Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

2019 Possible Launch Date

RVT-802 RVT-802 Enzyvant/Roivant Tissue-based therapy Congenital athymia Implant Filed NDA 12/2019 Yes Yes

Zimhi naloxone Adamis opioid antagonist Opioid dependence IM Filed NDA 12/2019 No No

calcitonin gene-related MK-1602 (AGN-241689) ubrogepant Allergan/ Merck peptide (CGRP) receptor Acute migraines PO Filed NDA 12/2019 No No antagonist

IDP-123 IDP-123 Bausch Health Acne TOP Filed NDA 12/22/2019 No No

Brinavess vernakalant Correvio potassium channel blocker Arrhythmia IV Filed NDA 12/24/2019 Yes No

E-2006 lemborexant Eisai/ Purdue orexin receptor antagonist Insomnia PO Filed NDA 12/27/2019 No No

ITI-007 lumateperone Intra-Cellular Therapies antipsychotic Schizophrenia PO Filed NDA 12/27/2019 No No

HIV integrase inhibitor/ cabotegravir (long-acting)/ non-nucleoside reverse TMC-278-LA ViiV Healthcare HIV IM Filed NDA 12/29/2019 Yes No rilpivirine (long- acting) transcriptase inhibitor (NNRTI)

S-265744 (S/GSK- 1265744) cabotegravir ViiV Healthcare HIV integrase inhibitor HIV PO Filed NDA 12/29/2019 Yes No

NRL-1 Neurelis Seizures Intranasal Filed NDA 2H2019 No Yes

2020 Possible Launch Date

integrin receptor 12/2019 - Entyvio (SC formulation) vedolizumab Takeda Ulcerative colitis SC Filed sBLA Yes No antagonist 1/2020

methyltransferase EZH2 E-7438 (EPZ- 6438) tazemetostat Epizyme/ Eisai Sarcoma PO Filed NDA 1/23/2020 Yes Yes inhibitor

CastiaRx.com // p: 866.516.2121 1 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Schizophrenia/ Rykindo risperidone ER Luye atypical antipsychotic IM Filed NDA 1/28/2020 Yes No Schizoaffective disorder

gonadotropin-releasing FP-001 (LMIS) leuprolide mesylate Foresee Prostate cancer SC Filed NDA 1/29/2020 Yes No hormone (GnRH) analog

AR-101 AR-101 Aimmune peanut protein capsule Peanut allergy PO Filed BLA 1/2020 Yes No

selective KIT and PDGFRa Gastrointestinal stromal BLU-285 avapritinib Blueprint Medicines PO Filed NDA 2/14/2020 Yes Yes inhibitor tumors

ethinyl estradiol/ hormonal combination Twirla Agile Therapeutics Pregnancy prevention TOP Filed NDA 2/16/2020 No No levonorgestrel contraceptive

calcitonin gene-related BMS-927711 (BHV-3000) rimegepant sulfate Biohaven peptide (CGRP) receptor Acute migraines PO Filed NDA 2/20/2020 No No antagonist

calcitonin gene-related ALD-403 eptinezumab Alder peptide (CGRP) receptor Migraine prevention IV Filed BLA 2/21/2020 No No antagonist

ETC-1002 bempedoic acid Esperion Therapeutics ATP citrate lyase inhibitor Hypercholesterolemia PO Filed NDA 2/21/2020 No No

ATP citrate lyase inhibitor/ ETC-1002/ bempedoic acid/ ezetimibe Esperion Therapeutics cholesterol absorption Hypercholesterolemia PO Filed NDA 2/26/2020 No No ezetimibe inhibitor

dopamine receptor APD-421 amisulpride Acacia Nausea/ vomiting IV Filed NDA 2/26/2020 No No antagonist

insulin-like growth factor 1 RV-001 (Roche- 1, R-1507) teprotumumab Horizon Therapeutics Thyroid eye disease IV Filed BLA 3/8/2020 Yes Yes (IGF-1) receptor antagonist

ITCA-650 exenatide sustained- glucagon-like peptide-1 (sustained release Intarcia Diabetes mellitus SC implant Filed NDA 3/9/2020 No No release (GLP-1) receptor agonist exenatide)

VS-EBOV (rVSV- EBOV; rVSV- ZEBOV Merck/ NewLink Genetics Ebola IM Filed BLA 3/14/2020 Yes No ZEBOV-GP)

CastiaRx.com // p: 866.516.2121 2 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

naloxone naloxone Insys Therapeutics opioid antagonist Opioid dependence Intranasal Filed NDA 3/15/2020 No No

ASG-22M6E enfortumab vedotin Astellas/ Seattle Genetics nectin-4 antagonist Bladder cancer IV Filed BLA 3/15/2020 Yes No

ET-105 lamotrigine Eton anticonvulsant Epilepsy PO Filed NDA 3/17/2020 No No

sphingosine 1-phosphate 1 ozanimod ozanimod Celgene (S1PR1) and 5 (S1PR5) Multiple sclerosis PO Filed NDA 3/25/2020 Yes No receptor modulator

farnesoid X receptor (FXR) Nonalcoholic obeticholic acid obeticholic acid Intercept Pharmaceuticals PO Filed NDA 3/26/2020 Yes No agonist steatohepatitis

IntelGenx / Red Hill Rizaport (VersaFilm) rizatriptan triptans Acute migraines PO Filed NDA 3/26/2020 No No Biopharma

anesthetic/ Nonsteroidal HTX-011 bupivacaine/ meloxicam Heron Therapeutics Anti-inflammatory Drug Pain Instillation Filed NDA 3/26/2020 No No (NSAID)

Sun Pharma Advanced paclitaxel injection Breast Cancer; Lung SPARC-1028 Research Company taxane IV Filed NDA 1Q2020 No No concentrate for suspension Cancer; Pancreatic Cancer (SPARC)

SABER- Posidur Novartis/ Durect local anesthetic Pain SC Filed NDA 1Q2020 No No bupivacaine CR

aldosterone synthase LCI-699 osilodrostat Novartis Cushing's syndrome PO Filed NDA 1Q2020 No Yes inhibitor

sodium glucose co- transporter-2 (SGLT-2) empagliflozin, linagliptin, empagliflozin, linagliptin, Eli Lilly/ Boehringer inhibitor, dipeptidyl Diabetes mellitus PO Filed NDA 1Q2020 No No metformin XR metformin XR Ingelheim peptidase 4 (DPP4) inhibitor, biguanide

bimatoprost sustained bimatoprost sustained Allergan prostaglandin agonist Glaucoma Implant Filed NDA 4/1/2020 No No release release

