Brand Pipeline Forecast 1st Quarter 2020

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

2020 Possible Launch Date

gene-related BMS-927711 (BHV-3000) sulfate Biohaven (CGRP) Migraine PO Filed NDA 2/2020 No No antagonist

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

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

gonadotropin-releasing FP-001 (LMIS) leuprolide mesylate Foresee Prostate cancer SC Filed NDA 2/2020 Yes No hormone (GnRH) analog

ITCA-650 sustained- -like peptide-1 (sustained release Intarcia Diabetes mellitus SC implant Filed NDA 3/9/2020 No No release (GLP-1) receptor agonist exenatide) naloxone nasal spray naloxone Insys Therapeutics antagonist Opioid dependence Intranasal Filed NDA 3/15/2020 No No

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

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

serotonin receptor Fintepla fenfluramine Zogenix Dravet syndrome PO Filed NDA 3/25/2020 Yes Yes agonist

Rizaport rizatriptan IntelGenx triptan Acute migraines PO Filed NDA 3/26/2020 No No

CastiaRx.com // p: 866.516.2121 1 Brand Pipeline Forecast 1st Quarter 2020

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

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

injection Sun Pharma Advanced Breast Cancer; Lung SPARC-1028, SPARC- concentrate for Research Company Cancer; Pancreatic IV Filed NDA 1Q2020 No No 1210, SPARC-1023 suspension (SPARC) Cancer

Aximris XR oxycodone ER IntelliPharmaCeutics opioid agonist Pain PO Filed NDA 1Q2020 No No

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

non-steroid anti- 3/2020 - E-58425 / Esteve inflammatory Acute pain PO Filed NDA No No 4/2020 drug/opioid bimatoprost sustained bimatoprost sustained Allergan prostaglandin agonist Glaucoma Implant Filed NDA 4/1/2020 No No release release

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

MitoGel UroGen alkylating agent Urothelial cancer Intravesical Filed NDA 4/18/2020 No Yes

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

CastiaRx.com // p: 866.516.2121 2 Brand Pipeline Forecast 1st Quarter 2020

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

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

Pulmonary arterial Trevyent treprostinil United Therapeutics prostacyclin analog SC Filed NDA 4/27/2020 Yes Yes hypertension isatuximab isatuximab Sanofi CD38 antagonist Multiple myeloma IV Filed BLA 4/30/2020 Yes Yes

selective MEK kinase 4/2020 to selumetinib selumetinib AstraZeneca/ Merck Neurofibromatosis PO Filed NDA Yes Yes inhibitor 5/2020

SEP-225289 dasotraline Sunovion triple reuptake inhibitor PO Filed NDA 5/14/2020 No No

non-ergoline dopamine APL-130277 apomorphine Sunovion Parkinson's disease PO Filed NDA 5/21/2020 No No agonist

Roche/ PTC RG-7916 (RO- 7034067) risdiplam SMN2 splicing modifier Spinal muscular atrophy PO Filed NDA 5/24/2020 Yes Yes Therapeutics

Amphora Amphora Evofem Biosciences spermicidal agent Pregnancy prevention VG Filed NDA 5/25/2020 No No

selective FGFR1/2/3 INCB-54828 pemigatinib Incyte Biliary tract cancer PO Filed NDA 5/30/2020 Yes Yes inhibitor nadofaragene nadofaragene 5/2020 - Ferring Pharmaceuticals gene therapy Bladder cancer Intravesical Filed BLA Yes No firadenovec firadenovec 6/2020

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

RS7-SN-38 antibody- IMMU-132 sacituzumab govitecan Immunomedics Breast cancer IV Filed BLA 6/2/2020 Yes No drug conjugate

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

CastiaRx.com // p: 866.516.2121 3 Brand Pipeline Forecast 1st Quarter 2020

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

EM-100 ketotifen Eton Allergic conjunctivitis OP Filed NDA 6/11/2020 No No

Contepo fosfomycin Nabriva Therapeutics cell wall inhibitor Bacterial infections IV Filed NDA 6/19/2020 Yes No

EVK-001 Evoke Pharma antidopiminergics Diabetic gastroparesis Intranasal Filed NDA 6/19/2020 No No

