sNDA/sBLA (sNDA) for Ravicti in pediatric patients ages two months to 04/01/2016-06/30/2016 Horizon Pharma plc HZNP Ravicti Urea Cycle Disorder (UCD) Approved sNDA Filing - Age 2 118368 Filing two years with Urea Cycle Disorder by the second quarter of 2016.

Progress Update - Horizon expects a product launch of Ravicti for Urea Cycle 01/01/2017-12/31/2017 Horizon Pharma plc HZNP Ravicti Urea Cycle Disorder (UCD) Approved Product Launch - Europe 118366 Product Launch (Europe) Disorder in Europe in 2017.

Horizon expects to file a supplemental New Drug Application Regulatory - sNDA/sBLA (sNDA) for Ravicti in pediatric patients from the age of birth 01/01/2018-03/31/2018 Horizon Pharma plc HZNP Ravicti Urea Cycle Disorder (UCD) Approved sNDA Filing - Birth 118370 Filing to two months with Urea Cycle Disorder by the first quarter of 2018. Waldenstrom Idera announced that they expect to release updated results Idera Pharmaceuticals, Macroglobulinemia (WM) / Trial Data - Updated 01/13/2016-12/31/2016 IDRA IMO-8400 I/II Phase I/II - Updated Results from the Phase I/II study of IMO-8400 for Waldenstrom 118347 Inc. Lymphoplasmacytic Lymphoma Results macroglobulinemia in 2016. (LPL) - NHL

Indivior announced that a decision in the lawsuits against Actavis and Par involving the Orange Book-listed patents for Patent - Expiration of 30- Suboxone Film November and December 2015 is expected 2/28/2016 Indivior plc INDV:LN Suboxone Film Drug Addiction Approved Expiration of 30-Month Stay 118414 Month Stay soon. As is Actavis’ 30 month stay of FDA approval expires February 28th 2016. Par’s 30 month stay of FDA approval expires on September 25th 2016.

Infinity expects to report initial data from CONTEMPO a Infinity Pharmaceuticals, Indolent Non-Hodgkin's Trial Data - Top-Line Phase Ib/II CONTEMPO - Top- 07/01/2016-12/31/2016 INFI Duvelisib III Phase Ib/II study in treatment-naïve patients with follicular 118397 Inc. Lymphoma - NHL Results Line Results lymphoma in 2H16. Pulmonary Arterial Trial Data - Top-Line Now-12/31/2016 Insmed, Inc. INSM INS-1009 Preclinical Phase I - Top-Line Results Insmed expects Phase I data on INS1009 during 2016. 118329 Hypertension (PAH) Results

Trial Announcement - Insys reported that clinical studies for Buprenorphine in Now-12/31/2016 INSYS Therapeutics, Inc. INSY Buprenorphine Spray Chronic Pain Program Hold Clinical Trials to Start 118403 Initiation acute and chronic pain are expected to initiate in 2016.

Insys reported that clinical studies of Cannabidiol for Infantile Spasms (West Trial Announcement - Now-12/31/2016 INSYS Therapeutics, Inc. INSY Cannabidiol IND Clinical Trials to Start Infantile Spasms (West Syndrome; Epilepsy) are expected to 118398 Syndrome; Epilepsy) Initiation be initiated in 2016.

Insys reported that Phase II studies of Liposomal Entrapped Trial Announcement - Now-12/31/2016 INSYS Therapeutics, Inc. INSY LEP-ETU Breast Cancer II Phase II - Trial to Start Paclitaxel-Easy to Use (LEP-ETU) for metastatic breast cancer 118404 Initiation are expected to begin in 2016.

RESOLVE Bioabsorbable Trial Data - Top-Line Phase III RESOLVE II - Top-Line 01/01/2017-12/31/2017 Intersect ENT XENT Allergy III Intersect expects results from the RESOLVE II study in 2017. 118239 Drug-eluting Implant Results Results

Intrexon announced that they plan to initiate a Phase II Trial Announcement - 01/13/2016-12/31/2016 Intrexon Corporation XON AG013 Mucositis I Phase II - Trial to Start clinical trial for the treatment of oral mucositis with 118337 Initiation ActoBiotics in 2016. Intrexon announced that they will assess CAR-T cells Trial Announcement - Phase I Hematological Tumors - 01/13/2016-12/31/2016 Intrexon Corporation XON Non-Viral CAR-T Program Hematologic Cancer I targeting hematological tumors in preclinical and clinical 118392 Hold Lifted Trial to Start studies in 2016. Trial Announcement - Phase I Myeloid Malignancies - Intrexon announced that a clinical trial for CAR-T targeting 01/13/2016-12/31/2016 Intrexon Corporation XON Non-Viral CAR-T Program Hematologic Cancer I 118395 Initiation Trial to Start myeloid malignancies is planned for 2016.

Biomedtracker August 2016 / 119 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Trial Announcement - Phase I Solid Tumors - Trial to Intrexon announced that they will test next-generation CAR- 01/01/2017-12/31/2017 Intrexon Corporation XON Non-Viral CAR-T Program Solid Tumors Preclinical 118393 Initiation Start T cells for efficacy in solid tumors in 2017.

Intrexon announced that they anticipate an IND filing for the Wet Age-Related Macular Ocular Disease Program next-gen RTS controlled gene therapy for the treatment of 07/01/2016-12/31/2016 Intrexon Corporation XON Degeneration (Wet AMD) Preclinical Regulatory - IND Filing IND Filing 118402 (Intrexon/Sun) wet age-related macular degeneration (Wet AMD) in the (Ophthalmology) second half of 2016.

Ionis Pharmaceuticals, Dyslipidemia / Trial Data - Top-Line Ionis anticipates releasing top-line results from the Phase I/II 07/01/2016-12/31/2016 IONS IONIS-ANGPTL3Rx I Phase II - Top-Line Results 118125 Inc. Hypercholesterolemia Results study of ANGPTL2-LRx in the second half of 2016.

Ionis anticipates initiating a Phase II trial of APO(a)-LRx for Ionis Pharmaceuticals, Trial Announcement - very high Lp(a) with recurring CVD in mid-2016 and a Phase 04/01/2016-09/30/2016 IONS IONIS-APO(a)-LRx Atherosclerosis I Phase II - Study to Start 118122 Inc. Initiation II trial for high Lp(a) with calcific aortic valve stenosis in the second half of 2016. Ionis anticipates initiating a Phase II trial of APO(a)-LRx for Ionis Pharmaceuticals, Trial Announcement - very high Lp(a) with recurring CVD in mid-2016 and a Phase 07/01/2016-12/31/2016 IONS IONIS-APO(a)-LRx Atherosclerosis I Phase II - Study to Start 118124 Inc. Initiation II trial for high Lp(a) with calcific aortic valve stenosis in the second half of 2016.

Ionis Pharmaceuticals, Dyslipidemia / Trial Announcement - Ionis anticipates enrolling the first patients in an extension 01/11/2016-03/31/2016 IONS Volanesorsen III Phase III - OLE Study to Start 118107 Inc. Hypercholesterolemia Initiation study of volanesorsen for FCS in the first quarter of 2016.

Ionis Pharmaceuticals, Dyslipidemia / Trial Announcement - Ionis announced that it anticipates initiating a pediatric study 07/01/2016-09/30/2016 IONS Volanesorsen III Pediatric Study to Start 118114 Inc. Hypercholesterolemia Initiation in FCS in the third quarter of 2016. Trial Announcement - Ionis announced that patient enrollment for the Phase II/II Ionis Pharmaceuticals, Phase II/III BROADEN - Pt. 01/01/2017-06/30/2017 IONS Volanesorsen Lipodystrophy II/III Patient Enrollment BROADEN study is expected to be completed in the first half 118121 Inc. Enrollment Complete Completed of 2017.

Ionis Pharmaceuticals, Trial Announcement - Ionis announced that it plans to initiate an extension study 10/01/2016-12/31/2016 IONS Volanesorsen Lipodystrophy II/III Phase III - OLE Study to Start 118115 Inc. Initiation of volanesorsen in FPL in the fourth quarter of 2016.

Ironwood announced that the Company is finalizing Ironwood Gastroesophageal Reflux Trial Data - Top-Line preparations for the IW-3718 dose-ranging Phase IIb study 01/01/2017-12/31/2017 IRWD IW-3718 II Phase IIb - Top-Line Results 118007 Pharmaceuticals, Inc. Disease (GERD) Results for the potential treatment of refractory GERD. The study is expected to read out in 2017.

Ironwood Pharmaceuticals announced that the Phase IIb study of linaclotide colonic release formulations for the Ironwood Linaclotide Colonic Trial Announcement - 01/01/2017-12/31/2017 IRWD Irritable Bowel Syndrome (IBS) IIb Phase III Trial to Start treatment of irritable bowel syndrome with constipation is 118000 Pharmaceuticals, Inc. Delivery Initiation expected to release data in the second half of 2016. A Phase III trial is expected to begin in 2017.

Sinusoidal Obstruction Trial Announcement - Prevention of VOD Trial to Jazz plans to have the first patient treated in its prevention 07/01/2016-09/30/2016 Jazz Pharmaceuticals plc JAZZ Defibrotide Syndrome (Veno-Occlusive NDA 117961 Initiation Start of VOD study of Defibrotide in the third quarter of 2016. Disease / VOD) Human Abuse Liability Data Jazz expects to have human abuse liability data results on 04/01/2016-09/30/2016 Jazz Pharmaceuticals plc JAZZ JZP-110 Narcolepsy III Trial Data - Other 117962 Results JZP-110 in mid-2016. Jazz expects to complete enrollment and have preliminary Trial Data - Top-Line 10/01/2016-12/31/2016 Jazz Pharmaceuticals plc JAZZ JZP-110 Narcolepsy III Phase III - Top-Line Results data results from its Phase III program of JZP-110 in 117958 Results narcolepsy in the fourth quarter of 2016.

Jazz expects to complete enrollment and have preliminary Trial Data - Top-Line 10/01/2016-12/31/2016 Jazz Pharmaceuticals plc JAZZ JZP-110 Sleep Apnea III Phase III - Top-Line Results data results from its Phase III program of JZP-110 in 117959 Results obstructive sleep apnea in the fourth quarter of 2016.

Trial Announcement - Phase III Children and Jazz expects to complete enrollment of its Phase III trial in 07/01/2016-12/31/2016 Jazz Pharmaceuticals plc JAZZ Xyrem Narcolepsy Approved Patient Enrollment Adolescents - Patient pediatric patients who have narcolepsywith cataplexy in the 117956 Completed Enrollment Completed second half of 2016. Genmab expects to report results from the Phase III CASTOR Trial Data - Top-Line Phase III CASTOR - Topline 01/14/2016-12/31/2016 Johnson & Johnson JNJ Darzalex Multiple Myeloma (MM) Approved trial of Darzalex in relapsed/ refractory multiple myeloma in 118468 Results Results 2016. Genmab expects to announce results from the Phase III Trial Data - Top-Line Phase III MMY3003 - Topline 01/14/2016-12/31/2016 Johnson & Johnson JNJ Darzalex Multiple Myeloma (MM) Approved Pollux study of Darzalex in relapsed/ refractory multiple 118470 Results Results myeloma patients in 2016. MorphoSys announced that data from the three pivotal Trial Data - Top-Line Phase III - NAVIGATE - Top-Line 04/01/2016-09/30/2016 Johnson & Johnson JNJ Guselkumab Psoriasis III studies of guselkumab for psoriasis are expected in mid- 118440 Results Data 2016. MorphoSys announced that data from the three pivotal Trial Data - Top-Line Phase III - VOYAGE 1 - Top-Line 04/01/2016-09/30/2016 Johnson & Johnson JNJ Guselkumab Psoriasis III studies of guselkumab for psoriasis are expected in mid- 118441 Results Data 2016. MorphoSys announced that data from the three pivotal Trial Data - Top-Line Phase III - VOYAGE 2 - Top-Line 04/01/2016-09/30/2016 Johnson & Johnson JNJ Guselkumab Psoriasis III studies of guselkumab for psoriasis are expected in mid- 118442 Results Results 2016. Karyopharm expects to reach the recommended Phase II Trial Data - Top-Line dose for KPT-8602 by the end of 2016. As such we expect 09/01/2016-12/31/2016 Karyopharm Therapeutics KPTI KPT-8602 Multiple Myeloma (MM) Preclinical Phase I - Top-Line Results 118346 Results results for the Phase I study of KPT-8602 within this time frame.

Karyopharm expects to reach the recommended Phase II Acute Myelogenous Leukemia Trial Data - Top-Line 04/01/2017-09/30/2017 Karyopharm Therapeutics KPTI KPT-9274 Preclinical Phase I - Top-Line Results dose for KPT-9274 by mid-2017. As such we expect results 118348 (AML) Results for the Phase I study of KPT-9274 within this time frame.

Acute Myelogenous Leukemia Regulatory - MAA Karyopharm expects to file an MAA for Selinexor in AML in 01/01/2017-06/30/2017 Karyopharm Therapeutics KPTI Selinexor II MAA Filing 118330 (AML) Submission (Europe) the first half of 2017.

Chronic Lymphocytic Leukemia Karyopharm expects final results for the Phase II SIRRT study 09/01/2017-12/31/2017 Karyopharm Therapeutics KPTI Selinexor (CLL)/Small Cell Lymphocytic II Trial Data - Final Results Phase II SIRRT - Final Results 118349 of Selinexor for Richter's Transformation by the end of 2017. Lymphoma (SLL) - NHL

Diffuse Large B-Cell Lymphoma Regulatory - MAA Karyopharm expects to file an MAA for Selinexor in DLBCL in 04/01/2017-09/30/2017 Karyopharm Therapeutics KPTI Selinexor II MAA Filing 118331 (DLBCL) - NHL Submission (Europe) mid-2017. Diffuse Large B-Cell Lymphoma Karyopharm reported that final results for the SADAL study 04/01/2017-09/30/2017 Karyopharm Therapeutics KPTI Selinexor II Trial Data - Final Results Phase II SADAL - Final Results 118341 (DLBCL) - NHL of Selinexor are expected in mid-2017.

Trial Data - Top-Line Phase II SCORE - Top-Line Karyopharm reported that an interim analysis for the Phase 04/01/2017-09/30/2017 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb 118320 Results Results II SCORE study of Selinexor for MM is expected in mid-2017.

Karyopharm plans to file an MAA/NDA for Selinexor in MM Regulatory - MAA 07/01/2017-12/31/2017 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb MAA Filing in the second half of 2017 based on results of the STORM 118325 Submission (Europe) study.

Phase IIb STORM - Final Karyopharm reported that final data for the STORM study of 07/01/2017-12/31/2017 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb Trial Data - Final Results 118339 Results Selinexor is expected in the second half of 2017.

Regulatory - sNDA/sBLA Karyopharm plans to file an MAA/NDA for Selinexor in MM 04/01/2018-09/30/2018 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb sNDA Filing 118327 Filing in mid-2018 based on results of the SCORE study. Regulatory - Karyopharm plans to file an MAA/NDA for Selinexor in MM 04/01/2018-09/30/2018 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb Supplemental Filing Supplemental EU Filing 118328 in mid-2018 based on results of the SCORE study. (Europe) Trial Data - Top-Line Phase II/III SEAL - Top-Line Karyopharm expects the Phase II/IIII SEAL study of Selinexor 10/01/2016-12/31/2016 Karyopharm Therapeutics KPTI Selinexor Sarcoma II/III 118332 Results Results to read out in the fourth quarter of 2016. Acute Lymphocytic Leukemia Trial Data - Top-Line Phase I/II - ZUMA-3 - Top-Line Kite expects data from the Phase I/II ZUMA-3 study of KTE- 01/01/2017-12/31/2017 Kite Pharma, Inc. KITE KTE-C19 I/II 118456 (ALL) Results Results C19 for ALL in adults in 2017. Acute Lymphocytic Leukemia Trial Data - Top-Line Phase I/II - ZUMA-4 - Top-Line Kite expects data from the Phase I/II ZUMA-3 study of KTE- 01/01/2017-12/31/2017 Kite Pharma, Inc. KITE KTE-C19 I/II 118457 (ALL) Results Results C19 for ALL in pediatric patients in 2017.

Biomedtracker August 2016 / 120 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Diffuse Large B-Cell Lymphoma Trial Announcement - European Clinical Studies to Kite intends to initiate KTE-C19 clinical studies in Europe in 01/14/2016-12/31/2016 Kite Pharma, Inc. KITE KTE-C19 I/II 118478 (DLBCL) - NHL Initiation Start 2016. Diffuse Large B-Cell Lymphoma Trial Data - Updated Phase I/II - ZUMA-1 - Primary Kite expects primary data from the Phase I/II ZUMA-1 study 01/01/2017-12/31/2017 Kite Pharma, Inc. KITE KTE-C19 I/II 118453 (DLBCL) - NHL Results Data of KTE-C19 in 2017. Trial Data - Top-Line Phase II - ZUMA-2 - Top-Line Kite expects data from the Phase II ZUMA-2 study of KTE-C19 01/01/2017-12/31/2017 Kite Pharma, Inc. KITE KTE-C19 Mantle Cell Lymphoma - NHL II 118455 Results Data for MCL in 2017. MannKind has announced that they are currently seeking a 01/13/2016-12/31/2016 MannKind Corporation MNKD Afrezza Diabetes Mellitus, Type I Approved Partnership - New New Partnership new partner for Afrezza. We await an update in the time 118408 frame above. MannKind has announced that they are currently seeking a 01/13/2016-12/31/2016 MannKind Corporation MNKD Afrezza Diabetes Mellitus, Type II Approved Partnership - New New Partnership new partner for Afrezza. We await an update in the time 118409 frame above.

MannKind announced that they are pursuing development of an inhaled version of Treprostinil using their proprietary Pulmonary Arterial Technosphere technology. They have currently tested three 10/01/2016-12/31/2016 MannKind Corporation MNKD Treprostinil (MannKind) Preclinical Regulatory - IND Filing IND Filing 118411 Hypertension (PAH) formulations and anticipate conducting IND enabling studies throughout 2016 with the aim of submitting an IND in the fourth quarter of 2016.

Medivation announced that they anticipate initiating a Trial Announcement - 01/11/2016-12/31/2016 Medivation, Inc. MDVN Talazoparib Breast Cancer III Phase III - Study to Start pivotal study of talazoparib for breast cancer beyond gBRCA 118077 Initiation mutations in 2016. Trial Announcement - Medivation anticipates initiating a Phase II study of 01/11/2016-12/31/2016 Medivation, Inc. MDVN Talazoparib Ovarian Cancer I Phase II - Study to Start 118078 Initiation talazoparib for late stage ovarian cancer in 2016. Medivation anticipates that pivotal studies in multiple other Trial Announcement - 01/11/2016-12/31/2016 Medivation, Inc. MDVN Talazoparib Small Cell Lung Cancer (SCLC) I Pivotal Trial to Start indications (non-BRCA breast prostate lung) for talazoparib 118032 Initiation will begin in 2016.

Merck announced they have submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration for KEYTRUDA for NSCLC based on Non-Small Cell Lung Cancer Regulatory - PDUFA for PDUFA for sBLA - KEYNOTE- 10/24/2016-10/31/2016 Merck & Co., Inc. MRK Keytruda Approved findings from KEYNOTE-010 at the end of 2015. Based on a 118090 (NSCLC) sNDA/sBLA 010 standard 10-month supplemental BLA filing under PDUFA V guidelines the PDUFA decision should occur in late October 2016.

Trial Announcement - First Line MCC and RCC - Trials Merck KGaA plans to initiate an additional clinical study of 01/11/2016-12/31/2016 Merck KGaA MKGAY Avelumab Merkel Cell Carcinoma II 117731 Initiation to Start Avelumab in first line MCC and RCC in 2016.

Trial Announcement - Merck KGaA announced that expansion cohorts of the Phase 07/01/2016-12/31/2016 Merck KGaA MKGAY M7824 Solid Tumors I Expansion Cohorts to Start 117736 Other I study of M7824 will begin in the second half of 2016.

Hepatocellular (Liver) Cancer Merck KGaA announced that Phase II data in hepatocellular Trial Data - Top-Line 07/01/2016-12/31/2016 Merck KGaA MKGAY Tepotinib (HCC) (including secondary I/II Phase II - Top-Line Results carcinoma for Teporinib are expected in the second half of 117727 Results metastases) 2016.

Merrimack Trial Data - Top-Line Merrimack expects to report results from the Phase I study 01/12/2016-12/31/2016 MACK Onivyde Breast Cancer - Imaging I Phase I - Top-Line Results 118253 Pharmaceuticals, Inc. Results of Onivyde for imaging of breast cancer in 2016.

MorphoSys expects to release updated data from the Phase Trial Data - Updated Phase I/IIa - Updated Data at 06/03/2016-06/07/2016 MorphoSys AG MOR:GR MOR202 Multiple Myeloma (MM) I/II I/IIa trial of MOR202 for multiple myeloma (MM) at ASCO 118445 Results ASCO 2016. Phase II - Combo Safety Study Diffuse Large B-Cell Lymphoma Trial Announcement - MorphoSys expects a Phase II combo safety study of 04/01/2016-09/30/2016 MorphoSys AG MOR:GR MOR208 II w/Bendamustine - Trial to 118443 (DLBCL) - NHL Initiation MOR208 and bendamustine for DLBCL will start in mid-2016. Start

Respiratory Syncytial Virus Trial Data - Top-Line Phase II Eldery Rollover Trial - Data from the Phase II rollover trial of the RSV F vaccine in 07/01/2016-12/31/2016 Novavax, Inc. NVAX RSV F Vaccine (Novavax) III 118426 (RSV) Results Top-Line Results older adults is expected in the second half of 2016.

Respiratory Syncytial Virus Regulatory - NDA/BLA Novavax intends to submit a Biologics License Application 01/01/2017-06/30/2017 Novavax, Inc. NVAX RSV F Vaccine (Novavax) III BLA Filing 118424 (RSV) Filing (BLA) for its RSV F vaccine during the first half of 2017.

OncoMed Trial Data - Updated OncoMed announced that brontictuzumab hematology data 07/01/2016-12/31/2016 OMED Brontictuzumab Hematologic Cancer I Phase I - Updated Results 118498 Pharmaceuticals, Inc. Results will be available in the second half of 2016.

Opko expects to commence a Phase IIa study of its long- Hemophilia A and B - General Trial Announcement - Now-03/31/2016 Opko Health OPK Factor VIIa-CTP II Phase IIa to Start acting Factor VIIa-CTP for hemophilia A and B during the first 118264 Clotting Products Initiation quarter of 2016. Opko expects to launch Rayaldee in the early part of the Hyperparathyroidism Progress Update - 07/01/2016-09/30/2016 Opko Health OPK Rayaldee NDA US Product Launch second half of 2016. We await an update in the third 118263 (Secondary) Product Launch quarter.

Autism Spectrum Disorders Development Trial Data - Top-Line OptiNose announced that the Phase IIa trial for OPN300 for 01/11/2016-06/30/2016 OptiNose AS OPN300 Phase IIa - Top-Line Results 118095 (Autism) Outside U.S. Results autism is expected to readout in the first half of 2016.

OptiNose announced that the New Drug Application (NDA) Regulatory - NDA/BLA 04/01/2016-09/30/2016 OptiNose AS OPN-375 Nasal Polyposis III NDA Filing for OPN-375 for CRS with nasal polyps is expected in mid- 118093 Filing 2016. A 10-month PDUFA review period is expected.

OptiNose announced that the Phase III trial for OPN-375 for Trial Announcement - Phase III - Chronic Sinusitis 07/01/2016-12/31/2016 OptiNose AS OPN-375 Nasal Polyposis III chronic sinusitis is expected to begin in the second half of 118094 Initiation Trial to Start 2016. Otonomy expects Phase III results for OTO-104 in the second Regulatory - NDA/BLA 01/01/2018-06/30/2018 Otonomy, Inc. OTIC OTO-104 Meniere's Disease III NDA Filing half of 2017 and if positive an NDA submission in the first 118257 Filing half of 2018. ProNAi announced that they expect to present results from Chronic Lymphocytic Leukemia the Phase II Brighton trial of PNT2258 for Richter's Trial Data - Top-Line Phase II - BRIGHTON Top-Line 12/03/2016-12/06/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 (CLL)/Small Cell Lymphocytic II transformation at the American Society of Hematology 118454 Results results at ASH Lymphoma (SLL) - NHL Annual Meeting which will take place from December 3-6 2016. ProNAi announced that they anticipate presenting results from the Phase II Wolverine trial of PNT2258 for Diffuse Large B-Cell Lymphoma Trial Data - Top-Line Phase II Wolverine - Top-Line 06/03/2016-06/07/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 II relapsed/refractory 3rd-line DLBCL at the 2016 American 118458 (DLBCL) - NHL Results Results at ASCO Society of Clinical Oncology meeting which will take place from June 3-7 2016.

ProNAi announced that they anticipate initiating the Phase II Diffuse Large B-Cell Lymphoma Trial Announcement - Phase II GRANITE - Trial to GRANITE clinical trial of PNT2258 for the treatment of 04/01/2016-09/30/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 II 118466 (DLBCL) - NHL Initiation Start relapsed/refractory DLBCL patients who are transplant ineligible and who are also receiving BR in mid-2016.

Puma expects top-line results for the PB272 Phase III trial for Trial Data - Top-Line Phase III NALA - Top-Line 10/01/2016-04/30/2017 Puma Biotechnology, Inc. PBYI Neratinib Breast Cancer III third line HER2+ MBC (PUMA-NER-1301) in late 2016 or early 117949 Results Results 2017. Puma expects top-line results from the Phase II study of Trial Data - Top-Line Phase II HER2+ w/Loperamide - Now-06/30/2016 Puma Biotechnology, Inc. PBYI Neratinib Breast Cancer III neratinib with loperamide prophylaxis for HER2+ breast 117948 Results Top-Line Results cancer in the first half of 2016. Puma expects to report results for the Phase II FB-7 Trial Data - Updated Phase II NSABP FB-7 - Updated Now-06/30/2016 Puma Biotechnology, Inc. PBYI Neratinib Breast Cancer III neoadjuvant HER2+ breast cancer trial using MammaPrint in 117957 Results Results the first half of 2016. Radius expects to readout data from the human replicate 04/01/2016-09/30/2016 Radius Health, Inc. RDUS Abaloparatide-TD Osteoporosis / Osteopenia II Trial Data - Other Human Replicate Study Data 117986 study of abaloparatide-TD in mid-2016. Trial Announcement - Phase IIb Vasomotor Radius expects to complete enrollment of its Phase IIb Menopause (including Hormone 10/01/2016-12/31/2016 Radius Health, Inc. RDUS RAD1901 II Patient Enrollment Symptoms - Patient Enrollment Vasomotor Symptom study of RAD1901 in the fourth quarter 117987 Replacement Therapy [HRT]) Completed Completed of 2016.

Biomedtracker August 2016 / 121 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Regeneron expects to initiate a Phase III study of Dupilumab Regeneron Trial Announcement - Phase III Pediatric Study to Now-06/30/2016 REGN Dupilumab Atopic Dermatitis (Eczema) III in pediatric patients with atopic dermatitis in the first half of 118302 Pharmaceuticals, Inc. Initiation Start 2016. Regeneron expects to have data from its Phase II pediatric Regeneron Trial Data - Top-Line Phase IIa Pediatric - Top-Line Now-06/30/2016 REGN Dupilumab Atopic Dermatitis (Eczema) III study of dupilumab in patients with atopic dermatitis in the 118303 Pharmaceuticals, Inc. Results Results first half of 2016. Regeneron Diabetic Retinopathy Trial Announcement - Regeneron plans to initiate a Phase III study of Eylea in Now-03/31/2016 REGN Eylea Approved Phase III to Start 118287 Pharmaceuticals, Inc. (Ophthalmology) Initiation diabetic retinopathy in the first quarter of 2016.

Regeneron expects that the Diabetic Retinopathy Clinical Regeneron Diabetic Retinopathy Trial Announcement - Now-04/30/2016 REGN Eylea Approved Protocol-W Trial to Start Research Network's Protocol-W study of Eylea in patients 118290 Pharmaceuticals, Inc. (Ophthalmology) Initiation with diabetic retinopathy is expected to begin in early 2016.

Regeneron expects to have data from the 16 week Phase Regeneron Trial Data - Top-Line Now-06/30/2016 REGN Fasinumab Chronic Pain II/III Phase II/III - Top-Line Results II/III study of Fasinumab in patients with osteoarthritis pain 118314 Pharmaceuticals, Inc. Results in the first half of 2016.

Based on discussions with the FDA Regeneron intends to Regeneron Trial Announcement - Now-12/31/2016 REGN Fasinumab Chronic Pain II/III Phase III Trials to Start intiaite Phase III trials (>16 weeks) of Fasinumab in patietns 118312 Pharmaceuticals, Inc. Initiation with chronic pain in the first half of 2016.

Sanofi announced that a second interim analysis of Praluent Regeneron Dyslipidemia / Trial Data - Updated Phase III ODYSSEY Outcomes - from the Phase III ODYSSEY OUTCOMES study will be 07/01/2016-12/31/2016 REGN Praluent Approved 118190 Pharmaceuticals, Inc. Hypercholesterolemia Results Second Interim Results released in the second half of 2016. This analysis will include futility and efficacy when ~75% of events have occurred.

Sanofi announced that a final data set of Praluent from the Regeneron Dyslipidemia / Phase III ODYSSEY Outcomes - 07/01/2017-12/31/2017 REGN Praluent Approved Trial Data - Final Results Phase III ODYSSEY OUTCOMES study is expected in the 118185 Pharmaceuticals, Inc. Hypercholesterolemia Final Results second half of 2017. Regeneron Trial Data - Top-Line Regeneron expects to have initial data from its CD20-CD3 Now-12/31/2016 REGN REGN1979 Hematologic Cancer I Phase I - Top-Line Results 118319 Pharmaceuticals, Inc. Results bispecific program in 2016. Wet Age-Related Macular Regeneron expects to have topline data from its Phase II Regeneron Trial Data - Top-Line 10/01/2016-12/31/2016 REGN REGN2176-3 Degeneration (Wet AMD) II Phase II - Top-Line Results data of Eylea in combination with PDGFR-beta by year end 118292 Pharmaceuticals, Inc. Results (Ophthalmology) 2016. Trial Announcement - Regeneron expects to complete enrollment of its Phase III Regeneron Respiratory Syncytial Virus Phase III NURSERY-Pre-term - 01/01/2017-12/31/2017 REGN REGN2222 III Patient Enrollment NURSERY-Pre-term study of REGN2222 in infants with RSV in 118316 Pharmaceuticals, Inc. (RSV) Patient Enrollment Completed Completed 2017.

Regeneron Trial Data - Top-Line Regeneron expects to have initial data from its PD-1 Now-06/30/2016 REGN REGN2810 Cancer I Phase I - Top-Line Results 118317 Pharmaceuticals, Inc. Results antibody collaboration with Sanofi in the first half of 2016.

Regeneron expects to initiate Phase II studies of Eylea in Regeneron Other Ophthalmological Trial Announcement - Now-06/30/2016 REGN REGN910-3 I Phase II Studies to Start combination with an ANG2 antibody in Wet AMD and 118294 Pharmaceuticals, Inc. Indications (Ophthalmology) Initiation Diabetic macular edema in the first half of 2016.

Rigel plans to initiate a Phase II study of fostamatinib for the Rigel Pharmaceuticals, Trial Announcement - 01/14/2016-03/31/2016 RIGL Fostamatinib Anemia Preclinical Phase II AIHI - Trial to Start treatment of autoimmune hemolytic anemia in the first 118475 Inc. Initiation quarter of 2016.

Rigel plans to release stage 1 results from a Phase II study of Rigel Pharmaceuticals, Trial Data - Top-Line 10/01/2016-12/31/2016 RIGL Fostamatinib Anemia Preclinical Phase II AIHI - Stage 1 Results fostamatinib for the treatment of autoimmune hemolytic 118476 Inc. Results anemia in the fourth quarter of 2016.

Rigel announced that they plan to file an IND for their IRAK Rigel Pharmaceuticals, Acute Myelogenous Leukemia 01/14/2016-12/31/2016 RIGL IRAK Inhibitor Program Preclinical Regulatory - IND Filing IND Filing inhibitor for the treatment of acute myeloid leukemia (AML) 118481 Inc. (AML) and myelodysplastic syndrome (MDS) in 2016.

Rigel announced that they plan to start clinical trials for their Rigel Pharmaceuticals, Acute Myelogenous Leukemia Trial Announcement - 01/14/2016-12/31/2016 RIGL IRAK Inhibitor Program Preclinical Clinical Trials to Start IRAK inhibitor for the treatment of acute myeloid leukemia 118482 Inc. (AML) Initiation (AML) and myelodysplastic syndrome (MDS) in 2016.

Roche announced that it has initiated a rolling NDA for the Regulatory - Rolling approval of Atezolizumab for the treatment of bladder 01/12/2016-06/30/2016 Roche Holding AG RHHBF Atezolizumab Bladder Cancer NDA Rolling NDA Completion 118193 NDA/BLA Completion cancer in the US. We await on the completion of the rolling NDA submission through the first half of 2016.

Roche announced that it has initiated a rolling NDA for the Non-Small Cell Lung Cancer Regulatory - Rolling approval of Atezolizumab for the treatment of NSCLC in the 01/12/2016-06/30/2016 Roche Holding AG RHHBF Atezolizumab NDA Rolling NDA Completion 118174 (NSCLC) NDA/BLA Completion US. We await on the completion of the rolling NDA submission through the first half of 2016.

Biosimilar Adalimumab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Rheumatoid Arthritis (RA) III BLA Filing 118106 (Samsung Bioepis) Filing biosimilar Humira.

Biosimilar Etanercept Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Ankylosing Spondylitis III BLA Filing 118118 (Samsung Bioepis) Filing biosimilar Enbrel. Biosimilar Etanercept Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Psoriasis III BLA Filing 118117 (Samsung Bioepis) Filing biosimilar Enbrel. Biosimilar Etanercept Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Psoriatic Arthritis (PA) III BLA Filing 118119 (Samsung Bioepis) Filing biosimilar Enbrel. Biosimilar Etanercept Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Rheumatoid Arthritis (RA) III BLA Filing 118116 (Samsung Bioepis) Filing biosimilar Enbrel. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Ankylosing Spondylitis I BLA Filing 118111 (Samsung Bioepis) Filing biosimilar Remicade. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Crohn's Disease I BLA Filing 118110 (Samsung Bioepis) Filing biosimilar Remicade. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Psoriasis I BLA Filing 118109 (Samsung Bioepis) Filing biosimilar Remicade. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Psoriatic Arthritis (PA) I BLA Filing 118113 (Samsung Bioepis) Filing biosimilar Remicade. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Rheumatoid Arthritis (RA) III BLA Filing 118108 (Samsung Bioepis) Filing biosimilar Remicade. Biosimilar Infliximab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Ulcerative Colitis (UC) I BLA Filing 118112 (Samsung Bioepis) Filing biosimilar Remicade.

Biosimilar Trastuzumab Regulatory - NDA/BLA Merck announced that a filing is anticipated in 2016 for 01/11/2016-12/31/2016 Samsung Bioepis Co., Ltd. Breast Cancer III BLA Filing 118105 (Samsung Bioepis) Filing biosimilar Herceptin.

Mucopolysaccharidosis I (MPS I; Trial Announcement - Sangamo plans to initiate the Phase I/II SB-318-1502 clinical Now-12/31/2016 Sangamo Biosciences, Inc. SGMO SB-318 Preclinical Phase I/II SB-318-1502 to Start 118232 Hurler Syndrome) Initiation trial of SB-318 for the treatment of Hurler syndrome in 2016.

Sangamo expects to have data from an additional 5 subjects Trial Data - Updated Phase I/II 1101 - Updated 07/01/2016-12/31/2016 Sangamo Biosciences, Inc. SGMO SB-728-T HIV / AIDS II in Cohort 3 from its Phase II SB-728-1101 study in the second 118230 Results Results half of 2016. Trial Data - Top-Line Sangamo expects to have initial data from its Phase I studies 10/01/2016-12/31/2016 Sangamo Biosciences, Inc. SGMO SB-FIX Hemophilia B IND Phase I/II - Top-Line Results 118227 Results of SB-FIX by the end of 2016. Sangamo plans to file an IND for SB-LSD4 in the second half 07/01/2016-12/31/2016 Sangamo Biosciences, Inc. SGMO SB-LSD4 Fabry's Disease Preclinical Regulatory - IND Filing IND Filing 118225 of 2016. Sanofi announced that they expect to initiate a Phase III trial Trial Announcement - 04/01/2016-06/30/2016 Sanofi SNY GZ402666 Pompe Disease II/III Phase III - Trial to Start of NeoGAA for the treatment of Pompe disease in the 118186 Initiation second quarter of 2016.

Biomedtracker August 2016 / 122 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Sanofi announced that they expect to initiate a Phase III Trial Announcement - 10/01/2016-12/31/2016 Sanofi SNY Isatuximab Multiple Myeloma (MM) I/II Phase III - Trial to Start study of isatuximab for the treatment of multiple myeloma 118189 Initiation (MM) in the fourth quarter of 2016.

Diffuse Large B-Cell Lymphoma Trial Data - Updated Phase II w/RCHOP or RCHP - Seattle Genetics expects to report Phase II data of Adcetris in 01/11/2016-12/31/2016 Seattle Genetics, Inc. SGEN Adcetris II 118035 (DLBCL) - NHL Results Updated Results 2016 for the treatment of frontline and relapsed DLBCL.