CNS-7056 Cosmo benzodiazepine Procedural sedation IV Filed NDA 4/3/2020 Yes No

CastiaRx.com // p: 866.516.2121 3 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

meningococcal polysaccharide (serogroups Men Quad TT Sanofi antibacterial meninococcus/ tetanus IM Filed BLA 4/25/2020 No No A, C, Y, and W135) tetanus toxoid conjugate vaccine

catechol-O- Neurocrine Biosciences/ Ongentys opicapone methyltransferase (COMT) Parkinson's disease PO Filed NDA 4/26/2020 No No Bial/ Ono inhibitor

Pulmonary arterial Trevyent treprostinil SteadyMed prostacyclin analog SC Filed NDA 4/27/2020 Yes Yes hypertension

HER2-targeting antibody- DS-8201 trastuzumab deruxtecan Daiichi Sankyo Breast cancer IV Filed BLA 4/29/2020 Yes No drug conjugate

Multiple myeloma/ Acute isatuximab isatuximab Sanofi/ ImmunoGen CD38 antagonist lymphoblastic leukemia or IV Filed BLA 4/30/2020 Yes Yes lymphoblastic lymphoma selective MEK kinase 4/2020 – selumetinib selumetinib AstraZeneca/ Merck Neurofibromatosis PO Filed NDA Yes Yes inhibitor 5/2020

SEP-225289 (DSP-225289, Sumitomo Dainippon/ dasotraline triple reuptake inhibitor Eating disorders PO Filed NDA 5/14/2020 No No SEP-289) Sunovion

selective FGFR1/2/3 INCB-54828 pemigatinib Incyte Biliary tract cancer PO Filed NDA 5/2020 Yes Yes inhibitor

FMX-103 minocycline Foamix tetracyclines Rosacea TOP Filed NDA 6/2/2020 No No

morpholino antisense Duchenne muscular NS-065 viltolarsen Nippon Shinyaku IV Filed BLA 6/2/2020 Yes Yes oligonucleotide dystrophy

Tentative Bafiertam monomethyl fumarate Banner Life Sciences Nrf2 pathway activator Multiple sclerosis PO 6/20/2020 Yes No Approval

Age-related macular 6/2020 – abicipar pegol abicipar pegol Allergan VEGF-A inhibitor Intravitreal Filed BLA Yes No degeneration 7/2020

Neuromyelitis optica 6/13/2020 - MEDI-551 inebilizumab Viela Bio CD-19 antagonist IV Filed BLA Yes Yes spectrum disorder 7/13/2020

CastiaRx.com // p: 866.516.2121 4 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

EVK-001 metoclopramide Evoke Pharma antidopiminergics Diabetic gastroparesis Intranasal CRL 2Q2020 No No

osmotic gradient enhancer; Bronchitol mannitol Pharmaxis Cystic fibrosis INH CRL 2Q2020 No Yes mucus clearance enhancer

Rexista XR oxycodone ER IntelliPharmaCeutics opioid agonist Pain PO Not Approved 1H2020 No No

insulin glargine insulin glargine Mylan/ Biocon Long-acting insulin Diabetes mellitus SC CRL 1H2020 No No

RS7-SN-38 antibody-drug IMMU-132 sacituzumab govitecan Immunomedics Breast cancer IV CRL 1H2020 Yes No conjugate

NKTR-181 NKTR-181 Nektar opioid agonist Pain PO Not Approved 1H2020 No No

morpholino antisense Duchenne muscular SRP-4045 Sarepta IV InTrial Mid-2020 Yes Yes oligonucleotide dystrophy

Contepo fosfomycin Nabriva Therapeutics cell wall inhibitor Bacterial infections IV CRL Mid-2020 Yes No

RG-7916 Roche/ PTC Therapeutics SMN2 splicing modifier PO InTrial Mid-2020 Yes Yes

Apealea (Paclical) paclitaxel Oasmia taxane Ovarian cancer IV InTrial Mid-2020 Yes Yes

Diffuse large B-cell phosphoinositide-3 kinase TGR-1202 umbralisib TG Therapeutics/ Rhizen lymphoma/ Chronic PO InTrial Mid-2020 Yes Yes (PI3K) delta inhibitor lymphocytic leukemia

Mesoblast/ JCR/ Prochymal remestemcel-L Mallinckrodt/ Osiris mesenchymal stem cells Graft vs. Host disease IV InTrial Mid-2020 Yes Yes Therapeutics

MOR-208 (MOR-00208, Diffuse large B-cell tafasitamab MorphoSys/ Xencor CD-19 antagonist IV InTrial Mid-2020 Yes Yes XmAB-5574) lymphoma

CastiaRx.com // p: 866.516.2121 5 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Solid tumors; non- small LOXO-292 selpercatinib Eli Lilly/ Loxo Oncology RET inhibitor cell lung cancer; thyroid PO InTrial Mid-2020 Yes No cancer

Amphora Amphora Evofem Biosciences spermicidal agent Pregnancy prevention VG CRL Mid-2020 No No

C-C 5 PRO-140 leronlimab CytoDyn HIV; Graft vs. host disease SC InTrial Mid-2020 Yes Yes (CCR5) antagonist

long-acting beta 2 adrenergic receptor indacaterol/ agonist (LABA)/ long- QVM-149 glycopyrronium bromide/ Novartis/ Sosei Asthma INH InTrial Mid-2020 No No acting muscarinic receptor mometasone furoate antagonist (LAMA)/ corticosteroid

chimeric antigen receptor Diffuse large B-cell JCAR-017 lisocabtagene maraleucel Juno/ Celgene IV InTrial Mid-2020 Yes Yes (CAR) T cell therapy lymphoma

mometasone furoate/ corticosteroid/ GSP-301 Glenmark Allergic rhinitis Intranasal CRL Mid-2020 No No olopatadine HCl antihistamine

lurbinectidin PharmaMar/ Myriad Zepsyre alkylating agent Small cell lung cancer IV InTrial Mid-2020 Yes Yes (lurbinectedin) Genetics