Intercept farnesoid X receptor Nonalcoholic obeticholic acid obeticholic acid PO Filed NDA 6/26/2020 Yes No Pharmaceuticals (FXR) agonist steatohepatitis

Mycapssa (Octreolin) octreotide Chiasma somatostatin analog Acromegaly PO Filed NDA 6/2020 Yes Yes

CD20 monoclonal OMB-157 ofatumumab Novartis Multiple sclerosis SC Filed BLA 6/2020 Yes No antibody

osmotic gradient Bronchitol mannitol Pharmaxis enhancer; mucus Cystic fibrosis INH Filed NDA 2Q2020 No Yes clearance enhancer glargine Mylan/ Biocon Long-acting insulin Diabetes mellitus SC CRL Mid-2020 No No

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

Neuromyelitis optica 6/2020 – MEDI-551 inebilizumab Viela Bio CD-19 antagonist IV Filed BLA Yes Yes spectrum disorder 7/2020 nonsteroidal anti- 6/2020 – DFN-15 celecoxib Dr. Reddy inflammatory drug Migraine PO Filed NDA No No 7/2020 (NSAID) collagenase clostridium collagenase clostridium Endo protease enzyme Cellulite SC Filed BLA 7/6/2020 Yes No histolyticum histolyticum

CastiaRx.com // p: 866.516.2121 4 Brand Pipeline Forecast 1st Quarter 2020

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

daratumumab (with humanized anti-CD38 Darzalex recombinant human Janssen Multiple myeloma SC Filed BLA 7/12/2020 Yes Yes monoclonal antibody hyaluronidase)

VP-102 VP-102 Verrica antiviral Molluscum TOP Filed NDA 7/13/2020 No No

Osmotica/ Vertical alpha-adrenergic Acquired blepharoptosis RVL-1201 oxymetazoline OP Filed NDA 7/16/2020 No No Pharmaceuticals receptor agonist (droopy eyelid)

calcipotriene/ vitamin D analog/ MC2-01 MC2 Therapeutics Psoriasis TOP Filed NDA 7/20/2020 No No betamethasone corticosteroid

sodium oxybate dopamine receptor JZP-258 Jazz Narcolepsy PO Filed NDA 7/21/2020 Yes Yes extended-release agonist

acetylcholinesterase Corplex donepezil donepezil Corium International Alzheimer's disease TOP Filed NDA 7/30/2020 No No inhibitor

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

Non-small cell lung 7/2020 - LOXO-292 selpercatinib Eli Lilly/ Loxo Oncology RET inhibitor PO Filed NDA Yes No cancer; thyroid cancer 8/2020

nucleoside metabolic Myelodysplastic 7/2020 - ASTX-727 and E- 7727 Astex Pharmaceuticals PO Filed NDA Yes Yes inhibitor syndrome 8/2020

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

BMS-663068 fostemsavir Bristol-Myers Squibb HIV attachment inhibitor HIV PO Filed NDA 8/5/2020 Yes No

CastiaRx.com // p: 866.516.2121 5 Brand Pipeline Forecast 1st Quarter 2020

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

chimeric antigen KTE-X19 KTE-X19 Gilead receptor (CAR) T cell IV Filed BLA 8/10/2020 Yes Yes therapy

TRV-130 oliceridine Trevena agonist Pain IV Filed NDA 8/10/2020 Yes No

BCX-7353 berotralstat BioCryst kallikrein inhibitor Hereditary angioedema PO Filed NDA 8/11/2020 Yes Yes

Non-small cell lung INCB-028060 (INC-280) capmatinib Novartis/ Incyte cMET inhibitor PO Filed NDA 8/11/2020 Yes Yes cancer

Pedmark (STS) sodium thiosulfate Fennec reducing agent Hearing loss IV Filed NDA 8/11/2020 Yes Yes

PDGFR-alpha kinase Gastrointestinal stromal DCC-2618 ripretinib Deciphera PO Filed NDA 8/14/2020 Yes Yes inhibitor tumors