Seattle Genetics announced that the Company expects to Acute Myelogenous Leukemia Trial Data - Updated Phase I Dose Escalation - 01/11/2016-12/31/2016 Seattle Genetics, Inc. SGEN SGN-CD33A I/II report additional data in 2016 from the Phase I trial of SGN- 118048 (AML) Results Updated Results CD33A in combination with HMAs.

Seattle Genetics plans on reporting data in 2016 from the Acute Myelogenous Leukemia Trial Data - Top-Line Phase Ib w/Cytarabine & 01/11/2016-12/31/2016 Seattle Genetics, Inc. SGEN SGN-CD33A I/II Phase I trial of SGN-CD3A in combination with cytarabine 118050 (AML) Results Daunorubicin - Topline Results and daunorubicin.

Seattle Genetics announced that the Company plans to Acute Myelogenous Leukemia Trial Announcement - 01/11/2016-12/31/2016 Seattle Genetics, Inc. SGEN SGN-CD33A I/II Phase I/II MDS - Trial to Start initiate a Phase I/II study of SGN-CD33A in frontline MDS. We 118064 (AML) Initiation await an update in the time frame above.

Seattle Genetics announced that the Company expects to Acute Myelogenous Leukemia Trial Announcement - Phase III Older AML - Trial to 07/01/2016-09/30/2016 Seattle Genetics, Inc. SGEN SGN-CD33A I/II initiate a pivotal Phase III trial of SGN-CD33A in older AML by 118043 (AML) Initiation Start the third quarter of 2016. Shire reports that Phase III top-line data from their Japan Shire Pharmaceuticals Trial Data - Top-Line Phase III Japan - Top-Line 04/01/2016-06/30/2016 SHPG Firazyr Hereditary Angioedema (HAE) Approved study of Hereditary Angioedema is expected to be available 118280 Group PLC Results Results in the second quarter of 2016. Shire Pharmaceuticals Progress Update - Shire expects a U.S. product launch of Lifitegrast in Dry Eye 07/01/2016-09/30/2016 SHPG Lifitegrast Dry Eye (Ophthalmology) III U.S. Product Launch 118276 Group PLC Product Launch (U.S.) in the third quarter of 2016. Shire reports that top-line results for the Phase III pediatric Shire Pharmaceuticals Attention Deficit Hyperactivity Trial Data - Top-Line Phase III Study 305 - Top-Line Now-03/31/2016 SHPG SHP-465 III trial of SHP-465 for ADHD is expected in the first quarter of 118279 Group PLC Disorder (ADHD) Results Results 2016. Trial Announcement - Shire expects the SHP607 Phase II Retinopathy of Shire Pharmaceuticals Other Retinopathy Phase II ROP - Patient Now-03/31/2016 SHPG SHP607 II Patient Enrollment Prematurity trial to be fully enrolled in the first quarter of 118278 Group PLC (Ophthalmology) Enrollment Completion Completed 2016. Other Congenital Blindness Trial Data - Top-Line Phase I/II Choroideremia - Spark Therapeutics expects first efficacy readout for SPK- 07/01/2016-12/31/2016 Spark Therapeutics, LLC ONCE SPK-CHM I/II 118085 Indications Results Topline Results CHM in the second half of 2016. Spark Therapeutics expects to announce initial efficacy data Trial Data - Top-Line 04/01/2016-09/30/2016 Spark Therapeutics, LLC ONCE SPK-FIX Hemophilia B I/II Phase I/II - Topline Results from a Phase I/II study of SPK-FIX for the trewatment of 118096 Results hemophilia B in mid-2016. Spark Therapeutics expects to file an IND for SPK-FVIII in the Trial Data - Top-Line 01/01/2017-06/30/2016 Spark Therapeutics, LLC ONCE SPK-FVIII Hemophilia A Preclinical Clinical Trial Results second half of 2016 with initial efficacy data in the first half 118102 Results of 2017. Spark Therapeutics expects to file an IND for SPK-FVIII in the 07/01/2016-12/31/2016 Spark Therapeutics, LLC ONCE SPK-FVIII Hemophilia A Preclinical Regulatory - IND Filing IND Filing second half of 2016 with initial efficacy data in the first half 118101 of 2017.

Leber's Congenital Amaurosis Regulatory - MAA Spark Therapeutics expects to complete a MAA filing in early 01/01/2017-04/30/2017 Spark Therapeutics, LLC ONCE SPK-RPE65 III MAA Filing 118120 (Ophthalmology) Submission (Europe) 2017.

Spark Therapeutics announced that the Company expects to Leber's Congenital Amaurosis Trial Data - Top-Line 04/01/2016-06/30/3016 Spark Therapeutics, LLC ONCE SPK-RPE65 III Phase III 302 - Topline Results announce data from a Phase III 302 study (Phase III 118123 (Ophthalmology) Results crossover subjects) in the second quarter of 2016.

Leber's Congenital Amaurosis Trial Data - Updated Spark Therapeutics expects to present 2-year data form the 07/01/2016-09/30/2016 Spark Therapeutics, LLC ONCE SPK-RPE65 III Phase III 301 - Updated Results 118092 (Ophthalmology) Results Phase III 301 study of SPK-RPE65 in third quarter of 2016.

Spark Therapeutics expects to announce additional Leber's Congenital Amaurosis Trial Data - Updated Phase I/II 102 - Updated 10/01/2016-12/31/2016 Spark Therapeutics, LLC ONCE SPK-RPE65 III durability data from the Phase I/II 102 cohort (4 year data) in 118091 (Ophthalmology) Results results the fourth quarter of 2016.

Leber's Congenital Amaurosis Regulatory - Meeting with Spark Therapeutics announced that the Company has Now-03/31/2016 Spark Therapeutics, LLC ONCE SPK-RPE65 III Meeting with FDA 118128 (Ophthalmology) FDA scheduled a Pre-BLA meeting with the FDA in 1Q 2016

Spark Therapeutics expects an IND filing for SPK-TPP1 in the Neuronal Ceroid Lipofuscinosis Trial Data - Top-Line 01/01/2017-06/30/2017 Spark Therapeutics, LLC ONCE SPK-TPP1 Preclinical Phase I Study - Topline results second half of 2016 with initial efficacy data in the first half 118104 (NCL) Results of 2017. Spark Therapeutics expects an IND filing for SPK-TPP1 in the Neuronal Ceroid Lipofuscinosis 07/01/2016-12/31/2016 Spark Therapeutics, LLC ONCE SPK-TPP1 Preclinical Regulatory - IND Filing IND Filing second half of 2016 with initial efficacy data in the first half 118103 (NCL) of 2017. Stemline expects to report data from their Phase I/II clinical Stemline Therapeutics, Acute Myelogenous Leukemia Trial Data - Top-Line Phase I/II Pts Who Achieved CR 04/01/2016-09/30/2016 STML SL-401 I/II study of SL-401 in acute myelogenous leukemia (in CR 118499 Inc. (AML) Results with MRD - Topline Results patients) in mid-2016. Stemline announced that the Company plans to report data Stemline Therapeutics, Hypereosinophilic syndrome Trial Data - Top-Line 07/01/2016-12/31/2016 STML SL-401 I/II Phase I/II - Topline Results from the the Phase I/II MPN study of SL-401 in the second 118509 Inc. (HES) Results half of 2016. Stemline announced that the Company plans to report data Stemline Therapeutics, Myelodysplastic Syndrome Trial Data - Top-Line Phase I/II HES/MF/CMML - 07/01/2016-12/31/2016 STML SL-401 I/II from the the Phase I/II MPN study of SL-401 in the second 118510 Inc. (MDS) Results Topline Results half of 2016. Stemline announced that the Company plans to report data Stemline Therapeutics, Trial Data - Top-Line 07/01/2016-12/31/2016 STML SL-401 Myelofibrosis (MF) I/II Phase I/II - Topline Results from the the Phase I/II MPN study of SL-401 in the second 118497 Inc. Results half of 2016. Stemline announced plans to initiate enrollment for clinical Stemline Therapeutics, Trial Announcement - Phase I Hematology - Trials to 07/01/2016-12/31/2016 STML SL-801 Cancer IND studies of SL-801 in advanced heme tumors in the second 118500 Inc. Initiation Start half of 2016. Stemline announced plans to report topline results for their Stemline Therapeutics, Trial Data - Top-Line Phase I Solid Tumors - Topline 07/01/2016-12/31/2016 STML SL-801 Cancer IND Phase I study of SL-801 in solid tumors in the second half of 118495 Inc. Results Results 2016.

Regulatory - Filing for Takeda announced that they anticipate filing for approval for Takeda Pharmaceutical Filing for Approval (Emerging 01/12/2016-06/30/2016 4502:JP Ninlaro Multiple Myeloma (MM) Approved Approval (Emerging Ninlaro in emerging markets in the near future. We await an 118209 Company Ltd Markets) Markets) update in the time frame above.

Takeda Pharmaceutical Dengue Fever - Vaccines and Trial Announcement - Takeda announced that they anticipate initiating a Phase III 01/12/2016-12/31/2016 4502:JP TAK-003 II Phase III - Study to Start 118207 Company Ltd Treatments Initiation trial of their dengue vaccine in 2016.

Chronic Lymphocytic Leukemia Teva Pharmaceutical Progress Update - Eagle Pharmaceuticals and Teva expect to launch Bendeka 01/11/2016-05/31/2016 TEVA Bendeka (CLL)/Small Cell Lymphocytic Approved U.S. Product Launch 117966 Industries Ltd. Product Launch by May 2016. Lymphoma (SLL) - NHL

Teva Pharmaceutical Indolent Non-Hodgkin's Progress Update - Eagle Pharmaceuticals and Teva expect to launch Bendeka 01/11/2016-05/31/2016 TEVA Bendeka Approved U.S. Product Launch 117970 Industries Ltd. Lymphoma - NHL Product Launch by May 2016. TG Therapeutics announced that the Phase IIb/III UNITY- Diffuse Large B-Cell Lymphoma Trial Announcement - Phase IIb/III - UNITY-DLBCL - 01/14/2016-06/30/2016 TG Therapeutics, Inc. TGTX TG-1303 I DLBCL study of TG-1303 is expected to start in the first half 118471 (DLBCL) - NHL Initiation Trail to Start of 2016.

Chronic Lymphocytic Leukemia Trial Announcement - Phase III - GENUINE - Patient TG Therapeutics expects to complete enrollment in the 01/14/2016-12/31/2016 TG Therapeutics, Inc. TGTX Ublituximab (CLL)/Small Cell Lymphocytic III Patient Enrollment 118459 Enrollment Completed Phase III GENUINE study by the end of 2016. Lymphoma (SLL) - NHL Completed

Trial Announcement - TG Therapeutics expects to initiate a Phase I/II study of TG- 01/14/2016-06/30/2016 TG Therapeutics, Inc. TGTX Ublituximab Multiple Sclerosis (MS) I/II Phase I/II - MS Study to Start 118472 Initiation 1101 in MS in the first half of 2016.

True North Therapeutics, Complement Deficiencies / Trial Announcement - True North Therapeutics anticipates opening a cold 01/01/2017-12/31/2017 TNT009 I CAD Registration Trial to Start 118241 Inc. Abnormalities Initiation agglutinin disease registration trial in 2017 for TNT009.

Biomedtracker August 2016 / 123 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM New Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Link Ultragenyx expects to have Phase III data from its pediatric Ultragenyx X-Linked Hypophosphatemia Trial Data - Top-Line Phase III Pediatric - Top-Line 01/01/2017-12/31/2018 RARE KRN23 III study of KRN23 in patients with XLH in 2017 or after. We 118214 Pharmaceutical Inc. (XLH) Results Results await an update in the time frame above.

Ultragenyx expects to have Phase III data from its adult Ultragenyx X-Linked Hypophosphatemia Trial Data - Top-Line Phase III CL303 - Top-Line 01/01/2017-12/31/2018 RARE KRN23 III study of KRN23 in patients with XLH in 2017 or after. We 118215 Pharmaceutical Inc. (XLH) Results Results await an update in the time frame above.

Ultragenyx expects to have data from its Phase II study of Ultragenyx Trial Data - Top-Line Phase II CL201 - Top-Line 07/01/2016-12/31/2016 RARE Triheptanoin Metabolic - General II Triheptanoin in patients with Glucose Transporter Type 1 118221 Pharmaceutical Inc. Results Results Deficiency Syndrome in the second half of 2016.

United Therapeutics reported that in December 2015 they filed a supplemental new drug application (sNDA) for United Therapeutics Pulmonary Arterial Regulatory - PDUFA for Remodulin for pulmonary arterial hypertension under a 10 10/01/2016-10/31/2016 UTHR Remodulin (injection) Approved PDUFA for sNDA - First Review 118099 Corporation Hypertension (PAH) sNDA/sBLA month review time frame.Based on a standard 10-month supplemental NDA filing under PDUFA V guidelines the PDUFA decision should occur in October 2016.

Valeant plans to launch Relistor Oral for the treatment of Valeant Pharmaceuticals Opioid Induced Constipation Progress Update - 04/20/2016-12/31/2016 VRX Relistor (Oral) NDA Product Launch (US) opioid induced constipation with chronic non-cancer pain in 118335 International, Inc. (OIC) Product Launch (U.S.) the US in 2016.

Valeant expects to launch Latanoprostene Bunod for the Valeant Pharmaceuticals Glaucoma / Ocular Progress Update - reduction of intraocular pressure in patients with open angle 07/22/2016-12/31/2016 VRX Vesneo NDA Product Launch (US) 118336 International, Inc. Hypertension (Ophthalmology) Product Launch (U.S.) glaucoma and ocular hypertension in 2016 sometime after the PDUFA decision on July 21 2016.

Trial Data - Top-Line Vectura reported that top-line data for the Phase I study of Now-06/30/2016 Vectura Group VEC:LN VR942 Respiratory Disease I Phase I - Top-Line Results 118416 Results VR942 for asthma will be available in the first half of 2016.

Vectura reported that the selection of the final Phase II Trial Announcement - Now-06/30/2016 Vectura Group VEC:LN VR942 Respiratory Disease I Phase II - Trial Announcement clinical study design is anticipated to be released in the first 118417 Other half of 2016. VentiRx Pharmaceuticals, Trial Data - Top-Line Celgene expects to have Phase II data for motolimod in Now-12/31/2016 Motolimod Head and Neck Cancer II Phase II - Top-Line Results 117756 Inc. Results patients with SCCHN in 2016. VentiRx Pharmaceuticals, Trial Data - Top-Line Celgene expects to have Phase II data for motolimod in Now-12/31/2016 Motolimod Ovarian Cancer II Phase II - Top-Line Results 117755 Inc. Results ovarian cancer in 2016.

Vertex recently began clinical development of two next- generation correctors known as VX-152 and VX-440. Both VX- Vertex Pharmaceuticals Trial Data - Top-Line 152 and VX-440 are being evaluated alone and as part of a 04/01/2016-09/30/2016 VRTX VX-152 Cystic Fibrosis (CF) I Phase I - Top-Line Results 118126 Incorporated Results triple combination with VX-661 and ivacaftor in ongoing Phase I studies in healthy volunteers. Safety data are expected in mid-2016.

Vertex recently began clinical development of two next- generation correctors known as VX-152 and VX-440. Both VX- Vertex Pharmaceuticals Trial Data - Top-Line 152 and VX-440 are being evaluated alone and as part of a 04/01/2016-09/30/2016 VRTX VX-440 Cystic Fibrosis (CF) I Phase I - Top-Line Results 118127 Incorporated Results triple combination with VX-661 and ivacaftor in ongoing Phase I studies in healthy volunteers. Safety data are expected in mid-2016.

ZIOPHARM Oncology announced that an abstract has been Brain Cancer (malignant glioma; 05/04/2016-05/07/2016 ZIOPHARM Oncology, Inc. ZIOP Ad-RTS IL-12 I Trial Data - Other Preclinical Data at ASGCT submitted to be presented at the 2016 ASGCT meeting in 118410 AA and GBM) May for Ad-RTS IL-12.

Biomedtracker August 2016 / 124 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Refined (01/11/2016) - Acorda expects to have Trial Data - Top-Line Phase III - MILESTONE - Acorda announced that interim results from the Phase III 07/01/2016-09/30/2016 Acorda Therapeutics, Inc. ACOR Ampyra Ischemic Stroke III an interim analysis of the Phase III post-stroke trial of 114242 Results Top-Line Results post-stroke study of dalfampridine are expected in 2016. dalfampridine in the third quarter of 2016.

Acorda announced that the Company has made progress in their collaborations to develop a once daily or QD Date Range Delayed (12/28/2015) - We await an update. formulation of dalfampridine. Acorda now has three Trial Announcement - Phase I - QD Formulation Catalyst Occurred (01/11/2016) - Acorda announced that Now-04/30/2016 Acorda Therapeutics, Inc. ACOR Ampyra Ischemic Stroke III prototypes from three independent collaborators that 114241 Initiation Trials to Start three prototypes of its QD formulation of dalfampridine is in appear promising based on in vitro testing and modeling. Phase I PK studies. The Company expects to move all three into Phase I clinical testing before the end of 2015.

Date Range Refined (08/14/2014) - Acorda expects to announce GGF2 heart failure trial results in 2H 2015.New Information (08/17/2015) - In June 2015 Acorda announced that it had stopped enrollment in the Phase Ib clinical trial of cimaglermin alfa based on the occurrence of a case of hepatotoxicity (liver injury) meeting Hy’s Law criteria based on blood test results. The Company also received a notification of clinical hold from the FDA following the Congestive Heart Failure Trial Data - Top-Line Phase Ib - Top-Line Acorda expects the Phase Ib trial for GGF2 in heart failure to submission of this information. The 23 patients who were 01/11/2016-04/30/2016 Acorda Therapeutics, Inc. ACOR Cimaglermin Program Hold 99131 (CHF) Results Results read out in 2015. dosed in the trial will complete the pre-planned one year of follow up. The Company expects to complete an analysis of data from the three-month follow up by the end of 2015.Date Range Delayed (12/15/2015) - We await an update. Date Range Delayed (01/11/2016) - Acorda announced that data analyses of the topline data from the Phase I Cimaglermin alfa study is ongoing and plans to meet with FDA to discuss data and evaluate next steps. We await an update through early 2016.

Acorda reported that a pivotal Phase III study of CVT-301 for treatment of OFF episodes in people with PD is expected to Date Range Delayed (01/11/2016) - Acorda plans to file an Regulatory - NDA/BLA 01/01/2017-03/31/2017 Acorda Therapeutics, Inc. ACOR CVT-301 Parkinson's Disease (PD) III 505(b)(2) NDA Filing begin enrolling in early 2015 and if successful a filing NDA for CVT-301 for the treatment of Parkinson's disease in 103594 Filing [505(b)(2)] for regulatory approval in the United States is the first quarter of 2017. expected by the end of 2016. Date Range Delayed (12/03/2015) - We await an update. Date Range Delayed (01/12/2016) - Actelion announced Actelion announced that the results from the Phase II study that it has the results from its Phase II MELODY study of in combined pre- and post-capillary pulmonary Actelion Pharmaceuticals Pulmonary Arterial Trial Data - Top-Line Phase II - MELODY-1 - Top- Opsumit and is currently reviewing for Phase III evaluation. 01/12/2016-06/30/2016 ALIOF Opsumit Approved hypertension due to left ventricular dysfunction (CpcPH- 113346 Ltd. Hypertension (PAH) Results Line Results We await an update on the release of the data in the first LVD) with macitentan are expected before the end of the half of 2016.Catalyst Occurred (01/12/2016) - Actelion year. announced that at the end of 2015 the initial results of the Phase II MELODY study with macitentan became available.

Date Range Expedited (10/16/2015) - A Phase II open-label safety and efficacy evaluation of CRS-207 in combination with epacadostat in patients with platinum resistant ovarian fallopian or peritoneal cancer is not yet open for Trial Announcement - Phase I/II - w/Epacadostat Aduro expects to initiate the Phase I/II study of CRS-207 in participant recruitment. The study is expected to begin in 01/14/2016-03/31/2016 Aduro Biotech ADRO CRS-207 Ovarian Cancer I/II 114836 Initiation to Start combination with Epacadostat in early 2016. December 2015. Date Range Delayed (12/28/2015) - This study is not yet open for participant recruitment. We await an update. Date Range Delayed (01/14/2016) - Aduro expects the Phase I study of CRS-207 and epacadostat for ovarian cancer will start in the first quarter 2016.

Aduro expects to complete enrollment for the Phase IIb ECLIPSE clinical trial of CRS-207 in combination with GVAX Date Range Refined (01/14/2016) - Aduro expects that top Trial Data - Top-Line Phase IIb ECLIPSE - Top- 05/15/2016-06/30/2016 Aduro Biotech ADRO CRS-207 Pancreatic Cancer IIb Pancreas in patients with metastatic pancreatic cancer in line data from the Phase IIb ECLIPSE study of CRS-207 in 110347 Results Line Results the third quarter of 2015 and to report top line results in pancreatic cancer late in the second quarter 2016. the first half of 2016.

Aduro expects to complete enrollment for the Phase IIb ECLIPSE clinical trial of CRS-207 in combination with GVAX Date Range Refined (01/14/2016) - Aduro expects that top Trial Data - Top-Line Phase IIb ECLIPSE - Top- 05/15/2016-06/30/2016 Aduro Biotech ADRO GVAX Pancreatic Vaccine Pancreatic Cancer IIb Pancreas in patients with metastatic pancreatic cancer in line data from the Phase IIb ECLIPSE study of CRS-207 in 112946 Results Line Results the third quarter of 2015 and to report top line results in pancreatic cancer late in the second quarter 2016. the first half of 2016. New Information (06/12/2013) - Aegerion's study of lomitapide in Japanese HoFH patients continues. The company initiated enrollment of Japanese and Caucasian subjects into a PK/PD study late last year and timing with this study is on track. To file formarketing authorization in Japan Aegerion will also need to conduct a small bridging study of lomitapide in Japanese HoFH patients which it expects tobe up to 10 patients in size. We await additional updates on the timing of the Japan submission.Date Range Delayed (08/20/2013) - Aegerion announced that in Q4 During the fourth quarter of 2012 Aegerion initiated 2013 the Company plans to submit a base report to the enrollment of Japanese subjects into a Phase I bridging Japanese regulatory authorities and to conduct a small study of the pharmacokinetic and pharmacodynamic therapeutic study of lomitapide in Japanese adult HoFH Aegerion Dyslipidemia / Regulatory - J-NDA Filing properties of JUXTAPID. Following the outcome of this study patients studying Juxtapid. Assuming that the Company is Now-04/30/2016 AEGR Juxtapid Approved J-NDA Filing 84066 Pharmaceuticals, Inc. Hypercholesterolemia (Japan) Aegerion plans to conduct a small therapeutic study of able to file following the completion of the 26-week efficacy JUXTAPID in Japanese HoFH patients in support of a planned study they plan to be able to submit the J-NDA as early as filing for marketing authorization in Japan. We expect an the first half of 2015. We await an update.New Information update on the filing in 2013. (01/13/2014) - Aegerion announced that they will submit the new drug application (NDA) in Japan for Juxtapid once the 26-week efficacy phase in the planned therapeutic study in Japanese adults has been completed. We continue to await an update in the first half of 2015. Date Range Delayed (04/09/2014) - Aegerion anticipates that the filing for marketing approval in Japan could happen as early as mid-2015.Date Range Delayed (05/26/2015) - Aegerion expects to file for a Japanese new drug application for lomitapide in adult HoFH patients late in the fourth quarter of 2015 or early in the first quarter of 2016.Catalyst Date Range Delayed (01/16/2014) - AstraZeneca plans to file for approval of metreleptin in Europe for the treatment of lidodystrophy in 2015.Date Range Delayed (01/12/2015) - AstraZeneca reported that the Company plans to file for EU Aegerion Regulatory - MAA Aegerion anticipates filing an MAA for Myalept for 10/01/2016-12/31/2016 AEGR Myalept Lipodystrophy Approved MAA Filing approval of metreleptin for the treatment of lipodystrophy 89469 Pharmaceuticals, Inc. Submission (Europe) generalized lipodystrophy in 2016.Date Range Refined during the first half of 2014. (01/14/2016) - Aegerion expects to file an MAA for Lojuxta for the treatment of generalized and severe lipodystrophy in the fourth quarter of 2016.

Date Range Delayed (05/07/2015) - Agios intends to initiate a global registration-enabling Phase III study of AG-120 in Agios plans to initiate a global registration program for AG- AML patients that harbor an IDH1 mutation in the first half Agios Pharmaceuticals, Acute Myelogenous Trial Announcement - Phase III Front-Line Trial 07/01/2016-12/31/2016 AGIO AG-120 I 120 in hematologic malignances that harbor an IDH1 of 2016.Date Range Delayed (01/11/2016) - Agios expects 105288 Inc. Leukemia (AML) Initiation to Start mutation by early 2016. to initiate a global registration-enabling Phase III study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016.

Biomedtracker August 2016 / 125 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link

Date Range Delayed (09/04/2015) - Alexion Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its Priority Review of the Company's Biologics License Application (BLA) for Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL-D). The previously disclosed September 8 2015 PDUFA date has been extended by the standard extension period of three months. As such we now await a Alexion announced that the company plans on launching product launch update in the time frame above. Date Range Kanuma for lysosomal acid lipase deficiency in the US in the Alexion Pharmaceuticals, Lysosomal Acid Lipase Progress Update - Refined (12/08/2015) - Alexion Pharmaceuticals announced Now-01/21/2016 ALXN Kanuma Approved US Product Launch second half of 2015. Because the PDUFA date is expected on 113946 Inc. Deficiency Product Launch (U.S.) that the U.S. Food and Drug Administration (FDA) has September 8 2015 we now expect to see the product launch approved Kanuma (sebelipase alfa) for the treatment of through the last 4 months of the year. patients of all ages with a diagnosis of lysosomal acid lipase deficiency (LAL-D). Alexion is preparing to serve patients in the U.S. with Kanuma and expects that Kanuma will become available commercially during the first week of January 2016. The Company’s expanded access program will remain open to enable patients with LAL-D in the U.S. to access Kanuma until commercial product is available.Catalyst Occurred (01/12/2016) - Alexion announced that Kanuma has been launched in the U.S.

Date Range Refined (01/12/2015) - Synageva expects preliminary data from the Phase I/II study of SBC-2014 in Synageva BioPharma announced that the investigational the second half of 2015.Date Range Refined (11/05/2015) - new drug (IND) application to the U.S. Food & Drug Alexion expects preliminary data from the Phase I/II trial of Administration (FDA) to evaluate SBC-103 as a treatment Mucopolysaccharidosis IIIB SBC-103 for patients with mucopolysaccharidosis IIIB in the Alexion Pharmaceuticals, Trial Data - Top-Line for mucopolysaccharidosis IIIB (MPS IIIB also known as Now-03/31/2016 ALXN SBC-103 (MPS IIIB; Sanfilippo B I/II Phase I/II Top-Line Data fourth quarter of 2015. Date Range Delayed (12/11/2015) - 105402 Inc. Results Sanfilippo B syndrome) is active. The company plans to start Syndrome) Alexion expects to have 6-month data from its Phase I/II enrolling patients with MPS IIIB in a Phase I/II study study of SBC-103 for the treatment of MPS IIIB in the first investigating intravenous administration of SBC-103 shortly quarter of 2016.Catalyst Occurred (01/12/2016) - Alexion and to report preliminary data from this study in 2015. announced preliminary data from the Phase I/II study of SBC- 103 for patients with mucopolysaccharidosis IIIB.

Date Range Refined (01/12/2016) - Alexion announced that Alexion Pharmaceuticals, Delayed Graft Function Trial Data - Top-Line Phase II/II PROTECT - Top- Alexion expects preliminary data from the Phase II/II study enrollment has been completed in the registrational study 07/01/2016-12/31/2016 ALXN Soliris II/III 116366 Inc. (DGF) Results Line Results of eculizumab for delayed graft function in 2016. of eculizumab for DGF and the Company expects preliminary data to be available in the second half of 2016.

Date Range Refined (03/02/2015) - Alkermes reported that top-line results from the Phase III FORWARD-5 efficacy study of ALKS 5461 for major depressive disorder are expected in the first quarter of 2016.Date Range Delayed Alkermes reported that data from the Phase III FORWARD-5 (12/16/2015) - Alkermes announced that the three core Major Depressive Disorder Trial Data - Top-Line Phase III FORWARD-5 - 07/01/2016-09/30/2016 Alkermes plc ALKS ALKS 5461 III efficacy study of ALKS 5461 for major depressive disorder efficacy studies for ALKS 5461 remain on track to read out in 102406 (MDD) Results Top-Line Results are expected in 2016. 2016 with data from the third study anticipated in mid- 2016. Date Range Refined (01/12/2016) - Alkermes announced that they anticipate data from the Phase III FORWARD-5 study of ALKS 5461 for the treatment of major depressive disorder (MDD) in the third quarter of 2016.

Date Range Delayed (06/19/2015) - We await an update on the IND filing for ALKS 7119 through the first half of the third quarter of 2015.Date Range Delayed (08/03/2015) - As Alkermes announced a new drug candidate ALKS 7119 for Alkermes expects to initiate the first clinical study of ALKS the treatment of agitation in patients with alzheimer’s 7119 in early Q1 2016 we look for a potential IND filing disease major depressive disorder (MDD) and other central towards the end of the year.Date Range Delayed Now-01/31/2016 Alkermes plc ALKS ALKS 7119 Alzheimer's Disease (AD) Preclinical Regulatory - IND Filing IND Submission nervous system (CNS) indications. Alkermes intends to file 109063 (12/15/2015) - We await an update through January 2016. an Investigational New Drug (IND) application with the U.S. New Information (01/12/2016) - Alkermes expects to Food and Drug Administration (FDA) in the second quarter initiate a Phase I study of ALKS 7119 with an initial focus on 2015. agitation in Alzheimer's disease (AD) in the first quarter of 2016. As such we continue to await an IND filing through January 2016.

Alkermes announced a new drug candidate ALKS 7119 for Date Range Delayed (08/03/2015) - Alkermes expects to the treatment of agitation in patients with alzheimer’s initiate the first clinical study of ALKS 7119 in early Q1 disease major depressive disorder (MDD) and other central Trial Announcement - 2016.Date Range Refined (01/12/2016) - Alkermes expects 01/12/2016-03/31/2016 Alkermes plc ALKS ALKS 7119 Alzheimer's Disease (AD) Preclinical Phase I - Trial to Start nervous system (CNS) indications. Alkermes has successfully 109065 Initiation to initiate a Phase I study of ALKS 7119 with an initial focus completed preclinical development and expects to initiate on agitation in Alzheimer's disease (AD) in the first quarter the first clinical study of ALKS 7119 in the third quarter of of 2016. 2015.

Date Range Delayed (01/11/2016) - Alnylam plans to Alnylam Pharmaceuticals expects that following approval of present initial clinical data from the Phase I study of ALN- Alnylam Pharmaceuticals, Trial Data - Top-Line Phase I/II - Top-Line 04/01/2016-09/30/2016 ALNY ALN-AAT Liver Failure / Cirrhosis I/II the CTA it will initiate the Phase I/II study in late 2015 with AAT an investigational RNAi therapeutic targeting alpha-1 111781 Inc. Results Results initial data expected to be reported in early 2016. antitrypsin (AAT) for the treatment of AAT deficiency- associated liver disease in mid-2016.

Alnylam Pharmaceuticals announced that the company has transitioned to Part B of the Phase I study with ALN-AS1 for Date Range Refined (01/11/2016) - Alnylam plans to Alnylam Pharmaceuticals, Trial Data - Updated the treatment of acute hepatic porphyrias in which subjects present data from the ongoing Phase I study of ALN-AS1 - 09/01/2016-12/31/2016 ALNY ALN-AS1 Porphyria I Phase I - Updated Results 114999 Inc. Results are receiving monthly subcutaneous dosing of 0.35 mg/kg including initial data in patients that experience recurring and initial data from this part of the study is expected in porphyria attacks - in late 2016. 2016. Alnylam announced that it initiated the final phase (Part C) Date Range Delayed (01/11/2016) - Alnylam plans to of its Phase I/II clinical trial with ALN-CC5. The Company Alnylam Pharmaceuticals, Paroxysmal Nocturnal Trial Data - Updated Phase I/II - Updated PNH present data from Part C of the Phase I/II trial of ALN-CC5 - 04/01/2016-12/31/2016 ALNY ALN-CC5 I/II expects to report initial PNH patient data from this ongoing 117408 Inc. Hemoglobinuria (PNH) Results Data which is being conducted in paroxysmal nocturnal clinical study of ALN-CC5 in mid-2016 and initiate Phase III hemoglobinuria (PNH) patients - in mid- and late 2016. studies in 2017.

Date Range Refined (06/12/2014) - Alnylam plans to file an investigational new drug application at or around year-end 2015.New Information (12/12/2014) - Alnylam announced that it has selected a Development Candidate (DC) for its ALN-HBV. The ALN-HBV DC is an ESC-GalNAc-siRNA targeting the HBV genome. In a rodent model of HBV a single subcutaneous dose at 3 mg/kg resulted in an up to Alnylam expects to name a ALN-HBV Development Alnylam Pharmaceuticals, Hepatitis B (HBV) Treatment 3.9 log10 reduction in hepatitis B surface antigen (HBsAg) 01/11/2016-04/30/2016 ALNY ALN-HBV Preclinical Regulatory - IND Filing IND Filing Candidate by the end of this year and file an IND or IND 100635 Inc. (Antiviral) levels (mean 1.8 log10 reduction).The company expects to equivalent around the end of 2015. file an investigational new drug (IND) application or IND equivalent for this program in late 2015. Date Range Delayed (12/18/2015) - We await an update.Date Range Delayed (01/11/2016) - Alnylam plans to a file Clinical Trial Application (CTA) for ALN-HBV an investigational RNAi therapeutic targeting the hepatitis B viral (HBV) genome for the treatment of chronic HBV infection in early 2016.

Date Range Delayed (01/11/2016) - Alnylam plans to start Alnylam Pharmaceuticals, Hemophilia A and B - Trial Announcement - Alnylam Pharmaceuticals announced plans to proceed to two Phase III trials of fitusiran in severe hemophilia A and B 04/01/2016-12/31/2016 ALNY Fitusiran I Phase III Trials to Start 112851 Inc. General Clotting Products Initiation pivotal Phase III studies of ALN-AT3 in mid-2016. patients with and without inhibitors in mid- and late 2016 respectively. New Information (10/08/2015) - Alnylam Pharmaceuticals announced that it has initiated a Phase I open-label extension (OLE) study with ALN-AT3 for the treatment of hemophilia and rare bleeding disorders. The company plans Alnylam Pharmaceuticals announced plans to to present to report clinical data from the Phase I OLE study at least Alnylam Pharmaceuticals, Hemophilia A and B - Trial Data - Top-Line Phase I Extension - Top- 09/01/2016-12/31/2016 ALNY Fitusiran I data from a Phase I - Extension Study on an ongoing basis at once per year beginning in 2016.Date Range Refined 112853 Inc. General Clotting Products Results Line Results least once per year beginning in 2016. (01/11/2016) - Alnylam plans to present initial data from the ongoing Phase I OLE study of fitusiran (ALN-AT3) an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD) in late 2016.

Biomedtracker August 2016 / 126 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (01/11/2016) - Alnylam plans to start two Phase III trials of fitusiran in severe hemophilia A and B patients with and without inhibitors in mid- and late 2016 Alnylam Pharmaceuticals, Hemophilia A and B - Trial Announcement - Alnylam Pharmaceuticals announced plans to proceed to 07/01/2016-09/30/2016 ALNY Fitusiran I Phase III Trials to Start respectively.Date Range Refined (01/12/2016) - Sanofi 112851 Inc. General Clotting Products Initiation pivotal Phase III studies of ALN-AT3 in mid-2016. announced that they expect to initiate a Phase III study of fitusiran for the treatment of hemophilia in the third quarter of 2016.

Date Range Expedited (01/11/2016) - Alnylam announced that the Company has enrolled over 200 patients in the Transthyretin-related Phase III APOLLO study of patisiran for the treatment of TTR- Trial Announcement - Alnylam expects to complete enrollment in the APOLLO Alnylam Pharmaceuticals, Hereditary Amyloidosis Phase III APOLLO - mediated amyloidosis (ATTR amyloidosis) in patients with 01/11/2016-01/31/2016 ALNY Patisiran III Patient Enrollment Phase III trial of patisiran over the next three to four 116249 Inc. (Familial Amyloid Enrollment Complete familial amyloidotic polyneuropathy (FAP). Based on the Completed months. Polyneuropathy) number of patients currently in screening the Company expects the APOLLO trial to be over-enrolled. Alnylam expects enrollment to complete by the end of January 2016.

Date Range Delayed (01/11/2016) - Alnylam expects to be Transthyretin-related Alnylam expects data from the Phase III APOLLO study of in a position to report data from the Phase III APOLLO trial Alnylam Pharmaceuticals, Hereditary Amyloidosis Trial Data - Top-Line Phase III APOLLO - Top- 01/01/2017-12/31/2017 ALNY Patisiran III Patisiran in 2.5 to 3.5 years. We look for data in the above of patisiran for the treatment of TTR-mediated amyloidosis 96891 Inc. (Familial Amyloid Results Line Results date range. (ATTR amyloidosis) in patients with familial amyloidotic Polyneuropathy) polyneuropathy (FAP) in 2017.