MitoGel mitomycin C UroGen alkylating agent Bladder cancer Intravesical InTrial Mid-2020 No Yes

3-F8 (Hu-3F8) naxitamab Y-mAbs Therapeutics GD2 antagonist Neuroblastoma IV InTrial Mid-2020 Yes Yes

lentiviral beta- globin gene Zynteglo (LentiGlobin) Bluebird Bio Beta-thalassemia IV InTrial Mid-2020 Yes Yes transfer

CastiaRx.com // p: 866.516.2121 6 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Hutchinson-Gilford Progeria Syndrome (HGPS EBP-994 (rEBP- 994) lonafarnib Eiger Biopharmaceuticals prenylation inhibitor or progeria) and progeroid PO InTrial Mid-2020 Yes Yes laminopathies; Hepatitis D (HDV)

daratumumab (with Johnson & Johnson / humanized anti-CD38 Multiple myeloma/ Darzalex recombinant human SC Filed BLA 7/10/2020 Yes Yes Genmab monoclonal antibody Amyloidosis hyaluronidase)

Fintepla fenfluramine Zogenix serotonin receptor agonist Dravet syndrome PO Filed NDA 7/24/2020 Yes Yes

calcipotriene/ analog/ MC2-01 MC2 Therapeutics Psoriasis TOP Filed NDA 7/24/2020 No No betamethasone corticosteroid

Ultragenyx/ Baylor Glucose transport type 1 UX-007 triheptanoin Research Institute/ medium chain fatty acid PO Filed NDA 7/31/2020 Yes Yes deficiency syndrome Uniquest

Viaskin Peanut Viaskin Peanut DBV Technologies Immunotherapy Peanut allergy TOP Filed BLA 8/5/2020 No No

TRC-101 veverimer Tricida carrier protein modulator Chronic kidney disease PO Filed NDA 8/22/2020 Yes No

Winlevi clascoterone Cassiopea androgen antagonist Acne vulgaris TOP Filed NDA 8/27/2020 No No

-6 (IL-6) 8/2020 – SA-237 (RG- 6168) satralizumab Roche/ Chugai Neuromyelitis optica SC Filed BLA Yes Yes monoclonal antibody 9/2020

collagenase clostridium collagenase clostridium Endo protease Cellulite SC Filed BLA 9/6/2020 Yes No histolyticum histolyticum

CastiaRx.com // p: 866.516.2121 7 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

VP-102 VP-102 Verrica antiviral Molluscum TOP Filed NDA 9/16/2020 No No

NNC-0195- 0092 (NN- recombinant human Growth hormone somapacitan Novo Nordisk SC Filed BLA 9/21/2020 Yes No 8640) growth hormone (rhGH) deficiency

LY-900014 (URLi) insulin lispro Eli Lilly insulins Diabetes mellitus SC Filed BLA 3Q2020 No No

valoctocogene BMN-270 BioMarin gene therapy Hemophilia IV InTrial 3Q2020 Yes Yes roxaparvovec

non-nucleoside reverse Tentative efavirenz efavirenz Micro Labs transcriptase inhibitor HIV PO 3Q2020 Yes No Approval (NNRTI)

GlaxoSmithKline/ Seattle anti-BCMA antibody-drug GSK-2857916 belantamab mafodotin Multiple myeloma SC InTrial 3Q2020 Yes Yes Genetics conjugate

delta-9- tetrahydrocannab PPP-001 PhytoPain Pharma cannabinoid product Pain INH InTrial 3Q2020 Yes Yes inol/ cannabidiol

BCX-7353 BCX-7353 BioCryst kallikrein inhibitor Hereditary angioedema PO InTrial 3Q2020 Yes Yes

granulocyte colony- SPI-2012 eflapegrastim Spectrum/ Hanmi Neutropenia SC Filed BLA 10/25/2020 Yes No stimulating factor (GCSF)

opioid receptor agonist Tentative CAM-2038 buprenorphine Camurus/ Braeburn Opioid use disorder/ Pain SC 11/1/2020 Yes No (partial) Approval

selective norepinephrine Attention deficit SPN-812 SPN-812 Supernus PO Filed NDA 11/11/2020 No No reuptake inhibitor hyperactivity disorder

CastiaRx.com // p: 866.516.2121 8 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Multiple myeloma/ Non- alkylating agent/ DNA Melflufen (Ygalo) melphalan- flufenamide Oncopeptides AB small cell lung cancer/ IV InTrial 4Q2020 No Yes synthesis inhibitor Ovarian cancer

PDGFR-alpha kinase Gastrointestinal stromal DCC-2618 ripretinib Deciphera PO InTrial 4Q2020 Yes Yes inhibitor tumors

proprotein convertase The Medicines Company/ ALN-PCSsc (PCSK9si) inclisiran subtilisin/kexin 9 (PCSK- Hyperlipidemia SC InTrial 4Q2020 Yes Yes Alnylam 9) inhibitor

Seattle Genetics/ Array ErbB-2 (Her-2/neu) ARRY-380 (ONT-380) tucatinib Breast cancer PO InTrial 4Q2020 Yes Yes BioPharma inhibitor

BLU-667 pralsetinib Blueprint Medicines RET inhibitor Non-small cell lung cancer PO InTrial 4Q2020 Yes Yes

janus associated kinase-1 GLPG-0634 Gilead/ Galapagos Rheumatoid arthritis PO InTrial 4Q2020 Yes No (JAK) inhibitor

Pulmonary arterial LIQ-861 treprostinil Liquidia Technologies prostacyclin analog INH InTrial 4Q2020 Yes No hypertension

ofatumumab (OMB-157) ofatumumab Novartis CD20 monoclonal antibody Multiple sclerosis SC InTrial 4Q2020 Yes No

Infacort hydrocortisone Diurnal Group corticosteroid Adrenal insufficiency PO InTrial 4Q2020 No Yes

gonadotropin-releasing Myovant Sciences/ Roivant Uterine fibroids/ TAK-385 TAK-385 hormone (GnRH) receptor PO InTrial 4Q2020 Yes No Sciences/ Takeda Endometriosis antagonist

tramadol tramadol Avenue Therapeutics opioid receptor agonist Pain IV InTrial 4Q2020 No No