GlaxoSmithKline/ Seattle anti-BCMA antibody- GSK-2857916 belantamab mafodotin Multiple myeloma SC Filed BLA 8/16/2020 Yes Yes Genetics drug conjugate

Zepsyre lurbinectidin PharmaMar alkylating agent Small cell lung cancer IV Filed NDA 8/16/2020 Yes Yes

janus associated kinase- GLPG-0634 filgotinib Gilead Rheumatoid arthritis PO Filed NDA 8/18/2020 Yes No 1 (JAK) inhibitor

chimeric antigen lisocabtagene Bristol-Myers Squibb/ Diffuse large B-cell JCAR-017 receptor (CAR) T cell IV Filed BLA 8/18/2020 Yes Yes maraleucel Celgene lymphoma therapy

HER2 oncoprotein MAGH-22 margetuximab MacroGenics Breast cancer IV Filed BLA 8/19/2020 Yes No antagonist

CastiaRx.com // p: 866.516.2121 6 Brand Pipeline Forecast 1st Quarter 2020

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

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

ErbB-2 (Her-2/neu) ARRY-380 (ONT-380) tucatinib Seattle Genetics Breast cancer PO Filed NDA 8/23/2020 Yes Yes inhibitor

valoctocogene BMN-270 BioMarin gene therapy Hemophilia A IV Filed BLA 8/23/2020 Yes Yes roxaparvovec

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

Diffuse large B-cell MOR-208 tafasitamab MorphoSys/ Xencor CD-19 antagonist IV Filed BLA 8/28/2020 Yes Yes lymphoma

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

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

LJPC-0118 artesunate La Jolla Pharmaceutical protozoacide Malaria Undisclosed Filed NDA 9/25/2020 No Yes

Libervant diazepam Aquestive Therapeutics benzodiazepine Seizures PO Filed NDA 9/27/2020 No Yes

Prochymal remestemcel-L Mesoblast mesenchymal stem cells Graft vs. Host disease IV Filed BLA 9/30/2020 Yes Yes

LY-900014 Eli Lilly Diabetes mellitus SC Filed BLA 3Q2020 No No

Infacort hydrocortisone Diurnal Group corticosteroid Adrenal insufficiency PO Filed NDA 10/2/2020 No Yes

CastiaRx.com // p: 866.516.2121 7 Brand Pipeline Forecast 1st Quarter 2020

Route of Estimated Release Specialty Drug Name Generic Name Company Drug Class Therapeutic Use Regulatory Status Orphan Drug Administration Date Drug tramadol tramadol Avenue Therapeutics opioid receptor agonist Pain IV Filed NDA 10/9/2020 No No

Qtrypta zolmitriptan Zosano triptan Acute migraines TOP Filed NDA 10/20/2020 No No

granulocyte colony- SPI-2012 eflapegrastim Spectrum/ Hanmi Neutropenia SC Filed BLA 10/24/2020 Yes No stimulating factor (GCSF)

selective norepinephrine Attention deficit SPN-812 Supernus PO Filed NDA 11/8/2020 No No reuptake inhibitor hyperactivity disorder

dopamine receptor olanzapine/ Schizophrenia/ Bipolar ALKS-3831 Alkermes antagonist/ opioid PO Filed NDA 11/15/2020 No No samidorphan disorder

Glabellar lines (frown RT-002 (Daxi) daxibotulinumtoxi nA Revance Therapeutics botulinum toxins IM Filed BLA 11/25/2020 Yes No lines)

Pulmonary arterial LIQ-861 treprostinil Liquidia Technologies prostacyclin analog INH Filed NDA 11/27/2020 Yes No hypertension

anti-parasitic, anti- nifurtimox nifurtimox Bayer Chagas disease PO Filed NDA 11/30/2020 No Yes protozoal

trastuzumab/ HER2/neu receptor 11/2020 - RG-6264 Roche Breast cancer SC Filed BLA Yes No pertuzumab antagonist 12/2020

hypoxia-inducible factor FG-4592 roxadustat FibroGen/ AstraZeneca prolyl hydroxylase (HIF- Anemia PO Filed NDA 12/23/2020 Yes No PHI)

