Biomedtracker Pharma intelligence | HEALTHCARE CONFERENCE: CONFERENCE REVIEW Healthcare Conference:JP Morgan Healthcare Conference Conference: Review Review Biomedtracker August 2016 / 2 Pharma intelligence | QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 1 Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review 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. ABOUT THE AUTHORS BioMedTracker is an independent research service that offers proprietary clinical assessments and patient-based revenue forecasts of developmental drugs within a comprehensive and intuitive drug information database. 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EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 2 Biomedtracker August 2016 / 3 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review 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. QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 3 Biomedtracker August 2016 / 4 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review 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. QUESTIONS? EMAIL [email protected] FOR OUR DISCLOSURES, PLEASE SEE BIOMEDTRACKER’s RESEARCH STANDARDS 4 Biomedtracker August 2016 / 5 Pharma intelligence | Healthcare Conference: Conference Review JP Morgan Healthcare Conference: Review 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