CastiaRx.com // p: 866.516.2121 9 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

dehydrated human Plantar fasciitis/ Achilles AmnioFix amnion/chorion MiMedx amniotic tissue membrane INJ InTrial 4Q2020 Yes No tendonitis/ Osteoarthritis membrane (dHACM)

Estelle estetrol/ drospirenone Mithra/ Fuji/ Zhejian Xianju estrogen receptor agonist Pregnancy prevention PO/SL InTrial 4Q2020 No No

Qtrypta zolmitriptan Zosano triptans Acute migraines TOP InTrial 4Q2020 No No

selective Fibrodysplasia ossificans R-667 (RG-667) Clementia/ Roche receptor agonist (RAR- PO InTrial 4Q2020 Yes Yes progressiva (FOP) gamma)

dopamine receptor Schizophrenia/ Bipolar ALKS-3831 / samidorphan Alkermes antagonist/ opioid PO InTrial 4Q2020 No No disorder receptor antagonist

REGN-EB3 REGN-EB3 Regeneron anti-Ebola virus Ebola IV InTrial 4Q2020 Yes Yes

hypoxia-inducible factor FG-4592 roxadustat FibroGen/ AstraZeneca prolyl hydroxylase Anemia PO InTrial 2H2020 Yes No (HIF-PHI)

Laboratorios Doria risperidone atypical antipsychotic Schizophrenia IM InTrial 2H2020 Yes No Farmacéuticos Rovi

Acute respiratory distress Traumakine -beta - 1a Faron/ Maruishi interferon IV InTrial 2H2020 Yes No syndrome

NX-1207 (NYM-4805, REC Benign prostatic fexapotide triflutate Nymox pro-apoptotic Intratumoral InTrial 2H2020 Yes No 0482) hyperplasia

CastiaRx.com // p: 866.516.2121 10 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

chimeric antigen receptor bb-2121 idecabtagene vicluecel Celgene/ Bluebird Bio Multiple myeloma IV InTrial 2H2020 Yes Yes (CAR) T cell therapy

131I-8H9 omburtamab Y-mAbs Therapeutics B7-H3 antagonist Brain cancer Undisclosed InTrial 2H2020 Yes Yes

IdeS imlifidase Hansa Medical bacterial enzyme Kidney transplant IV InTrial 2H2020 Yes Yes

Otsuka/ Astex nucleoside metabolic ASTX-727 decitabine and E- 7727 Myelodysplastic syndrome PO InTrial 2H2020 Yes Yes Pharmaceuticals inhibitor

Sci-B-Vac hepatitis B vaccine VBI vaccine Hepatitis B IM InTrial 2H2020 No No

LJPC-0118 LJPC-0118 La Jolla Pharmaceutical protozoacide Malaria Undisclosed InTrial 2H2020 No No

BMS-663068 fostemsavir Bristol-Myers Squibb HIV attachment inhibitor HIV PO InTrial 2H2020 Yes No

Zeftera ceftobiprole Basilea cephalosporin antibiotic Bacterial infections IV InTrial 2H2020 Yes No

Aromatic L-amino acid AGIL-AADC AGIL-AADC Agilis Biotherapeutics gene therapy Intracerebral InTrial 2H2020 Yes Yes decarboxylase deficiency

chimeric antigen receptor KTE-X19 KTE-X19 Gilead Mantle cell lymphoma IV InTrial 2H2020 Yes Yes (CAR) T cell therapy

CLS-1001 triamcinolone acetonide Clearside corticosteroid Macular edema intraocular/ subretinal CRL 2H2020 Yes No

Allergic conjunctivitis/ Dry EM-100 ketotifen Eton antihistamine OP CRL 2H2020 No No eyes

HER2 oncoprotein MAGH-22 margetuximab MacroGenics/ Green Cross Breast cancer IV InTrial 2H2020 Yes No antagonist

CastiaRx.com // p: 866.516.2121 11 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

KPI-121 0.25% loteprednol etabonate Kala corticosteroid Dry eyes OP CRL 2H2020 No No

PharmaEssentia/ AOP ropeginterfero n alfa-2b ropeginterferon alfa-2b interferon Polycythemia vera SC InTrial 2H2020 Yes Yes Orphan

D-threo- methylphenidate Attention deficit KP-415 KemPharm CNS stimulant PO InTrial 2H2020 No No controlled-release hyperactivity disorder

iodine I 131 monoclonal anti-CD45 monoclonal Acute myeloid leukemia/ Iomab-B Actinium IV InTrial 2H2020 Yes Yes antibody BC8 antibody Myelodysplastic syndrome

FLT-3 receptor tyrosine quizartinib Daiichi Sankyo Acute myeloid leukemia PO CRL 2H2020 Yes Yes kinase inhibitor

dexamethasone phosphate EGP-437 EyeGate corticosteroid Uveitis OP InTrial 2H2020 Yes No (iontophoretic)

Libervant diazepam Aquestive Therapeutics benzodiazepine Seizures SL InTrial 2H2020 No Yes

TRV-130 oliceridine Trevena opioid receptor agonist Pain IV CRL 2H2020 No No

Niemann-Pick Disease/ Sporadic Inclusion Body arimoclomol arimoclomol Orphazyme cytoprotectives PO InTrial 2H2020 Yes Yes Myositis/ Amyotrophic lateral sclerosis

Mycapssa (Octreolin) octreotide Chiasma somatostatin analog Acromegaly PO CRL 2H2020 Yes Yes

End stage renal diease/ human elastase Peripheral artery disease/ PRT-201 vonapanitase Proteon Therapeutics TOP InTrial 2H2020 Yes Yes (recombinant) Vascular access in hemodialysis

PRX-102 alpha galactosidase Protalix enzyme replacement Fabry disease IV InTrial 2H2020 Yes No

CastiaRx.com // p: 866.516.2121 12 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

INCB-028060 (INC-280) capmatinib Novartis/ Incyte cMET inhibitor Non-small cell lung cancer PO InTrial 2H2020 Yes Yes

ET-103 levothyroxine Eton Pharmaceuticals L-thyroxine Hypothyroidism PO InTrial 2H2020 No No

anti-ECAM exotoxin A Vicinium (VB-4- 845) oportuzumab monatox Eleven Biotherapeutics Bladder cancer Intravesical InTrial 2H2020 Yes No fusion protein