CastiaRx.com // p: 866.516.2121 8 Brand Pipeline Forecast 1st Quarter 2020

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

serotonin- norepinephrine- Major depressive LY-03005 ansofaxine Luye Pharma PO Filed NDA 12/26/2020 No No dopamine triple disorder reuptake inhibitor

selective beta 3 MK-4618 (KRP- 114V, vibegron Urovant Sciences adrenergic receptor Overactive bladder PO Filed NDA 12/29/2020 No No RVT-901) agonist

siRNA targeting The Medicines proprotein convertase inclisiran inclisiran Hyperlipidemia SC Filed NDA 12/2020 Yes Yes Company/ Novartis subtilisin kexin type 9 (PCSK9)

nerve growth factor tanezumab tanezumab Pfizer/ Eli Lilly Osteoarthritis SC Filed BLA 12/2020 Yes No antibody

phosphoinositide-3 Marginal zone TGR-1202 umbralisib TG Therapeutics kinase (PI3K) delta lymphoma/ follicular PO InTrial 4Q2020 Yes Yes inhibitor lymphoma

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

C-C chemokine receptor PRO-140 leronlimab CytoDyn HIV SC InTrial 4Q2020 Yes Yes 5 (CCR5) antagonist

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

CastiaRx.com // p: 866.516.2121 9 Brand Pipeline Forecast 1st Quarter 2020

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

lentiviral beta- globin Zynteglo (LentiGlobin) Bluebird Bio gene therapy Beta-thalassemia IV InTrial 4Q2020 Yes Yes gene transfer

131I-8H9 omburtamab Y-mAbs Therapeutics B7-H3 antagonist Brain cancer Undisclosed InTrial 4Q2020 Yes Yes

3-F8 (Hu-3F8) naxitamab Y-mAbs Therapeutics GD2 antagonist Neuroblastoma IV InTrial 4Q2020 Yes Yes

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

mometasone furoate/ corticosteroid/ GSP-301 Glenmark Allergic rhinitis Intranasal CRL 2H2020 No No olopatadine HCl antihistamine

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

Hutchinson-Gilford Eiger Progeria Syndrome EBP-994 (rEBP- 994) lonafarnib prenylation inhibitor PO InTrial 2H2020 Yes Yes Biopharmaceuticals (HGPS or progeria) and progeroid laminopathies

HIV integrase inhibitor/ cabotegravir (long- non-nucleoside reverse TMC-278-LA acting)/ rilpivirine (long- ViiV Healthcare HIV IM CRL 2020 Yes No transcriptase inhibitor acting) (NNRTI)

S-265744 (S/GSK- cabotegravir ViiV Healthcare HIV integrase inhibitor HIV PO CRL 2020 Yes No 1265744)

CastiaRx.com // p: 866.516.2121 10 Brand Pipeline Forecast 1st Quarter 2020

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

glycolate oxidase ALNG-01 lumasiran Alnylam Hyperoxaluria Intranasal InTrial Late 2020 Yes Yes antagonist

RVT-802 RVT-802 Enzyvant/Roivant Tissue-based therapy Congenital athymia Implant CRL Late 2020 Yes Yes

alpha galactosidase PRX-102 Protalix enzyme replacement Fabry disease IV InTrial Late 2020 Yes No (pegunigalsidase alfa)

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

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

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

CLS-1001 triamcinolone acetonide Clearside corticosteroid Macular edema Intraocular CRL Late 2020 Yes No

CastiaRx.com // p: 866.516.2121 11 Brand Pipeline Forecast 1st Quarter 2020

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

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

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

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

BridgeBio Pharma/ molybdenum cofactor Molybdenum cofactor ALXN-1101 fosdenopterin IV InTrial Late 2020 Yes Yes Origin Biosciences stimulant deficiency

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

Zimhi naloxone Adamis opioid antagonist Opioid dependence IM CRL Late 2020 No No

receptor Ulcerative colitis/ Entyvio (SC formulation) vedolizumab Takeda SC CRL Late 2020 Yes No antagonist Crohn's disease

PharmaEssentia/ AOP ropeginterfero n alfa-2b ropeginterferon alfa-2b interferon Polycythemia vera SC InTrial Late 2020 Yes Yes Orphan