Date Range Refined (01/11/2016) - Alnylam expects to be in a position to report data from the Phase III APOLLO trial of Transthyretin-related patisiran for the treatment of TTR-mediated amyloidosis Alnylam Pharmaceuticals, Hereditary Amyloidosis Regulatory - NDA/BLA Depending on positive results from its Phase III APOLLO 09/01/2017-12/31/2017 ALNY Patisiran III NDA Filing (ATTR amyloidosis) in patients with familial amyloidotic 98710 Inc. (Familial Amyloid Filing study Alnylam will file an NDA for Patisiran in 2017. polyneuropathy (FAP) in 2017 and to submit a New Drug Polyneuropathy) Application (NDA) and Marketing Authorisation Application (MAA) for patisiran in late 2017. Date Range Delayed (08/03/2015) - Amgen reported that the Phase IIb migraine prophylaxis study is expected to Migraine and Other Trial Data - Top-Line Phase IIb Prophylaxis Top- Amgen reported that the Phase IIb migraine prophylaxis report top-line data in 2016.Date Range Refined 07/01/2016-12/31/2016 Amgen, Inc. AMGN AMG 334 III 99866 Headaches Results Line Data study is expected to report top-line data in 2015. (01/12/2016) - Amgen reported that Phase IIb data for AMG334 for migraine prophylaxis are expected in the second half of 2016.

Date Range Delayed (10/23/2012) - Amgen today announced the issuance of U.S. Patent No. 8063182 related to Enbrel (etanercept). This patent is owned by Hoffman-La Roche (Roche) and exclusively licensed to Amgen. Immunex Corporation (acquired by Amgen in 2002) originally licensed this patent application from Roche in 1999 and in 2004 The patent for Enbrel is expected to expire on October 23 01/01/2029-12/31/2029 Amgen, Inc. AMGN Enbrel Ankylosing Spondylitis Approved Patent - Expiration Patent Expiration Amgen paid Roche a one-time payment and obtained an 8004 2012. exclusive fully paid-up license to the application which issued today as the '182 patent. The patent describes and claims the fusion protein that is etanercept and by statute the '182 patent has a term of 17 years from today. Date Range Delayed (01/12/2016) - Amgen maintains exclusivity for Enbrel through 2029.

Date Range Delayed (10/23/2012) - Amgen today announced the issuance of U.S. Patent No. 8063182 related to Enbrel (etanercept). This patent is owned by Hoffman-La Roche (Roche) and exclusively licensed to Amgen. Immunex Corporation (acquired by Amgen in 2002) originally licensed this patent application from Roche in 1999 and in 2004 Juvenile Rheumatoid The patent for Enbrel is expected to expire on October 23 01/01/2029-12/31/2029 Amgen, Inc. AMGN Enbrel Approved Patent - Expiration Patent Expiration Amgen paid Roche a one-time payment and obtained an 8006 Arthritis 2012. exclusive fully paid-up license to the application which issued today as the '182 patent. The patent describes and claims the fusion protein that is etanercept and by statute the '182 patent has a term of 17 years from today. Date Range Delayed (01/12/2016) - Amgen maintains exclusivity for Enbrel through 2029.

Date Range Delayed (10/23/2012) - Amgen today announced the issuance of U.S. Patent No. 8063182 related to Enbrel (etanercept). This patent is owned by Hoffman-La Roche (Roche) and exclusively licensed to Amgen. Immunex Corporation (acquired by Amgen in 2002) originally licensed this patent application from Roche in 1999 and in 2004 The patent for Enbrel is expected to expire on October 23 01/01/2029-12/31/2029 Amgen, Inc. AMGN Enbrel Psoriasis Approved Patent - Expiration Patent Expiration Amgen paid Roche a one-time payment and obtained an 8003 2012. exclusive fully paid-up license to the application which issued today as the '182 patent. The patent describes and claims the fusion protein that is etanercept and by statute the '182 patent has a term of 17 years from today. Date Range Delayed (01/12/2016) - Amgen maintains exclusivity for Enbrel through 2029.

Date Range Delayed (10/23/2012) - Amgen today announced the issuance of U.S. Patent No. 8063182 related to Enbrel (etanercept). This patent is owned by Hoffman-La Roche (Roche) and exclusively licensed to Amgen. Immunex Corporation (acquired by Amgen in 2002) originally licensed this patent application from Roche in 1999 and in 2004 The patent for Enbrel is expected to expire on October 23 01/01/2029-12/31/2029 Amgen, Inc. AMGN Enbrel Psoriatic Arthritis (PA) Approved Patent - Expiration Patent Expiration Amgen paid Roche a one-time payment and obtained an 8005 2012. exclusive fully paid-up license to the application which issued today as the '182 patent. The patent describes and claims the fusion protein that is etanercept and by statute the '182 patent has a term of 17 years from today. Date Range Delayed (01/12/2016) - Amgen maintains exclusivity for Enbrel through 2029.

Date Range Delayed (10/23/2012) - Amgen today announced the issuance of U.S. Patent No. 8063182 related to Enbrel (etanercept). This patent is owned by Hoffman-La Roche (Roche) and exclusively licensed to Amgen. Immunex Corporation (acquired by Amgen in 2002) originally licensed this patent application from Roche in 1999 and in 2004 The patent for Enbrel is expected to expire on October 23 01/01/2029-12/31/2029 Amgen, Inc. AMGN Enbrel Rheumatoid Arthritis (RA) Approved Patent - Expiration Patent Expiration Amgen paid Roche a one-time payment and obtained an 8002 2012. exclusive fully paid-up license to the application which issued today as the '182 patent. The patent describes and claims the fusion protein that is etanercept and by statute the '182 patent has a term of 17 years from today. Date Range Delayed (01/12/2016) - Amgen maintains exclusivity for Enbrel through 2029.

Date Range Refined (01/12/2016) - Amgen expects data Dyslipidemia / Trial Data - Top-Line Phase III GLAGOV - Top- 07/01/2016-12/31/2016 Amgen, Inc. AMGN Repatha Approved Amgen expects data from the GLAGOV study in 2016. from its Phase III coronary imaging study for Repatha in the 104955 Hypercholesterolemia Results Line Results second half of 2016.

New Information (01/14/2014) - UCB expects Phase III romosozumab PMO data in 2015. We continue to expect data towards the end of the year as previously guided.Date Range Delayed (01/30/2014) - Amgen reported that the Phase III data of romosozumab for the treatment of Results from a Phase III study of AMG 785 in postmenopausal osteoporosis is expected to be released in Trial Data - Top-Line Phase III FRAME (PMO) - Now-03/31/2016 Amgen, Inc. AMGN Romosozumab Osteoporosis / Osteopenia III postmenopausal osteoporosis are expected towards the end the first half of 2016.Date Range Delayed (07/30/2014) - 62074 Results Top Line Data of 2015. UCB expects Phase III romosozumab PMO data in the second half of 2016.Date Range Expedited (10/27/2014) - UCB expects Phase III romosozumab PMO data in the first half of 2016.Date Range Expedited (01/12/2016) - Amgen expects data from its ~7200 patient PMO study of romosozumab in the first quarter of 2016.

Biomedtracker August 2016 / 127 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (12/17/2014) - Scioderm has not provided an update on the potential filing for Zorblisa in the US. We await an update through the first half of 2015.Date Range Delayed (12/18/2014) - Scioderm intends to initiate trial of Zorblisa (SD-101) for the treatment of Epidermolysis Bullosa (EB) in the first quarter of 2015 and expects to have top-line results in the second half of 2015. Based on this information we expect to see a potential filing in late 2015/early 2016.Date Range Refined (07/14/2015) - Scioderm announced that it has agreed with the U.S. Food and Drug Administration (FDA) to submit a rolling New Drug Scioderm believes its partnership options are open based on Application (NDA) submission for Zorblisa for the treatment Regulatory - Rolling its potential to file SD-101 in 2014 in the U.S. We expect an of blisters and lesions in patients with Epidermolysis Bullosa Now-04/30/2016 Amicus Therapeutics, Inc. FOLD Zorblisa Epidermolysis Bullosa III Rolling NDA Initiation 97321 NDA/BLA Initiation update on a potential filing in the second half of 2014 (EB). Based on discussion with and agreement by the following the completion of the Phase IIb/III trial. Agency Scioderm plans to submit the nonclinical components of the NDA in the third quarter of 2015 followed by submission of the chemistry manufacturing and control (CMC) components by early fourth quarter of this year. Date Range Delayed (08/31/2015) - Amicus reports that that FDA has agreed to a rolling NDA submission for Zorblisa for epidermolysis bullosa beginning in 4Q15.Date Range Delayed (12/18/2015) - We await an update through early 2016.Catalyst Occurred (01/12/2016) - Amicus reported that the Company's rolling NDA for Zorblisa for the treatment of epidermolysis bullosa was initiated in the fourth quarter of 2015.

Antares announced that the last patient will complete their final visit in a dose-blinded multiple-dose concentration Phase III STEADY (Safety Date Range Refined (01/14/2016) - Antares expects the last Trial Announcement - controlled 26-week safety and pharmacokinetic study of 04/01/2016-06/30/2016 Antares Pharma, Inc. ATRS Vibex QST Hypogonadism III Study 005) - Dosing patient out from the six-month supplemental safety study 116250 Dosing Completed QuickShot Testosterone (QS T) administered Complete QST-15-005 of Vibex QST in the second quarter of 2016. subcutaneously once each week to adult males with hypogonadism by the end of the second quarter 2016. Date Range Refined (01/16/2014) - Antares anticipates filing the NDA for VIBEX QS-T in the middle of 2015.Date Range Delayed (01/13/2015) - Antares Pharma announced that it has received written recommendations from the U.S. Food and Drug Administration (FDA) related to its clinical development program for QuickShot Testosterone (QS T). Based on a single reported occurrence of hives in the Company’s Phase II study which the FDA characterized as an apparent allergic reaction as well as the known safety experience with other parenteral testosterone products the FDA is recommending that the Company create a larger safety database including approximately 350 subjects exposed to QS T with 200 subjects exposed for six months The Vibex QST development program for testosterone Regulatory - NDA/BLA and 100 subjects exposed for a year. The Company is 10/01/2016-03/31/2017 Antares Pharma, Inc. ATRS Vibex QST Hypogonadism III NDA Filing deficiency (or low T) is entering its pivotal clinical studies in 93534 Filing assessing the FDA’s comments in the advice letter and their preparation for a potential NDA filing in 2015. impact on the timing of the filing of a New Drug Application for QS T with the FDA. As such we expect that it will take an additional year to reach enrollment and obtain data analysis for more patients with 6-month exposure to Vibex.New Information (06/02/2015) - Antares announced that it has received a written update from the U.S. Food and Drug Administration (FDA) related to its clinical development program for QuickShot Testosterone (QS T). The Company believes that with the update just received from FDA there is an agreed upon path forward for the completion of an additional study to support the filing of a New Drug Application for QS T. The Company is now finalizing the protocol for the study and expects to initiate the trial in the Date Range Delayed (09/12/2014) - Apogenix announced that the first results of the Phase I study of Apocept for MDS are expected by the end of 2014.Date Range Delayed (12/17/2014) - Apogenix continues to expect the first results of the Phase I study of Apocept for MDS by the end Myelodysplastic Syndrome Trial Data - Top-Line Phase I (Germany) - Top- Results for the Phase I trial of Apocept are expected in mid- of 2014. We now await an update through early 2015.Date Now-03/31/2016 Apogenix GmbH Apocept I 85237 (MDS) Results Line Results 2014. Range Delayed (01/26/2015) - Per a Company representative results of the Phase I trial are expected to be reported by the end of 2015.Date Range Delayed (12/10/2015) - We await an update.New Information (01/11/2016) - Apogenix announced that final results from the Phase I trial are expected in the first quarter of 2016.

Arena plans to have top-line results from its Phase II study Date Range Delayed (01/14/2016) - Arena plans to have top- Arena Pharmaceuticals, Pulmonary Arterial Trial Data - Top-Line 01/01/2017-04/30/2017 ARNA Ralinepag II Phase II - Top-Line Results of Ralinepag in patients with pulmonary arterial line results from its Phase II study of Ralinepag in patients 106378 Inc. Hypertension (PAH) Results hypertension in 2016. with pulmonary arterial hypertension in early 2017.

Date Range Delayed (11/12/2014) - Array plans to provide an update on the LMNA_related DCM program of ARRY-797 early next year. We look for a potential data update in the same timeframe.Date Range Delayed (01/14/2015) - Array expects to release results for the Phase II study of ARRY-797 for the treatment of dilated cardiomyopathy (DCM) in Topline results from the Phase II study of ARRY-797 in Congestive Heart Failure Trial Data - Top-Line 2015.Date Range Delayed (12/15/2015) - Array continues to Now-03/31/2016 Array BioPharma, Inc. ARRY ARRY-797 II Phase II - Top-line Results patients with LMNA-related dilated cardiomyopathy (DCM) 98415 (CHF) Results progress select other wholly-owned programs including two are expected in late 2014. Phase II trials of filanesib in multiple myeloma and a Phase II trial of ARRY-797 in a rare cardiovascular disease. We continue to await an update.New Information (01/13/2016) - Array announced that the Phase II trial of ARRY-797 for LMNA-related dilated cardiomyopathy is ongoing. Data is expected to be presented in the near future.

Results from the NEMO trial including progression free survival overall survival objective response rate safety and Date Range Refined (01/13/2016) - Array BioPharma Trial Data - Updated Phase III NEMO - Updated 04/01/2016-09/30/2016 Array BioPharma, Inc. ARRY Binimetinib Melanoma III prespecified subgroup analyses including outcomes in announced that the updated results from the Phase III 117374 Results Results patients who received prior treatment with immunotherapy NEMO study of binimetinib will be presented in mid-2016. will be presented at a medical meeting in 2016.

Date Range Refined (01/13/2016) - Array BioPharma Array expects to submit regulatory filings for MEK162 based Regulatory - sNDA/sBLA Supplemental NDA Filing expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Binimetinib Melanoma III on the 900-patient COLUMBUS study in BRAF-mutant 92663 Filing (BRAF) encorafenib based on the 900-patient COLUMBUS study in melanoma patients in 2016. BRAF-mutant melanoma patients in the second half of 2016.

Date Range Refined (01/13/2016) - Array BioPharma Regulatory - Array expects to submit regulatory filings for MEK162 based Supplemental JNDA Filing expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Binimetinib Melanoma III Supplemental Filing on the 900-patient COLUMBUS study in BRAF-mutant 92664 (BRAF) encorafenib based on the 900-patient COLUMBUS study in (Japan) melanoma patients in 2016. BRAF-mutant melanoma patients in the second half of 2016.

Date Range Refined (01/13/2016) - Array BioPharma Regulatory - Array expects to submit regulatory filings for MEK162 based Supplemental MAA Filing expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Binimetinib Melanoma III Supplemental Filing on the 900-patient COLUMBUS study in BRAF-mutant 92665 (BRAF) encorafenib based on the 900-patient COLUMBUS study in (Europe) melanoma patients in 2016. BRAF-mutant melanoma patients in the second half of 2016.

Date Range Refined (01/13/2016) - Array BioPharma Regulatory - MAA Novartis plans on filing MEK162 + LGX818 for approval for expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Encorafenib Melanoma III MAA Filing (BRAF) 98255 Submission (Europe) BRAF mutant melanoma in 2016. encorafenib based on the 900-patient COLUMBUS study in BRAF-mutant melanoma patients in the second half of 2016.

Date Range Refined (01/13/2016) - Array BioPharma Regulatory - J-NDA Filing Novartis plans on filing MEK162 + LGX818 for approval for expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Encorafenib Melanoma III J-NDA Filing (BRAF) 98254 (Japan) BRAF mutant melanoma in 2016. encorafenib based on the 900-patient COLUMBUS study in BRAF-mutant melanoma patients in the second half of 2016.

Date Range Refined (01/13/2016) - Array BioPharma Regulatory - NDA/BLA Novartis plans on filing MEK162 + LGX818 for approval for expects to submit regulatory filings for binimetinib and 07/01/2016-12/31/2016 Array BioPharma, Inc. ARRY Encorafenib Melanoma III NDA Filing (BRAF) 98253 Filing BRAF mutant melanoma in 2016. encorafenib based on the 900-patient COLUMBUS study in BRAF-mutant melanoma patients in the second half of 2016.

Biomedtracker August 2016 / 128 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Refined (01/11/2016) - Medivation announced Trial Data - Top-Line Phase II HER2+ - Top Line Astellas expects to present data from the Her2+ study in 07/01/2016-12/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Breast Cancer II that they anticipate releasing data from the Phase II HER2+ 106119 Results Data 2016. trial in the second half of 2016. Date Range Delayed (01/11/2016) - Medivation announced Trial Data - Top-Line Phase II HR+ - Top Line Astellas expects to have a read out from the Phase II trial 07/01/2016-12/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Breast Cancer II that they anticipate releasing data from the Phase II 106118 Results Results with exemestane in September 2016. ER/PgR+ and HER2 trial in the second half of 2016.

Catalyst Occurred (01/14/2016) - Medivation and Astellas announced that results from the Phase II TERRAIN trial of Medivation announced that they anticipate the publication enzalutamide compared to bicalutamide in metastatic Trial Data - Published Phase II TERRAIN - Now-03/31/2016 Astellas Pharma, Inc. 4503:JP Xtandi Prostate Cancer Approved of data from the Phase II TERRAIN trial of MDV3100 for castration-resistant prostate cancer (CRPC) were published 118044 Results Published Results prostate cancer in the first quarter of 2016. in the Lancet Oncology. The article is titled Efficacy and Safety of enzalutamide Versus bicalutamide for Patients with Metastatic Prostate Cancer (TERRAIN).

Date Range Delayed (02/13/2014) - AstraZeneca expects an external representation of results for the Phase III SELECT-1 study for selumetinib in patients with non-small cell lung cancer in the second quarter of 2017. Date Range Expedited Array expects primary data from the 634-patient KRAS- (05/02/2014) - Array expects primary data from the SELECT- Non-Small Cell Lung Cancer Trial Data - Top-Line Phase III SELECT-1 - Top- 04/01/2016-09/30/2016 AstraZeneca PLC AZN Selumetinib III mutant NSCLC study of Selumetinib (SELECT-1) to be 1 study of Selumetinib in July 2016.Date Range Delayed 92661 (NSCLC) Results Line Results available in July 2016. (08/24/2015) - Array expects data from the SELECT-1 study in the second half of 2016.Date Range Refined (01/13/2016) - Array expects data from the Phase III SELECT-1 study of selumetinib in patients with non-small cell lung cancer in mid-2016.

Atara Biotherapeutics expects to commence a multicenter New Information (01/11/2016) - Atara expects to initiate an Expanded Access Trial Atara Biotherapeutics, Trial Announcement - pilot study of EBV-CTL in post transplant expanded access trial with EBV-CTL in refractory EBV-PTLD Now-06/30/2016 ATRA EBV-CTL Hematologic Cancer II (EBV-PTLD after HCT) to 115872 Inc. Initiation lymphoproliferateive disorders (EBV-PTLD) after patients after HCT during the first half of 2016 followed by a Start hematopoietic cell transplant (HCT) in the first half of 2016. pivotal trial.

Atara Biotherapeutics expects to commence a multicenter pilot study of EBV-CTL in post transplant New Information (01/11/2016) - Atara expects to initiate an lymphoproliferateive disorders (EBV-PTLD) after solid organ Atara Biotherapeutics, Trial Announcement - Expanded Access Trial expanded access trial with EBV-CTL in refractory EBV Now-09/30/2016 ATRA EBV-CTL Hematologic Cancer II transplant (SOT) following the initiation of EBV-PTLD after 115873 Inc. Initiation (EBV after SOT) to Start patients after SOT during 2016 followed by a pivotal trial. hematopoietic cell transplant HCT studies (which is We continue to await an update in the time frame above. expectected to begin in the first half of 2016). As such we await an update in the time frame above. Date Range Refined (09/10/2015) - aTyr Pharma announced that data from the Phase Ib/II study of resolaris for the treatment of FSHD are expected in the fourth quarter of 2015 or first quarter of 2016. Date Range Refined aTyr Pharma expect to report initial results from its Phase Trial Data - Top-Line Phase Ib/II - Top-Line (01/13/2016) - aTyr Pharma announced that it has Now-03/31/2016 aTyr Pharma LIFE Resolaris Muscular Dystrophy I/II Ib/II clinical trial of Resolaris in adult patients with FSHD in 110096 Results Results completed patient dosing for its Phase Ib/II trial for the fourth quarter of 2015 or early 2016. Resolaris in adult FSHD patients and is in the process of database curation and analysis. The Company expects to report data at approximately the end of the first quarter of 2016. Date Range Delayed (01/13/2016) - aTyr plans to initiate a clinical program in rare pulmonary diseases with an Trial Announcement - Phase Ib/II RPIC - Trial to aTyr expects to initiate a Phase Ib/II study of Resolaris for immune component (RPIC) in patients with interstitial lung Now-12/31/2016 aTyr Pharma LIFE Resolaris Muscular Dystrophy I/II 110100 Initiation Start RPIC in the first half of 2016. disease (ILD). By the end of 2016 the Company will make a determination as to whether to move forward with an exploratory Phase Ib/II trial for Resolaris.

Baxalta announced initial results from a Phase III clinical trial of ADYNOVATE for the treatment for hemophilia A in New Information (01/12/2016) - Nektar reports that they previously-treated patients (PTPs) with severe hemophilia A Regulatory - sNDA/sBLA intend to file a European BLA submission for Adynovate in Now-04/30/2016 Baxalta Incorporated BXLT Adynovate Hemophilia A Approved sBLA Filing younger than 12 years of age. With the study results the 117448 Filing hemophilia A in the second half of 2016. We continue to company plans to file for marketing authorization in Europe expect a pediatric application in the U.S. in early 2016. and aims to file for a pediatric indication in the U.S. in early 2016.

Date Range Refined (11/13/2013) - Based on the results of the Phase II/III PROLONG-ATE study Baxter expects to complete the trial and file for regulatory approval late in 2014. We expect a EU filing to occur in a similar timeframe.Date Range Delayed (01/13/2014) - Baxter expects to file for European approval for BAX 855 in 2016.Date Range Refined (08/21/2014) - Baxter is initiating a Phase III study of BAX 855 among 60 previously treated patients under the age of 12 with severe hemophilia A. Baxter expects to file a Marketing Authorization Application with the EMA upon the completion of the pediatric study. According to the National Institute of Health (NIH) clinical Regulatory - MAA 07/01/2016-12/31/2016 Baxalta Incorporated BXLT Adynovate Hemophilia A Approved MAA Filing Regulatory filings for BAX 855 are planned for 2014. trial registry the primary completion of the study is 84237 Submission (Europe) expected in April 2016. We expect an update on the MAA filing for BAX 855 from the time of the expected primary completion in April 2016 to the end of 2016.New Information (02/11/2015) - Baxter expects to file for marketing authorization with the European Medicines Agency in 2016 upon completion of their pediatric study. Date Range Expedited (12/07/2015) - Baxalta expects to file for marketing authorization for ADYNOVATE to treat hemophilia A in Europe in early 2016.Date Range Delayed (01/12/2016) - �Nektar reports that they intend to file a European BLA submission for Adynovate in hemophilia A in the second half of 2016.

Date Range Delayed (08/09/2014) - Nektar reported that Amakacin Inhale is being evaluated in gram-negative pneumonias and ventilated patients and Bayer expects Phase III data in early 2016. Date Range Delayed (05/08/2015) - Nektar Therapeutics announced that the Hospital Acquired Phase III trials of Amikacin for the treatment of hospital Trial Data - Top-Line Phase III INHALE 1 - Top- Nektar expects to release Phase III INHALE 1 top-line results 01/01/2017-04/30/2017 Bayer AG BAYRY Amikacin Inhale (Nosocomial) Pneumonia III acquired pneumonia is expected to be completed in the first 97082 Results Line Data during mid-2015. (HAP) (Antibacterial) part of 2016. As such we expect an update a top-line data update in the time frame above. Date Range Delayed (01/12/2016) - Nektar reported that they intend to release top-line results from the Phase III INHALE program of Amikacin Inhale for Gram-Negative Pneumonia in early 2017.

Date Range Delayed (08/09/2014) - Nektar reported that Amakacin Inhale is being evaluated in gram-negative pneumonias and ventilated patients and Bayer expects Phase III data in early 2016. Date Range Delayed (05/08/2015) - Nektar Therapeutics announced that the Hospital Acquired Phase III trials of Amikacin for the treatment of hospital Trial Data - Top-Line Phase III INHALE 2 - Top- Nektar expects to release Phase III INHALE 2 top-line results 01/01/2017-04/30/2017 Bayer AG BAYRY Amikacin Inhale (Nosocomial) Pneumonia III acquired pneumonia is expected to be completed in the first 97083 Results Line Data during mid-2015. (HAP) (Antibacterial) part of 2016. As such we expect an update a top-line data update in the time frame above.Date Range Delayed (01/12/2016) - Nektar reported that they intend to release top-line results from the Phase III INHALE program of Amikacin Inhale for Gram-Negative Pneumonia in early 2017. Date Range Delayed (10/07/2014) - Nektar reported that Amakacin Inhale is being evaluated in gram-negative pneumonias and ventilated patients and Bayer expects Phase III data in early 2016. As such we expect regulatory Hospital Acquired Regulatory - MAA Nektar announced that they have planned filings with the filings in mid- to late-2016. Date Range Delayed 04/01/2017-12/31/2017 Bayer AG BAYRY Amikacin Inhale (Nosocomial) Pneumonia III MAA Filing 97086 Submission (Europe) FDA and EMA for Amikacin Inhale in Q4 2015. (01/12/2016) - Nektar reported that they intend to release (HAP) (Antibacterial) top-line results from the Phase III INHALE program of Amikacin Inhale for Gram-Negative Pneumonia in early 2017. As such we expect regulatory filings in mid- to late- 2017.

Biomedtracker August 2016 / 129 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (10/07/2014) - Nektar reported that Amakacin Inhale is being evaluated in gram-negative pneumonias and ventilated patients and Bayer expects Phase III data in early 2016. As such we expect regulatory Hospital Acquired Regulatory - NDA/BLA Nektar announced that they have planned filings with the filings in mid- to late-2016. Date Range Delayed 04/01/2017-12/31/2017 Bayer AG BAYRY Amikacin Inhale (Nosocomial) Pneumonia III NDA Filing 97085 Filing FDA and EMA for Amikacin Inhale in Q4 2015. (01/12/2016) - �Nektar reported that they intend to (HAP) (Antibacterial) release top-line results from the Phase III INHALE program of Amikacin Inhale for Gram-Negative Pneumonia in early 2017. As such we expect regulatory filings in mid- to late- 2017.

Date Range Delayed (08/09/2014) - Nektar reported that Cipro DPI is being evaluated in non-cystic-fibrosis bronchiectasis and Bayer expects Phase III results in the latter part of 2015. Date Range Delayed (11/18/2014) - Nektar expects potential Phase III data readouts for Ciprofloxacin DPI in non-cystic fibrosis bronchiectasis Trial Data - Top-Line Phase III RESPIRE 1 Top- Nektar expects to have Phase III RESPIRE 1 top-line data in Now-06/30/2016 Bayer AG BAYRY Ciprofloxacin DPI Bronchiectasis III throughout 2016. Date Range Refined (01/13/2015) - 97087 Results Line Data Q3 2015. Nektar expects potential Phase III data readouts for Ciprofloxacin DPI in non-cystic fibrosis bronchiectasis in mid- 2016.Date Range Expedited (01/12/2016) - Nektar reported that they intend to release Phase III RESPIRE 1 top-line data of Cirpofloxacin DPI for Non-Cystic Fibrosis Bronchiectasis in the first half of 2016.

Date Range Delayed (08/09/2014) - Nektar reported that Cipro DPI is being evaluated in non-cystic-fibrosis bronchiectasis and Bayer expects Phase III results in the latter part of 2015. Date Range Delayed (11/18/2014) - Nektar expects potential Phase III data readouts for Ciprofloxacin DPI in non-cystic fibrosis bronchiectasis Trial Data - Top-Line Phase III RESPIRE 2 Top- Nektar expects to have Phase III RESPIRE 2 top-line data in 07/01/2016-12/31/2016 Bayer AG BAYRY Ciprofloxacin DPI Bronchiectasis III throughout 2016. Date Range Refined (01/13/2015) - 97089 Results Line Data� Q3 2015. Nektar expects potential Phase III data readouts for Ciprofloxacin DPI in non-cystic fibrosis bronchiectasis in mid- 2016.Date Range Delayed (01/12/2016) - Nektar reported that they intend to release Phase III RESPIRE 2 top-line data of Cirpofloxacin DPI for Non-Cystic Fibrosis Bronchiectasis in the second half of 2016.

Bayer HealthCare announced the expansion of the clinical Date Range Delayed (12/31/2015) - We await an update in development programme for finerenone with three Phase III the first quarter of 2016.Catalyst Occurred (01/13/2016) - Congestive Heart Failure Trial Announcement - Phase III FINESSE-HF to studies. The studies will investigate the efficacy and safety Now-03/31/2016 Bayer AG BAYRY Finerenone III Bayer announced that Phase III trials of finerenone for 114548 (CHF) Initiation Start of finerenone in patients with chronic heart failure (CHF) diabetic kidney disease and chronic heart failure have been and patients with diabetic kidney disease (DKD) with the initiated. first patients expected to be enrolled by the year-end.

Date Range Delayed (10/08/2015) - BioCryst Pharmaceuticals announced that the randomized placebo- controlled Phase I clinical trial of orally-administered BCX7353 in healthy volunteers successfully met all of its objectives. A Phase II four week dose ranging trial to evaluate the safety tolerability pharmacokinetics BioCryst expects to initiate a Phase II proof of concept study BioCryst Pharmaceuticals, Hereditary Angioedema Trial Announcement - Phase II APeX-1 - Trial to pharmacodynamics and efficacy of BCX7353 as a 01/13/2016-03/31/2016 BCRX BCX7353 I of its second generation HAE compound (BCX7353) by year 106360 Inc. (HAE) Initiation Start preventative treatment to reduce the frequency of attacks end 2015. in HAE patients is expected to begin by late 2015 or early 2016.New Information (01/13/2016) - BioCryst announced that the Phase II dose-ranging study of BCX7353 for the treatment of hereditary angioedema (HAE) is expected to start in the first quarter of 2016 following regulatory review and IRB approvals.

BioDelivery Sciences International and its partner Endo Pharmaceuticals a subsidiary of Endo International announced that the U.S. Food and Drug Administration (FDA) approved Belbuca (CIII) (buprenorphine HCl) buccal Date Range Refined (01/11/2016) - Endo announced that BioDelivery Sciences Progress Update - 02/15/2016-02/29/2016 BDSI Belbuca Chronic Pain Approved U.S. Product Launch film for use in patients with chronic pain severe enough to the launch of Belbuca for chronic pain is anticipated in late 115916 International, Inc. Product Launch (U.S.) require daily around-the-clock long-term opioid treatment February 2016. for which alternative treatment options are inadequate. It is anticipated that Belbuca may become commercially available in the U.S. during the first quarter of 2016.

Date Range Delayed (01/16/2014) - Sangamo plans to file an IND for the Biogen Idec-partnered ZFP beta-thalassemia drug for the treatment of sickle cell anemia in 2015. New Information (01/12/2015) - Sangamo announced that they expect the initiation in the first half of 2015 of a Phase I clinical trial of its zinc finger nuclease (ZFN)-modified Biogen Idec and Sangamo BioSciences announced an hematopoietic stem cell approach for the potential cure of exclusive worldwide collaboration and license agreement beta-thalassemia. The Company also expects that an focused on the development of therapeutics for investigational new drug (IND) application for this approach hemoglobinopathies inherited conditions that result from in sickle cell disease will be filed by the end of the year. Both the abnormal structure or underproduction of hemoglobin. programs are partnered with Biogen Idec. Date Range 07/01/2016-12/31/2016 Biogen, Inc. BIIB ZFP - Beta-Thalassemia Sickle Cell Anemia Preclinical Regulatory - IND Filing IND Filing Sangamo is responsible for all research and development 96735 Delayed (08/05/2015) - Sangamo announced that they activities through the first clinical proof of concept trial in expect to file a new Investigational New Drug (IND) beta-thalassemia and both companies will perform application for its zinc finger nuclease (ZFN)-mediated activities to enable submission of an Investigational New genome editing programs targeting beta-thalassemia and Drug (IND) application for SCD. We await an update on an sickle cell disease (SCD) in 2016. Date Range Delayed IND filing for SCD through 2014. (11/03/2015) - Biogen is now expecting to file an IND in 2017 for its sickle cell product being developed in collaboration with Sangamo.Date Range Expedited (01/12/2016) - Sangamo plans to file an IND for its partnered Sickle cell disease program with Biogen in the second half of 2016.

Date Range Delayed (12/15/2015) - We await an update. Biomarin announced that the complete data from the Phase Date Range Delayed (01/11/2016) - BioMarin announced BioMarin Pharmaceutical 04/01/2016-04/30/2016 BMRN BMN 111 Achondroplasia II Trial Data - Final Results Phase II - Final Results II study of BMN 111 will be presented at a medical meeting that 1 year data from the Phase II study of BMN 111 for the 112738 Inc. later in 2015. treatment of achondroplasia at the 15 µg/kg/QD dose will be presented at the R&D Day in April 2016.

New Information (09/18/2015) - BioMarin expects to be able to start a Phase III study of BMN 111 for the treatment BioMarin is preparing for discussions with health of achondroplasia in the middle of 2016.Date Range BioMarin Pharmaceutical Trial Announcement - authorities on design and planning for registration enabling Delayed (01/11/2016) - BioMarin announced that pending 10/01/2016-12/31/2016 BMRN BMN 111 Achondroplasia II Phase III - Trial to Start 114776 Inc. Initiation studies of vosoritide for the treatment of achondroplasia health authority discussions on study design and potential which the Company hopes to begin in the first half of 2016. timelines the Company anticipates initiating a Phase III study of BMN 111 for the treatment of achondroplasia in the fourth quarter of 2016.

New Information (09/07/2015) - BioMarin reported that its product pipeline products is on track and expected to produce important results over the next six months to nine months. This includes data from the ongoing Phase I/II study of cerliponase alfa for the treatment of CLN2 disorder.Date BioMarin Pharmaceutical Neuronal Ceroid Trial Data - Updated Phase I/II - Complete BioMarin expects complete results from the Phase I/II study 3/2/2016 BMRN BMN 190 I/II Range Delayed (12/15/2015) - BioMarin expects to release 106049 Inc. Lipofuscinosis (NCL) Results Results of BMN-190 in Q4 2015. results from the Phase I/II study of cerliponase alfa for the treatment of CLN2 in the first quarter of 2016. Exact Date (01/11/2016) - BioMarin announced that study results of BMN 190 for the treatment of CLN2 disorder will be presented at WORLD Symposium 2016 on March 2 2016.

Biomedtracker August 2016 / 130 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link

BioMarin Pharmaceutical announced the submission of a New Information (06/25/2015) - BioMarin announced the Marketing Authorization Application (MAA) to the European European Medicines Agency (EMA) has validated the Medicines Agency (EMA) for drisapersen an investigational Marketing Authorization Application (MAA) for drisapersen antisense oligonucleotide drug candidate for the treatment for the treatment of Duchenne Muscular Dystrophy of the largest subset of Duchenne muscular dystrophy amenable to exon 51 skipping. Validation of the MAA (DMD) amenable to single exon skipping. Based on an confirms that the submission is complete and starts the BioMarin Pharmaceutical Regulatory - CHMP internal analysis of the centralized European approval EMA's standard review process. Day 120 questions will be 04/01/2016-06/30/2016 BMRN Kyndrisa Muscular Dystrophy NDA CHMP Opinion 112461 Inc. (European Panel) Results procedure we estimate the European marketing received on October 22 2015 leading to a potential CHMP authorization for this drug for this indication will be granted opinion in the first half of 2016. We continue to await an in approximately 11-17 months. As the approval decision is update in the time frame above.Date Range Refined normally issued 67 days from adoption of a positive (01/11/2016) - BioMarin announced that a CHMP opinion Committee for Medicinal Products for Human Use (CHMP) for Kyndrisa for the treatment of Duchenne Muscular opinion we then estimate the CHMP opinion to occur Dystrophy (exon 51) is expected in the second quarter of between March 2016 and September 2016. 2016.

Date Range Refined (06/25/2015) - BioMarin announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA BioMarin Pharmaceutical announced the submission of a confirms that the submission is complete and starts the Marketing Authorization Application (MAA) to the European EMA's standard review process. Day 120 questions will be Medicines Agency (EMA) for drisapersen an investigational received on October 22 2015 leading to a potential CHMP antisense oligonucleotide drug candidate for the treatment opinion in the first half of 2016 and a European Commission BioMarin Pharmaceutical Regulatory - Approval European Approval of the largest subset of Duchenne muscular dystrophy 06/01/2016-08/31/2016 BMRN Kyndrisa Muscular Dystrophy NDA Decision by the third quarter of 2016. We continue to await 112463 Inc. Decision (Europe) Decision (DMD) amenable to single exon skipping. Based on an an update in the time frame above. Date Range Refined internal analysis of the centralized European approval (01/11/2016) - BioMarin announced that a CHMP opinion procedure we estimate the European marketing for Kyndrisa for the treatment of Duchenne Muscular authorization for this drug for this indication will be granted Dystrophy (exon 51) is expected in the second quarter of in approximately 11-17 months. 2016. As the approval decision is normally issued 67 days from adoption of a positive Committee for Medicinal Products for Human Use (CHMP) opinion we then estimate the approval decision to occur between June and August 2016.