RT-002 daxibotulinumtoxi nA Revance Therapeutics botulinum toxins Glabellar lines (frown lines) IM InTrial 2H2020 Yes Yes

nonsteroidal anti- N-1539 meloxicam Recro Pharma/ Alkermes Pain IV CRL 2020 Yes No inflammatory drug (NSAID)

indacaterol maleate/ long-acting beta 2 agonist/ QMF-149 Novartis/ Merck Asthma INH InTrial 2020 No No mometasone furoate corticosteroid

dopamine precursor/ dopa- ND-0612L levodopa/ carbidopa NeuroDerm Parkinson's disease SC InTrial 2020 Yes No decarboxylase inhibitor

ornithine decarboxylase Familial adenomatous CPP-1X/ Cancer Prevention inhibitor/ non-steroidal eflornithine/ sulindac polyposis/ Colorectal PO InTrial 2020 Yes Yes sulindac (DFMO) Pharma/ Zeria anti-inflammatory drug cancer (NSAID)

Zalviso sufentanil, ARX-01 AcelRx opioid analgesic Pain SL CRL 2020 No No

mycolic acid biosynthesis Deltyba delamanid Otsuka Tuberculosis PO InTrial 2020 No No inhibitor

Pedmark (STS) sodium thiosulfate Fennec reducing agent Hearing loss IV InTrial 2020 Yes Yes

dopamine precursor/ dopa- ND-0612H levodopa/ carbidopa NeuroDerm Parkinson's disease SC InTrial 2020 Yes No decarboxylase inhibitor

DS-200 DS-200 Eton undisclosed Ophthalmological disease SC InTrial 2020 No No

CastiaRx.com // p: 866.516.2121 13 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

ELI-200 oxycodone/ naltrexone Elite opioid agonist Pain PO CRL 2020 No No

astodrimer sodium (SPL- VivaGel Starpharma viral attachment inhibitor Bacterial infections VG CRL 2020 No No 7013) Blepharitis/ Ocular Dexasite dexamethasone InSite Vision corticosteroid TOP InTrial 2020 No No inflammation Chronic lymphocytic ublituximab (LFB-R603, leukemia/ Small cell TG20, TGTX- 1101, TG- CD-20 monoclonal ublituximab TG Therapeutics lymphocytic lymphoma/ IV InTrial 2020 Yes Yes 1101, antibody Mantle cell lymphoma/ Utuxin) Multiple sclerosis

CCP-08 CCP-08 Tris Pharma undisclosed Viral rhinitis PO CRL 2020 No No

autologous chondrocyte autologous chondrocyte NeoCart Histogenics/ Purpose Joint repair Undisclosed InTrial 2020 Yes No tissue implant tissue implant

APN-311 dinutuximab beta EUSA Pharma disialoganglioside Neuroblastoma SC InTrial 2020 Yes Yes

alpha-adrenergic Benign prostatic tamsulosin DRS tamsulosin delayed-release Veru PO InTrial 2020 No No antagonist hyperplasia

Oralair Mites (Actair) dust mite peptide Stallergenes/ Shionogi vaccine Dust mite allergic rhinitis SL InTrial 2020 Yes No

Travivo gepirone ER GSK/Fabre-Kramer 5-HT-1A receptor agonist Major depressive disorder PO CRL 2020 No No

MNK-812 oxycodone Mallinckrodt opioid agonist Pain PO CRL 2020 No No

Sumitomo Dainippon/ non-ergoline dopamine APL-130277 apomorphine Parkinson's disease SL CRL 2020 No No MonoSol Rx/ Sunovion agonist

phosphodiesterase-5 (PDE- tadalafil VersaFilm tadalafil VersaFilm IntelGenx Erectile dysfunction PO Filed NDA 2020 Yes No 5) inhibitor

CastiaRx.com // p: 866.516.2121 14 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Leukocyte Interleukin (CS- Head and Neck cancer/ Multikine CEL-SCI immunomodulator SC InTrial 2020 Yes Yes 001P3) Squamous cell carcinoma

phosphodiesterase-5 (PDE- APC-8000 tadalafil Adamis Erectile dysfunction PO CRL 2020 Yes No 5) inhibitor

Corplex donepezil donepezil Corium International anticholinergic Alzheimer's disease TOP InTrial Late 2020 No No

Acute myeloid leukemia/ CC-486 azacitidine Celgene DNA methylation inhibitor Myelodysplastic PO InTrial Late 2020 Yes Yes syndromes

Non-cystic fibrosis Linhaliq ciprofloxacin Aradigm/ Grifols fluoroquinolone bronchiectasis/ Cystic INH CRL Late 2020 Yes Yes fibrosis

Acute myeloid leukemia/ Nedd 8 Activating Enzyme Chronic myelogenous MLN-4924 (TAK-92) pevonedistat Takeda PO InTrial Late 2020 Yes No (NAE) antagonist leukemia/ Myelodysplastic syndrome

neurotrophic tyrosine kinase receptor type 1 tanezumab tanezumab Pfizer/ Eli Lilly Osteoarthritis/ Pain IV/SC InTrial Late 2020 Yes No (TrkA) antagonist (monoclonal antibody)

complement C1s BIVV-009 (TNT- 009) sutimlimab Sanofi Cold agglutinin disease IV InTrial Late 2020 Yes Yes subcomponent inhibitor

Orphan Therapeutics/ Lucassin V-1 () agonist Hepato-renal syndrome IV CRL Late 2020 Yes Yes Ikaria

tetrahydrofolate iclaprim iclaprim Motif Bio Bacterial infections IV CRL Late 2020 Yes Yes dehydrogenase inhibitor

CastiaRx.com // p: 866.516.2121 15 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

morpholino antisense Duchenne muscular SRP-4053 Sarepta IV CRL Late 2020 Yes Yes oligonucleotide dystrophy

NS-2 (ALDX- 1E1, ALDX-1E2, Allergic conjunctivitis/ Dry reproxalap Aldeyra Therapeutics aldehyde antagonist OP InTrial Late 2020 No No ADX-102) eyes

Ryplazim human plasminogen ProMetic/ Hematech plasminogen Plasminogen deficiency IV CRL Late 2020 Yes Yes

Hematopoietic stem cell anti-MASP-2 monoclonal transplant- associated OMS-721 narsoplimab Omeros IV/SC InTrial Late 2020 Yes Yes antibody thrombotic microangiopathy