Neutrolin (CRMD-003, citrate/ taurolidine/ antimicrobial agent/ Catheter-related CorMedix IV InTrial Late 2020 No No CRMD-004) heparin anticoagulant infections

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

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

CastiaRx.com // p: 866.516.2121 12 Brand Pipeline Forecast 1st Quarter 2020

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

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

2021 Possible Launch Date

PD-1 checkpoint TSR-042 dostarlimab GlaxoSmithKline Endometrial cancer IV Filed BLA 1/14/2021 Yes No inhibitor

gene transcription Duchenne muscular Translarna ataluren PTC Therapeutics PO CRL 1Q2021 Yes Yes modulator dystrophy

StrataGraft Skin Tissue StrataGraft Skin Tissue Mallinckrodt autologous skin tissue Burn injury TOP InTrial 1Q2021 Yes Yes

KPI-121 0.25% loteprednol etabonate Kala corticosteroid Dry eyes OP CRL 1Q2021 No No

ET-103 levothyroxine Eton Pharmaceuticals L-thyroxine Hypothyroidism PO InTrial 1Q2021 No No

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

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

HM-30181A/ P-glycoprotein pump Oraxol Athenex Breast cancer PO InTrial 1Q2021 Yes No paclitaxel inhibitor/ taxane

Aromatic L-amino acid AGIL-AADC AGIL-AADC Agilis Biotherapeutics gene therapy Intracerebral InTrial 1Q2021 Yes Yes decarboxylase deficiency

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

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

CastiaRx.com // p: 866.516.2121 13 Brand Pipeline Forecast 1st Quarter 2020

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

DNA methylation CC-486 Celgene Acute myeloid leukemia PO InTrial 1Q2021 Yes Yes inhibitor

D-threo- Attention deficit KP-415 KemPharm CNS stimulant PO InTrial 1Q2021 No No hyperactivity disorder controlled-release

Myovant Sciences/ gonadotropin-releasing Uterine fibroids/ TAK-385 relugolix Roivant Sciences/ hormone (GnRH) PO InTrial 1H2021 Yes No Endometriosis Takeda receptor antagonist

sphingosine 1 phosphate RG-3477 (ACT- 128800) ponesimod Johnson & Johnson Multiple sclerosis PO InTrial 1H2021 Yes No (S1P) receptor agonist

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

C5a receptor (C5aR) CCX-168 avacopan ChemoCentryx/ Galencia Vasculitis PO InTrial 1H2021 Yes Yes antagonist arimoclomol arimoclomol Orphazyme cytoprotectives Niemann-Pick Disease PO InTrial 1H2021 Yes Yes

NX-1207 (NYM-4805, Benign prostatic fexapotide triflutate Nymox pro-apoptotic Intratumoral InTrial 1H2021 Yes No REC 0482) hyperplasia sulopenem sulopenem Iterum Therapeutics carbapenem Bacterial infection IV/PO InTrial 1H2021 No No

anti-ECAM exotoxin A Vicinium (VB-4- 845) oportuzumab monatox Sesen Bio Bladder cancer Intravesical InTrial 1H2021 Yes No fusion protein

glucan synthase SCY-078 (MK- 3118) ibrexafungerp Scynexis Fungal infections IV/PO InTrial 1H2021 No Yes inhibitors

CastiaRx.com // p: 866.516.2121 14 Brand Pipeline Forecast 1st Quarter 2020

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

Luveniq voclosporin Aurinia Pharmaceuticals calcineurin inhibitor Lupus nephritis PO InTrial 1H2021 Yes No

C-type natriuretic BMN-111 vosoritide (vasoritide) BioMarin/ Chugai SC InTrial 1H2021 Yes Yes peptide (CNP) analog

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

complement C1s BIVV-009 sutimlimab Sanofi Cold agglutinin disease IV InTrial Mid-2021 Yes Yes subcomponent inhibitor

ZYN-002 ZYN-002 Zynerba cannabinoid product Fragile X syndrome TOP InTrial Mid-2021 Yes Yes