New Information (06/25/2015) - BioMarin announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy BioMarin Pharmaceutical announced the submission of a amenable to exon 51 skipping. Validation of the MAA Marketing Authorization Application (MAA) to the European confirms that the submission is complete and starts the Medicines Agency (EMA) for drisapersen an investigational EMA's standard review process. Day 120 questions will be antisense oligonucleotide drug candidate for the treatment received on October 22 2015 leading to a potential CHMP of the largest subset of Duchenne muscular dystrophy opinion in the first half of 2016. We continue to await an (DMD) amenable to single exon skipping. Based on an update in the time frame above.Date Range Refined BioMarin Pharmaceutical Regulatory - CHMP internal analysis of the centralized European approval (01/11/2016) - BioMarin announced that a CHMP opinion 04/01/2016-06/30/2016 BMRN Kyndrisa Muscular Dystrophy III CHMP Opinion 112461 Inc. (European Panel) Results procedure we estimate the European marketing for Kyndrisa for the treatment of Duchenne Muscular authorization for this drug for this indication will be granted Dystrophy (exon 51) is expected in the second quarter of in approximately 11-17 months. As the approval decision is 2016.New Information (01/14/2016) - BioMarin anticipates normally issued 67 days from adoption of a positive that the Committee for Medicinal Products for Human Use Committee for Medicinal Products for Human Use (CHMP) (CHMP) of the European Medicines Agency (EMA) will opinion we then estimate the CHMP opinion to occur provide an opinion for the company's Marketing between March 2016 and September 2016. Authorization Application (MAA) for Kyndrisa for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping in the first half of 2016. We continue to look for a decision in Q2 as guided at the JP Morgan Healthcare Conference.

Date Range Refined (06/25/2015) - BioMarin announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA's standard review process. Day 120 questions will be received on October 22 2015 leading to a potential CHMP opinion in the first half of 2016 and a European Commission BioMarin Pharmaceutical announced the submission of a Decision by the third quarter of 2016. We continue to await Marketing Authorization Application (MAA) to the European an update in the time frame above. Date Range Refined Medicines Agency (EMA) for drisapersen an investigational (01/11/2016) - BioMarin announced that a CHMP opinion antisense oligonucleotide drug candidate for the treatment for Kyndrisa for the treatment of Duchenne Muscular BioMarin Pharmaceutical Regulatory - Approval European Approval of the largest subset of Duchenne muscular dystrophy Dystrophy (exon 51) is expected in the second quarter of 06/01/2016-08/31/2016 BMRN Kyndrisa Muscular Dystrophy III 112463 Inc. Decision (Europe) Decision (DMD) amenable to single exon skipping. Based on an 2016. As the approval decision is normally issued 67 days internal analysis of the centralized European approval from adoption of a positive Committee for Medicinal procedure we estimate the European marketing Products for Human Use (CHMP) opinion we then estimate authorization for this drug for this indication will be granted the approval decision to occur between June and August in approximately 11-17 months. 2016. New Information (01/14/2016) - BioMarin anticipates that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will provide an opinion for the company's Marketing Authorization Application (MAA) for Kyndrisa for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping in the first half of 2016. The EC is expected to render a final decision for Kyndrisa in the second half of 2016. However we continue to expect a potential European decision in the above date range given

New Information (06/29/2015) - BioMarin announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping and the Prescription Drug User Fee Act (PDUFA) goal date for a BioMarin announced completion of the rolling submission decision is December 27 2015. As this falls on the weekend of a New Drug Application (NDA) to the United States Food of a federal holiday we continue to expect a decision on and Drug Administration (FDA) for drisapersen for the Thursday December 24 2015.Date Range Delayed Regulatory - treatment of a genetically defined subset of Duchenne (12/18/2015) - BioMarin announced that the U.S. Food and BioMarin Pharmaceutical PDUFA for NDA - First Now-01/15/2016 BMRN Kyndrisa Muscular Dystrophy III PDUFA/Approval Decision muscular dystrophy (DMD). Assuming a standard 8-month Drug Administration (FDA) has notified the Company that 110804 Inc. Review (US) priority review timeline under PDUFA V guidelines the they had not yet completed their review process and would PDUFA decision should occur on December 27 2015. As this be unable to take an action by the Prescription Drug User falls on the weekend of a federal holiday we expect a Fee Act (PDUFA) action date for Kyndrisa (drisapersen) of decision on Thursday December 24 2015. December 27 2015 and anticipate taking action in early January 2016. Catalyst Occurred (01/14/2016) - BioMarin announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company's New Drug Application (NDA) for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.

Date Range Refined (12/02/2015) - bluebird bio plans to present data from the Starbeam study of LentiD in CCALD in bluebird bio expects to present data from its Phase II/III the first half of 2016.Date Range Refined (01/12/2016) - Adrenomyeloneuropathy Trial Data - Top-Line Phase II/III Starbeam - Top- 04/15/2016-04/21/2016 bluebird bio BLUE Lenti-D II/III Starbeam study of childhood cerebral bluebird bio announced that they plan to present data from 106377 (Adrenoleukodystrophy) Results Line Results at AAN adrenoleukodystrophy (CCALD) program during 2016. the Starbeam study of LentiD in CCALD at the American Academy of Neurology Annual meeting that takes place from April 15-21 2016.

Biomedtracker August 2016 / 131 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link

New Information (06/10/2015) - bluebird announced the completion of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee’s (RAC) public review of the HGB-208 pediatric study protocol for bluebird bio’s LentiGlobin BB305 product candidate in beta- thalassemia major. The RAC recommendation was to delay initiation of the study in the United States for one to two bluebird bio announced that it has reached general years. This recommendation has no effect on the HGB-207 agreement with the U.S. Food and Drug Administration protocol plan. We continue to await an update through mid- Trial Announcement - HGB-207 Trial 07/01/2016-12/31/2016 bluebird bio BLUE LentiGlobin Anemia I/II (FDA) on the design of its planned clinical trials HGB-207 2015.Date Range Delayed (09/30/2015) - We await an 111829 Initiation (Adolescents) to Start and HGB-208. We await an update on the initiation of these update through the rest of 2015.Date Range Delayed trials through mid-2015. (12/02/2015) - bluebird bio currently plan to initiate two new clinical trials of LentiGlobin called HGB-207 for adult and adolescent patients with beta-thalassemia major and HGB-208 for pediatric patients with beta-thalassemia major. We await an update through the first half of 2016.Date Range Delayed (01/12/2016) - bluebird bio announced that it plans to initiate the HB-207 study of LentiGlobin in the second half of 2016.

Date Range Delayed (12/16/2014) - We await an update.Date Range Delayed (03/27/2015) - We continue to The evaluation of safety and efficacy in the treatment arms await an update.Date Range Delayed (09/24/2015) - We Phase III POSTURE - Trial Data - Updated of the POSTURE study is ongoing and the results of the study continue to await an update.Date Range Delayed Now-12/31/2016 Celgene Corporation CELG Otezla Ankylosing Spondylitis III Updated Results to be 101948 Results will be presented at an upcoming medical meeting. We look (12/23/2015) - We continue to await an update.Date Range Presented for an update through 2014. Delayed (01/11/2016) - Celgene expects to present long- term data from its Phase III study of Otezla in patients with ankylosing spondylitis in 2016. Date Range Delayed (01/11/2016) - Celgene expects to have Trial Data - Top-Line Celgene expects Phase II data for Otezla for ulcerative colitis Now-12/31/2016 Celgene Corporation CELG Otezla Atopic Dermatitis (Eczema) II Phase II - Top-Line Results data from its Phase II study Otezla in patients with atopic 105995 Results and atopic dermatitis beginning in H1 2016. dermatitis in 2016. Date Range Delayed (01/11/2016) - Celgene expects to have Trial Data - Top-Line Celgene expects Phase II data for Otezla for ulcerative colitis 09/01/2016-04/30/2017 Celgene Corporation CELG Otezla Ulcerative Colitis (UC) II Phase II - Top-Line Results data from its Phase II study of Otezla for ulcerative colitis in 105994 Results and atopic dermatitis beginning in H1 2016. late 2016/early 2017. Date Range Delayed (01/12/2015) - Celgene expects to Chronic Lymphocytic complete patient enrollment in the Phase III CONTINUUM Leukemia (CLL)/Small Cell Trial Data - Top-Line Phase III - CLL-002 Top- Celgene expects data from the Phase III CLL-002 study of study of Revlimid in H2 2015. As such we look for a Now-12/31/2016 Celgene Corporation CELG Revlimid III 70263 Lymphocytic Lymphoma Results Line Results Revlimid beginning in 2015. potential data read-out in the first half of 2016.Date Range (SLL) - NHL Delayed (01/11/2016) - Celgene expects to have data from its Phase III CONTINUUM study of Revlimid in 2016.

Date Range Delayed (12/14/2015) - Celgene announced that data is expected from the Revlimid lymphoma trials Indolent Non-Hodgkin's Trial Data - Top-Line Phase III - RELEVANCE Top- Celgene expects data from the Phase III RELEVANCE study of beginning in 2017. We expect an update in the time frame 01/01/2017-06/30/2017 Celgene Corporation CELG Revlimid III 70261 Lymphoma - NHL Results Line Results Revlimid beginning in 2015. above. Date Range Refined (01/11/2016) - Celgene expects data from its Phase III RELEVANCE study of Revlimid in the first half of 2017.

Date Range Delayed (10/30/2015) - We await an update.Date Range Delayed (11/19/2015) - The EMA does not list Revlimid under its November 16-19 2015 CHMP Celgene expects an EU approval decision for Revlimid for meeting agenda. We continue to await an update.Date Regulatory - CHMP relapsed/refractory mantle cell lymphoma in Q4 2015. As Range Delayed (12/14/2015) - The EMA does not list Mantle Cell Lymphoma - (European Panel) CHMP Opinion - the approval decision is normally issued 67 days from Revlimid under its December 14-17 2015 CHMP meeting 01/11/2016-06/30/2016 Celgene Corporation CELG Revlimid Approved 105987 NHL Supplemental Filing Relapsed/Refractory MCL adoption of a positive Committee for Medicinal Products for agenda. We continue to await an update.Date Range Results Human Use (CHMP) opinion we then estimate the CHMP Delayed (01/11/2016) - Celgene expects to receive a opinion to occur in the time frame above. decision from the Committee for Medicinal Products for Human Use (CHMP) on the submission of REVLIMID for relapsed/refractory mantle cell lymphoma in Europe in 2016. We await an update through the first half of 2016.

Date Range Delayed (11/19/2015) - The EMA does not list Revlimid under its November 16-19 2015 CHMP meeting agenda. As the approval decision is normally issued 67 days from adoption of a positive Committee for Medicinal Products for Human Use (CHMP) opinion we then estimate the approval decision to occur in January to February 2016 Regulatory - Supplemental EU Mantle Cell Lymphoma - Celgene expects an EU approval decision for Revlimid for assuming a December 2015 CHMP opinion. Date Range 07/01/2016-12/31/2016 Celgene Corporation CELG Revlimid Approved Supplemental Approval Approval Decision - 105984 NHL relapsed/refractory mantle cell lymphoma in Q4 2015. Delayed (01/11/2016) - Celgene expects to receive a (Europe) Relapsed/Refractory MCL decision from the Committee for Medicinal Products for Human Use (CHMP) on the submission of REVLIMID for relapsed/refractory mantle cell lymphoma in Europe in 2016. We await an update on the CHMP opinion through the first half of 2016 and an approval decision in the second half of 2016.

Date Range Refined (10/05/2015) - A Phase II/III open-label non-randomized single-arm multi-center study to evaluate oral sodium fusidate (CEM-102) for the treatment of staphylococcal bone or joint infections in subjects for whom chronic antibiotic suppressive therapy is indicated has been announced but is not yet open for participant recruitment. The study is expected to begin in October 2015. Date Range Delayed (10/28/2015) - The Phase II/III study of CEM-102 for the treatment of bone or joint infections is not yet open Bone and Joint Infections Trial Announcement - Cempra expects to initiate Phase III development of Taksta for participant recruitment. We await an update in the time Now-03/31/2016 Cempra, Inc. CEMP Taksta II Phase III - Trial to Start 109330 (Antibacterial) Initiation in refractory bone and joint infections in 2015. frame above.Date Range Delayed (11/16/2015) - The Phase II/III study of CEM-102 for the treatment of bone or joint infections is not yet open for participant recruitment. We await an update in the time frame above.Date Range Delayed (12/08/2015) - Cempra plans to initiate an exploratory trial for Taksta early next year in patients with refractory bone or joint infections. Date Range Refined (01/13/2016) - Cempra announced plans to initiate a Phase III study of Taksta for the treatment of bone or joint infections in the first quarter of 2016.

ChemoCentryx announced that patient recruitment in the Date Range Refined (01/14/2016) - ChemoCentryx plans to Trial Data - Top-Line Phase Ib - Overall Phase Ib trial of CCX872 is ramping up having reached 20 report overall response data from the Phase Ib clinical trial Now-06/30/2016 ChemoCentryx, Inc. CCXI CCX872 Pancreatic Cancer I 116297 Results Response Data percent of the target enrollment. ChemoCentryx looks of CCX872 in patients with pancreatic cancer in the first half forward to efficacy data from this trial in 2016. of 2016. Date Range Delayed (01/12/2016) - Chimerix announced that the company will review data from the AdVise study Regulatory - NDA/BLA Chimerix plans to submit the NDA for Brincidofovir using with Regulatory agencies to determine a path for approval. 09/01/2016-12/31/2016 Chimerix, Inc. CMRX Brincidofovir Antiviral - Other Treatments III NDA Filing 106365 Filing both the SUPRESS and AdVise data in early 2016. The Company aims to determine its development plan by the summer of 2016. As such we await a NDA filing in late 2016.

Date Range Delayed (12/16/2015) - Chimerix announced Chimerix expects that final results from its pivotal study of that the Company expects to review data from the animal an animal model for smallpox including data on the rule study of brincidofovir for the treatment of smallpox Final Preclinical Results at incidence and severity of clinical and laboratory events in 02/08/2016-02/10/2016 Chimerix, Inc. CMRX Brincidofovir Smallpox I Trial Data - Other with the FDA in the first half of 2016.Date Range Refined 113408 ASM each cohort are expected by the fourth quarter of 2015 and (01/12/2016) - Chimerix will announce full data from the will be submitted to an upcoming medical conference and Animal Rule Study at the upcoming ASM Biodefense and to the FDA for discussion of next steps. Emerging Diseases Research Meeting in February 2016.

Biomedtracker August 2016 / 132 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (12/14/2015) - Results from the Clovis sponsored U.S. Phase II study of lucitanib was not presented at the 2015 San Antonio Breast Cancer Symposium. As such we await an update in the time frame above. Date Range Clovis expects preliminary data from its Phase II breast Delayed (01/13/2016) - Clovis announced an overview of Trial Data - Top-Line Phase II (Clovis) - Top-Line cancer study of lucitinib by year-end 2015 presumably at lucitanib at the 34th annual JP Morgan Healthcare 01/13/2016-03/31/2016 Clovis Oncology, Inc. CLVS Lucitanib Breast Cancer II 106057 Results Results the San Antonio Breast Cancer Symposium (SABCS) to be Conference. There is an unclear regulatory path as held December 8-12 2015. monotherapy and future development is focused on combination studies. Enrollment (n=160) completion is expected the first quarter of 2016 for the Phase II trial of lucitanib for breast cancer. We await an update on the initial data in the same time frame. Date Range Delayed (12/28/2015) - We await an update.Date Range Delayed (01/13/2016) - Clovis Clovis announced that a Phase II study of rociletinib in Non-Small Cell Lung Cancer Trial Announcement - Phase II (TIGER-J2) to announced that they are delaying the TIGER-J2 Phase II 01/13/2016-09/30/2016 Clovis Oncology, Inc. CLVS Rociletinib NDA Japanese patients agreed upon with Japanese regulatory 111246 (NSCLC) Initiation Start study in Japanese patients and enrolling Asian patients in authorities is expected to initiate in the second half of 2015. other ongoing studies. We await an update on the start of this trial.

Date Range Delayed (12/28/2015) - A Phase Ib/II study of the safety and efficacy of rociletinib (CO-1686) Clovis Oncology announced that they have entered into a administered in combination with MPDL3280A in patients clinical trial collaboration with Genentech a member of the with activating EGFR mutation-positive (EGFRm) advanced Roche Group to evaluate a novel combination therapy of or metastatic non-small cell lung cancer (NSCLC) is not yet Phase Ib/II Genentech’s investigational cancer immunotherapy open for participant recruitment. We await an Non-Small Cell Lung Cancer Trial Announcement - Now-01/31/2016 Clovis Oncology, Inc. CLVS Rociletinib NDA w/Atezolizumab - Trial to atezolizumab (MPDL3280A; anti-PDL1) and rociletinib for update.Catalyst Occurred (01/13/2016) - Clovis announced 114046 (NSCLC) Initiation Start the treatment of advanced EGFR-mutant non-small cell lung that the Phase Ib/II study of the safety and efficacy of cancer (NSCLC). The Phase Ib/II trial of rociletinib in rociletinib (CO-1686) administered in combination with combination with atezolizumab is planned to begin atezolizumab in patients with activating EGFR mutation- enrolling patients before the end of 2015. positive (EGFRm) advanced or metastatic non-small cell lung cancer (NSCLC) has begun clinical activities this week starting with the first clinical site.

Date Range Delayed (06/29/2015) - We await an update in the third quarter of 2015.Date Range Delayed (09/30/2015) - This study is not yet open for participant recruitment. We await an update.Date Range Delayed (11/18/2015) - This Trial Announcement - Phase II RUBY (Unicancer) Clovis plans to initiate a Phase II trial called the RUBY Study study is not yet open for participant recruitment. We 01/13/2016-04/30/2016 Clovis Oncology, Inc. CLVS Rucaparib Breast Cancer II 106062 Initiation Study to Start during the second quarter of 2015. continue to await an update.Date Range Delayed (01/13/2016) - Clovis plans to initiate the Phase II RUBY trial in non-known gBRCA and BRCA-like HER2-negative ER/PR- positive breast cancer patients with rucaparib in 2016. We await an update.

New Information (04/06/2015) - Clovis Oncology announced that the ARIEL2 trial was recently expanded into a registration study (the ARIEL2 extension) which will include an additional approximately 300 women with recurrent disease after at least three prior lines of chemotherapy. Data from this study are planned to serve as the basis of a New Drug Application (NDA) filing for the Regulatory - Rolling Clovis is planning an NDA submission for rucaparib in treatment of ovarian cancer in 2016.Date Range Refined Now-03/31/2016 Clovis Oncology, Inc. CLVS Rucaparib Ovarian Cancer III Rolling NDA Initiation 106054 NDA/BLA Initiation ovarian cancer in 2016. (01/13/2016) - Clovis is planning a U.S. NDA submission in the advanced tumor tissue BRCA mutant (germline and somatic mutations) ovarian cancer population. The NDA will include platinum sensitive and platinum resistant patients. The initial NDA for tBRCAmut patients with advanced disease plans to start by a rolling submission to initiate in the first quarter of 2016 and to complete by the second quarter of 2016.

Date Range Refined (01/12/2015) - Coherus announced Coherus is enrolling two Phase III clinical trials of CHS-0214 during their 2015 J.P. Morgan Healthcare Conference that (biosimilar etanercept) for rheumatoid arthritis and Biosimilar Etanercept Regulatory - MAA the MAA filing of Biosimilar Etanercept for Psoriasis will be 07/01/2016-12/31/2016 Coherus BioSciences, Inc. CHRS Psoriasis III MAA Filing psoriasis and expects the results of these studies if positive 103619 (Coherus) Submission (Europe) filed in mid-2016.Date Range Delayed (01/11/2016) - to support the filing of a marketing application in Europe in Coherus expects to file a MAA for CHS-0214 in Europe in the 2016. second half of 2016. Coherus is enrolling two Phase III clinical trials of CHS-0214 (biosimilar etanercept) for rheumatoid arthritis and Biosimilar Etanercept Regulatory - MAA Date Range Refined (01/11/2016) - Coherus expects to file a 07/01/2016-12/31/2016 Coherus BioSciences, Inc. CHRS Rheumatoid Arthritis (RA) III MAA Filing psoriasis and expects the results of these studies if positive 103618 (Coherus) Submission (Europe) MAA for CHS-0214 in Europe in the second half of 2016. to support the filing of a marketing application in Europe in 2016. Date Range Refined (01/11/2016) - A randomized single- blind single-dose 2-period crossover Phase I study in healthy Coherus BioSciences announced the Company plans to subjects to assess PK PD and safety (including Biosimilar Pegfilgrastim Trial Announcement - Phase I PK/PD Follow-On 01/11/2016-01/31/2016 Coherus BioSciences, Inc. CHRS Neutropenia / Leukopenia I initiate a follow-on study of CHS-1701 in healthy volunteers. immunogenicity) of a single 6mg subcutaneous injection of 117244 (Coherus) Initiation Trial to Start As such we await an update in the time frame above. CHS-1701 (Coherus pegfligrastim) compared with a single 6mg SC dose of Neulasta (pegfilgrastim) is expected to start in January 2016. Date Range Delayed (12/28/2015) - We await an Coherus announced that the Phase I immunogenicity study Biosimilar Pegfilgrastim Trial Data - Top-Line Phase I - Immunogenicity update.New Information (01/11/2016) - The 01/11/2016-03/31/2016 Coherus BioSciences, Inc. CHRS Neutropenia / Leukopenia I is projected to be concluded in 2015 to support submission 112686 (Coherus) Results Top-Line Results immunogenicity study of CHS-1701 is expected to readout of the BLA. in the first quarter of 2016. Ignyta's strategic priorities for 2016 with respect to RXDX- 105 are achieving clinical proof of concept in patients with Date Range Refined (01/14/2016) - Ignyta announced that Trial Announcement - Now-03/31/2016 Daiichi Sankyo Co., Ltd. DSKYF RXDX-105 Colorectal Cancer (CRC) I/II Phase Ib Portion to Start activating RET alterations and initiating the Phase Ib portion the Phase Ib portion of the umbrella basket study is planned 118074 Other of the ongoing clinical trial in patients with solid tumors to start in the first quarter of 2016. harboring RET or BRAF alterations.

Date Range Delayed (01/15/2015) - Dynavax expects to begin the Phase IIa study in Asthma patients in the second or third quarter of 2015. Date Range Delayed (04/07/2015) - Dynavax is currently working with AstraZeneca to design Dynavax Technologies announced that the Company and the new Phase IIa trial which AstraZeneca will fully fund and AstraZeneca signed an amendment to the existing Research Dynavax will conduct beginning in the second half of Collaboration and License Agreement under which 2015.Date Range Delayed (07/21/2015) - Dynavax Dynavax Technologies Trial Announcement - Now-12/31/2016 DVAX AZD1419 Asthma I Phase IIa - Trial to Start AstraZeneca will fully fund and Dynavax will conduct a announced that a Phase II study for the asthma therapeutic 105284 Corporation Initiation Phase IIa safety and efficacy trial of AZD1419 in patients program in partnership with AstraZeneca is expected to with asthma. Dynavax intends to initiate the Phase IIa study start in early 2016. Date Range Delayed (09/09/2015) - in asthma patients in the first half of 2015. Dynavax announced that the Company is currently working with AstraZeneca to design a Phase II study for AZD1419 that is expected to begin in the first half of 2016.Date Range Delayed (01/14/2016) - Dynavax expects to begin Phase II development for AZD1419 in 2016.

Merck and Dynavax Technologies announced they have entered into two clinical trial collaboration agreements to investigate the potential synergistic effect of combining Date Range Delayed (12/16/2015) - We await an update. immunotherapies from both companies’ pipelines: Merck’s Dynavax Technologies Trial Announcement - Phase I w/MK1966 - Trial Date Range Delayed (01/14/2016) - Dynavax expects to Now-06/30/2016 DVAX SD-101 (Dynavax) Hematologic Cancer I/II KEYTRUDA (pembrolizumab) and MK-1966 with Dynavax’s 112263 Corporation Initiation to Start initiate the Phase I study of SD-101 in combination with MK- SD-101. A Phase I study exploring the safety and efficacy of 1966 in the first half of 2016. combining SD-101 with MK-1966 in patients with solid or hematological malignancies is expected to be initiated in the second half of 2015.

Date Range Refined (09/17/2015) - Incyte expects a submission of the NDA filing for baricitinib by Lilly in late Regulatory - NDA/BLA Eli Lilly plans to file for approval of Baricitinib for Now-04/30/2016 Eli Lilly & Company LLY Baricitinib Rheumatoid Arthritis (RA) III NDA Filing 2015 or early 2016. Date Range Refined (01/12/2016) - Eli 106223 Filing rheumatoid arthritis in 2015. Lilly expects to file regulatory submissions for Baricitinib for the treatment of rheumatoid arthritis in early 2016.

Date Range Delayed (09/17/2015) - Incyte expects a submission of the NDA filing for baricitinib by Lilly in late Regulatory - MAA Eli Lilly plans to file for approval of Baricitinib for 2015 or early 2016. We expect an EU filing in a similar time Now-04/30/2016 Eli Lilly & Company LLY Baricitinib Rheumatoid Arthritis (RA) III MAA Submission 106224 Submission (Europe) rheumatoid arthritis in 2015. frame.Date Range Refined (01/12/2016) - Eli Lilly expects to file regulatory submissions for Baricitinib for the treatment of rheumatoid arthritis in early 2016.

Biomedtracker August 2016 / 133 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Based on the ongoing discussions with the FDA Eli Lilly intends to submit U.S. and European regulatory applications Date Range Delayed (01/12/2016) - Eli Lilly announced Regulatory - MAA for Olaratumab in soft tissue sarcoma using the Phase II Now-12/31/2016 Eli Lilly & Company LLY LY3012207 Sarcoma III MAA Filing plans for a European regulatory submission for Olaratumab 113546 Submission (Europe) data. The Company aims to complete the U.S. submission for the treatment of soft-tissue sarcoma in 2016. before the end of 2015. We look for an update on the European filing through the first half of 2016.

Based on the ongoing discussions with the FDA Eli Lilly Date Range Delayed (12/18/2015) - We await an intends to submit U.S. and European regulatory applications update.Catalyst Occurred (01/12/2016) - Eli Lilly announced Regulatory - NDA/BLA Now-01/31/2016 Eli Lilly & Company LLY LY3012207 Sarcoma III NDA Filing for Olaratumab in soft tissue sarcoma using the Phase II that the Company has begun regulatory submission for 113547 Filing data. The Company aims to complete the U.S. submission olaratumab for the treatment of soft tissue sarcoma in late before the end of 2015. 2015.

New Information (01/10/2016) - Emergent BioSolutions Emergent BioSolutions announced that its Board of announced that it has received a favorable private letter Directors has authorized management to pursue a tax-free ruling from the Internal Revenue Service related to the spin-off of the company's Biosciences business into a planned spin-off of its biosciences business. Subject to its separate stand-alone publicly-traded company. The spin-off terms the ruling confirms that certain aspects of the is expected to create two independent public companies planned transaction including those related to the with distinct strategic plans growth strategies and qualification of the business as an active trade or business operational and development priorities.The new will not preclude the spin-off from qualifying as tax-free to Biosciences company to be named at a later date will focus Emergent and its stockholders. The Company's next steps Emergent BioSolutions, Company - Biosciences Spin-Off 04/01/2016-09/30/2016 EBS on providing novel oncology and hematology therapeutics. include selecting the Aptevo senior management team filing 113922 Inc. Divestment/Spinoff Complete The core technology of the Biosciences company will be its with and obtaining clearance from the Securities and ADAPTIR platform applied to immuno-oncology. Emergent Exchange Commission of a Form 10 Registration Statement BioSolutions will continue to operate as a global specialty and completing the distribution of Aptevo shares to biopharmaceutical company whose core business is focused stockholders which Emergent expects to be completed by on providing specialty products for civilian and military the middle of 2016. New Information (01/11/2016) - populations that address intentional and naturally emerging Emergent Biosolutions announced that the Company is public health threats. The transaction is expected to be pursuing a spin-off of the Company's Bioscience business completed in mid-2016. into a separate stand-alone and publicly-traded company named Aptevo Therapeutics.

New Information (01/13/2016) - Enanta announced the Enanta announced that in their NASH program the Company Enanta Pharmaceuticals, Non-alcoholic Trial Announcement - nomination of EDP-305 as the developmental candidate for Now-12/31/2016 ENTA EDP-305 Preclinical Clinical Trials to Start expects to select the candidate by the end of 2015 and 111422 Inc. Steatohepatitis (NASH) Initiation the study of NASH and PBC. The Company is currently on initiate clinical studies in 2016. tract to initiate clinical trials in 2016.

Date Range Delayed (12/14/2015) - Enanta announced that they are working on several compounds that selectively bind to and activate the farnesoid X receptor. Enanta plans to develop these compounds for use in the treatment of non- Enanta Pharmaceuticals, Non-alcoholic Progress Update - Enanta expects to select a candidate for its FXR agonist alcoholic steatohepatitis or NASH and possibly primary Now-12/31/2016 ENTA EDP-305 Preclinical Candidate Selection 108480 Inc. Steatohepatitis (NASH) Development Review program for NASH in 2015. biliary cholangitis or PBC. Clinical trials of an FXR agonist candidate are expected to initiate in 2016. We await an update.Catalyst Occurred (01/13/2016) - Enanta announced the selection of EDP-305 as the product candidate to enter clinical trials for the treatment of NASH and PBC.

Date Range Delayed (06/29/2015) - We await an update through the third quarter of 2015.Date Range Delayed (08/13/2015) - Endo expects to initiate Phase IIb studies of XIAFLEX in Frozen Shoulder Syndrome/AdhesiveCapsulitis Auxilium looks forward to initiating a planned Phase IIb and Cellulite by end of 2015.Date Range Delayed Trial Announcement - clinical trial of Xiaflex for the treatment of edematous (12/28/2015) - Endo plans to initiate Phase IIb studies for 01/11/2016-02/29/2016 Endo International plc ENDP Xiaflex Cellulite II Phase IIb - Trial to Start 102970 Initiation fibrosclerotic panniculopathy (EFP) in the second quarter of CCH in adhesive capsulitis and cellulite shortly. We await an 2015. update through the first quarter of 2016.Date Range Delayed (01/11/2016) - Endo announced that they held a meeting with the FDA in December 2015 for Xiaflex for cellulite. A Phase IIb clinical trial initiation is expected in the near-term.

Endo International announced that they are conducting Date Range Expedited (08/13/2015) - Endo expects to additional analyses to determine the path forward for initiate Phase IIb studies of XIAFLEX in Frozen Shoulder Xiaflex for the treatment of frozen shoulder syndrome Syndrome/AdhesiveCapsulitis and Cellulite by end of based on the results from the first Phase IIb study. initiate Phase IIb studies for CCH in adhesive capsulitis and 01/11/2016-04/30/2016 Endo International plc ENDP Xiaflex II Phase IIb - Trial to Start 112154 (Adhesive Capsulitis) Initiation The Company expects to pursue additional testing in this cellulite shortly. We await an update through the first indication most likely in the form of a Phase IIb trial to quarter of 2016.Date Range Delayed (01/11/2016) - Endo provide proof for the top-line results seen in the Phase IIa expects to meet with the FDA in the first quarter of 2016 to trial. As this study is not expected to occur in 2015 we await discuss Xiaflex for adhesive capsulitis. Trial initiation is an update in early 2016. anticipated after that discussion.

Esperion reported that specifics of the Phase III Date Range Refined (01/13/2016) - Esperion Therapeutics Esperion Therapeutics, Dyslipidemia / Progress Update - Progress Update - Phase development program for ETC-1002 for the treatment of 04/01/2016-06/30/2016 ESPR ETC-1002 III will provide details of the full Phase III global development 115253 Inc. Hypercholesterolemia Development Review III Program Update patients with hypercholesterolemia are anticipated to be strategy in the second quarter of 2016. finalized by the first half of 2016.

Date Range Delayed (01/13/2016) - Esperion Therapeutics announced the start of a global Phase III long-term safety and tolerability study (ETC-1002-040) of bempedoic acid in Phase III Long-Term Esperion expects to start a Phase III long-term safety study Esperion Therapeutics, Dyslipidemia / Trial Data - Top-Line patients with hyperlipidemia whose LDL-C is not adequately 10/01/2017-12/31/2017 ESPR ETC-1002 III Safety Study - Top-Line of ETC-1002 in Q4 2015 with a potential for results in the 115277 Inc. Hypercholesterolemia Results controlled with low- and moderate-dose statins. This study Results first half of 2017. will enable the Company to understand the 52 week safety profile of bempedoic acid and top-line results are expected in the fourth quarter of 2017.

Date Range Delayed (12/23/2015) - We await an update. Trial Announcement - Genmab plans to bring HuMax-AXL ADC to the clinic this Date Range Delayed (01/14/2016) - Genmab announced 01/14/2016-12/31/2016 Genmab A/S GEN:DC HuMax-AXL ADC Solid Tumors Preclinical Phase I - Trial to Start 106469 Initiation year. that the Company plans to initiate clinical trials for HuMax- AXL-ADC in solid tumors in 2016.

Gilead announced that they plan to begin clinical studies of Date Range Delayed (12/28/2015) - We await an update. Non-alcoholic Trial Announcement - GS-9674 for the treatment of non-alcoholic steatohepatitis Catalyst Occurred (01/11/2016) - Gilead lists GS-9674 in Now-02/29/2016 Gilead Sciences, Inc. GILD GS-9674 I Clinical Trials to Start 111034 Steatohepatitis (NASH) Initiation (NASH). The Company expects to start the trial before the Phase I development for the treatment of non-alcoholic end of the year 2015. steatohepatitis (NASH).

New Information (07/28/2014) - Per a company representative data for the Phase II studies of simtuzumab in cirrhosis and non-cirrhosis patients with NASH are expected in mid-2015.Date Range Delayed (01/13/2015) - Gilead expects Phase II 48-week NASH data for Simtuzumab Gilead announced that data for a Phase II study of in Q4 2015.New Information (07/31/2015) - Gilead expects Non-alcoholic Trial Data - Top-Line Phase IIb Cirrhosis - Top- simtuzumab for NASH is expected in the middle of 2015. We a 48-week interim analysis for futility by the DSMB for 01/11/2016-06/30/2016 Gilead Sciences, Inc. GILD Simtuzumab IIb 102312 Steatohepatitis (NASH) Results Line Results expect data to be available for the Phase II study of patients Simtuzumab in Q4 2015. We assume this will be applicable with compensated cirrhosis secondary to NASH. to both ongoing Phase II studies.Date Range Delayed (12/18/2015) - We await an update.Date Range Delayed (01/11/2016) - Gilead announced that top-line data from Phase II studies of simtuzumab for the treatment of non- alcoholic steatohepatitis (NASH) are expected in 2016. We await an update in the first half of 2016.

Date Range Delayed (01/13/2015) - Gilead expects Phase II 48-week NASH data for Simtuzumab in Q4 2015.New Information (07/31/2015) - Gilead expects a 48-week interim analysis for futility by the DSMB for Simtuzumab in Per a company representative data for the Phase II studies Q4 2015. We assume this will be applicable to both ongoing Non-alcoholic Trial Data - Top-Line Phase IIb Advanced 01/11/2016-06/30/2016 Gilead Sciences, Inc. GILD Simtuzumab IIb of simtuzumab in cirrhosis and non-cirrhosis patients with Phase II studies.Date Range Delayed (12/18/2015) - We 102339 Steatohepatitis (NASH) Results Fibrosis - Top-Line Results NASH are expected in mid-2015. await an update.Date Range Delayed (01/11/2016) - Gilead announced that top-line data from Phase II studies of simtuzumab for the treatment of non-alcoholic steatohepatitis (NASH) are expected in 2016. We await an update in the first half of 2016.

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BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Catalyst Occurred (01/12/2016) - Gilead announced that it has submitted a New Drug Application (NDA) to the U.S. Tenofovir alafenamide Hepatitis B (HBV) Treatment Regulatory - NDA/BLA Food and Drug Administration (FDA) for tenofovir Now-03/31/2016 Gilead Sciences, Inc. GILD NDA NDA Filing Gilead expects to submit an NDA for TAF in HBV in Q1 2016. 113739 fumarate (Antiviral) Filing alafenamide (TAF) 25 mg an investigational once-daily treatment for adults with chronic hepatitis B virus (HBV) infection.