Cerebral adreno- Lenti-D elivaldogene tavalentivec Bluebird Bio gene therapy IV InTrial Late 2020 Yes Yes myeloneuropathy

CD-20 monoclonal Chronic lymphocytic antibody/ leukemia/ Diffuse large B- TG-1303 ublituximab/ TGR- 1202 TG Therapeutics IV/PO InTrial Late 2020 Yes Yes phosphoinositide-3 kinase cell lymphoma/ Non- (PI3K) delta inhibitor Hodgkin lymphoma

MVA-MUC1- IL2 TG-4010 Transgene vaccine Non-small cell lung cancer SC InTrial Late 2020 No No

SHP-621 budesonide Shire corticosteroid Eosinophilic esophagitis PO InTrial Late 2020 Yes Yes

HuMax-TF ADC tisotumab vedotin Genmab/ Seattle Genetics tissue factor antibody Solid tumors Undisclosed InTrial Late 2020 Yes No

C-type natriuretic peptide BMN-111 vosoritide (vasoritide) BioMarin/ Chugai Achondroplasia SC InTrial Late 2020 Yes Yes (CNP) analog

TSR-042 dostarlimab AnaptysBio PD-1 checkpoint inhibitor Endometrial cancer IV InTrial Late 2020 Yes No

sodium oxybate extended- JZP-258 Jazz dopamine receptor agonist Narcolepsy PO InTrial Late 2020 Yes No release

gene Duchenne muscular Translarna PTC Therapeutics PO CRL Late 2020 Yes Yes modulator dystrophy

CastiaRx.com // p: 866.516.2121 16 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

corticosteroid/ long- acting budesonide/ muscarinic receptor Chronic obstructive BGF-MDI (PT- 010) glycopyrronium/ AstraZeneca antagonist (LAMA)/ long- INH CRL Late 2020 No No pulmonary disease formoterol acting beta 2 adrenergic receptor agonist (LABA)

HM-30181A/ P-glycoprotein pump Oraxol Athenex Breast cancer PO InTrial Late 2020 Yes Yes paclitaxel inhibitor/ taxane

melanocortin 4 receptor Rare genetic disorders of BIM-22493 (RM-493) setmelanotide Rhythm/ Camurus/ Ipsen SC InTrial Late 2020 Yes Yes (MC4R) agonist obesity

sphingosine 1 phosphate RG-3477 (ACT- 128800) ponesimod Johnson & Johnson Multiple sclerosis PO InTrial Late 2020 Yes No receptor agonist

2021 Possible Launch Date

glycolate oxidase ALNG-01 (ALN- G-01) lumasiran Alnylam Hyperoxaluria Intranasal InTrial 1Q2021 Yes Yes antagonist

MK-4618 (KRP- 114V, RVT- Roivant Sciences/ Urovant/ selective beta 3 adrenergic vibegron Overactive bladder PO InTrial 1Q2021 No No 901) Kissei/ Kyorin/ Merck receptor agonist

amyloid beta-protein BIIB-037 aducanumab Biogen Idec/ Eisai Alzheimer's disease IV InTrial 1Q2021 Yes No inhibitor

Src kinase and tubulin KX-01 (KX2- 391) tirbanibulin Athenex Actinic keratosis PO InTrial 1Q2021 Yes No inhibitor

ZP-4207 (ZP- GA-1) dasiglucagon Zealand Pharma glucagon analog Diabetes mellitus SC InTrial 1Q2021 No Yes

Furoscix furosemide scPharmaceuticals diuretic Heart failure SC CRL 1Q2021 Yes No

Sun Pharma Advanced SDP-037, SDN- 037 SDP-037, SDN-037 Research Company Corticosteroid Ocular inflammation/pain OP InTrial 2Q2021 No No (SPARC)

CastiaRx.com // p: 866.516.2121 17 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

SCY-078 ibrexafungerp Scynexis glucan synthase inhibitors Fungal infections IV/PO InTrial 1H2021 No Yes

Short stature/ growth ACP-001 TransCon Growth Hormone Ascendis growth hormone prodrug SC InTrial 1H2021 Yes No hormone deficiency

RGN-259 (GBT- 201; RGN- Neurotrophic keratitis/ Dry thymosin beta 4 RegeneRx actin regulating peptide OP InTrial 1H2021 No Yes 352) eyes

protein absorption CM-AT CM-AT Curemark Autism PO InTrial 1H2021 Yes No enhancer

selective peroxisome Non-alcoholic GFT-505 elafibranor Genfit proliferator-activated steatohepatitis/ Primary PO InTrial 1H2021 Yes No receptor (PPAR) modulator biliary cirrhosis

MediWound/ BL&H/ peptide hydrolase NexoBrid Burns/ Skin injury TOP InTrial 1H2021 No Yes CrystalGenomics/ Kaken replacement agent

PDR-001 PDR-001 Novartis PD-1 checkpoint inhibitor Melanoma IV InTrial 1H2021 Yes No

C5a receptor (C5aR) CCX-168 avacopan ChemoCentryx/ Galencia Vasculitis PO InTrial 1H2021 Yes Yes antagonist

StrataGraft Skin Tissue StrataGraft Skin Tissue Mallinckrodt autologous skin tissue Burn injury TOP InTrial 1H2021 Yes Yes

tumor vascular disrupting NPI-2358 plinabulin BeyondSpring Neutropenia IV InTrial 1H2021 Yes No agent

Niemann-Pick disease type GZ-402665 olipudase alfa Sanofi sphingomyelinase IV InTrial Mid-2021 Yes Yes B

X-linked myotubular AT-132 (AAV8- MTM1) AT-132 (AAV8- MTM1) Audentes Therapeutics gene therapy IV InTrial Mid-2021 Yes Yes myopathy

GZ-402666 (NeoGAA) avalglucosidase alfa Sanofi enzyme therapy Pompe disease IV InTrial Mid-2021 Yes No

CastiaRx.com // p: 866.516.2121 18 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Atara Biotherapeutics/ Lymphoproliferative EBV-CTL (ATA- 129) tabelecleucel Memorial Sloan- Kettering cell therapy IV InTrial Mid-2021 Yes Yes disorder Cancer Center

respiratory syncytial virus Respiratory syncytial virus RSV-F (ResVax) Novavax vaccine IM InTrial Mid-2021 Yes No vaccine infection

histone deacetylase entinostat entinostat Syndax Breast cancer PO InTrial Mid-2021 Yes No (HDAC) inhibitor