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

Acute myeloid iodine I 131 monoclonal anti-CD45 monoclonal leukemia/ Iomab-B Actinium IV InTrial Mid-2021 Yes Yes antibody BC8 antibody Myelodysplastic syndrome

vascular endothelial Recentin cediranib AstraZeneca Ovarian cancer PO InTrial Mid-2021 Yes Yes (VEGF) antagonist

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

CastiaRx.com // p: 866.516.2121 15 Brand Pipeline Forecast 1st Quarter 2020

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

CUTX-101 copper histidinate Fortress Biotech copper histidinate Menkes Disease SC InTrial Mid-2021 Yes Yes

non-ATP competitive Myelodysplastic Estybon rigosertib Onconova IV InTrial Mid-2021 Yes Yes kinase inhibitor syndrome

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

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

interleukin-17 (IL-17) UCB-4940 bimekizumab UCB Plaque psoriasis IV InTrial Mid-2021 Yes No receptor inhibitor

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

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

Lymphoproliferative EBV-CTL tabelecleucel Atara Biotherapeutics cell therapy IV InTrial Mid-2021 Yes Yes disorder

tavokinogene pIL-12 OncoSec Medical gene therapy Melanoma Intratumoral InTrial Mid-2021 Yes Yes telsaplasmid

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

CastiaRx.com // p: 866.516.2121 16 Brand Pipeline Forecast 1st Quarter 2020

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

PD-1 checkpoint PDR-001 spartalizumab Novartis Melanoma IV InTrial Mid-2021 Yes No inhibitor

recombinant human AT-GAA acid alpha- glucosidase + Amicus enzyme therapy Pompe disease IV InTrial Mid-2021 Yes Yes AT2220

BGJ-398 infigratinib BridgeBio FGFR inhibitor Biliary tract cancer PO InTrial Mid-2021 Yes Yes

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

elivaldogene cerebral adreno- Lenti-D Bluebird Bio gene therapy IV InTrial Mid-2021 Yes Yes tavalentivec myeloneuropathy

ROCK2 (Rho-associated KD-025 KD-025 Kadmon coiled-coiled kinase 2) Graft vs. Host disease PO InTrial Mid-2021 No Yes inhibitor

janus kinase 1 (JAK-1) PF-04965842 abrocitinib Pfizer Atopic dermatitis PO InTrial 3Q2021 Yes No inhibitor

C-C chemokine receptor Nonalcoholic TBR-652 (TAK- 652, CVC) cenicriviroc Allergan 5 (CCR5) and receptor 2 PO InTrial 4Q2021 Yes No steatohepatitis antagonist

Duchenne muscular VBP-15 vamorolone Santhera corticosteroid PO InTrial 4Q2021 Yes Yes dystrophy

CastiaRx.com // p: 866.516.2121 17 Brand Pipeline Forecast 1st Quarter 2020

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

nicotinic acetylcholine OS-01 nasal spray OC-01 Oyster Point Pharma receptor (nAChR) Dry eye disease Intranasal InTrial 4Q2021 No No agonist

opioid receptor OPNT-003 nalmefene Opiant Opioid overdose Intranasal InTrial 4Q2021 No No antagonist

Growth hormone MOD-401 somatrogon OPKO Health/ Pfizer enzyme replacement SC InTrial 2H2021 Yes Yes deficiency

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

NK-1 receptor MK-0594 (VPD- 737) serlopitant Menlo Pruritus PO InTrial 2H2021 Yes No antagonist

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

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

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

interleukin-13 (IL-13) CAT-354 tralokinumab Leo Pharma Atopic dermatitis SC InTrial 2H2021 Yes No inhibitor

tumor vascular NPI-2358 plinabulin BeyondSpring Neutropenia IV InTrial 2H2021 Yes No disrupting agent (tVDA)

CastiaRx.com // p: 866.516.2121 18 Brand Pipeline Forecast 1st Quarter 2020

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

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

chimeric antigen LCAR-B38M LCAR-B38M Johnson & Johnson receptor (CAR) T cell Multiple myeloma Undisclosed InTrial 2H2021 Yes Yes therapy

ARGX-113 efgartigimod Argen NV Fc antagonist Myasthenia gravis IV/SC InTrial 2H2021 Yes Yes