Following the recommendation by an independent Data Monitoring Committee (DMC) Gilead announced that its Phase III Study 115 evaluating Zydelig (idelalisib) added to Chronic Lymphocytic standard therapy in previously-treated chronic lymphocytic Leukemia (CLL)/Small Cell Regulatory - sNDA/sBLA leukemia (CLL) patients will be unblinded early. Detailed Date Range Refined (01/11/2016) - Gilead announced that a 01/11/2016-06/30/2016 Gilead Sciences, Inc. GILD Zydelig Approved sNDA Filing - Study 0115 116747 Lymphocytic Lymphoma Filing results from this study will be presented during a late- sNDA filing of Zydelig is planned for the first half of 2016. (SLL) - NHL breaking abstracts session (#LBA-5) at the Annual Meeting of the American Society of Hematology (ASH) meeting. Based on these results Gilead plans to submit supplemental regulatory filings in the U.S. and Europe early next year. New Information (01/13/2015) - Amicus reported that FDA regulatory interaction for the Amigal studies for the treatment of Fabry's disease is expected in the first quarter of 2015. We await an update on the timing of a NDA filing following the meeting with the FDA.Date Range Delayed (03/19/2015) - Amicus Therapeutics announced that it has met with regulatory authorities in the U.S. to discuss the approval pathways for migalastat HCl as a precision medicine monotherapy for Fabry patients who have amenable genetic mutations. Subsequently Amicus plans to schedule a pre-NDA meeting and to submit an accelerated approval NDA under Subpart H in the second half of Amicus expects to present results for Amigal studies for the 2015.Date Range Refined (09/15/2015) - Amicus treatment of Fabry's disease to the FDA in the fourth Regulatory - NDA/BLA Therapeutics announced that a Pre-NDA meeting was held Now-03/31/2016 GlaxoSmithKline plc GSK Galafold Fabry's Disease III NDA Filing quarter of 2014. As submission pre-submissions and 102944 Filing with the U.S. Food and Drug Administration (FDA) to discuss meetings occur in the second half of 2014 Amicus expects a migalastat for the treatment of Fabry disease. Amicus potential submission to the FDA in early 2015. remains on track to submit an NDA in the fourth quarter of 2015 under Accelerated Approval.Date Range Delayed (10/02/2015) - Amicus Therapeutics announced additional regulatory guidance from the U.S. Food and Drug Administration (FDA) on the oral small molecule pharmacological chaperone migalastat for the treatment of Fabry disease. In conjunction with the Agency Amicus is further evaluating several U.S. pathways including potentially generating additional data on migalastat's effect on gastrointestinal symptoms in Fabry disease to support submission requesting full approval as well as a Subpart H strategy. In addition the Agency has requested further

Amicus has submitted a marketing authorization application (MAA) to request full approval of Galafold (migalastat HCl) for Fabry patients who have amenable genetic mutations. New Information (06/25/2015) - Amicus Therapeutics Galafold is the first investigational Fabry drug to be granted announced that the European Medicines Agency (EMA) has Accelerated Assessment in the EU. Under Accelerated validated the Marketing Authorization Application (MAA) Assessment the Committee for Medicinal Products for and the Centralized Procedure has begun for the oral small Human Use (CHMP) may shorten the MAA review period molecule pharmacological chaperone Galafold (migalastat Regulatory - CHMP from 210 days under standard review to 150 days under HCl) for Fabry patients who have amenable genetic 02/01/2016-03/31/2016 GlaxoSmithKline plc GSK Galafold Fabry's Disease III CHMP Opinion 112326 (European Panel) Results Accelerated Assessment. Based on an internal analysis of mutations. As previously announced Galafold is the first the centralized European approval procedure we estimate investigational Fabry drug to be granted Accelerated the European marketing authorization for this drug for this Assessment in the EU. Date Range Expedited (01/12/2016) - indication will be granted in approximately 9-15 months. As Amicus expects a CHMP opinion on Galafold for Fabry's the approval decision is normally issued 67 days from disease in early 2016 likely during the February or March adoption of a positive Committee for Medicinal Products for meeting. Human Use (CHMP) opinion we then estimate the CHMP opinion to occur between January 2016 and July 2016.

New Information (01/13/2015) - Amicus reported that FDA regulatory interaction for the Amigal studies for the treatment of Fabry's disease is expected in the first quarter of 2015. We await an update on the timing of a NDA filing following the meeting with the FDA.Date Range Delayed (03/19/2015) - Amicus Therapeutics announced that it has met with regulatory authorities in the U.S. to discuss the approval pathways for migalastat HCl as a precision medicine monotherapy for Fabry patients who have amenable genetic mutations. Subsequently Amicus plans to schedule a pre-NDA meeting and to submit an accelerated approval NDA under Subpart H in the second half of Amicus expects to present results for Amigal studies for the 2015.Date Range Refined (09/15/2015) - Amicus treatment of Fabry's disease to the FDA in the fourth Regulatory - NDA/BLA Therapeutics announced that a Pre-NDA meeting was held Now-03/31/2016 GlaxoSmithKline plc GSK Galafold Fabry's Disease III NDA Filing quarter of 2014. As submission pre-submissions and 102944 Filing with the U.S. Food and Drug Administration (FDA) to discuss meetings occur in the second half of 2014 Amicus expects a migalastat for the treatment of Fabry disease. Amicus potential submission to the FDA in early 2015. remains on track to submit an NDA in the fourth quarter of 2015 under Accelerated Approval.Date Range Delayed (10/02/2015) - Amicus Therapeutics announced additional regulatory guidance from the U.S. Food and Drug Administration (FDA) on the oral small molecule pharmacological chaperone migalastat for the treatment of Fabry disease. In conjunction with the Agency Amicus is further evaluating several U.S. pathways including potentially generating additional data on migalastat's effect on gastrointestinal symptoms in Fabry disease to support submission requesting full approval as well as a Subpart H strategy. In addition the Agency has requested further

Date Range Delayed (12/29/2015) - Glaxo Kline Smith still plans to initiate a Phase III randomized double-blind placebo- controlled multicenter international study (CARDIO-TTR) in patients with familial amyloid cardiomyopathy (FAC) and wt- TTR amyloidosis who also have a history of heart failure. The study will measure the effects of ISIS-TTRRx on a clinical Transthyretin Amyloid composite outcome that includes mortality cardiac GlaxoSmithKline anticipates initiating a Phase III study of (ATTR) Cardiomyopathy Trial Announcement - Phase III - CARDIO-TTR to transplant and cardiovascular hospitalization. We await an 01/11/2016-04/30/2016 GlaxoSmithKline plc GSK IONIS-TTRrx II ISIS-TTRrx in patients with TTR-related cardiomyopathy 110662 (Familial Amyloidotic Initiation Start update regarding the initiation of the trial. Date Range later in 2015. Cardiomyopathy) Delayed (01/04/2016) - Isis announced that GSK plans to initiate the Phase III CARDIO-TTR outcome study enrolling all forms of TTR amyloid cardiomyopathy in the first half of 2016.Date Range Expedited (01/11/2016) - Ionis announced that GSK plans to initiate the Phase III CARDIO-TTR outcome study enrolling all forms of TTR amyloid cardiomyopathy in early 2016.

Date Range Delayed (12/29/2015) - Glaxo Kline Smith plans to initiate a small Phase III study evaluating ISIS-TTRRx in Transthyretin-related patients with FAP in Japan. We await an update regarding GlaxoSmithKline anticipates initiating a Phase III study of Hereditary Amyloidosis Trial Announcement - the initiation of the trial. Date Range Delayed (01/04/2016) - 04/01/2016-09/30/2016 GlaxoSmithKline plc GSK IONIS-TTRrx II/III Japanese Phase III to Start ISIS-TTRrx in patients with familial amyloid polyneuropathy 110661 (Familial Amyloid Initiation Isis expects the Phase III FAP Japan study of ISIS-TTRRx to (FAP) in Japan later in 2015. Polyneuropathy) start in 2016.Date Range Refined (01/11/2016) - Ionis expects the Phase III FAP Japan study of ISIS-TTRRx to start in mid-2016

Date Range Delayed (01/13/2014) - Isis announced that its Phase III study of ISIS-TTRRx is on track to complete Transthyretin-related enrollment in 2015.Date Range Delayed (12/29/2015) - This Trial Announcement - Hereditary Amyloidosis Phase II/III - Patient Isis expects to complete enrollment in their Phase III study is currently recruiting participants. We await an Now-01/31/2016 GlaxoSmithKline plc GSK IONIS-TTRrx II/III Patient Enrollment 93136 (Familial Amyloid Enrollment Completed program for ISIS-TTRrx in 2014. update. Catalyst Occurred (01/11/2016) - Ionis announced Completed Polyneuropathy) that patient enrollment was completed at the end of 2015 for the Phase III NEURO-TTR study of IONIS-TTRrx for the treatment of familial amyloid polyneuropathy.

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BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Glenmark Pharmaceuticals announced the completion of Phase I supporting studies and the submission of a clinical Date Range Delayed (12/23/2015) - We await an update. trial application to the Paul-Ehrlich Institute in Germany Catalyst Occurred (01/12/2016) - Glenmark reported that it Glenmark Trial Announcement - with a novel clinical development candidate GBR 1302. GBR Now-03/31/2016 GNP:IN GBR 1302 Cancer I Phase I - Trial to Start has initiated a Phase I trial of GBR-1302 in Germany. GBR- 115151 Pharmaceuticals Limited Initiation 1302 is a HER2xCD3 bi-specific antibody based on 1302 is listed in the pipeline with HER2-positive cancers are Glenmark's proprietary BEAT platform. Glenmark expects to the primary indications. obtain approval for the initiation of clinical studies with GBR1302 during this financial year.

Date Range Delayed (10/28/2015) - We await an Glenmark intends to open an US IND for clinical studies of Glenmark update.Catalyst Occurred (01/12/2016) - Glenmark Now-01/31/2016 GNP:IN GBR 830 Atopic Dermatitis (Eczema) II Regulatory - IND Filing IND Filing GBR 830 in Q2 FY 2015 – 16. We await an update in the 114730 Pharmaceuticals Limited reported that a Phase IIa trial of GBR 830 has been initiated time frame above. in the US with atopic dermatitis as the primary indication.

Glenmark has now completed clinical Phase I studies for GBR 830 in the Netherlands. GBR 830 was well tolerated and its safety and pharmacokinetics profile in healthy Date Range Delayed (12/28/2015) - We await an update. Glenmark Trial Announcement - volunteers fully support the transition into clinical Phase II Catalyst Occurred (01/12/2016) - Glenmark reported that a Now-02/29/2016 GNP:IN GBR 830 Atopic Dermatitis (Eczema) II Phase II - Trial to Start 114731 Pharmaceuticals Limited Initiation studies. Preparations for initiating Phase II studies in both Phase IIa trial of GBR 830 has been initiated in the US with atopic dermatitis and celiac disease in the US and Europe atopic dermatitis as the primary indication. are in order. Glenmark expects dosing to commence in the next few months. Date Range Delayed (01/11/2016) - Lundbeck announced Lundbeck announced that the Phase III program on Lu Trial Announcement - that the Phase III program on Lu AF35700 is planned to Now-06/30/2016 H. Lundbeck A/S HLUKF Lu AF35700 Schizophrenia I Phase III - Trial to Start AF35700 is planned to start during the first quarter of 2016 117287 Initiation commence in the first half of 2016 in treatment-resistant in treatment-resistant schizophrenia. schizophrenia.

Eisai and Halozyme Therapeutics announced that they have signed a clinical collaboration agreement to evaluate Eisai's Date Range Delayed (08/10/2015) - Halozyme announced agent eribulin mesylate (brand name: Halaven eribulin) in that they expect to initiate the Phase Ib/II clinical trial using combination with Halozyme's investigational drug PEGPH20 PEGPH20 in combination with HALAVEN (eribulin) in first (PEGylated recombinant human hyaluronidase) in first line line HER2-negative metastatic breast cancer patients with HER2-negative metastatic breast cancer. The companies Halozyme Therapeutics, Trial Announcement - high-HA in early 2016. Date Range Delayed (01/12/2016) - 01/12/2016-06/30/2016 HALO PEGPH20 Breast Cancer Preclinical Phase Ib/II Study to Start will jointly share the costs of a Phase Ib/II clinical trial to 113729 Inc. Initiation Halozyme announced that they expect to initiate the Phase assess whether or not eribulin in combination with Ib/II clinical trial using PEGPH20 in combination with PEGPH20 can improve overall response rate (ORR) -- the HALAVEN (eribulin) in first line HER2-negative metastatic proportion of women that have a predefined reduction in breast cancer patients with high-HA by the end of the tumor burden -- as compared with eribulin alone as a second quarter of 2016. therapy in women with advanced breast cancer. We await an update on the trial initiation by the end of 2015.

New Information (08/10/2015) - Halozyme announced that progress through the initial dose escalation cohorts is taking longer than anticipated likely due to the initial trial design which constrains the number of patients that could be screened and potentially enrolled at the same time. The design of the trial has recently been modified and five new sites have been initiated in order to address this problem. An update is expected in November 2015.Date Range Halozyme plans to complete patient enrollment in the on- Delayed (11/23/2015) - Halozyme is progressing into a Phase Ib/II PRIMAL - going global Phase Ib multi-center randomized clinical trial second dosing cohort in the Halozyme Phase Ib/II PRIMAL Halozyme Therapeutics, Non-Small Cell Lung Cancer Trial Announcement - Patient Enrollment (PRIMAL) evaluating PEGPH20 as a second-line therapy for study of PEGPH20 plus docetaxel in non-small cell lung 07/01/2016-12/31/2016 HALO PEGPH20 I/II 106286 Inc. (NSCLC) Other Completed (Phase Ib patients with locally advanced or metastatic NSCLC in the cancer patients. Actions initiated during the quarter have Portion) third quarter of 2015 pending the number of dose resulted in an increase in the number of patients screened escalation cohorts of PEGPH20. for the study. Once a maximum tolerated dose is determined the company plans to expand the study with additional sites outside the U.S. and screen patients prospectively for trial eligibility based on high levels of HA. We await an update through the first quarter of 2016.Date Range Delayed (01/12/2016) - Halozyme expects to advance to dose expansion phase in the Keytruda and PRIMAL studies pending the number of dose cohorts in the second half of 2016.

Date Range Refined (04/08/2015) - Based on FDA feedback Halozyme is targeting the end of first quarter 2016 to Halozyme plans to initiate patient enrollment and dosing for initiate the Phase III study of PEGPH20 in patients with Halozyme Therapeutics, Trial Announcement - a registration trial of PEGPH20 for pancreatic cancer in the 03/01/2016-03/31/2016 HALO PEGPH20 Pancreatic Cancer II Phase III to Start metastatic pancreatic cancer.Date Range Refined 106281 Inc. Initiation fourth quarter of 2015 or the first quarter of 2016 pending (01/12/2016) - Halozyme reported that the Phase III trial for regulatory feedback. PEG-PH20 in pancreatic cancer is now expected to start in March 2016. New Information (04/04/2014) - Halozyme announced that as a result of a recommendation received yesterday from an independent Data Monitoring Committee (DMC) it is temporarily halting patient enrollment and dosing of PEGPH20 in an ongoing Phase II trial (Study 202) evaluating PEGPH20 in patients with pancreatic cancer. The DMC is assessing clinical data that indicates a possible difference in the thromboembolic event rate between the group of patients treated with PEGPH20 nab-paclitaxel and gemcitabine versus the group of patients treated with nab- paclitaxel and gemcitabine without PEGPH20. The Company is halting enrollment and dosing of PEGPH20 as precautionary actions while the DMC's full evaluation of the Trial Announcement - Halozyme Therapeutics, Phase II HALO-202 - Halozyme reported that they expect enrollment to complete data is ongoing. We continue to await an update in the time Now-01/31/2016 HALO PEGPH20 Pancreatic Cancer II Patient Enrollment 97365 Inc. Enrollment Complete in their Phase II HALO-202 study in the second half of 2014. frame above.New Information (05/19/2014) - Halozyme Completed announced that they have provided the DMC with requested information and data and the DMC has informed that they support continued enrollment of patients and dosing of PEGPH20 in the Phase II trial. The Company is now providing information to the FDA so they can conduct their assessment of the request for the clinical hold to be lifted so that Halozyme may continue enrollment of patients and dosing of PEGPH20 in the Phase II trial. We continue to await an update in the time frame above.Date Range Delayed (06/04/2014) - Halozyme announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on patient enrollment and dosing of PEGPH20 in the ongoing Phase II trial (Study 202) evaluating PEGPH20 Ignyta's strategic priority for 2016 with respect to RXDX-106 Date Range Delayed (01/14/2016) - Ignyta plans to file an 07/01/2016-12/31/2016 Ignyta, Inc. RXDX RXDX-106 Solid Tumors Preclinical Regulatory - IND Filing IND Filing is filing an IND to begin clinical evaluation of this product 118076 IND for RXDX-106 in the second half of 2016. candidate. Ignyta's strategic priorities for 2016 with respect to RXDX- Date Range Refined (01/14/2016) - Ignyta expects to Trial Announcement - Phase Ia - Trial 107 are completing the Phase Ia portion of the ongoing 10/01/2016-12/31/2016 Ignyta, Inc. RXDX RXDX-107 Solid Tumors I complete the PHase Ia study of RXDX-107 in the fourth 118075 Trial Completion Completion clinical trial and identifying the recommended Phase II dose quarter of 2016. for this product candidate.

Ignyta's strategic priorities for 2016 with respect to taladegib are initiating one or more pivotal Phase II clinical Date Range Refined (01/14/2016) - Ignyta plans to initial Skin Cancer - Basal Cell Trial Announcement - 07/01/2016-12/31/2016 Ignyta, Inc. RXDX Taladegib I Phase II - Trial(s) to Start trials in advanced basal cell carcinoma as well as potentially Phase II studies of Taladegib in patients with advanced basal 118072 Carcinoma (BCC) Initiation initiating a Phase Ib basket study for patients with other cell carcinoma in the second half of 2016. solid tumors harboring a hedgehog pathway alteration.

Date Range Refined (01/12/2016) - ImmunoGen reports ImmunoGen announced that for coltuximab ravtansine that they intend to provide disclosure regarding the Diffuse Large B-Cell Trial Announcement - Combination Study to 04/01/2016-09/30/2016 ImmunoGen, Inc. IMGN Coltuximab Ravtansine II ImmunoGen is assessing alternative combination strategies combination regimen for their planned Phase II combination 114433 Lymphoma (DLBCL) - NHL Initiation Start and expects to initiate combination clinical testing in 2016. study in the first half of 2016 and initiate the Phase II combination study mid-year.

Date Range Delayed (12/07/2015) - The Phase II combination study of IMGN529 with rituximab for patients ImmunoGen announced that the Company is planning to with relapsed or refractory B-Cell lymphoma is not yet open Diffuse Large B-Cell Trial Announcement - Phase II w/ Rituximab - initiate a clinical trial to assess the combination of IMGN529 for participant recruitment. We await an update.Date Now-12/31/2016 ImmunoGen, Inc. IMGN IMGN529 I 111969 Lymphoma (DLBCL) - NHL Initiation Trial to Start with rituximab for the treatment of non-hodgkin lymphoma. Range Delayed (01/12/2016) - ImmunoGen reports that the We await an update in the time frame above. Phase II trial of IMGN529 in combination with rituximab for Diffuse Large B-Cell Lymphoma (DLBCL) is poised to start in 2016. We await an update on specific timing.

Biomedtracker August 2016 / 136 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (01/12/2016) - ImmunoGen expects to Acute Myelogenous Trial Announcement - ImmunoGen announced plans to start clinical testing for Now-06/30/2016 ImmunoGen, Inc. IMGN IMGN779 IND Clinical Trials to Start initiate the Phase I trial of IMGN779 for Acute Myelogenous 115084 Leukemia (AML) Initiation IMGN779 in early 2016. Leukemia (AML) in the first half of 2016.

Date Range Delayed (09/18/2015) - ImmunoGen announced ImmunoGen announced that the enrollment of patients into plans to release full data for the 46-patient cohort in the the Phase I expansion cohort of IMGN853 is expected to second quarter of 2016. New Information (01/12/2016) - Mirvetuximab Phase I Expansion Cohort - 04/01/2016-06/30/2016 ImmunoGen, Inc. IMGN Ovarian Cancer I Trial Data - Final Results complete by year end 2015 with presentation of the ImmunoGen reported that clinical data from the 40-patient 114432 soravtansine Final Results findings for the full 40-patient cohort anticipated in mid- cohort from the Phase I Mirvetuximab Soravtansine trial for 2016. ovarian cancer will be presented at a medical meeting in the second quarter of 2016.

Date Range Delayed (12/12/2013) - We await an update on a partnership for Rytary throughout the first half of 2014. Date Range Delayed (01/14/2014) - Impax reported that they plan to seek potential partnerships for the commercialization of Rytary outside of the United States. Impax intends to initiate activities to find a partner or As such we await an update through the end of 2014.Date partners for markets outside the U.S. looking to grow their Now-12/31/2016 Impax Laboratories, Inc. IPXL Rytary Parkinson's Disease (PD) Approved Partnership - New New Partnership Range Delayed (12/19/2014) - We continue to await an 87108 non-US neurology franchise. We look for a potential update on a potential licensing deal for Rytary.Date Range licensing deal for Rytary through 2013. Delayed (12/22/2015) - We continue to await an update.New Information (01/12/2016) - Impax announced that ex-U.S. partnering activities for Numient for the treatment of Parkinson's disease (PD) are ongoing. We continue to await an update through 2016.

New Information (01/11/2016) - The ECHO (Epacadostat Clinical development in Hematology and Oncology) Incyte and Merck known as MSD outside the United States program has been designed to investigate combinations of and Canada announced the expansion of the companies’ Incyte’s IDO1 inhibitor epacadostat across the full cycle of ongoing clinical collaboration to include a Phase III study anti-tumor immunity including with checkpoint blockade Trial Announcement - Phase III - w/Keytruda - Now-06/30/2016 Incyte Corporation INCY Epacadostat Melanoma I/II evaluating the combination of epacadostat with Keytruda vaccines and other modulators of the tumor immune 115588 Initiation Trial to Start (pembrolizumab) as first-line treatment for patients with response.The Phase III ECHO-301 study evaluating the advanced or metastatic melanoma. The Phase III study is combination of epacadostat with the anti-PD-1 antibody expected to begin in the first half of 2016. Keytruda (pembrolizumab) for the first-line treatment of patients with advanced or metastatic melanoma is expected to begin in the first half of 2016.

Date Range Delayed (12/29/2015) - A Phase Ib/II study of Duvelisib and Venetoclax in subjects with relapsed or refractory chronic lymphocytic leukemia small lymphocytic lymphoma or indolent or aggressive non-Hodgkin lymphoma who have not previously received a Bcl-2 or PI3K Infinity expects expects three company-sponsored clinical inhibitor has been announced but is not yet open for Infinity Pharmaceuticals, Indolent Non-Hodgkin's Trial Announcement - Combination Study studies of duvelisib to be initiated including the first clinical participant recruitment. The trial is expected to begin in 01/11/2016-01/31/2016 INFI Duvelisib III 106041 Inc. Lymphoma - NHL Initiation w/Venetoclax to Start study of duvelisib in combination with venetoclax (ABT-199) January 2016.New Information (01/11/2016) - Infinity in 2015. announced that the Phase Ib/II clinical study of duvelisib in combination with venetoclax is expected to begin this month. This study is designed to evaluate the safety and efficacy of duvelisib in combination with venetoclax in approximately 174 patients with relapsed or refractory CLL small lymphocytic lymphoma iNHL or aggressive NHL.

Date Range Delayed (05/06/2015) - Infinity announced they they expect to complete enrollment in the Phase II DYNAMO study of duvelisib in indolent non-Hodgkin's lymphoma in the second half of 2015. As such the Company now expects to report data from this study in 2016 and will provide more Infinity anticipates that it will report top line data from precise guidance on the timing of a data readout once DYNAMO a Phase II study of duvelisib (IPI-145) in patients enrollment is complete.Date Range Refined (10/06/2015) - Infinity Pharmaceuticals, Indolent Non-Hodgkin's Trial Data - Top-Line Phase II DYNAMO - Top- 07/01/2016-08/15/2016 INFI Duvelisib III with refractory indolent non-Hodgkin lymphoma (iNHL) in Infinity announced that the 120th patient has been enrolled 106023 Inc. Lymphoma - NHL Results Line Results the second half of 2015 following the completion of patient in DYNAMO a Phase II study in patients with refractory enrollment in the first half of 2015. indolent non-Hodgkin lymphoma (iNHL) and topline data from this study are anticipated in the third quarter of 2016.Date Range Refined (01/11/2016) - Infinity expects to report topline data from DYNAMO a Phase II study of duvelisib in patients with refractory indolent non-Hodgkin lymphoma (iNHL) early in the third quarter of 2016.

Date Range Delayed (05/07/2015) - Insmed announced that they expect to complete enrollment in the Phase III 212 study of Arikayce for the treatment of NTM lung infections Respiratory Tract Infections Trial Announcement - Phase III MAC-NTM - Insmed anticipates that the Arikayce Phase III global study in approximately twelve months from the initiation of the 10/01/2016-12/31/2016 Insmed, Inc. INSM Arikayce (Excluding Pneumonia) III Patient Enrollment Patient Enrollment for NTM will be fully enrolled in 2015. We expect full trial. Since the study initiated in January 2015 we await an 106299 (Antibacterial) Completed Complete enrollment in the second half of 2015. update on patient enrollment completion in the first quarter of 2016. Date Range Delayed (01/13/2016) - Insmed expects the Phase III CONVERT trial of Arikayce to be fully enrolled by the end of 2016.

Date Range Refined (12/22/2014) - According to clinicaltrials.gov the Phase III study of Cannabidiol in Dravet Syndrome is planned to initiate in April 2015.Date Range Delayed (03/20/2015) - Insys projects to file an IND for the use of cannabidiol for patients with epilepsy in 2015 and Insys Therapeutics intends to initiate a pivotal Phase III trial also plans to initiate a Phase III study later this year. We Trial Announcement - Phase III - Pivotal Trial to Now-12/31/2016 INSYS Therapeutics, Inc. INSY Cannabidiol Dravet Syndrome Preclinical of their pharmaceutical cannabidiol (CBD) candidate to now await an update on this Phase III study to start during 105345 Initiation Start treat Dravet Syndrome in 2015. the second half of 2015.Date Range Delayed (12/10/2015) - The Phase III study is not yet open for participant recruitment. We await an update. Date Range Delayed (01/13/2016) - Insys reported that clinical studies of Cannabidiol for Dravet Syndrome are expected to be initiated in 2016.

Date Range Refined (12/22/2014) - According to clinicaltrials.gov the Phase III study of Cannabidiol in LGS is planned to initiate in April 2015. Date Range Delayed (03/20/2015) - Insys projects to file an IND for the use of cannabidiol for patients with epilepsy in 2015 and also Insys Therapeutics intends to initiate a pivotal Phase III trial plans to initiate a Phase III study later this year. We now Lennox-Gastaut Syndrome Trial Announcement - Phase III - Pivotal Trial to Now-12/31/2016 INSYS Therapeutics, Inc. INSY Cannabidiol Preclinical of their pharmaceutical cannabidiol (CBD) candidate to await an update on this Phase III study to start during the 105346 (LGS; Epilepsy) Initiation Start treat Lennox-Gastaut Syndrome in 2015. second half of 2015.Date Range Delayed (12/10/2015) - The Phase II study is not yet open for participant recruitment. We await an update. Date Range Delayed (01/13/2016) - Insys reported that clinical studies of Cannabidiol for Lennox- Gastaut Syndrome (LGS; Epilepsy) are expected to be initiated in 2016. Trial Announcement - Phase III RESOLVE II - RESOLVE Bioabsorbable Intersect ENT announced it expects to complete patient Date Range Delayed (01/12/2016) - Intersect expects to 07/01/2016-12/31/2016 Intersect ENT XENT Allergy III Patient Enrollment Patient Enrollment 106226 Drug-eluting Implant enrollment for the RESOLVE II trial in 2016. complete enrollment in the RESOLVE II study in H2. Completed Completed Date Range Refined (05/04/2015) - Intra-Cellular plans to initiate a clinical trial evaluating ITI-007 for the treatment of behavioral disturbances in patients with dementia including Alzheimer's disease in the second half of 2015.Date Range Delayed (12/02/2015) - The Company plans to commence Intra-Cellular Therapies intends to initiate a Phase II clinical its late phase clinical program evaluating ITI-007 for the Intra-Cellular Therapies, Trial Announcement - trial in 2015 evaluating ITI-007 in patients with behavioral Now-06/30/2016 ITCI ITI-007 Alzheimer's Disease (AD) I/II Phase II - Trial to Start treatment of behavioral disturbances in patients with 103831 Inc. Initiation disturbances associated with dementia including dementia including Alzheimer's disease in late 2015 or Alzheimer's disease. during the first half of 2016.Date Range Refined (01/13/2016) - Intra-Cellular plans to initiate a clinical trial evaluating ITI-007 for the treatment of behavioral disturbances in patients with dementia in the first half of 2016.

Biomedtracker August 2016 / 137 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link New Information (09/21/2015) - Intra-Cellular Therapies is currently evaluating ITI-214 for several indications including cognition in patients with Parkinson's disease dementia Upon termination of the agreement with Takeda all rights schizophrenia and other CNS and non-CNS disorders.Date granted under the Takeda License Agreement were Range Delayed (12/09/2015) - We await an update. Date Intra-Cellular Therapies, Progress Update - ITI-214 Development 01/13/2016-12/31/2016 ITCI ITI-214 Schizophrenia I returned to Intra-Cellular. Intra-Cellular expects to finalize Range Delayed (01/13/2016) - Intra-Cellular Therapies 111117 Inc. Development Review Review their strategy for the PDE1 inhibitor program by the end of announced that the Company is currently evaluating ITI-214 2015. in several indications and expect to continue clinical development in 2016. As such we continue to await an update regarding a finalized strategy in the time frame above.

Date Range Refined (03/13/2015) - Isis expects to report clinical data from its Phase II study of ISIS-DMPK-2.5Rx in patients with myotonic dystrophy type 1 in late 2015/early Ionis Pharmaceuticals, Trial Data - Top-Line Phase I/II - Top-Line Phase II data on ISIS-DMPK-2.5Rx in myotonic dystrophy are Now-04/30/2016 IONS IONIS-DMPK-2.5Rx Muscular Dystrophy I/II 2016.Date Range Delayed (01/11/2016) - Ionis expects to 106131 Inc. Results Results expected in 2015/2016. report clinical data from its Phase II study of ISIS-DMPK- 2.5Rx in patients with myotonic dystrophy type 1 in the fourth quarter of 2016.

Isis plans to present detailed data from its Phase II study of Date Range Delayed (12/21/2015) - We continue to await ISIS-GCCRRx at a future medical meeting and are evaluating Ionis Pharmaceuticals, Trial Data - Updated Phase II CS2 - Updated an update. Date Range Delayed (01/11/2016) - Ionis Now-03/31/2016 IONS IONIS-GCCRRx Diabetes Mellitus, Type II II alternatives for further development in patients with type 2 114020 Inc. Results Results anticipates releasing updated results from the Phase II study diabetes and in patients with Cushing's disease. We await of IONIS-GCCRRx in the fourth quarter of 2016. an update through the rest of 2015. Date Range Delayed (01/11/2016) - Ionis announced that it Isis announced that it expects to complete enrollment in Trial Announcement - expects to complete enrollment in the Phase III ENDEAR Ionis Pharmaceuticals, Phase III ENDEAR - Patient both of its Phase III studies of nusinersen the generic name 07/01/2016-12/31/2016 IONS IONIS-SMNRx Spinal Muscular Atrophy III Patient Enrollment study of nusinersen the generic name for ISIS-SMNRx in 117564 Inc. Enrollment Completed for ISIS-SMNRx in infants and in children with spinal Completed infants and in children with spinal muscular atrophy by the muscular atrophy by early 2016. second half of 2016. Isis announced that it expects to complete enrollment in Catalyst Occurred (01/11/2016) - Ionis Pharmaceuticals Trial Announcement - Phase III CHERISH - Ionis Pharmaceuticals, both of its Phase III studies of nusinersen the generic name announced that enrollment was completed in the Phase III Now-04/30/2016 IONS IONIS-SMNRx Spinal Muscular Atrophy III Patient Enrollment Patient Enrollment 117563 Inc. for ISIS-SMNRx in infants and in children with spinal CHERISH study of IONIS-SMNRx for spinal muscular atrophy Completed Completed muscular atrophy by early 2016. at the end of 2015.

Date Range Delayed (01/12/2015) - Phase III data from the ISIS-APOCIIIRx program are planned for 2016/2017. Date Ionis Pharmaceuticals, Dyslipidemia / Trial Data - Top-Line Isis plans to begin Phase III trials in the first half of 2014 and 01/01/2017-06/30/2017 IONS Volanesorsen III Phase III Top-Line Results Range Refined (01/11/2016) - Ionis announced that top-line 90723 Inc. Hypercholesterolemia Results expects to release data in 2016. results from the Phase III study of volanesorsen are expected in the first half of 2017.

Date Range Refined (02/09/2015) - AstraZeneca plans to file for approval of Linaclotide in China for irritable bowel syndrome with constipation in the fourth quarter of 2015.Date Range Delayed (07/08/2015) - AstraZeneca and Ironwood Pharmaceuticals announced that the Companies Regulatory - Filing for intend to file for China Food and Drug Administration Ironwood Irritable Bowel Syndrome AstraZeneca plans to file for approval of Linaclotide in China Now-03/31/2016 IRWD Linzess Approved Approval (Emerging Chinese Filing (CFDA) approval to market linaclotide in early 2016. Date 97362 Pharmaceuticals, Inc. (IBS) for irritable bowel syndrome with constipation in 2015. Markets) Range Refined (11/03/2015) - Ironwood and AstraZeneca intend to file for approval to market linaclotide with the China Food and Drug Administration in the first quarter of 2016.Catalyst Occurred (01/11/2016) - Ironwood announced that Linzess is in review for approval for IBS-C in China.

Date Range Refined (01/11/2016) - Jazz announced that the Jazz announced that the three Phase III studies and one long- three Phase III studies and one long-term safety study Trial Announcement - Phase III Program - term safety study evaluating JZP-110 for the treatment of evaluating JZP-110 for the treatment of excessive daytime 10/01/2016-12/31/2016 Jazz Pharmaceuticals plc JAZZ JZP-110 Narcolepsy III Patient Enrollment Patient Enrollment excessive daytime sleepiness in narcolepsy or obstructive 114086 sleepiness in narcolepsy or obstructive sleep apnea are Completed Complete sleep apnea are expected to complete enrollment during expected to complete enrollment by the fourth quarter of the second half of 2016. 2016. Date Range Refined (01/11/2016) - Jazz announced that the Jazz announced that the three Phase III studies and one long- three Phase III studies and one long-term safety study Trial Announcement - Phase III Program - term safety study evaluating JZP-110 for the treatment of evaluating JZP-110 for the treatment of excessive daytime 10/01/2016-12/31/2016 Jazz Pharmaceuticals plc JAZZ JZP-110 Sleep Apnea III Patient Enrollment Patient Enrollment excessive daytime sleepiness in narcolepsy or obstructive 114087 sleepiness in narcolepsy or obstructive sleep apnea are Completed Complete sleep apnea are expected to complete enrollment during expected to complete enrollment by the fourth quarter of the second half of 2016. 2016.

New Information (08/05/2015) - Jazz announced that they are currently in the process of collecting and evaluating the available data of JZP-416 for the treatment of acute Jazz voluntarily suspended patient enrollment in the pivotal lymphoblastic leukemia (ALL). The Company plans to Phase II clinical trial of JZP-416 for the treatment of patients conduct additional research and analysis prior to Acute Lymphocytic Progress Update - Now-04/30/2016 Jazz Pharmaceuticals plc JAZZ JZP-416 II Development Review with acute lymphoblastic leukemia. The Company determining whether to resume the pivotal Phase II study 109185 Leukemia (ALL) Development Review anticipates that it will be able to determine next steps later and determining next steps regarding the development of this year or in early 2016. JZP-416. Catalyst Occurred (01/11/2016) - Based on the outcome of the analyses Jazz has decided that the pivotal Phase II clinical trial of JZP-416 for the treatment of acute lymphoblastic leukemia has been terminated.

Karyopharm plans to initiate clinical development in Date Range Delayed (01/13/2016) - Karyopharm reported Acute Myelogenous Trial Announcement - 04/01/2016-09/30/2016 Karyopharm Therapeutics KPTI KPT-9274 Preclinical Phase I - Trial to Start patients with heavily pretreated solid tumors or lymphoma that the first in human trial of KPT-9274 is anticipated in 117247 Leukemia (AML) Initiation in the first half of 2016. patients with solid tumors or NHL in mid-2016.

Diffuse Large B-Cell Regulatory - NDA/BLA Karyopharm expects to file an NDA for Selinexor in DLBCL Date Range Delayed (01/13/2016) - Karyopharm expects to 04/01/2017-09/30/2017 Karyopharm Therapeutics KPTI Selinexor II NDA Filing 106252 Lymphoma (DLBCL) - NHL Filing Richter's and AML in the first half of 2017. file an NDA for Selinexor in DLBCL in mid-2017.

Date Range Expedited (01/13/2016) - Karyopharm plans to Regulatory - NDA/BLA Karyopharm plans to file an NDA for Selinexor in MM in the 07/01/2017-12/31/2017 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb NDA Filing file an MAA/NDA for Selinexor in MM in the second half of 106258 Filing first half of 2018. 2017 based on results of the STORM study.