Diffuse large B-cell ADCT-402 loncastuximab tesirine ADC Therapeutics antibody drug conjugate IV InTrial Mid-2021 Yes Yes lymphoma

phosphodiesterase type 5 Benign prostatic TadFin tadalafil and finasteride Veru inhibitor /5-alpha- PO InTrial Mid-2021 No No hyperplasia reductase inhibitor

non-steroidal anti- AXS-07 meloxicam/rizatri ptan Axsome Therapeutics Migraine PO InTrial Mid-2021 No No inflammatory drug/triptan

ALN-APC (ALN- AT3) fitusiran Alnylam/ Sanofi RNAi therapeutic Hemophilia SC InTrial Mid-2021 Yes Yes

Duchenne muscular WVE-210201 suvodirsen Wave Life Sciences oligonucleotide IV InTrial Mid-2021 Yes Yes dystrophy

Sun Pharma Advanced PDP-716 brimonidine Research Company alpha-2 agonist Glaucoma OP InTrial 3Q2021 No No (SPARC)

MacroGenics/ Provention PRV-031 teplizumab CD3 antigen inhibitor Diabetes mellitus IV InTrial 4Q2021 Yes Yes Bio

C-C chemokine receptor 5 Tobira Therapeutics/ Non-alcoholic TBR-652 cenicriviroc (CCR5) and receptor 2 PO InTrial 4Q2021 Yes No Takeda steatohepatitis antagonist

CastiaRx.com // p: 866.516.2121 19 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

selective alpha 2a receptor Schizophrenia and bipolar BXCL-501 dexmedetomidine BioXcel Therapeutics PO InTrial 2H2021 No No agonist disorder

Psoriasis/ Psoriatic interleukin-17 (IL-17) arthritis/ Ankylosing UCB-4940 (CDP-4940) bimekizumab UCB IV InTrial 2H2021 Yes No receptor inhibitor spondylitis/ Rheumatoid arthritis

177Lu-PSMA- Lutetium Endocyte Radiopharmaceutical Prostate cancer IV InTrial 2H2021 Yes No 617

nicotinic acetylcholine OS-01 OC-01 Oyster Point Pharma Dry eye disease Intranasal InTrial 2H2021 No No receptor (nAChR) agonist

SHP-647 SHP-647 Shire MAdCAM-1 antagonist Irritable bowel disease IV/SC InTrial 2H2021 Yes Yes

IDP-124 pimecrolimus Bausch Health calcineurin Inhibitor Atopic dermatitis TOP InTrial 2H2021 No No

long- glatiramer acetate depot Mylan/ Mapi Pharma immunosuppressant Multiple sclerosis IM InTrial 2H2021 Yes No acting

ROCK2 (Rho-associated KD-025 KD-025 Kadmon coiled-coiled kinase 2) Graft vs. Host disease PO InTrial 2H2021 No Yes inhibitor

dextromethorpha n/ N-methyl-D-aspartate Treatment-resistant AXS-05 Axsome PO InTrial 2H2021 No No bupropion (NMDA) antagonist depression

dexamethasone sustained- Otividex Otonomy corticosteroid Meniere's disease Intratympanic InTrial 2H2021 Yes No release

interferon receptor Systemic lupus MEDI-546 AstraZeneca/ BMS IV InTrial 2H2021 Yes No antagonist erythematosus

CastiaRx.com // p: 866.516.2121 20 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

tumor infiltrating LN-145 lifileucel Iovance Biotherapeutics Cervical Cancer IV InTrial 2H2021 Yes No lymphocyte

POD- INP-104 dihydroergotamin e Impel/ 3M ergot derivative Acute migraines Intranasal InTrial 2H2021 No No mesylate (POD- DHE)

Attention deficit SPN-810 molindone Supernus atypical antipsychotic PO InTrial 2H2021 No No hyperactivity disorder

GS-010 GS-010 GenSight Biologics gene therapy Optic neuropathy Intraocular InTrial 2H2021 Yes Yes

TWIN (S6G5T- 1; S6G5T-3) benzoyl peroxide/ Sol-Gel Technologies retinoid Acne vulgaris TOP InTrial 2H2021 No No

digitalis-like factor AMAG-423 digoxin immune fab (DIF) AMAG/ Velo Preeclampsia IV InTrial 2H2021 Yes Yes antagonist

CR-845 difelikefalin Cara Therapeutics opioid receptor agonist Pruritus IV/PO InTrial 2H2021 No No

pIL-12 tavokinogene telsaplasmid OncoSec Medical gene therapy Melanoma Intratumoral InTrial 2H2021 Yes Yes

MD-1003 MD-1003 MedDay biotin Multiple sclerosis PO InTrial 2H2021 Yes No

Duchenne muscular VBP-15 vamorolone Santhera corticosteroid PO InTrial 2H2021 Yes Yes dystrophy

ZYN-002 ZYN-002 Zynerba cannabinoid product Fragile X syndrome TOP InTrial 2H2021 Yes Yes

Calliditas/ Kyowa Hakko PL-56 budesonide corticosteroid Nephropathy PO InTrial 2H2021 No No Kirin

fibroblast growth factor dovitinib dovitinib Oncology Venture receptor 3 (FGFR3) Renal cell carcinoma PO InTrial 2H2021 Yes No inhibitor

CastiaRx.com // p: 866.516.2121 21 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

alpha-2/beta-1 adrenergic vascular Ehlers-Danlos Edsivo celiprolol HCl Acer Therapeutics PO CRL 2021 Yes Yes agent Syndrome

Tlando testosterone Lipocine androgen Hypogonadism PO CRL 2021 No No

PRO-145223 etrolizumab IgG1 monoclonal antibody Ulcerative colitis SC InTrial 2021 Yes No

chemokine (CXCR4) POL-6326 balixafortide Polyphor Transplant/ Breast cancer IV InTrial 2021 Yes No antagonist

Duchenne muscular idebenone idebenone Santhera co-enzyme Q-10 analog PO InTrial 2021 Yes Yes dystrophy

Chronic myelogenous ABL-001 asciminib Novartis allosteric Bcr-Abl inhibitor PO InTrial 2021 Yes Yes leukemia