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

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

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

GSK-2696274 (OTL-200) GSK-2696274 (OTL-200) GlaxoSmithKline gene therapy Leukodystrophy IV InTrial 2H2021 Yes Yes

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

selective peroxisome proliferator-activated Nonalcoholic GFT-505 elafibranor Genfit PO InTrial 2H2021 Yes No receptor (PPAR) steatohepatitis modulator

Sci-B-Vac hepatitis B vaccine VBI Vaccines vaccine Hepatitis B IM InTrial 2H2021 No No

CastiaRx.com // p: 866.516.2121 19 Brand Pipeline Forecast 1st Quarter 2020

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

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

Dihydro- ergotamine INP-104 Impel/ 3M ergot derivative Acute migraines Intranasal InTrial 2H2021 No No mesylate

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

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

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

aldose reductase AT-007 AT-007 Applied Therapeutics Galactosemia Undisclosed InTrial 2H2021 Yes Yes inhibitor

Nedd 8 Activating Myelodysplastic MLN-4924 (TAK-92) pevonedistat Takeda Enzyme (NAE) IV InTrial 2021 Yes No syndrome antagonist

Prostate cancer; breast RG-7440 (GDC- 0068) ipatasertib Roche pan-Akt inhibitor PO InTrial 2021 Yes No cancer

Oralair Mites dust mite peptide Stallergenes/ Shionogi vaccine Dust mite allergic rhinitis PO InTrial 2021 Yes No

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

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

CastiaRx.com // p: 866.516.2121 20 Brand Pipeline Forecast 1st Quarter 2020

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

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

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

Ophthalmological DS-100 DS-100 Eton undisclosed SC InTrial 2021 Yes No disease ublituximab (LFB-R603, Chronic lymphocytic TG20, TGTX- 1101, TG- CD-20 monoclonal ublituximab TG Therapeutics leukemia/ multiple IV InTrial 2021 Yes Yes 1101, antibody sclerosis Utuxin)

S5G4T-1 benzoyl peroxide Sol-Gel Technologies benzoyl peroxide Rosacea TOP InTrial 2021 No No

receptor Estelle estetrol/ drospirenone Mithra Pregnancy prevention PO InTrial 2021 No No agonist

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

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

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

CaPre omega-3 fatty acids Acasti Pharma fatty acids Hypertriglyceridemia PO InTrial Late 2021 No No

CastiaRx.com // p: 866.516.2121 21 Brand Pipeline Forecast 1st Quarter 2020

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

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

GS-010 GS-010 GenSight Biologics gene therapy Optic neuropathy Intraocular InTrial Late 2021 Yes Yes

Fungal infections, ATI-1501 metronidazole Appili Therapeutics nitroimidazole anaerobic bacterial PO InTrial Late 2021 No No infections

digoxin immune fab digitalis-like factor AMAG-423 AMAG/ Velo Preeclampsia IV InTrial Late 2021 Yes Yes (DIF) antagonist

RGN-259 (GBT- 201; RGN- timbetasin RegeneRx actin regulating peptide Dry eyes OP InTrial Late 2021 No Yes 352)

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

DNA-directed DNA CMX-001 brincidofovir Chimerix Smallpox PO InTrial Late 2021 No Yes polymerase inhibitor

Wet age-related macular PDS-1.0 ranibizumab Roche/ Genentech anti-VEGF antibody Intravitreal implant InTrial Late 2021 Yes No degeneration

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

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

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

CastiaRx.com // p: 866.516.2121 22 Brand Pipeline Forecast 1st Quarter 2020

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

glatiramer acetate long- glatiramer acetate depot Mylan/ Mapi Pharma immunosuppressant Multiple sclerosis IM InTrial Late 2021 Yes No acting

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

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

apical sodium- dependent bile acid SHP-625 (LUM- 001) maralixibat Mirum Pharmaceuticals Alagille syndrome PO InTrial Late 2021 Yes Yes transporter (ABST) inhibitor

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

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

Mitsubishi Tanabe Erythropoietic MT-7117 MT-7117 Undisclosed PO InTrial Late 2021 Yes No Pharma protoporphyria 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 23