Date Range Expedited (12/07/2015) - Karyopharm plans to initiate a Phase II/III clinical study (SCORE) in early 2016 to evaluate the combination of selinexor carfilzomib and dexamethasone versus carfilzomib and dexamethasone in patients with relapsed/refractory multiple myeloma who Karyopharm is preparing to launch Phase III studies. As they Trial Announcement - Phase III SCORE - Trial to were previously treated with a proteasome inhibitor and an Now-04/30/2016 Karyopharm Therapeutics KPTI Selinexor Multiple Myeloma (MM) IIb did not provide a time frame we expect the initiation 106263 Initiation Start immunomodulatory drug.Catalyst Occurred (01/13/2016) - sometime in 2016. The Phase II study of selinexor plus carfilzomib (Kyprolis) plus low-dose dexamethasone versus placebo plus carfilzomib plus low-dose dexamethasone in patients with relapsed/refractory multiple myeloma is currently recruiting participants.

Date Range Refined (10/19/2015) - Karyopharm plans to initiate a randomized placebo-controlled Phase II/III trial of selinexor to treat liposarcoma during the fourth quarter of 2015. New Information (12/08/2015) - In solid tumors Karyopharm plans to initiate a randomized placebo- Karyopharm plans to initiate a registration-directed clinical Trial Announcement - Phase II/III SEAL - Trial to controlled Phase II/III trial of selinexor to treat liposarcoma Now-01/31/2016 Karyopharm Therapeutics KPTI Selinexor Sarcoma II/III trial of selinexor to treat liposarcoma in the second half of 112659 Initiation Start during the fourth quarter of 2015. Date Range Delayed 2015. (12/31/2015) - This study is not yet open for participant recruitment. We await an update.New Information (01/13/2016) - Karyopharm expects to initiate its Phase II/III SEAL study of Selinexor for liposarcoma shortly. We expect an update in the time frame above.

Date Range Delayed (12/04/2015) - Medgenics currently plans to submit an IND for a Phase I study of NFC-1 for 22q11.2 deletion syndrome in the fourth quarter of 2015. Attention Deficit IND Filing - 22q11.2 Medgenics plans to submit an IND for NFC-1 for 22q11.2 We await an update in the time frame above.Catalyst Now-01/15/2016 Medgenics Inc. MDGN NFC-1 Hyperactivity Disorder I Regulatory - IND Filing 114859 Deletion Syndrome deletion syndrome in Q4 2015. Occurred (01/14/2016) - Medgenics announced that the (ADHD) Company submitted an Investigational New Drug (IND) application for a Phase I/II trial of NFC-1 for 22q11.2 Deletion Syndrome in the fourth quarter of 2015.

Biomedtracker August 2016 / 138 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (07/20/2015) - BioMarin estimates the BioMarin announced that patient enrollment in the Phase III enrollment of the pivotal EMBRACA study of talazoparib will Trial Announcement - Phase III EMBRACA - trial of BMN 673 in patients with metastatic breast cancer be completed in the first half of 2016. Date Range Delayed 10/01/2016-12/31/2016 Medivation, Inc. MDVN Talazoparib Breast Cancer III Patient Enrollment 96949 Enrollment Completed (mBC) is expected to be completed in the second half of (01/11/2016) - Medivation expects the Phase III EMBRACA Completed 2015. study to complete patient enrollment by the end of the fourth quarter of 2016.

New Information (08/20/2015) - Merck expects to begin Phase III studies of combination regimens of grazoprevir and MK-3682 in 2015. Since both elbasvir and MK-8408 are being evaluated in Phase II studies with this combination we Merck expects to begin Phase III studies of of combination expect one of these two drugs will be included in the Phase Phase III w/MK-3682 + Trial Announcement - regimens of grazoprevir and MK-3682 (formerly IDX21437) III combination. As such we are maintaining a Phase III trial Now-01/31/2016 Merck & Co., Inc. MRK Grazoprevir Hepatitis C (HCV) (Antiviral) II Elbasvir or MK-8408 to 108350 Initiation with either elbasvir or MK-8408 for the treatment of HCV initiation catalyst in all four drug profiles until further Start infection in 2015. clarification from Merck. Date Range Delayed (12/16/2015) - We await an update. Date Range Delayed (01/11/2016) - Merck expects to begin Phase III studies of combination regimens in their HCV Triplet program in 2016. We await an update on the initiation of the trials.

Date Range Delayed (12/14/2015) - Merck plans to file the results from its Phase III KEYNOTE-010 study of Keytruda for Merck plans regulatory submissions for KEYTRUDA NSCLC in 2015. We await an update through the first half of Non-Small Cell Lung Cancer Regulatory - sNDA/sBLA (pembrolizumab) in previously treated advanced non-small- January 2016.Catalyst Occurred (01/11/2016) - Merck Now-01/15/2016 Merck & Co., Inc. MRK Keytruda Approved sNDA Filing 115929 (NSCLC) Filing cell lung cancer (NSCLC) whose tumors express PD-L1 in the announced they have submitted a supplemental Biologics U.S. in late 2015 and in the European Union in early 2016. License Application (sBLA) to the U.S. Food and Drug Administration for KEYTRUDA for NSCLC based on findings from KEYNOTE-010 at the end of 2015.

New Information (08/20/2015) - Merck expects to begin Phase III studies of combination regimens of grazoprevir and MK-3682 in 2015. Since both elbasvir and MK-8408 are being evaluated in Phase II studies with this combination we Merck expects to begin Phase III studies of of combination expect one of these two drugs will be included in the Phase Trial Announcement - Phase III w/Grazoprevir + regimens of grazoprevir and MK-3682 (formerly IDX21437) III combination. As such we are maintaining a Phase III trial Now-01/31/2016 Merck & Co., Inc. MRK MK-3682 Hepatitis C (HCV) (Antiviral) I/II 108349 Initiation Elbasvir/MK-8408 to Start with either elbasvir or MK-8408 for the treatment of HCV initiation catalyst in all four drug profiles until further infection in 2015. clarification from Merck. Date Range Delayed (12/16/2015) - We await an update. Date Range Delayed (01/11/2016) - Merck expects to begin Phase III studies of combination regimens in their HCV Triplet program in 2016. We await an update on the initiation of the trials.

New Information (08/20/2015) - Merck expects to begin Phase III studies of combination regimens of grazoprevir and MK-3682 in 2015. Since both elbasvir and MK-8408 are being evaluated in Phase II studies with this combination we Merck expects to begin Phase III studies of of combination expect one of these two drugs will be included in the Phase Trial Announcement - Phase III w/MK3682 + regimens of grazoprevir and MK-3682 (formerly IDX21437) III combination. As such we are maintaining a Phase III trial Now-01/31/2016 Merck & Co., Inc. MRK MK-8408 Hepatitis C (HCV) (Antiviral) II 108348 Initiation Grazoprevir to Start with either elbasvir or MK-8408 for the treatment of HCV initiation catalyst in all four drug profiles until further infection in 2015. clarification from Merck. Date Range Delayed (12/16/2015) - We await an update. Date Range Delayed (01/11/2016) - Merck expects to begin Phase III studies of combination regimens in their HCV Triplet program in 2016. We await an update on the initiation of the trials.

Date Range Delayed (09/24/2014) - The Phase II/III study of MK-8931 in subjects with mild to moderate Alzheimer's Disease (AD) is expected to complete in April 2020. Date Range Expedited (01/12/2015) - The Phase II/III study of MK- Trial Data - Top-Line Phase III - Top-Line Merck anticipates the Phase II/III study of MK-8931 to be 01/01/2017-12/31/2017 Merck & Co., Inc. MRK MK-8931 Alzheimer's Disease (AD) III 8931 in subjects with mild to moderate Alzheimer's Disease 85696 Results Results complete in 2016. (AD) is expected to complete in early 2018. Date Range Expedited (01/14/2016) - The Phase III study of MK-8931 in subjects with mild to moderate Alzheimer's Disease (AD) is expected to read-out in 2017. Date Range Delayed (01/11/2016) - Merck KGaA expects to Systemic Lupus Trial Data - Top-Line Phase II ADDRESS II - Top- Merck KGaA expects to have data readout from its Phase II 07/01/2016-12/31/2016 Merck KGaA MKGAY Atacicept II have data readout from its Phase II study of Atacicept in the 117615 Erythematosus (SLE) Results Line Results study of Atacicept in the first half of 2016. second half of 2016. Merck KGaA Pfizer and Syndax Pharmaceuticals announced that they have entered into a collaboration agreement to evaluate avelumab in combination with Syndax’s entinostat New Information (01/11/2016) - Merck KGaA announced Trial Announcement - Phase Ib/II w/Entinostat in patients with heavily pre-treated recurrent ovarian Now-12/31/2016 Merck KGaA MKGAY Avelumab Ovarian Cancer III that a Phase Ib/II collaboration study of avelumab with 117548 Initiation Study to Start cancer. The study will investigate the safety tolerability and Syndax is expected to begin in 2016. preliminary efficacy of avelumab and entinostat in advanced ovarian cancer. We await an update regarding the initiation of the trial.

Date Range Refined (01/12/2016) - Merrimack anticipates Merrimack anticipates results from HERMIONE the Phase II results from HERMIONE the Phase II clinical study of MM- Merrimack Trial Data - Top-Line Phase II HERMIONE - Top- clinical study of MM-302 in patients with HER2-positive 01/01/2017-06/30/2017 MACK MM-302 Breast Cancer II 302 in patients with HER2-positive metastatic breast cancer 116932 Pharmaceuticals, Inc. Results Line Results metastatic breast cancer designed to support a potential designed to support a potential Accelerated Approval Accelerated Approval application to the FDA in 2017. application to the FDA in the first half of 2017.

Chronic Lymphocytic MorphoSys annnnounced that they will commence a Phase Date Range Delayed (12/22/2015) - We await an update. Leukemia (CLL)/Small Cell Trial Announcement - Phase II w/ Idelalisib - II trial of MOR208 in chronic lymphocytic leukemia (CLL) in Date Range Refined (01/14/2016) - MorphoSys expects the 01/14/2016-03/31/2016 MorphoSys AG MOR:GR MOR208 II 114799 Lymphocytic Lymphoma Initiation Trial to Start the near future.We await an update through the end of Phase II study of MOR208 in combination with idelalisib for (SLL) - NHL 2015. CLL will start in the first quarter 2016.

Date Range Delayed (06/26/2015) - This study is not yet open for participant recruitment. We await an update.Date Range Delayed (07/31/2015) - This study is not yet open for participant recruitment. We continue to await an update.Date Range Delayed (08/31/2015) - This study is not yet open for participant recruitment. We continue to await A Phase II single-arm open-label multicentre study to an update.Date Range Delayed (09/30/2015) - The Phase II evaluate the safety and efficacy of lenalidomide combined Diffuse Large B-Cell Trial Announcement - Phase II w/Lenalidomide - study of MOR208 with lenalidomide is not yet open for 01/14/2016-03/31/2016 MorphoSys AG MOR:GR MOR208 II with MOR00208 in patients with relapsed or refractory 109809 Lymphoma (DLBCL) - NHL Initiation Trial to Start patient recruitment. We await an update.Date Range diffuse large B-cell lymphoma (R-R DLBCL) is expected to Delayed (11/24/2015) - MorphoSys expects the Phase II start in June 2015. combination study of MOR208 and lenalidomide in DLBCL to start during Q4 2015.Date Range Delayed (12/21/2015) - We await an update. Date Range Delayed (01/14/2016) - MoprhoSys expects the Phase II study of MOR208 in combination with lenalidomide to start in the first quarter 2016.

Date Range Delayed (01/12/2016) - Nektar reports that Trial Data - Top-Line Phase III - Top-Line Nektar plans on releasing the first Phase III trial results in they intend to release top-line data from the Phase III 07/01/2016-12/31/2016 Nektar Therapeutics NKTR NKTR-181 Chronic Pain III 97097 Results Results Q1 2016. SUMMIT-07 study of NKTR-181 for chronic lower back pain in the second half of 2016.

Date Range Expedited (11/18/2014) - Nektar expects to initiate the second Phase III study of NKTR-181 by the middle of 2015.New Information (02/25/2015) - Nektar Therapeutics announced that the SUMMIT Phase III (SUMMIT-12) program will also include a Phase III efficacy and safety trial in opiod-experienced patients with chronic Trial Announcement - Phase III SUMMIT-12 - 07/01/2016-12/31/2016 Nektar Therapeutics NKTR NKTR-181 Chronic Pain III Nektar plans to initiate a second Phase III trial in Q3 2015. lower back pain.Date Range Delayed (09/29/2015) - We 97096 Initiation Trial to Start await an update.Date Range Delayed (12/21/2015) - We await an update.Date Range Delayed (01/12/2016) - A second Phase III efficacy study is planned to initiate in the second half of 2016. The trial in opioid-experienced patients with chronic lower back pain (SUMMIT-12) will begin after the first efficacy study is completed.

New Information (01/11/2016) - Neurocrine announced Neurocrine will share additional details from the Kinect 3 Neurocrine Biosciences, Trial Data - Updated Phase III - Kinect 3 - that the Company has submitted data to AAN APA and 04/01/2016-09/30/2016 NBIX NBI-98854 Tardive Dyskinesia III study of NBI-98854 at upcoming scientific meetings starting 115522 Inc. Results Updated Results Movement Disorders scientific conferences. We await in mid-2016. further details on these presentations.

Biomedtracker August 2016 / 139 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link

Date Range Delayed (11/11/2015) - The catalyst below was entered in error. Neuroderm expects to initiate an EU bioequivalence study of ND0612H following results from its pilot dosing finding in the second quarter of 2016. As such we now expect an update on top-line results for the Phase I - EU NeuroDerm expects results from the EU bioequivalence bioequivalence study in the fourth quarter of 2016.Date Trial Data - Top-Line 04/01/2016-06/30/2016 NeuroDerm, Ltd. NDRM ND0612H Parkinson's Disease (PD) II Bioequivalence - Top-Line study of ND0612H in 20-40 healthy volunteers in the first Range Refined (11/11/2015) - NeuroDerm announced that 103413 Results Results half of 2016. topline results from the ongoing pharmacokinetic (PK) pilot dose finding study (EU trial) are expected in the second quarter of 2016.Date Range Expedited (01/14/2016) - NeuroDerm announced that they have initiated a Phase II PK equivalence study of ND0612H in Europe. They expect the results to read out in the second quarter of 2016.

NeuroDerm expects to receive marketing approval for New Information (01/14/2016) - NeuroDerm announced Regulatory - MAA 01/01/2017-06/30/2017 NeuroDerm, Ltd. NDRM ND0612H Parkinson's Disease (PD) II MAA Filing ND0612H in Europe by the first half of 2018. As such we that it anticipates filing for approval in Europe for ND0612H 103415 Submission (Europe) look for a potential filing in the first half of 2017. in the first half of 2017.

NeuroDerm expects to receive US marketing approval of New Information (01/14/2016) - NeuroDerm announced Regulatory - NDA/BLA ND0612H in the second half of 2018. As such we assume a 07/01/2017-12/31/2017 NeuroDerm, Ltd. NDRM ND0612H Parkinson's Disease (PD) II NDA Filing that the company anticipates filing for approval for 103414 Filing potential filing in the second half of 2017 in line with the ND0612H in the United States in the second half of 2017. company's guidance for Phase III results. Depending on clinical results NeuroDerm expects to receive New Information (01/14/2016) - NeuroDerm announced Regulatory - NDA/BLA marketing approval in the US and EU for ND0612L by the 07/01/2017-12/31/2017 NeuroDerm, Ltd. NDRM ND0612L Parkinson's Disease (PD) II NDA 505(b)(2) Filing that they expect to file for approval for NDO0612 in both 103416 Filing second half of 2018. As such we look for potential filings in Europe and the U.S. by the end of 2017. the second half of 2017. Depending on clinical results NeuroDerm expects to receive New Information (01/14/2016) - NeuroDerm announced Regulatory - MAA marketing approval in the US and EU for ND0612L by the 07/01/2017-12/31/2017 NeuroDerm, Ltd. NDRM ND0612L Parkinson's Disease (PD) II MAA Filing that they expect to file for approval for NDO0612 in both 103417 Submission (Europe) second half of 2018. As such we look for potential filings in Europe and the U.S. by the end of 2017. the second half of 2017.

Date Range Delayed (06/30/2015) - We await an update through the third quarter of 2015.Exact Date (09/17/2015) - Novavax will provide an update on its planned Phase I study Novavax announced during the 2015 J.P. Morgan of its RSV/Flu vaccine combination during its Analyst & RSV F / Seasonal Influenza Respiratory Syncytial Virus Trial Announcement - Healthcare Conference that a Phase I study testing RSV F+ 07/01/2016-12/31/2016 Novavax, Inc. NVAX Preclinical Phase I Trial to Start Investor Day on September 29 2015.Date Range Delayed 106347 Vaccine (RSV) Initiation seasonal influenza VLP as a pentavalent respiratory vaccine (09/29/2015) - We await an update.Date Range Delayed candidate is planned to begin in the second quarter of 2015. (12/29/2015) - �We await an update. Date Range Delayed (01/13/2016) - The Phase I combination respiratory (RSV + Flu) trial is expected to start in the second half of 2016.

Date Range Delayed (06/30/2015) - We await an update Novavax announced during the 2015 J.P. Morgan through the third quarter of 2015.Date Range Delayed RSV F / Seasonal Influenza Trial Announcement - Healthcare Conference that a Phase I study testing RSV F+ (09/29/2015) - We await an update.Date Range Delayed 07/01/2016-12/31/2016 Novavax, Inc. NVAX Seasonal Influenza Vaccines Preclinical Phase I Trial to Start 106369 Vaccine Initiation seasonal influenza VLP as a pentavalent respiratory vaccine (12/29/2015) - �We await an update. Date Range Delayed candidate is planned to begin in the second quarter of 2015. (01/13/2016) - The Phase I combination respiratory (RSV + Flu) trial is expected to start in the second half of 2016.

Date Range Delayed (12/30/2015) - We await an OncoMed Pharmaceuticals announced that the Company update.Catalyst Occurred (01/14/2016) - OncoMed OncoMed Trial Announcement - Phase Ib SIERRA Trial Now-03/31/2016 OMED Demcizumab Ovarian Cancer I/II expects to finish the Phase Ib portion of the Phase Ib/II trial Pharmaceuticals announced that the Phase Ib/II SIERRA 108518 Pharmaceuticals, Inc. Other Completion of demcizumab for ovarian cancer the second half of 2015. study of demcizumab for ovarian cancer completed the Phase Ib portion in the second half of 2015. Date Range Delayed (12/18/2015) - We await an update. Hepatocellular (Liver) In 2015 OncoMed plans to report data from Phase Ib OncoMed Trial Data - Top-Line Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Ipafricept Cancer (HCC) (including I Phase Ib - Top-line Data pancreatic ovarian and hepatocellular cancer trials of 105963 Pharmaceuticals, Inc. Results that ipafricept data will be available in the second half of secondary metastases) Ipafricept. 2016. Date Range Delayed (12/18/2015) - We await an update. In 2015 OncoMed plans to report data from Phase Ib OncoMed Trial Data - Top-Line Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Ipafricept Ovarian Cancer I Phase Ib - Top-line Data pancreatic ovarian and hepatocellular cancer trials of 105962 Pharmaceuticals, Inc. Results that ipafricept data will be available in the second half of Ipafricept. 2016. Date Range Delayed (12/18/2015) - We await an update. In 2015 OncoMed plans to report data from Phase Ib OncoMed Trial Data - Top-Line Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Ipafricept Pancreatic Cancer I Phase Ib - Top-line Data pancreatic ovarian and hepatocellular cancer trials of 105961 Pharmaceuticals, Inc. Results that ipafricept data will be available in the second half of Ipafricept. 2016. OncoMed announced that they will present Phase Ib study Date Range Delayed (12/18/2015) - We await an update. OncoMed Trial Data - Top-Line data of Vantictumab in breast cancer non-small cell lung Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Vantictumab Breast Cancer I Phase Ib - Top-line Data 108523 Pharmaceuticals, Inc. Results cancer and pancreatic cancer during the second half of that vantictumab data will be available in the second half of 2015. 2016. OncoMed announced that they will present Phase Ib study Date Range Delayed (12/18/2015) - We await an update. OncoMed Non-Small Cell Lung Cancer Trial Data - Top-Line data of Vantictumab in breast cancer non-small cell lung Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Vantictumab I Phase Ib - Top-line Data 108522 Pharmaceuticals, Inc. (NSCLC) Results cancer and pancreatic cancer during the second half of that vantictumab data will be available in the second half of 2015. 2016. OncoMed announced that they will present Phase Ib study Date Range Delayed (12/18/2015) - We await an update. OncoMed Trial Data - Top-Line data of Vantictumab in breast cancer non-small cell lung Date Range Delayed (01/14/2016) - OncoMed announced 07/01/2016-12/31/2016 OMED Vantictumab Pancreatic Cancer I Phase Ib - Top-line Data 108521 Pharmaceuticals, Inc. Results cancer and pancreatic cancer during the second half of that vantictumab data will be available in the second half of 2015. 2016.

Date Range Delayed (03/17/2014) - Ophthotech expects to advance to a Phase II/III clinical trial of Zimura for treatment of geographic atrophy in late 2014 or early 2015. Date Range Delayed (03/04/2015) - Ophthotech expects to advance to a Phase II/III clinical trial of Zimura for treatment of geographic atrophy in the second half of 2015.Date Range Refined (08/11/2015) - Ophthotech expects to Dry Age-Related Macular Ophthotech announced that a Phase II/III trial of Zimura Trial Announcement - initiate the Phase II/III dry AMD program with Zimura in the Now-01/31/2016 Ophthotech Corp. OPHT Zimura Degeneration (Dry AMD) II/III Phase II/III Trial to Start monotherapy in Dry AMD is currenlty planned. We expect 96871 Initiation fourth quarter of 2015.Date Range Delayed (12/31/2015) - (Ophthalmology) further updates through mid-2014. We await an update.Catalyst Occurred (01/11/2016) - Ophthotech has initiated a Phase II/III geographic atrophy study for Zimura. The study is estimated to last from 18 to 24 months. The primary analysis will be the mean change in best corrected visual acuity and the main secondary analysis will be the mean rate of change in geographic atrophy.

Date Range Delayed (10/07/2015) - OPKO announced that based on the recent Phase II clinical data the Company confirms its plan to initiate a global pivotal Phase III study in prepubertal GHD children in 2016 following supply of the Pivotal Phase III Trial Opko announced that a non-inferiority Phase III study of Short Stature / Growth Trial Announcement - product by Pfizer in a pen device evaluating a single dose of 07/01/2016-12/31/2016 Opko Health OPK Lagova III (Prepubertal GHD Lagova for the treatment of growth hormone deficiency is 101729 Hormone Deficiency Initiation hGH-CTP versus daily injections of growth hormone. We Children) to Start anticipated to start in 2015. await an update in the first half of 2016. Date Range Delayed (01/12/2016) - Opko expects to initiate the pediatric Phase III clinical trial of hGH-CTP during the second half of 2016.

Date Range Delayed (07/10/2015) - OPKO has completed enrollment in the single pivotal Phase III trial of its long acting human growth hormone (hGH-CTP) in growth Opko stated that there is a milestone related an adult Phase hormone deficient (GHD) adults. The study is expected to Short Stature / Growth Trial Data - Top-Line Phase III 005 - Top-Line III study of hGH-CTP for H2 2015. Given the expected 07/01/2016-12/31/2016 Opko Health OPK Lagova III end toward the second half of 2016. As such we now 90428 Hormone Deficiency Results Results completion date of the ongoing Phase III study we assume anticipate top-line results from this study in the first half of this to be referring to top-line results for the study. 2016. Date Range Delayed (01/12/2016) - Opko expects top- line data in adults Phase III of hGH-CTP in adults to be available in the second half of 2016.

Date Range Expedited (05/09/2014) - Lundbeck announced A 52-week multicenter open-label study to evaluate the that currently two studies are ongoing using Abilify effectiveness of an intramuscular depot formulation of Maintena in bipolar I disorder with a total of 1600 patients. Trial Data - Top-Line Phase III ATLAS - Top-line 07/01/2016-12/31/2016 Otsuka Holdings Co., Ltd. 4768:JP Abilify Maintena Bipolar Disorder III aripiprazole (OPC-14597) as maintenance treatment in This program is expected to be finalised in 2016.Date Range 84600 Results Data patients with bipolar I disorder is expected to be completed Refined (01/11/2016) - Lundbeck announced that the Phase in January 2017. III program of Abilify Maintena for bipolar I disorder is expected to finalize in the second half of 2016. Lundbeck announced that currently two studies are ongoing Date Range Refined (01/11/2016) - Lundbeck announced Trial Data - Top-Line Phase III Maintenance - using Abilify Maintena in bipolar I disorder with a total of 07/01/2016-12/31/2016 Otsuka Holdings Co., Ltd. 4768:JP Abilify Maintena Bipolar Disorder III that the Phase III program of Abilify Maintena for bipolar I 100514 Results Top-line Data 1600 patients. This program is expected to be finalised in disorder is expected to finalize in the second half of 2016. 2016.

Biomedtracker August 2016 / 140 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (11/09/2015) - We await an update.Date Range Delayed (11/13/2015) - Otsuka announced that the New Drug Application (NDA) for AVP- Avanir announced that a New Drug Application (NDA) for 825 for acute migraine is currently being reviewed. We Regulatory - AVP-825 has been accepted and is currently under review Migraine and Other PDUFA for NDA - Second continue to await an update.Date Range Delayed 01/11/2016-06/30/2016 Otsuka Holdings Co., Ltd. 4768:JP AVP-825 NDA PDUFA/Approval Decision by the U.S. Food and Drug Administration (FDA) with a 112719 Headaches Review (12/21/2015) - We continue to await an update.Date Range (US) Prescription Drug User Fee Act (PDUFA) goal date of Delayed (01/11/2016) - OptiNose announced that the New November 6 2015. Drug Application (NDA) for AVP-825 for acute migraine is currently being reviewed and a FDA approval decision is expected in the first half of 2016.

Date Range Delayed (12/21/2015) - We await an update.Date Range Delayed (01/11/2016) - Lundbeck Regulatory - J-NDA Filing Otsuka expects a regulatory filing for brexpiprazole for 01/11/2016-06/30/2016 Otsuka Holdings Co., Ltd. 4768:JP Rexulti Schizophrenia Approved J-NDA Filing announced that the first filings for Rexulti in non-US 112222 (Japan) schizophrenia in Japan during 2015. countries will begin in the first half of 2016. We await an update on a submission in Japan in that time frame.

Pacira Pharmaceuticals announced that it completed an End- of-Review process with the U.S. Food and Drug Administration (FDA) regarding its supplemental New Drug Application (sNDA) for the use of EXPAREL (bupivacaine liposome injectable suspension) for administration as a nerve block to provide postsurgical analgesia. Based upon Date Range Delayed (12/28/2015) - We await an the FDA guidance that the expected use of EXPAREL will be update.Date Range Delayed (01/11/2016) - Pacira Pacira Pharmaceuticals, Trial Announcement - Now-02/29/2016 PCRX Exparel Anesthesia III Phase III Studies to Start for a broad spectrum of nerve blocks and not limited to the announced that two nerve block studies in the lower 112127 Inc. Initiation narrow indication of a single nerve block Pacira plans to extremity and upper extremity are planned. We await an conduct additional Phase III studies for upper extremity and initiation update in the time frame above. lower extremity nerve blocks that together would cover the majority of nerve blocks performed in the U.S. Pacira anticipates working with the FDA to finalize the design of the Phase III trials and expects to initiate the studies by the end of 2015.

Date Range Refined (01/11/2016) - Pacira Pharmaceuticals Pacira Pharmaceuticals, Regulatory - sNDA/sBLA Pacira anticipates submission of U.S. sNDAs for Exparel for expects to file an sNDA for Exparel in femoral nerve block Now-12/31/2016 PCRX Exparel Anesthesia III sNDA Filing 115549 Inc. Filing oral surgery and nerve block in 2016. and brachial plexus in the second and third quarters of 2016.

Date Range Refined (10/05/2015) - A Phase II double blind randomized cross- over study examining efficacy of Pf- 06372865 in a photosensitivity epilepsy study using lorazepam as a positive control is not yet open for participant recruitment. The study is expected to initiate in October 2015. Date Range Delayed (10/28/2015) - A Phase Trial Announcement - Pfizer is planning for a potential Phase II study start for its II double blind randomized cross- over study examining 01/14/2016-02/29/2016 Pfizer Inc. PFE PF-06372865 Seizure Disorders (Epilepsy) Preclinical Phase II to Start 106279 Initiation GABA-A receptor PF-06372865 in epilepsy during 2015. efficacy of Pf-06372865 in a photosensitivity epilepsy study using lorazepam as a positive control is not yet open for participant recruitment. We await an update. Date Range Delayed (11/24/2015) - This study is not yet open for participant recruitment. We continue to await an update.Date Range Delayed (01/14/2016) - This study is not yet open for participant recruitment. We await an update.

Date Range Delayed (01/14/2016) - ProNAi announced that ProNAi announced plans to initiate a Phase II trial of they anticipate initiating the Phase II CYPRESS clinical trial Diffuse Large B-Cell Trial Announcement - Phase II CYPRESS - Trial to PNT2258 for the treatment of relapsed or refractory second- 04/01/2016-06/30/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 II of PNT2258 for the treatment of relapsed/refractory DLBCL 112654 Lymphoma (DLBCL) - NHL Initiation Start line DLBCL in combination with therapeutic agent or patients who are transplant eligible and who are also treatment regimen in the first quarter of 2016. receiving R-ICE in the second quarter of 2016.

ProNAi announced plans to initiate a second Phase II combination trial (in addition to the Phase II trial of Date Range Delayed (01/14/2016) - ProNAi announced that Phase II (other DLBCL) Diffuse Large B-Cell Trial Announcement - PNT2258 for the treatment of relapsed or refractory second- they anticipate initiating a Phase II combination trial of 10/01/2016-12/31/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 II Combiination Trial to 112681 Lymphoma (DLBCL) - NHL Initiation line DLBCL in combination with therapeutic agent or PNT2258 and another targeted agent for DLCBL in the Start treatment regimen) of PNT2258 for the treatment of other fourth quarter of 2016. DLBCL in the third quarter of 2016.

ProNAi announced plans to initiate a single-agent Phase II Date Range Delayed (01/14/2016) - ProNAi announced trial evaluating PNT2258’s potential in hematological plans to initiate a single-agent Phase II trial evaluating Trial Announcement - 07/01/2016-09/30/2016 ProNAi Therapeutics, Inc. DNAI PNT2258 Hematologic Cancer II Phase II Trial to Start malignancies (such as acute myeloid leukemia acute PNT2258’s potential in hematological malignancies (such as 112680 Initiation lymphoblastic leukemia and multiple myeloma) in the acute myeloid leukemia acute lymphoblastic leukemia and second quarter 2016. multiple myeloma) in the third quarter of 2016.

Date Range Refined (09/16/2014) - PTC expects to complete enrollment in the ACT CF study in the second half of 2015 Trial Data - Top-Line Phase III ACT CF - Top-line PTC expect to complete the Phase III CF confirmatory trial of with data expected a year later. As such we look for top-line 01/01/2017-04/30/2017 PTC Therapeutics, Inc. PTCT Translarna Cystic Fibrosis (CF) III 88612 Results Results ataluren and have initial top-line data available in 2016. results in H2 2016.Date Range Delayed (01/13/2016) - PTC reported that the Phase III study of Translarna in cystic fibrosis is ongoing with data expected in early 2017. PTC Therapeutics announced the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna Date Range Expedited (01/13/2016) - PTC Therapeutics Regulatory - (ataluren) for the treatment of nonsense mutation expects a US approval decision for Translarna in Duchenne PDUFA for NDA - First 9/8/2016 PTC Therapeutics, Inc. PTCT Translarna Muscular Dystrophy NDA PDUFA/Approval Decision Duchenne muscular dystrophy (nmDMD). Based on a muscular dystrophy in mid-2016. As such assuming the FDA 117686 Review (US) standard 12-month review timeline the PDUFA decision grants an 8-month priority review we expect a PDUFA should occur on January 8 2017. As this date falls on a decision on September 8 2016. weekend we expected the PDUFA decision on the Friday before. Date Range Delayed (12/22/2015) - Puma anticipates completing the ongoing Phase II trial of PB272 in patients with HER2-positive metastatic breast cancer that has Puma announced that they expect to complete the ongoing metastasized to the brain in the first half of 2016. As such Brain Cancer (secondary; Trial Data - Updated Phase II trial of PB272 in patients with HER2-positive 10/01/2016-12/31/2016 Puma Biotechnology, Inc. PBYI Neratinib Investigator Initiated Phase II - Updated Results we await updated results from this trial in the same time 104758 metastases) Results metastatic breast cancer that has metastasized to the brain frame.Date Range Delayed (01/11/2016) - Puma reported with the potential to report data in the second half of 2015. that potential results for the Phase II study of neratinib for HER2+ MBC with brain metastases are expected in the fourth quarter of 2016.

Date Range Delayed (03/31/2014) - We await an update.Date Range Delayed (04/01/2014) - Puma announced that based on the results from the Phase II I-SPY 2 trial neratinib is eligible for the upcoming I-SPY 3 Phase III trial. We await an update on the start of I-SPY 3 in mid- 2014.Date Range Delayed (09/30/2014) - We continue to Puma reported that based on the results from this I-SPY 2 await an update. Date Range Delayed (12/30/2014) - We Trial Announcement - Phase III I-SPY 3 Trial to TRIAL neratinib is now eligible for the upcoming I-SPY 3 Now-12/31/2016 Puma Biotechnology, Inc. PBYI Neratinib Breast Cancer III continue to await an update.Date Range Delayed 92816 Initiation Start Phase III trial. We await an update through the first quarter (03/04/2015) - Puma stated that they should be starting the of 2014. I-SPY3 study this year.Date Range Delayed (12/29/2015) - We continue to await an update on the I-SPY3 trial.Date Range Delayed (01/11/2016) - Puma expects a neratinib neoadjuvant Phase III study for breast cancer to initiate in 2016. As such we expect an update on the I-SPY3 study by the end of 2016.

Date Range Delayed (12/19/2014) - We await an update on the release of results from the Phase II study through the first quarter of 2015.Date Range Delayed (03/04/2015) - Puma expects to report data from their Phase II trial of PB272 in HER2-negative breast cancer patients who have a HER2 mutation in the second half of 2015.Date Range Puma anticipates that more detailed data of the Phase II Refined (08/10/2015) - Puma announced that they expect Trial Data - Top-Line Phase II HER2(-) - Top- 10/01/2016-12/31/2016 Puma Biotechnology, Inc. PBYI Neratinib Breast Cancer III study of neratinib in patients with HER2- breast cancer will to report data from the Phase II trial of PB272 in HER2 non- 93494 Results Line Results be released at a medical conference in 2014. amplified breast cancer that has a HER2 mutation in the fourth quarter of 2015. Date Range Delayed (12/30/2015) - We await an update.Date Range Delayed (01/11/2016) - Puma expects to report data from the Phase II study of neratinib plus fulvestrant in patients with HER2 non- amplified breast cancer that has a HER2 mutation in the fourth quarter of 2016.

Biomedtracker August 2016 / 141 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Refined (11/17/2015) - Radius intends to commence a Phase IIb trial for low-dose RAD1901 for Menopause (including Radius anticipates initiating a Phase IIb trial of RAD1901 for vasomotor symptoms in December 2015. Date Range Trial Announcement - Now-01/31/2016 Radius Health, Inc. RDUS RAD1901 Hormone Replacement II Phase IIb to Start the treatment of vasomotor symptoms in the second half of Delayed (12/29/2015) - We await an update.Catalyst 105367 Initiation Therapy [HRT]) 2015. Occurred (01/11/2016) - Radius announced that the Phase IIb study of RAD1901 in vasomotor symptoms was initiated in December 2015.

Date Range Delayed (10/08/2015) - Regeneron Pharmaceuticals and Sanofi announced that results from Phase III studies of dupilumab for the treatment of adults with moderate-to-severe atopic dermatitis that is not adequately controlled with topical atopic dermatitis Regeneron Trial Data - Top-Line Phase III SOLO Studies - Sanofi expects top-line Phase III results for dupilumab in medications are expected in the first half of 2016. Date Now-06/30/2016 REGN Dupilumab Atopic Dermatitis (Eczema) III 113794 Pharmaceuticals, Inc. Results Top-Line Results atopic dermatitis during the first quarter of 2016. Range Refined (10/30/2015) - Sanofi continues to expect Phase III top-line results for dupilumab in atopic dermatitis during Q1 2016. Date Range Delayed (01/13/2016) - Regeneron expects to have data from the Phase III SOLO studies of dupilumab in patients with atopic dermatitis in the first half of 2016.

Date Range Delayed (12/22/2015) - Regeneron reported that fasinumab is still under partial clinical hold by the FDA. Regeneron announced that the Company expects to hear We await an update.Catalyst Occurred (01/13/2016) - Regeneron Trial Announcement - FDA Response to Partial from the FDA in the second half of 2015 about the partial Now-02/29/2016 REGN Fasinumab Chronic Pain II/III Regeneron announced that the partial hold on Fasinumab 114336 Pharmaceuticals, Inc. Hold Lifted Hold clinical hold which if lifted will allow the Company to has been lifted. Based on discussions with the FDA Phase III conduct studies that are longer than 16 weeks in duration. trials (>16 weeks) are expected to begin in the first half of 2016.