PPP-002 PPP-002 Tetra Bio-Pharma botanical drug Pain Undisclosed InTrial 2021 No No

sodium-dependent glucose transporter 1 (SGLT-1) and Zynquista sotagliflozin Sanofi/ Lexicon Diabetes mellitus PO CRL 2021 No No SGLT-2 inhibitor

JNJ-3872 (VX- 787) pimodivir Johnson & Johnson/ Vertex viral protein inhibitor Influenza PO InTrial 2021 No No

V1A RG-7314 (RO- 5285119) balovaptan Roche Autism spectrum disorder PO InTrial 2021 Yes No antagonist

HER2/neu receptor RG-6264 trastuzumab/ pertuzumab Roche Breast cancer SC InTrial 2021 Yes No antagonist

toll-like receptor 9 (TLR- IMO-2125 tilsotolimod Idera Melanoma SC/ intratumoral InTrial 2021 Yes Yes 9) agonist

CastiaRx.com // p: 866.516.2121 22 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

ARYx Therapeutics/ vitamin K epoxide ATI-5923 tecarfarin Anticoagulation PO InTrial 2021 No No Armetheon reductase

Acute myelogenous RG-7388 (RO- 5503781) idasanutlin Roche MDM2 antagonist PO InTrial 2021 Yes No leukemia

brincidofovir DNA-directed DNA Adenovirus/ CMX-001 Chimerix PO InTrial 2021 No Yes hexadecyloxyprop yl ester polymerase inhibitor Cytomegalovirus/ Smallpox

neurokinin 1 receptor (NK- LY-686017 tradipitant Vanda Pharmaceuticals Motion sickness PO InTrial 2021 No No 1R) antagonist

Prostate cancer; breast RG-7440 ipatasertib Roche pan-Akt inhibitor PO InTrial 2021 Yes No cancer

c-Met receptor tyrosine EMD-1214063 tepotinib Merck Non-small cell lung cancer PO InTrial 2021 Yes No kinase inhibitor

RTA-408 omaveloxolone Reata Pharmaceuticals Nrf2 activator Friedreich's ataxia PO InTrial 2021 Yes Yes

vascular endothelial Recentin cediranib AstraZeneca growth factor receptor Ovarian cancer PO InTrial 2021 Yes Yes (VEGF) antagonists

Growth hormone MOD-401 somatrogon OPKO Health/ Pfizer enzyme replacement SC InTrial 2021 Yes Yes deficiency

DS-100 DS-100 Eton undisclosed Ophthalmological disease SC InTrial 2021 No No

S5G4T-1 (DER- 45-EV) benzoyl peroxide Sol-Gel Technologies benzoyl peroxide Rosacea TOP InTrial 2021 No No

ADA-transduced Adenosine deaminase- OTL-101 autologous stem cell Orchard Therapeutics gene therapy deficient severe combined Undisclosed InTrial 2021 Yes Yes therapy immunodeficiency

CastiaRx.com // p: 866.516.2121 23 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

Duchenne muscular CAT-1004 edasalonexent Catabasis NF-kB inhibitor PO InTrial Late 2021 Yes Yes dystrophy

wet age-related macular ONS-5010 bevacizumab Outlook Therapeutics anti-VEGF antibody Intravitreal InTrial Late 2021 Yes No degeneration

multivalent group B PF-06482077 Pfizer vaccine Bacterial infection IM InTrial Late 2021 Yes No streptococcus vaccine

angiopoietin-like 3 REGN-1500 evinacumab Regeneron Hyperlipidemia IV/SC InTrial Late 2021 Yes No (ANGPTL3) antagonist

etranacogene AMT-061 uniQure gene therapy Hemophilia B IV InTrial Late 2021 Yes No dezaparvovec

Anti-VEGF (vascular Wet age-related macular PDS-1.0 ranibizumab Roche/ Genentech Intravitreal implant InTrial Late 2021 Yes No endothelial growth factor) degeneration

End-stage renal disease/ Humacyl human acellular vessel Humacyte cellular therapy Implant InTrial Late 2021 Yes No Peripheral artery disease

insulin-like growth factor 1 Rett syndrome/ Fragile X NNZ-2566 trofinetide Neuren IV/PO InTrial Late 2021 Yes Yes (IGF-1) derivative syndrome/ Brain injury

MK-0594 (VPD- 737) serlopitant Menlo NK-1 receptor antagonist Pruritus PO InTrial Late 2021 Yes No

chemoattractant receptor- QAW-039 (NVP-QAW- 039) fevipiprant Novartis homologous molecule Asthma PO InTrial Late 2021 Yes No (CRTH2) antagonist

CastiaRx.com // p: 866.516.2121 24 Brand Pipeline Forecast 4th Quarter 2019

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug

tripotassium citrate Distal rental tubular ADV-7103 monohydrate/ potassium Advicenne Undisclosed PO InTrial Late 2021 Yes No acidosis hydrogen carbonate

PW-4142 (T- 111) nalbuphine ER Trevi Therapeutics/ Endo opioid agonist/ antagonist Prurigo nodularis PO InTrial Late 2021 No No

GSK-2696274 (OTL-200) GSK-2696274 (OTL-200) GlaxoSmithKline gene therapy Leukodystrophy IV InTrial Late 2021 Yes Yes

paroxysmal nocturnal Ultomiris SC ravulizumab-cwvz Alexion C5 complement inhibitor hemoglobinuria; Hemolytic SC InTrial Late 2021 Yes Yes uremic syndrome

1 (JAK-1) PF-04965842 Pfizer Atopic dermatitis PO InTrial Late 2021 Yes No inhibitor

COR-003 levoketoconazole Strongbridge azole antifungal Cushing's syndrome PO InTrial Late 2021 No Yes

hypoxia-inducible factor- AKB-6548 vadadustat Akebia Therapeutics prolyl hydroxylase (HIF- Anemia PO InTrial Late 2021 Yes No PH) inhibitor

IM = intramuscular, INH = inhalation, INJ = injection, IUD = intrauterine device, IV = intravenous, OP = ophthalmic, PO = oral, SC = subcutaneous, SL = sublingual, SPR = spray, TOP = topical, VG = vaginal, NSCLC = Non-small cell lung cancer, COPD = Chronic Obstructive Pulmonary Disease

CastiaRx.com // p: 866.516.2121 25