Date Range Expedited (09/28/2015) - Results from the ODYSSEY OUTCOMES trial are anticipated in 2017.Date Range Expedited (01/12/2016) - Sanofi announced that an interim analysis of Praluent from the Phase III ODYSSEY Phase III ODYSSEY Regeneron Dyslipidemia / Trial Data - Top-Line Regeneron expects the Phase III ODYSSEY Outcomes study OUTCOMES study will be analyzed when ~50% of events 01/12/2016-09/30/2016 REGN Praluent Approved Outcomes - Top-Line 103135 Pharmaceuticals, Inc. Hypercholesterolemia Results of alirocumab for dyslipidemia to read out in 2018. have occurred. The Company anticipates releasing a second Results interim analysis for futility and efficacy when ~75% of events have occurred in the second half of 2016. As such we anticipate top-line results through the third quarter of 2016. Regeneron Pharmaceuticals announced that the Company Date Range Delayed (01/13/2016) - Regeneron plans to Regeneron Regeneron BARDA Trial Announcement - Now-06/30/2016 REGN Ebola Preclinical Clinical Trials to Start plans to initiate a Phase I study of the Regeneron BARDA initiate a Phase I study of its Ebola program in healthy 115100 Pharmaceuticals, Inc. Program Initiation Program in January 2016. volunteers in the first half of 2016. Date Range Delayed (01/13/2016) - Regeneron expects to Regeneron Trial Data - Top-Line Phase III MONARCH - Top- Sanofi expects the MONARCH Phase III results in 07/01/2016-12/31/2016 REGN Sarilumab Rheumatoid Arthritis (RA) BLA have Phase III MONARCH study data of Sarilumab vs. 116169 Pharmaceuticals, Inc. Results Line Results rheumatoid arthritis during Q3 2016. adalimumab in the second half of 2016. Date Range Refined (07/16/2014) - Rigel Pharmaceuticals announced the initiation of a Phase III clinical program for fostamatinib in patients with ITP (immune thrombocytopenic purpura). The focus of these clinical studies is to evaluate the potential of fostamatinib to increase the platelet counts of patients with chronic ITP. Results are expected at year-end 2015.Date Range Delayed (12/03/2014) - Rigel expects that the top line results of the Rigel representatives met with the FDA for an end-of-Phase two Phase III FIT studies will be reported separately; with II meeting for fostamatinib in development for patients with the US/UK-based study results expected by the end of 2015 immune thrombocytopenic purpura (ITP). The Company and the other study results expected in the first quarter of expects to initiate two pivotal Phase III studies in the first 2016.Date Range Delayed (12/03/2014) - await an half of 2014. Each of these trials are expected to enroll update.Date Range Delayed (01/15/2015) - Rigel expects to approximately 75 patients who would be treated for six Rigel Pharmaceuticals, Immune Thrombocytopenic Trial Data - Top-Line Phase III FIT (048) - Top- report results from the first FIT study of Fostamatinib in Q1 07/01/2016-12/31/2016 RIGL Fostamatinib III months and have the option to enroll in an extension study. 91580 Inc. Purpura (ITP) Results Line Data 2016 and the second study shortly thereafter. We look for a These trials will be randomized placebo-controlled and will data readout in the date range above.Date Range Delayed enroll verified ITP patients with platelet counts below (04/16/2015) - Rigel expects to separately report top line 30000 platelets per microliter of blood. The goal of the results of the studies of fostamatinib for the treatment of trials will be to achieve a durable platelet count increase to immune thrombocytopenic purpura (ITP) with the first over 50000 platelets per microliter of blood. Rigel expects study reporting in the first quarter of 2016 and the other top line data from these studies in 2015. study in the second quarter of 2016. Date Range Delayed (06/08/2015) - Rigel announced that the Company expects top-line data from both Phase III studies of Fostamatinib for the treatment of Immune Thrombocytopenic Purpura in mid- 2016.Date Range Refined (01/14/2016) - Rigel announced that data from the second Phase III trial of fostamatinib for the treatment of immune thrombocytopenic purpura (ITP) are expected shortly after results from the first Phase III Date Range Delayed (01/14/2016) - Rigel plans to file an Rigel Pharmaceuticals, Immune Thrombocytopenic Regulatory - NDA/BLA Rigel plans to file an NDA for Fostamatinib for ITP towards 01/01/2017-04/30/2017 RIGL Fostamatinib III NDA Filing NDA for fostamatinib for the treatment of immune 106487 Inc. Purpura (ITP) Filing the end of 2016. thrombocytopenic purpura (ITP) in the first part of 2017.

Date Range Delayed (01/15/2015) - Rigel expects Phase II data for the IgA nephropathy study of Fostamatinib in 2016.Date Range Delayed (04/16/2015) - Rigel expects to report results from its Phase II clinical trial of fostamatinib in patients with IgAN clinical trial of fostamatinib in patients Phase II - IgA Rigel announced that the Company expects to have top-line Rigel Pharmaceuticals, Renal Disease / Renal Trial Data - Top-Line with IgAN.Date Range Delayed (06/08/2015) - Rigel 10/01/2016-12/31/2016 RIGL Fostamatinib II Nephropathy Top-Line results from the Phase II study of Fostamatinib for the 97339 Inc. Failure Results Pharmaceuticals announced that the Company expects to Results treatment of IgA Nephropathy in the second half of 2015. report results for the Phase II SIGN clinical trial of fostamatinib in the second half of 2016.Date Range Refined (01/14/2016) - Rigel announced that Phase II results of fostamatinib for the treatment of IgA nephropathy are expected in the fourth quarter of 2016.

Roche plans on announcing topline results for the Phase III Date Range Expedited (01/12/2016) - Roche expects to have Trial Data - Top-Line Phase III IMvigor211 - Top- Now-12/31/2016 Roche Holding AG RHHBF Atezolizumab Bladder Cancer NDA IMvigor211 study of atezolizumab for the treatmtent of data from its Phase III IMvigor 211 study of Atezolizumab 115289 Results Line Results bladder cancer in 2017. for the treatment of bladder cancer in 2016. Date Range Refined (09/29/2015) - Roche plans to file for regulatory approval of atezolizumab for bladder cancer in Regulatory - NDA/BLA Roche plans to file for regulatory approval of RG7446 for early 2016. Catalyst Occurred (01/12/2016) - Roche Now-04/30/2016 Roche Holding AG RHHBF Atezolizumab Bladder Cancer NDA NDA Filing 102274 Filing bladder cancer in 2016. announced that it has initiated a rolling NDA for the approval of Atezolizumab for the treatment of bladder cancer in the US. Date Range Refined (09/28/2015) - Roche expects a U.S. filing for atezolizumab for the treatment of NSCLC in early Non-Small Cell Lung Cancer Regulatory - NDA/BLA Roche stated that they could potentially file for approval of Now-04/30/2016 Roche Holding AG RHHBF Atezolizumab NDA NDA Filing - 2nd/3rd Line 2016. Catalyst Occurred (01/12/2016) - Roche announced 89467 (NSCLC) Filing their anti-PDL1 (RG7446) for NSCLC in 2016. that it has initiated a rolling NDA for the approval of Atezolizumab for the treatment of NSCLC in the US.

New Information (01/12/2015) - Sangamo announced an update on the progress of Sangamo's proprietary programs Sangamo announced that SB-LSD is in preclinical in the lysosomal storage disorders (LSDs) that use its In Vivo development and will be execute preclinical efficacy as well Protein Replacement Platform (IVPRP) approach. The as in vivo proof of concept studies. The Company also has Mucopolysaccharidosis I Company expects to file IND applications for its first two Now-03/31/2016 Sangamo Biosciences, Inc. SGMO SB-318 Preclinical Regulatory - IND Filing IND Filing plans to scale up manufacturing to prepare for clinical 84256 (MPS I; Hurler Syndrome) programs in Hunter's and Hurler's disease by the end of development. In addition toxicology studies will be 2015. Date Range Delayed (12/30/2015) - We await an conducted in relevant animal models. Two INDs are planned update.Catalyst Occurred (01/12/2016) - Sangamo filed an for 2015. IND application for SB-318 for the treatment of Hurler Syndrome (MPS I) at the end of 2015.

Date Range Delayed (12/24/2015) - We await an update.Date Range Delayed (01/12/2016) - Sanofi Trial Announcement - Hanmi expects to initiate a Phase III study for HM11260C 10/01/2016-12/31/2016 Sanofi SNY Efpeglenatide Diabetes Mellitus, Type II II Phase III to Start announced that they expect to initiate a Phase III study of 106420 Initiation (LAPS-CA-Exendin-4) during 2015. efpeglenatide for the treatment of diabetes in the fourth quarter of 2016.

Biomedtracker August 2016 / 142 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link

Date Range Delayed (02/26/2015) - This study is not yet open for participant recruitment. We await an update.Date Range Delayed (03/30/2015) - This study is not yet open for participant recruitment. We continue to await an update.Date Range Delayed (04/28/2015) - �This study is not yet open for participant recruitment. We continue to await an update. Date Range Delayed (06/04/2015) - Genzyme announced that the Company is preparing to initiate enrollment for a Phase II/III adult study of olipudase alfa in the second half of 2015.Date Range Refined Genzyme announced plans to begin enrolling patients in a (08/03/2015) - The Phase II/III study of olipudase alfa in Trial Announcement - Phase II/III study of SAR402665 for the treatment of patients with acid sphingomyelinase deficiency is expected 01/12/2016-03/31/2016 Sanofi SNY Olipudase Alfa Niemann-Pick Disease I/II Phase II/III - Trial to Start 108684 Initiation Niemann-Pick Type B in 2015. The trial is expected to to start in August 2015.Date Range Delayed (08/31/2015) - initiate in February 2015. The Phase II/III study of olipudase alfa in patients with acid sphingomyelinase deficiency is not yet open for participant recruitment. We await an update.Date Range Delayed (09/28/2015) - This study is not yet open for participant recruitment. We continue to await an update.Date Range Delayed (11/25/2015) - This study is not yet open for participant recruitment. We continue to await an update.Date Range Refined (01/12/2016) - Sanofi announced that they expect to initiate a pivotal Phase II/III study of olipudase alfa in Niemann Pick type B in the first quarter of 2016.

Date Range Refined (01/12/2016) - Sanofi announced that Trial Announcement - Lexicon announced that Phase III trials of sotagliflozin 10/01/2016-12/31/2016 Sanofi SNY Sotagliflozin Diabetes Mellitus, Type II III Phase III - Trial to Start they expect to initiate a Phase III study of sotagliflozin for 116512 Initiation (LX4211) in type 2 diabetes are expected to initiate in 2016. the treatment of diabetes in the fourth quarter of 2016.

Seres announced that they plan to meet with the FDA to present Phase II safety and efficacy results and a proposed protocol for the Phase III clinical trial following analysis of Clostridium difficile- Phase II study data. The Company expects to initiate a Phase New Information (01/13/2016) - Seres Therapeutics Associated Trial Announcement - 07/01/2016-12/31/2016 Seres Health, Inc. MCRB SER-109 II Phase III - Trial to Start III clinical trial of SER-109 for the treatment of clostridium announced that they anticipate initiating a Phase III trial of 112174 Diarrhea/Infection Initiation difficile-associated diarrhea/infection (CDAD/CDI) in 2016. SER-109 for C. difficile by the end of 2016. (CDAD/CDI) Since data read-out is expected in mid 2016 we await an update on the initiation of a Phase III study in the second half of 2016.

Shire announced that it has reached an agreement with the U.S. Food and Drug Administration (FDA) on a clear regulatory path for SHP465 (triple-bead mixed Attention Deficit amphetamine salts - MAS) being evaluated as a potential Date Range Expedited (01/12/2016) - Shire reports that Shire Pharmaceuticals Regulatory - NDA/BLA 10/01/2016-12/31/2016 SHPG SHP-465 Hyperactivity Disorder III NDA Resubmission treatment for Attention-Deficit/Hyperactivity Disorder their FDA refiling of SHP-465 for ADHD is now expected in 110094 Group PLC Filing (ADHD) (ADHD) in adults. The Company now expects to submit to the fourth quarter of 2016. the FDA by second quarter 2017 a Class 2 resubmission for approval of SHP465 as a treatment for ADHD in adults which typically entails a 6-month review.

Date Range Delayed (11/18/2013) - Shire reported that the Phase II headline data is expected to be released in the first half of 2015.Date Range Delayed (10/27/2014) - Shire reported that the Phase II headline data is expected to be released towards later 2015.Date Range Expedited Shire reported that the Phase II data of HGT-ROP-001 for (02/17/2015) - Shire reported that the Phase II headline Shire Pharmaceuticals Other Retinopathy Trial Data - Top-Line Phase II ROP - Top-Line 04/01/2016-06/30/2016 SHPG SHP607 II the treatment of retinopathy of prematurity is expected to data is expected to be released in the second half of 88403 Group PLC (Ophthalmology) Results Data be released in the second half of 2014. 2015.Date Range Delayed (12/03/2015) - Shire reported that the Phase II headline data is expected to be released in mid-2016.Date Range Expedited (01/12/2016) - Shire reported that Phase II SHP607 top-line data for their Retinopathy of Prematurity trial is expected in the second quarter of 2016.

Date Range Delayed (02/10/2015) - Spark has announced they anticipate their BLA filing in 2016. New Information (10/05/2015) - Spark Therapeutics announced positive top- line results from the Phase III pivotal trial of its lead gene therapy product candidate SPK-RPE65 for the treatment of Leber's Congenital Regulatory - NDA/BLA RPE65-mediated inherited retinal dystrophies (IRDs). Based 07/01/2016-12/31/2016 Spark Therapeutics, LLC ONCE SPK-RPE65 III BLA Filing Spark anticipates filing a BLA for AAV2-hRPE65v2 in 2015. 97324 Amaurosis (Ophthalmology) Filing on these results Spark intends to file a Biologics License Application with the U.S. Food and Drug Administration in 2016 as the first step in executing its global regulatory and commercialization strategy.Date Range Refined (01/11/2016) - Spark Therapeutics expects to complete the BLA filing for SPK-RPE65 in the second half of 2016.

Date Range Expedited (04/08/2014) - Stemline announced that SL-401 is on track for an IND filing and initiation of clinical studies this year in a wide range of hematologic malignancies. Date Range Delayed (12/30/2014) - The catalyst below was entered in error. Stemline reports the myeloma trials for SL-401 are planned. We continue to expect update through early 2015.Date Range Delayed (04/30/2015) - Additional SL-401 studies are currently planned in myeloma and certain other lymphomas and Stemline expects to start a Phase I/II trial of SL-401 in leukemias. We await an update.Date Range Delayed Stemline Therapeutics, Trial Announcement - Now-06/30/2016 STML SL-401 Multiple Myeloma (MM) Preclinical Phase I/II to Start relapsed/refractory myeloma in the second half of 2014 or (06/15/2015) - We await an update.Date Range Delayed 97376 Inc. Initiation early 2015. (07/29/2015) - We continue to await an update. Date Range Delayed (09/23/2015) - Stemline continues to anticipate opening a trial of SL-401 in myeloma. We await an update in the time frame above.Date Range Delayed (11/25/2015) - Stemline announced that additional SL-401 studies are currently planned in myeloma and certain other lymphomas and leukemias. We continue to await an update through the first half of 2016.Date Range Refined (01/14/2016) - Stemline announced plans to initiate enrollment for SL401 in myeloma in the first half of 2016.

Date Range Delayed (12/22/2015) - SL-801 is a novel oral small molecule reversible XPO1 inhibitor that is expected to enter clinical development in early 2016 for both solid and Stemline Therapeutics, Trial Announcement - Phase I Solid Tumors - Stemline plans to initiate a clinical trial of SL-801 by the end Now-03/31/2016 STML SL-801 Cancer IND hematologic cancers.Date Range Refined (01/14/2016) - 106465 Inc. Initiation Clinical Trial to Start of 2015. Stemline announced plans to initiate a Phase I clinical trial for SL-801 in advanced solid tumors in the first quarter of 2016.

Date Range Delayed (11/21/2014) - Takeda anticipates filing MLN9708 (ixazomib) for approval during its 2015 financial year ending in March 31 2016.Date Range Refined (05/21/2015) - Takeda announced that the Company plans Takeda is targeting regulatory filings in the US EU and Japan Takeda Pharmaceutical Regulatory - J-NDA Filing to submit Ixazomib for approval within the first half of fiscal 01/12/2016-09/30/2016 4502:JP Ninlaro Multiple Myeloma (MM) Approved J-NDA Filing for MLN9708 in multiple myeloma during its fiscal year 76873 Company Ltd (Japan) 2015 ending March 31 2016 Date Range Delayed 2014 ending March 31 2015. (09/22/2015) - We await an update.Date Range Delayed (12/08/2015) - We continue to await an update.Date Range Delayed (01/12/2016) - Takeda announced that they anticipate filing for approval in Japan for Ninlaro in FY2016.

Date Range Delayed (01/13/2016) - Mesoblast reported that the Phase III trial of NeoFuse for Congestive Heart Interim analysis results for Revascor expected in early 2016 Teva Pharmaceutical Congestive Heart Failure Trial Data - Top-Line Failure is recruiting across North America. A first interim 04/01/2016-06/30/2016 TEVA NeoFuse III Phase III - Top-Line Data will follow the initial cohort completing six months of follow- 108680 Industries Ltd. (CHF) Results analysis will be performed during the first quarter of 2016 up. while the results will be available during the second quarter of 2016.

Biomedtracker August 2016 / 143 Pharma intelligence | Healthcare Conference: Conference Review

BioMedTracker JPM Updated Catalysts Expected Date Range Company Symbol Drug Indication Phase Expected Catalyst Catalyst Title Analysis Updated Analysis Link Date Range Delayed (11/12/2015) - The Medicines Ongoing clinical studies have expanded for ABP-700 and Company announced that they anticipated beginning Phase Trial Announcement - Now-06/30/2016 The Medicines Company MDCO ABP-700 Anesthesia I Phase II - Study to Start additional clinical trials are expected to launch in the II trials of ABP-700 in 2016. Date Range Refined 108342 Initiation second half of this year. (01/11/2016) - The Medicines Company expects to start a Phase II study for ABP-700 in the first half of 2016. Alnylam reported that Phase II study of ALN-PCsc for Date Range Refined (01/11/2016) - The Medicines Company Dyslipidemia / Trial Data - Top-Line Phase II ORION 1 - Top- 10/01/2016-12/31/2016 The Medicines Company MDCO ALN-PCSsc II dyslipidemia is expected to read out in the second half of expects top-line results for the Phase II study of PCSK9 si in 114572 Hypercholesterolemia Results Line Results 2016. the fourth quarter of 2016. The development leadership of ALN-PCSsc now transitions from Alnylam to The Medicines Company who together announce the initiation of the ORION development program Date Range Expedited (01/11/2016) - The Medicines Dyslipidemia / Trial Announcement - 10/01/2016-12/31/2016 The Medicines Company MDCO ALN-PCSsc II Phase III - Trial to Start with an initial Phase II study planned to begin by end-2015 Company expects a Phase III study of its PCSK9 to initiate in 114560 Hypercholesterolemia Initiation and a Phase III study expected to begin by end-2017. ORION the fourth quarter of 2016. is also expected to include a comparative study of ALN- PCSsc with anti-PCSK9 MAbs.

Date Range Refined (11/12/2015) - ThromboGenics announced that the FDA has accepted its Investigational New Drug (IND) Application for its CIRCLE study a Phase II randomized double-masked sham-controlled multi-center study that will evaluate the efficacy and safety of multiple doses of ocriplasmin in inducing total posterior vitreous detachment (PVD) in subjects with non-proliferative ThromboGenics expects the first patient in the Jetrea US Diabetic Retinopathy Trial Announcement - Phase II CIRCLE - Trial to diabetic retinopathy (NPDR). ThromboGenics remains on Now-01/31/2016 ThromboGenics NV THR:BB Jetrea II Phase IIa trial in proliferative diabetic retinopathy to be 106473 (Ophthalmology) Initiation Start track to recruit the first patient in the United States around enrolled in the second half of 2015. year-end 2015. Date Range Delayed (12/23/2015) - We await an update.Catalyst Occurred (01/13/2016) - ThromboGenics announced that the first patient has been enrolled in its Phase II CIRCLE study evaluating the efficacy and safety of multiple doses of ocriplasmin in inducing total posterior vitreous detachment (PVD) in patients with non- proliferative diabetic retinopathy (NPDR).

True North announced that they will present the results of Catalyst Occurred (01/12/2016) - True North Therapeutics the Phase Ia study validating TNT009’s proof-of-mechanism True North Therapeutics, Complement Deficiencies / Trial Data - Updated Phase Ia - Updated Data announced more results at the 34th annual JP Morgan 1/12/2016 TNT009 I along with the overall development plan for TNT009 at the 117576 Inc. Abnormalities Results at J.P. Morgan Healthcare Conference from their Phase Ia/Ib study of 34th Annual J.P. Morgan Healthcare Conference on Tuesday TNT009. January 12 2016.

Date Range Delayed (04/02/2015) - UCB announced that Phase II studies for UCB5857 are expected to begin in the Trial Announcement - UCB announced that Phase II studies for UCB5857 are second half of 2015.Date Range Delayed (12/30/2015) - We Now-03/31/2016 UCB SA UCB:BB UCB5857 Sjogren's Syndrome II Phase II - Study to Start 102431 Initiation expected to begin in early 2015. await an update.Catalyst Occurred (01/13/2016) - The Phase II study of UCB5857 in patients with primary Sjogren's syndrome is currently recruiting participants.

Ultragenyx Pharmaceutical announced the initiation of a new development program for the human monoclonal anti- Date Range Delayed (04/07/2015) - Ultragenyx expects to FGF23 antibody KRN23 (UX023) in tumor-induced have interim Phase II data of KRN23 targeting Tumor- Ultragenyx Bone Complications Trial Data - Top-Line Phase II TIO or ENS - Top- Now-06/30/2016 RARE KRN23 II osteomalacia (TIO). Ultragenyx intends to initiate a Phase II Induced Osteomalacia (TIO) in late 2015 or early 2016.Date 114825 Pharmaceutical Inc. (including bone metastases) Results Line Results study of KRN23 in six adult TIO patients in the first half of Range Delayed (01/12/2016) - Ultragenyx expects to have 2015 and anticipates interim safety and efficacy data from Phase II TIO interim data for KRN23 in the first half of 2016. the study by the end of 2015.

Ultragenyx and Kyowa Hakko Kirin plan to proceed with a pediatric Phase III study in 2016. The exact design details are yet to be determined but will likely utilize RGI-C as the Date Range Refined (01/12/2016) - Ultragenyx expects to Ultragenyx X-Linked Hypophosphatemia Trial Announcement - Phase III - Pediatric Trial primary endpoint and would include a standard of care 04/01/2016-09/30/2016 RARE KRN23 III initiate its Phase III study of KRN23 in pediatric patients for 117069 Pharmaceutical Inc. (XLH) Initiation to Start reference arm. This study is expected to be required for XLH in mid-2016. potential approval in the US and could also serve as a confirmatory study in the EU if a conditional marketing authorization were granted.

New Information (12/02/2015) - Ultragenyx Pharmaceutical and Kyowa Hakko Kirin announced interim data through 40 weeks from the first 36 patients in the ongoing pediatric Phase II study of KRN23 for the treatment of X-linked Feedback from the EMA indicates that conditional approval hypophosphatemia (XLH). Ultragenyx and Kyowa Hakko filing for KRN23 may be possible based on ongoing pediatric Kirin plan to file for a conditional marketing authorization in Ultragenyx X-Linked Hypophosphatemia Regulatory - MAA and adult studies. Ultragenyx stated that the decision to file the EU on the basis of these data and on prior feedback 07/01/2016-12/31/2016 RARE KRN23 III MAA Filing 114214 Pharmaceutical Inc. (XLH) Submission (Europe) based on 40-week rickets data from pediatric Phase II in Q4 from the EMA suggesting that the companies could do so if 2015 (original 36 patients) or in mid-2016 (expanded the data indicated a positive benefit-to-risk profile. We n=~50). await an update in the time frame above. Date Range Delayed (01/12/2016) - Ultragenyx plans to file a conditional marketing authorization with the EMA. Pending future discussions the filing will occur in the second half of 2016.

Phase II LC FAOD - Ultragenyx announced that 78 week results for the Phase II Date Range Delayed (01/12/2016) - Ultragenyx expects to Ultragenyx Fatty Acid Oxidation Trial Data - Updated 04/01/2016-09/30/2016 RARE Triheptanoin II Updated Results (78 study of UX007 in fatty acid oxidation disorders is expected have the Phase II 78-week data for Triheptanoin in mid- 115591 Pharmaceutical Inc. Disorders (FAOD) Results Weeks) in the second half of 2016. 2016. Ultragenyx announced that the Company intends to begin planning for a Phase III study of UX007 in LC-FAOD. An Date Range Refined (01/12/2016) - Ultragenyx expects to Ultragenyx Fatty Acid Oxidation Trial Announcement - update on the design and timing of the Phase III study will 07/01/2016-12/31/2016 RARE Triheptanoin II Phase III - Trial to Start enroll the first patient in its Phase III study of UX007 in 115592 Pharmaceutical Inc. Disorders (FAOD) Initiation be provided after discussions with regulatory authorities in patients with FAOD in the second half of 2016. the first half of 2016. As such we await a Phase III initiation update in the time frame above.

Date Range Refined (12/01/2015) - United Therapeutics and In March 2015 the FDA requested that Medtronic amend its Medtronic met with the FDA in the third quarter of 2015. PMA for the Remodulin Implantable System to reflect an Based on the outcome of this meeting the current plan is amendment to the SynchroMed II PMA separately that Medtronic will file an amendment to the PMA in submitted by Medtronic’s neuromodulation business unit December that is responsive to all queries to-date. and issued a refuse-to-file letter with respect to the NDA for United Therapeutics Pulmonary Arterial Regulatory - sNDA/sBLA sNDA Filing - Remodulin Subsequently United Therapeutics will re-file the NDA also Now-01/31/2016 UTHR Remodulin (injection) Approved the Remodulin Implantable System. Medtronic has 114170 Corporation Hypertension (PAH) Filing Implantable System in the December timeframe.Date Range Delayed amended its PMA and United Therapeutics is working to (12/30/2015) - We await an update.Catalyst Occurred resubmit the NDA to address the FDA’s requests. Once the (01/11/2016) - United Therapeutics reported that they have amendment and resubmission have been completed they filed a supplemental new drug application (sNDA) for anticipate a ten-month review process by FDA. We await an Remodulin for Pulmonary Arterial Hypertension in update on the sNDA filing in the time frame above. December 2015 under a 10 month review time frame.

Biomedtracker August 2016 / 144 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link

Abiomed announced that it has submitted U.S Food & Drug Administration (FDA) pre-market approval Impella 2.5, Impella 5.0, (PMA) supplemental submissions requesting to and Impella CP expand Impella 2.5 PMA approval to all of the Date Range Delayed (01/13/2016) - Abiomed announced 01/13/2016-09/30/2016 Abiomed, Inc. ABMD Impella 2.5 Congestive Heart Failure (CHF) Approved Supplemental PMA Impella family of devices (Impella 2.5 Impella CP and that it hopes to have approval for the cardiogenic chock 114227 Approval - Cardiogenic Impella 5.0/LD). Under the FDA’s Medical Device indication by summer 2016. Shock User Fee Amendment of 2012 (MDUFA III) an approval decision on this PMA application is expected to be made within 180 days of filing.

Date Range Refined (01/13/2016) - Abiomed announced Impella 2.5 Japanese Abiomed estimates launching the Impella 2.5 in 06/01/2016-09/30/2016 Abiomed, Inc. ABMD Impella 2.5 Congestive Heart Failure (CHF) Approved that the PMA in Japan is pending and the company expects 114076 Approval Japan around mid calendar year 2016. to receive approval during the summer of 2016.

Date Range Refined (08/17/2015) - Abiomed announced that it has submitted U.S Food & Drug Administration (FDA) pre-market approval (PMA) supplemental submissions requesting to expand Impella 2.5 PMA approval to all of the Impella 2.5, Impella 5.0, Abiomed plans to submit supplemental PMAs for Impella family of devices (Impella 2.5 Impella CP and and Impella CP Impella 5.0 and Impella CP indicated for emergency Impella 5.0/LD). We expect U.S. Food and Drug 01/13/2016-09/30/2016 Abiomed, Inc. ABMD Impella 5.0 Congestive Heart Failure (CHF) Approved Supplemental PMA 114064 use in Q2 FY2016 and expects to receive approval in Administration Approval to occur during this time-range. Approval - Cardiogenic Summer 2016. User Fee Amendment of 2012 (MDUFA III) an approval Shock decision on this PMA application is expected to be made within 180 days of filing.Date Range Delayed (01/13/2016) - Abiomed announced that it hopes to have approval for the cardiogenic chock indication by summer 2016.

Date Range Refined (08/17/2015) - Abiomed announced that it has submitted U.S Food & Drug Administration (FDA) pre-market approval (PMA) supplemental submissions requesting to expand Impella 2.5 PMA approval to all of the Impella 2.5, Impella 5.0, Impella family of devices (Impella 2.5 Impella CP and Abiomed plans to submit supplemental PMAs for and Impella CP Impella 5.0/LD). We expect U.S. Food and Drug Impella 5.0 and Impella CP indicated for emergency 01/13/2016-09/30/2016 Abiomed, Inc. ABMD Impella CP Congestive Heart Failure (CHF) Approved Supplemental PMA Administration Approval to occur during this time- 114071 use in Q2 FY2016 and expects to receive approval in Approval - Cardiogenic range.User Fee Amendment of 2012 (MDUFA III) an Summer 2016. Shock approval decision on this PMA application is expected to be made within 180 days of filing.Date Range Delayed (01/13/2016) - Abiomed announced that it hopes to have approval for the cardiogenic chock indication by summer 2016.

Accelerate said it hopes to launch the ID/AST System Accelerate Diagnostics, ID/AST Blood Culture Approved in 07/01/2016-12/31/2016 AXDX Infectious Disease Product Launch (US) in late 2016 assuming it receives FDA clearance 118163 Inc. Assay Europe earlier in 2016. Date Range Refined (10/19/2015) - AngioDynamics said in its Q2 FY2015 earnings call that it plans to file an application Celerity with Navigation - AngioDynamics announced that it expects to Now-05/31/2016 AngioDynamics, Inc. ANGO Celerity System Surgical Tools (General) Approved with the FDA for Celerity with Navigation within a month or 113351 Product Launch (US) launch a second-generation Celerity Navigation product in the second half of FY2016. two and expects to launch the product by the end of the fiscal year. Next Generation AngioDynamics expects to launch a next-generation 06/01/2017-05/30/2018 AngioDynamics, Inc. ANGO NanoKnife System Solid Tumors Approved NanoKnife System - 118401 NanoKnife System during FY2018. Product Launch New Information (05/01/2015) - Boston Scientific said that it anticipates receiving CE Mark approval for Eluvia in the Boston Scientific announced that it expects the Boston Scientific Eluvia Drug-Eluting Peripheral Arterial Disease first half of 2016.New Information (01/12/2016) - Boston 01/12/2016-06/30/2016 BSX IDE CE Mark Approval Eluvia Drug-Eluting Stent System to receive CE Mark 110863 Corporation Vascular Stent System (PAD) Scientific announced that it expects to launch Eluvia in approval in 2016. Europe in the first half of 2016. Given this estimate we expect CE Mark approval in the same time frame.

Boston Scientific said it anticipates launching Date Range Expedited (01/12/2016) - Boston Scientific Boston Scientific Eluvia Drug-Eluting Peripheral Arterial Disease Eluvia in Europe in 2016. Given the companies 01/12/2016-06/30/2016 BSX IDE Product Launch (EU) announced that it expects to launch Eluvia in Europe in the 110998 Corporation Vascular Stent System (PAD) anticipation of CE Mark approval in the first half of 2016 we expect the launch of the product to first half of 2016. occur in the second half of 2016.

Date Range Delayed (01/12/2016) - Boston Scientific said it Boston Scientific Ventricular Tachycardia or EMBLEM MRI S-ICD Boston Scientific expects to launch the EMBLEM MRI 07/01/2016-12/31/2016 BSX S-ICD System Approved expects to launch the next-generation Emblem MRI-safe 111043 Corporation Fibrillation Product Launch (EU) S-ICD in Europe in 2016. device in Europe in the second half of 2016.

Date Range Delayed (10/13/2015) - The Watchman procedure was not granted a fiscal-year 2016 new- technology add-on payment by the Centers for Medicare and Medicaid Services (CMS) in its hospital inpatient rule put out in summer 2015 however Boston Scientific notes that CMS established new higher-paying categories (MD- DRGs) for percutaneous intracardiac procedures to include the left-atrial appendage closure device procedure. The Boston Scientific said that it will understand more US Reimbursement - Chief Medical Officer of Boston Scientific said CMS's Boston Scientific Stroke Prevention in Atrial about the national coverage decision as well as the 2/8/2016 BSX WATCHMAN Approved National Coverage Final decision to include Watchman in the MS-DRGs will mean 113453 Corporation Fibrillation (SPAF) new technology add-on payment in the back-half of Decision hospitals will on average receive 19% more in Medicare 2015. reimbursements. Boston Scientific is now waiting on CMS to establish a national coverage policy for the Watchman procedure. The agency opened a national coverage analysis earlier this year in response to a request from the company. A proposal is expected in November and a final decision by next February.Date Range Refined (01/12/2016) - Boston Scientific said it expects to hear a decision on WATCHMAN reimbursement on February 8 2016.

EkoSonic Endovascular Pulmonary Arterial Improved Control Unit BTG will roll-out an improved version of the control 07/01/2016-12/31/2016 BTG plc BTC:LN Approved 118400 System Hypertension (PAH) Launch unit for the EKOS System in H2 2016. BTG announced that they plan to complete patient EkoSonic Endovascular Pulmonary Arterial OPTALYSE PE - Patient 10/01/2016-12/31/2016 BTG plc BTC:LN Approved enrollment in the OPTALYSE PE study of EKOS at the 118343 System Hypertension (PAH) Enrollment Complete end of 2016. Hepatocellular (Liver) Cancer BTG announced that they expect EU approval of DC 01/13/2016-06/30/2016 BTG plc BTC:LN LC Bead (HCC) (including secondary Approved CE Mark Decision 118350 Bead LUMI in the first half of 2016. metastases) Hepatocellular (Liver) Cancer BTG announced that they plan to commence studies Bioresorbable Bead - 01/13/2016-06/30/2016 BTG plc BTC:LN LC Bead (HCC) (including secondary Approved for a bioresorbable bead product in the first half of 118351 Studies to Start metastases) 2016. BTG said it is progressing its regulatory application in Date Range Refined (01/13/2016) - BTG announced that 01/13/2016-06/30/2016 BTG plc BTC:LN PneumRx Coil System Emphysema IDE PMA Filing to U.S. FDA the US which it anticipates submitting around mid- they intend to submit a U.S. PMA for PneumRx in the first 117328 2016. half of 2016. BTG announced that they expect to secure full 07/01/2016-12/31/2016 BTG plc BTC:LN PneumRx Coil System Emphysema IDE Reimbursement (France) reimbursement in France for PneumRx in the second 118288 half of 2016. BTG said it is progressing its regulatory application in Date Range Refined (01/13/2016) - BTG announced that PMA Approval Decision the US which it anticipates submitting around mid- 10/01/2016-12/31/2016 BTG plc BTC:LN PneumRx Coil System Emphysema IDE they expect a U.S. PMA decision for PneumRx at the end of 117327 U.S. 2016. Given this timeline for filing we expect an 2016. update on the approval in the time frame above. Cepheid announced that it has has targeted Development Date Range Delayed (01/13/2016) - Cepheid said it plans to 01/13/2016-12/31/2016 Cepheid CPHD Xpert Bladder Cancer Bladder Cancer Product Launch (EU) commercial release outside the US in late 2015 or 111345 Outside U.S. launch the Bladder test in Europe in 2016. early 2016. Development Cepheid said it plans to launch the Bladder test in the 01/01/2017-12/31/2018 Cepheid CPHD Xpert Bladder Cancer Bladder Cancer Product Launch (US) 118340 Outside U.S. U.S. in the 2017-2018 time frame.

Biomedtracker August 2016 / 145 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link

Date Range Delayed (12/15/2015) - Cepheid said that Xpert Carba-R was submitted to the FDA in the middle of In a Q1 2015 pipeline update Cepheid said it expects September. We await an update on the approval and launch Approved in 01/13/2016-12/31/2016 Cepheid CPHD Xpert Carba-R Antibacterial - General 510(k) Clearance commercial release of the Xpert CARBA-R test to in the time frame above.Date Range Delayed (01/13/2016) - 101490 Europe occur in the second half of 2015. Cepheid announced it plans to launch Xpert Carba-R in the U.S. in 2016. We await an update on the approval in the time frame above.

Cepheid announced that Xpert HBV has moved into Date Range Delayed (01/13/2016) - Cepheid announced it Hepatitis B (HBV) Treatment Development 01/01/2017-12/31/2018 Cepheid CPHD Xpert HBV Product Launch (EU) active development with targeted CE IVD release in expects to launch the Xpert HBV Viral Load assay in Europe 111344 (Antiviral) Outside U.S. the 2016-2017 timeframe. during the 2017-2018 time frame. Cepheid announced it expects to launch the Xpert Hepatitis B (HBV) Treatment Development 01/01/2017-12/31/2018 Cepheid CPHD Xpert HBV Product Launch (US) HBV Viral Load assay in the U.S. during the 2017- 118338 (Antiviral) Outside U.S. 2018 time frame. Cepheid announced that the Xpert HCV Viral Load test is in clinical trials and is expected to be released Date Range Delayed (01/13/2016) - Cepheid announced it Development 01/01/2017-12/31/2018 Cepheid CPHD Xpert HCV Viral Load Hepatitis C (HCV) (Antiviral) Product Launch (U.S.) in the U.S. in the 2016-2017 time frame. The expects to launch the Xpert HCV Viral Load assay in the U.S. 111340 Outside U.S. company said it will narrow that window as it gets during the 2017-2018 time frame. closer to the FDA submission.

Cepheid announced that the Xpert HIV Viral Load test is in clinical trials and is expected to be released Date Range Delayed (01/13/2016) - Cepheid announced it Approved in 01/01/2017-12/31/2018 Cepheid CPHD Xpert HIV-1 Viral Load HIV / AIDS Product Launch (U.S.) in the U.S. in the 2016-2017 time frame. The expects to launch the Xpert HIV-1 Viral Load assay in the 111341 Europe company said it will narrow that window as it gets U.S. during the 2017-2018 time frame. closer to the FDA submission.

Cepheid announced that it expects to launch the 01/13/2016-12/31/2016 Cepheid CPHD Xpert MTB/RIF Tuberculosis Approved Product Launch (EU) 118342 Xpert MTB/RIF Ultra assay in Europe in 2016.

Urinary Tract and Reproductive Trichomonas Male Cepheid announced it plans to launch a version of 01/13/2016-12/31/2016 Cepheid CPHD Xpert Trichomonas Approved 118334 Tract Infections (Antibacterial) Indication Launch Xpert Trichomonas for men in 2016. Dexcom G4 Platinum DexCom expects results from the DIaMonD study Phase IV DIaMonD - Top- 07/01/2016-09/30/2016 DexCom Inc. DXCM Continuous Glucose Diabetes Mellitus, Type II Approved early in the second half of 2016. We look for a 118261 Line Results Monitoring System potential data readout in Q3. Edwards Lifesciences US Early Feasibility Study Edwards said an early feasibility study in the US Now-03/31/2016 EW CardiAQ Edwards Valve Cardiac Valve Surgery IDE 117314 Corp. to Start is set to begin soon. We await an update in the time frame above. Edwards Lifesciences Edwards said the CE Mark trial for the CardiAQ valve 07/01/2017-12/31/2017 EW CardiAQ Edwards Valve Cardiac Valve Surgery IDE CE Mark Trial to Start 117313 Corp. is set to begin in mid-2016.

Date Range Delayed (02/10/2014) - Edwards Lifesciences reported that they expect to receive CE mark approval in mid-2015.New Information (02/26/2015) - Edwards reported that they expect to launch Centera in Europe in 2016 following CE Mark approval. Thus we await an update on the approval in the time frame above.Date Range Edwards Lifesciences CENTERA Transcatheter Development Edwards Lifesciences Corp. announced it expects to 10/01/2016-12/31/2016 EW Cardiac Valve Surgery CE Mark Approval Delayed (10/30/2015) - Edwards announced that given the 92937 Corp. Heart Valve Outside U.S. receive CE mark approval in 4Q 2014. strong adoption and success of its SAPIEN 3 valve the company has reprioritized the timing of CENTERA and now plans to launch in Europe in 2017. We expect an update on the CE Mark decision in the time frame above.Date Range Delayed (12/09/2015) - Edwards announced it expects CENTERA to receive CE Mark in the second half of 2017.

New Information (07/30/2015) - Edwards said that its activities in support of U.S. approval for INTUITY Elite Edwards Lifesciences announced it plans to receive remains on track and it expects the launch in 2016. We Edwards Lifesciences EDWARDS INTUITY Valve 01/11/2016-09/30/2016 EW Cardiac Valve Surgery IDE PMA FDA Approval U.S. Food and Drug Administration (FDA) approval expect an update on the PMA approval in the time frame 108582 Corp. System for its INTUITY Elite in 2016. above.Date Range Expedited (01/11/2016) - As Edwards expect a U.S. launch of INTUITY Elite in mid-2016 we are expediting our expected approval timeline.

Edwards Lifesciences EDWARDS INTUITY Valve Edwards expect a U.S. launch of INTUITY Elite in mid- 04/01/2016-09/30/2016 EW Cardiac Valve Surgery IDE US Product Launch 117944 Corp. System 2016. Edwards said that clinical study planning is underway Edwards Lifesciences Development Early Feasibility Clinical for an Early Feasibility Study in the United States as Date Range Refined (12/11/2015) - Edwards said it plans to Now-12/31/2016 EW FORMA Cardiac Valve Surgery 115655 Corp. Outside U.S. Trial to Start (US) well as a multi-center European and Canadian study. begin the CE Mark and US early feasibility studies in 2016. We await an update in the time frame above.

Edwards said that clinical study planning is Multi-Center Edwards Lifesciences Development underway for an Early Feasibility Study in the Date Range Refined (12/11/2015) - Edwards said it plans to Now-12/31/2016 EW FORMA Cardiac Valve Surgery European/Canadian 115656 Corp. Outside U.S. United States as well as a multi-center European begin the CE Mark and US early feasibility studies in 2016. Study to Start and Canadian study. We await an update in the time frame above. Edwards announced it is developing a surgical heart valve INSPIRE which is designed to improve future Edwards Lifesciences 01/01/2017-12/31/2017 EW INSPIRIS Heart Valve Cardiac Valve Surgery Development Product Launch (US) TAVR valve-in-valve procedures. The company 108740 Corp. expects to launch the product in Europe and the US in 2017. Edwards announced it is developing a surgical heart valve INSPIRE which is designed to improve future Edwards Lifesciences 01/01/2017-12/31/2017 EW INSPIRIS Heart Valve Cardiac Valve Surgery Development Product Launch (EU) TAVR valve-in-valve procedures. The company 108742 Corp. expects to launch the product in Europe and the US in 2017. Edwards announced that in 2016 it plans to initiate a Edwards Lifesciences trial to study patients with a lower risk indication Now-12/31/2016 EW SAPIEN 3 Cardiac Valve Surgery Approved Low-Risk Trial to Start 117298 Corp. (STS 0-4/5). The study will be a 1:1 randomized comparison to surgery. Edwards expects to submit an application to the U.S. Date Range Refined (07/30/2015) - Edwards said that it Food and Drug Administration (FDA) for the Sapien 3 expects to reach the 1-year endpoint for intermediate-risk Edwards Lifesciences PMA Approval Decision - for intermediate risk patients late in 2015. The patients with Sapien 3 (PARTNER II Trial; Intermediate-Risk 09/01/2016-12/31/2016 EW SAPIEN 3 Cardiac Valve Surgery Approved 109011 Corp. Intermediate Risk company assumes a 1-year review period followed S3 ARM) around the end of 2015. Assuming positive trial by approval in late 2016 but average FDA results and an expedited FDA review the company said it review/approval time could take until early 2017. expects to receive approval in late 2016.

Edwards said that the SAPIEN 3 Ultra System which includes the SAPIEN 3 Ultra delivery system the SAPIEN 3 Valve and the Axela next-generation sheath is expected to receive CE Mark in Europe in Q4 2016. Edwards Lifesciences SAPIEN 3 Ultra System CE 09/01/2016-12/31/2016 EW SAPIEN 3 Cardiac Valve Surgery Approved The SAPIEN 3 Ultra Delivery System is an on-balloon 117269 Corp. Mark system with 14F compatability for all valve sizes and the Axela Sheath is a next-generation sheath that the company says facilitates dynamic expansion and contraction.

Endologix expects to receive CE Mark approval for AFX Endovascular AAA 01/12/2016-06/30/2016 Endologix, Inc. ELGX Aortic Aneurysm Approved CE Mark Approval - AFX2 AFX2 in the first half of 2016 and launch the product 118183 System during Q3 2016.

Date Range Delayed (10/12/2015) - Endologix announced that it has received U.S. Food and Drug Administration (FDA) approval for the AFX2 Bifurcated Endograft System Endologix Inc. announced it expects to launch the AFX Endovascular AAA US Product Launch - AFX for the treatment of abdominal aortic aneurysms (AAA). 04/01/2016-06/30/2016 Endologix, Inc. ELGX Aortic Aneurysm Approved AFX2 delivery system for the bifurcated main body 98638 System 2 Endologix anticipates a commercial launch in the U.S. device by the end of 2015. expected to begin in the first quarter of 2016.Date Range Delayed (01/12/2016) - Endologix said it plans to launch the AFX2 in the U.S. during Q2 2016.

Biomedtracker August 2016 / 146 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link Endologix announced that In Europe AFX2 is AFX Endovascular AAA Product Launch (EU) - Date Range Delayed (01/12/2016) - Endologix said it 07/01/2016-09/30/2016 Endologix, Inc. ELGX Aortic Aneurysm Approved expected to be introduced in the first quarter of 116511 System AFX2 expects to launch the AFX2 in Europe during Q3 2016. 2016.

Date Range Delayed (11/20/2015) - Endologix announced that it expects it will submit Modular 2 of its modular PMA in February 2016.New Information (01/12/2016) - Nellix Endovascular Endologix announced that is expects to submit PMA Endologix announced that it has completed the submission 02/01/2016-02/29/2016 Endologix, Inc. ELGX Aortic Aneurysm PMA PMA Module 2 Filing 111250 Aneurysm Sealing System modules 2 and 3 later in 2015. of PMA modules 1 and 3. The company is currently working toward modular 2 which is expected in February and modular 4 (final module) which is expected in the March or April time frame.

New Information (06/17/2015) - Endologix announced that based on current assumptions and timelines the Company Endologix announced it completed patient anticipates submission of the premarket approval (PMA) enrollment in the EVAS FORWARD IDE study in application to the U.S. FDA in early 2016.Date Range November. The patients in this study will be followed Refined (11/20/2015) - Endologix announced that it expects for one year after which the company will submit the Nellix Endovascular PMA Module 4 (Final) to submit Modular 4 the final module for the Nellix PMA in 03/01/2016-04/30/2016 Endologix, Inc. ELGX Aortic Aneurysm PMA final module of the PMA to the FDA. Based on this 109361 Aneurysm Sealing System Submission March 2016.Date Range Delayed (01/12/2016) - Endologix timeline the company remains on track for potential announced that it has completed the submission of PMA FDA approval of Nellix in late 2016. As such we look modules 1 and 3. The company is currently working toward for a final PMA module submission sometime in the modular 2 which is expected in February and modular 4 first half of 2016. (final module) which is expected in the March or April time frame.

Date Range Delayed (11/06/2015) - Endologix announced Nellix Endovascular EU Launch - Next Endologix announced it plans to launch an enhanced 04/01/2016-06/30/2016 Endologix, Inc. ELGX Aortic Aneurysm PMA that the next generation of Nellix is scheduled for launch in 109362 Aneurysm Sealing System Generation version of Nellix in Europe by the end of 2015. Europe during the second quarter 2016.

Date Range Refined (01/12/2016) - Endologix announced Endologix announced that it expects to present 1- Nellix Endovascular IDE EVAS FORWARD 1- that it plans to present 1-year follow-up from the Nellix IDE 06/08/2016-06/11/2016 Endologix, Inc. ELGX Aortic Aneurysm PMA year clinical results from the Nellix IDE study in June 116887 Aneurysm Sealing System Year Data at SVS study at the Society for Vascular Surgery (SVS) meeting in 2016. June 2016.

Date Range Expedited (01/13/2011) - Endologix expects to launch the Nellix device in the United States in 2012 pending FDA approval.Date Range Delayed (08/18/2011) - Endologix Inc. announced it now expects FDA PMA approval in 2015.Date Range Delayed (10/26/2012) - Endologix Inc. now expects to be in position to receive FDA approval in 2016.Date Range Refined (11/04/2013) - Endologix Inc. now expects to achieve PMA approval in the U.S. in the second half of 2016.Date Range Delayed (08/05/2014) - Endologix Inc. announced it now expects PMA approval to occur in the U.S. by the end of 2016.New Information (11/19/2014) - Nellix Endovascular Endologix announced that it expects to receive PMA 09/01/2016-04/30/2017 Endologix, Inc. ELGX Aortic Aneurysm PMA PMA Approval Endologix announced that it anticipates FDA approval of 55630 Aneurysm Sealing System approval in the U.S. in 2015. the Nellix System in the U.S. before the end of 2016.New Information (04/29/2015) - Endologix announced that it is still on track for potential U.S. FDA approval in late 2016.New Information (06/17/2015) - Based on current assumptions and timelines the Company anticipates submission of the premarket approval (PMA) application to the U.S. FDA in early 2016 and is anticipating FDA approval of the Nellix System by the end of 2016.Date Range Delayed (01/12/2016) - Endologix announced that it expects to receive PMA approval for Nellix around the end of 2016 or early 2017.

Date Range Delayed (01/12/2016) - Endologix announced Nellix Endovascular Endologix announced it plans to launch Nellix in the that it expects to receive PMA approval for Nellix around 09/01/2016-04/30/2017 Endologix, Inc. ELGX Aortic Aneurysm PMA U.S. Product Launch 109360 Aneurysm Sealing System United States in late 2016. the end of 2016 or early 2017. The company expects the launch to occur around the same time frame.

Nellix Endovascular Endologix announced that it anticipates approval 01/01/2017-12/31/2017 Endologix, Inc. ELGX Aortic Aneurysm PMA Approval (Brazil) 116516 Aneurysm Sealing System and launch of Nellix in Brazil in 2017.

Nellix Endovascular Endologix announced that it anticipates approval 01/01/2017-12/31/2017 Endologix, Inc. ELGX Aortic Aneurysm PMA PMA Approval (Japan) 116514 Aneurysm Sealing System and launch of Nellix in Japan in 2017.

Nellix Endovascular Nellix CHEVAS - CE Mark Endologix anticipates approval and launch of Nellix 01/01/2018-12/31/2018 Endologix, Inc. ELGX Aortic Aneurysm PMA 118178 Aneurysm Sealing System Approval CHEVAS in Europe in 2018.

Endologix announced that for the China market its Nellix Endovascular Date Range Delayed (01/12/2016) - Endologix expects 01/01/2019-12/31/2019 Endologix, Inc. ELGX Aortic Aneurysm PMA Approval (China) current plans are to focus on commercializing Nellix 116515 Aneurysm Sealing System approval and launch of Nellix in China in 2019. in the 2018 time frame.

Nellix Endovascular Nellix CHIVAS - PMA Endologix anticipates approval and launch of Nellix 01/01/2020-12/31/2020 Endologix, Inc. ELGX Aortic Aneurysm PMA 118180 Aneurysm Sealing System Approval CHIVAS in the US in 2020.

Exact Sciences Final USPSTF Draft Exact Sciences announced that it expects to receive 09/01/2016-12/31/2016 EXAS Cologuard Colorectal Cancer (CRC) Approved 118446 Corporation Guidelines USPSTF final guidelines on Cologuard in late 2016. Date Range Delayed (04/16/2015) - GenMark Diagnsotics announced it now plans to submit its 510(k) application with GenMark expects to submit a 510(k) application to U.S. FDA 510(k) the FDA in the first quarter of 2016.Date Range Delayed 04/01/2016-06/30/2016 GenMark Diagnostics, Inc. GNMK ePlex Diagnostic Platforms Development the U.S. Food and Drug Administration (FDA) for the 106220 Submission (01/12/2016) - GenMark said that it expects a 510(k) ePlex system during the second half of 2015. submission to the FDA for ePlex in the second quarter of 2016. Date Range Delayed (01/12/2016) - GenMark said that it expects a 510(k) submission to the FDA for ePlex in the GenMark Diagnostics announced it plans to launch second quarter of 2016. Assuming an approval at the end of 09/28/2016-12/31/2016 GenMark Diagnostics, Inc. GNMK ePlex Diagnostic Platforms Development Product Launch - U.S. ePlex in the United States during the first half of 106234 a standard 90-day 510(k) review by the FDA from the end of 2016. Q2 we expect an update on the launch in the time frame above. HeartWare announced that the HVAD Lateral study Date Range Refined (01/11/2016) - Heartware announced HeartWare International, HeartWare Ventricular HVAD Lateral Enrollment 01/11/2016-03/31/2016 HTWR Congestive Heart Failure (CHF) Approved is currently at 60% enrollment. The company expects that it has reached 75% enrollment in the LATERAL study 116168 Inc. Assist System Completion to complete enrollment early in 2016. and expects to complete enrollment in Q1 2016.

New Information (05/11/2015) - HeartWare announced that it is still on track to file for Japanese approval in the middle of 2015.Date Range Refined (08/07/2015) - HeartWare announced that is still preparing its application to Japanese regulatory authorities seeking approval of HVAD for bridge to transplantation. We continue to await Japan Approval an update in the time frame above.Date Range Delayed HeartWare International, HeartWare Ventricular HeartWare International announced it plans to 01/11/2016-12/31/2016 HTWR Congestive Heart Failure (CHF) Approved Submission - Bridge-to- (09/21/2015) - The company has not yet provided any 106181 Inc. Assist System submit for Japanese approval in mid-2015. Transplant further information. We continue to await an update.Date Range Delayed (11/25/2015) - Per a communication with HeartWare the status of the Japanese submission has not changed since their Q2 earnings update. We continue to await an update on this submission.Date Range Delayed (01/11/2016) - Heartware announced that it expects to file for approval in Japan in 2016.

Biomedtracker August 2016 / 147 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link Heartware announced that the Lavare pulsatility HeartWare International, HeartWare Ventricular Lavare Algorithm FDA 01/11/2016-12/31/2016 HTWR Congestive Heart Failure (CHF) Approved algorithm is currently pending FDA approval. We 118025 Inc. Assist System Approval await an update in the time frame above.

HeartWare said it expects to complete one-year patient follow-up in the ENDURANCE II study next Date Range Refined (01/11/2016) - Heartware announced HeartWare International, HeartWare Ventricular ENDURANCE II 1-Year summer. Results from this study will be used to that it expects to complete one-year follow-up on the 02/01/2016-03/31/2016 HTWR Congestive Heart Failure (CHF) Approved 116136 Inc. Assist System Results support a Pre-Market Approval (PMA) application ENDURANCE II trial in August 2016. We expect an update on seeking a destination therapy indication. We await this data in the time frame above. an update on the results in the time frame above.

Date Range Refined (03/06/2015) - HeartWare International announced it plans to file with the U.S. Food and Drug Administration in late 2016.New Information (04/16/2015) - Heartware said it anticipates submitting a Premarket Approval (PMA) application to the FDA next year seeking FDA Supplemental PMA HeartWare International, HeartWare Ventricular HeartWare is targeting a U.S. FDA submission for approval of the HeartWare System for the Destination 04/01/2016-09/30/2016 HTWR Congestive Heart Failure (CHF) Approved Submission - DT 106180 Inc. Assist System destination therapy approval in 2016. Therapy indication.Date Range Refined (01/11/2016) - Indication Heartware announced that it expects to submit the PMA to the FDA in mis-2016. The company said it will submit 2-year results from ENDURANCE and a minimum of 6-month follow-up from ENDURANCE II to support the approval of HVAD for the destination therapy indication.

Heartware said it expects to complete 6-month HeartWare International, HeartWare Ventricular 6-Month Follow-Up follow-up in ENDURANCE II in February 2016. We 04/01/2016-09/30/2016 HTWR Congestive Heart Failure (CHF) Approved 118024 Inc. Assist System Complete expect an update on this data in the time frame above.

New Information (01/11/2016) - Heartware announced that HeartWare announced that is has suspended it is has completed the alterations to the controller and enrollment in the MVAdvantage study. The software updates. The company said it is now working to company announced that although HeartWare identify the cause of pump thrombus events that were may not re-initiate enrollment in the MVAD observed in December 2015 following the pause in the trial clinical trial in November as it previously due to an issue with the controller. The company believes expected HeartWare remains confident in its that the power and efficiency in the device is causing some MVAD System and the potential for the MVAD patients to experience sustained suction which appears to HeartWare International, MVAdvantage Study Re- Now-03/31/2016 HTWR MVAD System Congestive Heart Failure (CHF) IDE design to meaningfully improve outcomes for lead to thrombus. The company is looking into the qPulse 115614 Inc. Initiation ventricular assist patients. HeartWare intends to algorithm as a potential cause of the suction problem and is provide additional commentary related to the investigating potential solutions including a suction alarm MVAD clinical trial on its third quarter financial algorithm. The company hopes to provide regulators with results call and webcast scheduled for October positive results regarding the potential solutions as soon as 29 2015. It has been and remains the intention possible but has not given a definitive timeline on the start of the Company to present and publish clinical data following the completion of enrollment and of the trial. We expect an update in the time frame above. follow-up for all trial patients. The company has also noted that if design enhancements are required the company may need to begin a new trial.

Integra LifeSciences Integra Dermal Integra plans to launch the Omnigraft in summer 06/01/2016-09/30/2016 IART Diabetic Foot Ulcers Approved U.S. Product Launch 118394 Holdings Corporation Regeneration Template 2016. Phase IV PROGRESS - Intersect expects to complete enrollment in the 04/01/2016-06/30/2016 Intersect ENT XENT Nova Sinus Implant Allergy IDE Patient Enrollment 118234 NOVA study cohort of the PROGRESS study in Q2. Completed Phase IV PROGRSS - Top- Intersect expects data from the Nova study cohort of 07/01/2016-09/30/2016 Intersect ENT XENT Nova Sinus Implant Allergy IDE 118235 Line Results the PROGRESS study in Q3. Intersect expects to receive a supplemental PMA Approval Decision - 01/01/2017-12/31/2017 Intersect ENT XENT Nova Sinus Implant Allergy IDE premarket approval (PMA) from the U.S. Food and 111475 Frontal Drug Administrstion (FDA) for NOVA in 2017.

Date Range Refined (09/30/2015) - Intersect ENT announced that the company has submitted a supplemental premarket approval (PMA-s) submission to the U.S. Food Intersect ENT announced at the J.P. Morgan and Drug Administration (FDA) to seek approval to expand Regulatory PMA Healthcare Conference that it anticipates to receive the indication of the PROPEL mini steroid releasing sinus 01/12/2016-03/31/2016 Intersect ENT XENT PROPEL Sinus Implant Allergy Approved Supplemental Approval - 106493 PMA Supplemental approval for Propel-mini's frontal implant to the treatment of patients undergoing frontal Frontal Indication indication. sinus surgery. Given the FDA's standard 180-day review period we await an update in the time frame above.Date Range Delayed (01/12/2016) - Intersect expects approval of the frontal sinus indication for PROPEL Mini this quarter.

Novocure plans to open a Phase III trial the METIS Brain Cancer (secondary; Development trial for the treatment of brain metastases Now-12/31/2016 NovoCure Limited NVCR Optune Phase III to Start 117070 metastases) Outside U.S. originating from non-small cell lung cancer (NSCLC) in 2016 subject to FDA approval of the final protocol.

Development Phase II STELLAR - Novocure said it expects to enroll the last patient in 01/01/2017-12/31/2017 NovoCure Limited NVCR Optune Mesothelioma 117365 Outside U.S. Enrollment Completion the STELLAR trial in 2017. Non-Small Cell Lung Cancer Phase III LUNAR Study to Now-12/31/2016 NovoCure Limited NVCR Optune Development Novocure said it expects to begin the first patient 117381 (NSCLC) Start in the Phase III LUNAR trial in 2016. Development INNOVATE Enrollment Novocure said it expects to enroll the last patient in Now-12/31/2016 NovoCure Limited NVCR Optune Ovarian Cancer 117363 Outside U.S. Completion the INNOVATE trial in 2016.

Date Range Expedited (01/13/2016) - Novocure announced that 1st cohort data from the PANOVA trial is expected to Development PANOVA - 1st Cohort Top- Novocure said it expects to present results from the 1/19/2016 NovoCure Limited NVCR Optune Pancreatic Cancer be presented at the ASCO GI meeting in January 2016. The 117364 Outside U.S. Line Data at ASCO GI 1st cohort of the PANOVA trial in 2016. company noted that the data will be available on January 19 once the embargo is lifted.

Ovascience reports that it will proceed with a go/no- 10/01/2016-12/31/2016 OvaScience, Inc. OVAS OvaPrime Reproductive Disorder Preclinical Go/No-Go Decision go decision for the development of OvaPrime in the 118480 fourth quarter of 2016 based on available data.

OvaScience commenced a non-commercial preceptorship program with the OvaPrime treatment at an in vitro fertilization (IVF) clinic New Information (01/14/2016) - Ovascience reports that it outside of the United States to gain insight into the IVF Patient Experience will proceed with a go/no-go decision for the development 10/01/2016-12/31/2016 OvaScience, Inc. OVAS OvaPrime Reproductive Disorder Preclinical clinical efficacy and feasibility of the treatment. The 117220 Report of OvaPrime in the fourth quarter of 2016 based on Company expects that physicians using the available data in the same time frame. treatment will report patient experiences by the end of 2016 as clinically meaningful information becomes available.

Biomedtracker August 2016 / 148 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link

Date Range Delayed (09/23/2015) - REVA announced that it expects to complete enrollment in this trial prior to December 31 2015.New Information (10/01/2015) - Reva announced that it has enrolled the first cohort of 110 patients in the FANTOM II clinical trial. We continue to await an update on the second cohort in the time frame above.Date Range Delayed (12/29/2015) - REVA stated that Reva Medical said it plans to enroll the 110 patients they are enrolling an additional 110 supplemental patients Fantom Bioresorbable Development FANTOM II - Patient in the FANTOM II study in Q2 and Q3 of 2015. Thus 01/14/2016-04/30/2016 REVA Medical Inc. RVA:ASX Coronary Artery Disease with Fantom to obtain additional data for 109895 Scaffold Outside U.S. Enrollment Completion we expect enrollment in this study to be completed commercialization purposes. While no timeframe has been in the time frame above. given as the company enrolled the first 110 in about 7 months (March through September) we expect the second cohort of 110 in a similar time frame.New Information (01/14/2016) - REVA announced that it has enrolled 70 of the 110 expected patients for Cohort B of the FANTOM II Trial. We await further update on the completion of enrollment in this cohort.

Fantom Bioresorbable Development REVA plans to file for CE Mark approval of their Date Range Refined (01/14/2016) - REVA announced that it 07/01/2016-09/30/2016 REVA Medical Inc. RVA:ASX Coronary Artery Disease CE Mark Filing 105415 Scaffold Outside U.S. Fantom bioresorbable scaffold in mid-2016. plans to file for CE Mark approval in Q3 2016. REVA announced that it plans to file for CE Mark Fantom Bioresorbable Development 10/01/2016-12/31/2016 REVA Medical Inc. RVA:ASX Coronary Artery Disease CE Mark Approval approval in Q3 2016 and anticipates receiving CE 118474 Scaffold Outside U.S. Mark approval in Q4 2016. REVA announced that it anticipates receiving CE Fantom Bioresorbable Development 01/01/2017-06/30/2017 REVA Medical Inc. RVA:ASX Coronary Artery Disease Product Launch (EU) Mark approval in Q4 2016 and commercializing the 118477 Scaffold Outside U.S. product to market in the first half of 2017.

Halozyme announced that they anticipate finalizing the specifications and submiting an investigational device exemption for the companion diagnostic. The Date Range Refined (02/01/2016 - 02/29/2016) - Halozyme PEGPH20 Companion Regulatory - IDE 02/01/2016-02/29/2016 Roche Holding AG RHHBF Pancreatic Cancer Preclinical companion diagnostic will support initiation of announced that a U.S. IDE submission is anticipated in 114534 Diagnostic Submission patient enrollment in the planned Phase III study, February 2016 for the PEGPH20 companion diagnostic test. which is expected to begin by the end of the first quarter of 2016.

New Information (06/24/2015) - Spectranetics announced it submitted a CE mark application for its AngioSculpt product AngioSculptX Drug- Spectranetics announced that it is developing a Development and is targeting a mid-2016 European launch.Date Range 01/13/2016-12/31/2016 Spectranetics Corp. SPNC Coated PTCA Scoring Coronary Artery Disease CE Mark Approval coronary drug-coated angiosculpt balloon and 106406 Outside U.S. Delayed (01/13/2016) - Spectranetics expects CE mark Balloon Catheter expects to receive CE Mark approval in 2016. approval for the AngioSculptX drug-coated PTCA scoring balloon catheter in 2016.

ILLUMENATE GLOBAL - Spectranetics will present 12 month interim data Peripheral Arterial Disease 04/26/2016-04/29/2016 Spectranetics Corp. SPNC Stellarex DCB IDE Top-Line Results at from the ILLUMENATE Global study in April at the 118379 (PAD) Charing Cross Charing Cross International Symposium. Spectranetics will present 12 month data from the Peripheral Arterial Disease ILLUMENATE EU RCT - 09/18/2016-09/22/2016 Spectranetics Corp. SPNC Stellarex DCB IDE ILLUMENATE EU RCT study at the VIVA conference in 118380 (PAD) Top-Line Results at VIVA September.

Phase II ILLUMENATE Spectranetics will present 12 month data from the Peripheral Arterial Disease 10/29/2016-11/02/2016 Spectranetics Corp. SPNC Stellarex DCB IDE Pivotal Study (US) - Top- ILLUMENATE US pivotal study at the TCT meeting in 118362 (PAD) Line Results at TCT October. ILLUMENATE Global Spectranetics will present 12 month data from the Peripheral Arterial Disease 01/01/2017-01/31/2017 Spectranetics Corp. SPNC Stellarex DCB IDE Registry - Top-Line ILLUMENATE Global Registry study at the LINC 118382 (PAD) Results at LINC meeting in January 2017. EnSite Precision Cardiac St. Jude Medical announced that it expects a H1 Hemodynamic/Vital Sign 01/13/2016-06/30/2016 St. Jude Medical, Inc. STJ EnSite System Approved Mapping System - 2016 launch for the EnSite Precision Cardiac 118322 Monitoring Product Launch (US) Mapping System. T2 Biosystems expects a possible approval of the 01/01/2017-04/30/2017 T2 Biosystems, Inc. TTOO T2Bacteria Sepsis and Septic Shock Development 510(k) Clearance 118505 T2Bacteria Panel by early 2017. T2 plans to start an FDA clinical study with 04/01/2016-09/30/2016 T2 Biosystems, Inc. TTOO T2HemoStat Hemostasis Development Clinical Study to Start 118502 T2HemoStat in mid-2016.

Mallinckrodt announced that it has entered into a purchase agreement with The Medicines Company under which subsidiaries of Mallinckrodt will acquire a global portfolio of three commercial-stage topical hemostasis drugs – RECOTHROM Thrombin topical (Recombinant) PreveLeak Surgical Sealant and Preveleak Surgical Product Acquisition RAPLIXA (Fibrin Sealant) – for a total potential Now-03/31/2016 The Medicines Company MDCO Hemostasis Approved 117938 Sealant Complete - Mallinckrodt consideration of up to $410 million. Subject to customary terms and conditions including the expiration or termination of the waiting period under the Hart Scott Rodino Antitrust Improvements Act the transaction is expected to close in the first calendar quarter of 2016 Mallinckrodt's fiscal second quarter.

Date Range Delayed (02/25/2014) - The Company plans to file for FDA approval in Q4 2014. We are pushing back the date of estimated approval based on the expected industry average 5 month approval process for a 510(k). Date Range Delayed (11/06/2014) - TransEnterix now expects to submit a 510(k) application in mid-2015. We have delayed the expected approval to the end of 2015 due to TransEnterix's delay.New Information (06/01/2015) - TransEnterix announced on June 1 2015 that it submitted a 510(k) Company officials will seek market clearance for application with the U.S. Food and Drug Administration 01/14/2016-03/31/2016 TransEnterix, Inc. TRXC SurgiBot Surgical Tools (General) 510(k) 510(k) Approval Decision 97749 the SurgiBot single-site mobile robot from the (FDA) for clearance of the company's SurgiBot System. U.S. Food and Drug Administration in 2014. Given the FDA's typical 90-day review for 510(k) clearances we expect an update on the clearance decision in the time frame above.Date Range Delayed (08/30/2015) - TransEnterix has not provided an update on the 510(k) application for SurgiBot and there is no approval listed on the FDA website. We await an update.Date Range Delayed (10/15/2015) - We continue to await an update.Date Range Delayed (01/14/2016) - TransEnterix expects SurgiBot FDA clearance in Q1 2016.

TransEnterix expects to launch SurgiBot in the US in 04/01/2016-06/30/2016 TransEnterix, Inc. TRXC SurgiBot Surgical Tools (General) 510(k) Product Launch (U.S.) 118486 Q2 2016. Approved in TransEnterix expects to commence sales of ALF-X in 01/14/2016-03/31/2016 TransEnterix, Inc. TRXC Telelap ALF-X Surgical Tools (General) Product Launch (Europe) 118485 Europe Q1 2016. Approved in TransEnterix expects to launch ALF-X in the U.S. in 01/01/2017-12/31/2017 TransEnterix, Inc. TRXC Telelap ALF-X Surgical Tools (General) Product Launch (U.S.) 118487 Europe 2017. TriVascular Technologies, Ovation Abdominal Stent PMA Approval (Japan) - Now-12/31/2016 TRIV Aortic Aneurysm Approved Endologix anticipates approval and launch of 118184 Inc. Graft System Ovation Prime Ovation Prime in Japan in 2016. TriVascular Technologies, Endologix anticipates US approval of Ovation Alto in 01/01/2018-12/31/2018 TRIV Ovation Alto System Aortic Aneurysm Preclinical PMA Approval (US) 118188 Inc. 2018 or early 2019. TriVascular Technologies, Endologix anticipates European approval of Ovation 01/01/2018-04/30/2019 TRIV Ovation Alto System Aortic Aneurysm Preclinical CE Mark Approval 118187 Inc. Alto in 2018. Development Cardinal for Mitral Repair Heartware said the Cardinal is expected to launch in 01/11/2016-12/31/2016 Valtech Cardio, Ltd. Cardinal Cardiac Valve Surgery 118051 Outside U.S. Product Launch (EU) the EU in 2016.

Biomedtracker August 2016 / 149 Pharma intelligence | Healthcare Conference: Conference Review

MedDeviceTracker JPM Catalysts Expected Start Date Company Symbol Device Indication Phase Expected Catalyst Analysis Updated Analysis Link Development Cardinal for Mitral Repair Heartware said a Cardinal IDE study is expected to 01/01/2017-12/31/2017 Valtech Cardio, Ltd. Cardinal Cardiac Valve Surgery 118056 Outside U.S. IDE Study to Start begin in 2017. Development Cardinal for Mitral Repair Heartware said Cardinal for mitral valve repair is 01/01/2018-12/31/2018 Valtech Cardio, Ltd. Cardinal Cardiac Valve Surgery 118065 Outside U.S. PMA Approval expected to receive PMA approval in 2018.

HeartWare announced that there is an Approved in Now-12/31/2016 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery IDE Study to Start Invesitgational Device Exemption (IDE) submission 116163 Europe for a U.S. IDE trial planned for 2016.

HeartWare announced that it expects a first-in-man Date Range Refined (01/11/2016) - Heartware announced Approved in CardioBand Tricuspid 08/01/2016-09/30/2016 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery trial for CardioBand tricuspid treatment for tricuspid that the FIM for Cardioband for tricuspid repair is expected 116164 Europe FIM Study to Start regurgitation to begin in late 2016. in 2016. Approved in Cardioband Tricuspid IDE Heartware said it expects the Cardioband TR IDE 01/01/2017-12/31/2017 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery 118054 Europe to Start study to begin in 2017. Approved in Cardioband Mitral PMA Heartware the Cardioband for mitral repair is 01/01/2018-12/31/2018 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery 118058 Europe Approval expected to receive PMA approval in 2018. Approved in Cardioband Tricuspid CE Heartware said Cardioband for tricuspid repair is 01/01/2018-12/31/2018 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery 118062 Europe Mark expected to receive CE Mark approval in 2018.

Approved in Cardioband Tricuspid Heartware said the Cardioband for tricuspid repair is 01/01/2019-12/31/2019 Valtech Cardio, Ltd. Cardioband Cardiac Valve Surgery 118069 Europe PMA Approval expected to receive PMA approval in 2019. Development Heartware said the first-in-man study of Cardiovalve 01/01/2017-12/31/2017 Valtech Cardio, Ltd. CardioValve Cardiac Valve Surgery FIM Trial to Start 118052 Outside U.S. is expected to begin in 2017. Development Heartware said a Cardiovalve IDE study is expected 01/01/2018-12/31/2018 Valtech Cardio, Ltd. CardioValve Cardiac Valve Surgery IDE Study to Start 118067 Outside U.S. to begin in 2018. Development Heartware said Cardiovalve is expected to receive CE 01/01/2019-12/31/2019 Valtech Cardio, Ltd. CardioValve Cardiac Valve Surgery Cardiovalve CE Mark 118068 Outside U.S. Mark in 2019.

Veracyte announced that it is leveraging its technology used in the Percepta Bronchial Laboratory Idiopathic Pulmonary Idiopathic Pulmonary Fibrosis Genomic Classifier to develop a diagnostic 01/14/2016-12/31/2016 Veracyte, Inc. VCYT Developed Test Product Launch (US) 118493 Fibrosis Test (IPF) solution for patients who are at risk for idiopathic (LDT) pulmonary fibrosis that doesn't require diagnostic surgery. The company plans to launch the product by the end of 2016.

Biomedtracker August 2016 / 150 Pharma intelligence |