Phase 3 biotech companies
232 listed drug developers whose lead program is in Phase 3, largest first, with the next catalyst each has disclosed.
| Company | Ticker | Lead program | Indication | Next catalyst |
|---|---|---|---|---|
| AbbVie Inc. | ABBV | Rinvoq (upadacitinib) | multiple immune-mediated diseases (new submissions: non-segmental vitiligo, severe alopecia areata) | — |
| Merck & Co., Inc. | MRK | Keytruda (pembrolizumab) | Multiple cancers | — |
| Novartis AG | NVS | Entresto (sacubitril/valsartan) | Heart failure (HFrEF, HFpEF, pediatric) | date not stated — Planned FDA meeting on next steps in FSHD |
| Astrazeneca PLC | AZN | Marketed portfolio (e.g., Farxiga, Tagrisso, Imfinzi, Enhertu) | oncology, CVRM, respiratory & immunology, rare disease | H2 2026 — Phase III data readout |
| Gilead Sciences, Inc. | GILD | Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) | HIV-1 infection | December 23, 2026 — FDA action on BLA (PDUFA) |
| Novo Nordisk A/S | NVO | Semaglutide franchise (Ozempic, Wegovy, Rybelsus) and GLP-1/insulin portfolio | Type 2 diabetes and obesity | — |
| Pfizer Inc | PFE | Marketed portfolio (Eliquis, Vyndaqel family, Padcev, Lorbrena and others) | — | — |
| Vertex Pharmaceuticals Inc | VRTX | povetacicept | IgA nephropathy (accelerated approval, adults) | Q1 2027 — AMPLITUDE interim analysis data |
| Bristol Myers Squibb Co | BMY | Marketed portfolio (Opdivo, Eliquis, Reblozyl, Breyanzi, Camzyos, Cobenfy, others) | oncology, hematology, immunology, cardiovascular, neuroscience | 2026 — Registrational study readouts |
| GSK plc | GSK | Hibsago (bepirovirsen) | chronic hepatitis B virus infection | H2 2026 — GSK continues regulatory submissions for bepirovirsen across multiple geographies with key decisions anticipated in the coming months. |
| Sanofi | SNY | Dupixent (dupilumab; marketed portfolio) | Atopic dermatitis and other type 2 inflammatory diseases | 2026 — Start of Phase 3 in asthma |
| Regeneron Pharmaceuticals, Inc. | REGN | EYLEA HD / EYLEA (aflibercept) | Neovascular AMD, diabetic macular edema, other retinal diseases | Q4 2026 — Phase 3 results vs pembrolizumab in adjuvant melanoma |
| Moderna, Inc. | MRNA | Spikevax (mRNA-1273) | COVID-19 | 2026 — Phase 3 melanoma data |
| Takeda Pharmaceutical Co Ltd | TAK | ENTYVIO (vedolizumab) | Ulcerative colitis and Crohn's disease (IBD) | Q1 2027 — FDA PDUFA target action date (NDA under Priority Review) |
| Argenx SE | ARGX | VYVGART | generalized myasthenia gravis, CIDP, ITP | December 2026 — Phase 3 MMN data for empasiprubart are expected in December 2026. |
| BeOne Medicines Ltd. | ONC | BRUKINSA (zanubrutinib) | CLL, MCL, WM, MZL, FL (B-cell malignancies) | 2026 — Potential FDA accelerated approval submission based on Phase 2 in R/R CLL |
| Revolution Medicines, Inc. | RVMD | daraxonrasib | Previously treated metastatic pancreatic ductal adenocarcinoma | Q4 2026 — Global Phase 3 RASolve 307 of elironrasib plus standard of care expected to start in Q4 2026. |
| Biogen Inc. | BIIB | Leqembi (lecanemab) | Alzheimer's disease | — |
| Alnylam Pharmaceuticals, Inc. | ALNY | AMVUTTRA (vutrisiran) | hATTR amyloidosis with polyneuropathy; ATTR-CM | November 2026 — FDA target action date for NDA in generalized myasthenia gravis |
| Genmab A/S | GMAB | Epcoritamab (EPKINLY/TEPKINLY) | relapsed/refractory DLBCL and follicular lymphoma; frontline DLBCL combination in Phase 3 | — |
| Roivant Sciences Ltd. | ROIV | brepocitinib | dermatomyositis | H2 2026 — Phase 2 data |
| United Therapeutics Corporation | UTHR | Tyvaso DPI / Nebulized Tyvaso (treprostinil) | Pulmonary arterial hypertension (PAH) and PH-ILD | date not stated — FDA decision on IPF sNDA (submitted June 2026) |
| Incyte Corp | INCY | JAKAFI (ruxolitinib) and portfolio of marketed products | myelofibrosis, polycythemia vera, GVHD (plus multiple other marketed indications) | Q4 2026 — Potential EU approval (MAA submitted end of 2025) |
| BioNTech SE | BNTX | pumitamig | Multiple solid tumors incl. ES-SCLC, NSCLC, TNBC (Phase 3 ROSETTA trials) | October 2026 — New clinical data from late-stage oncology pipeline (16 abstracts) at ESMO 2026 |
| Insmed Incorporated | INSM | BRINSUPRI (brensocatib) / ARIKAYCE | non-cystic fibrosis bronchiectasis; MAC lung disease | H2 2026 — Initiate Phase 3 study in progressive pulmonary fibrosis |
| Ascendis Pharma A/S | ASND | TransCon CNP (navepegritide) | achondroplasia in children | Q4 2026 — European Commission decision on marketing authorisation application |
| Exelixis, Inc. | EXEL | zanzalintinib (+ atezolizumab, STELLAR-303) | previously treated metastatic colorectal cancer | December 3, 2026 — FDA PDUFA target action date |
| Neurocrine Biosciences Inc | NBIX | INGREZZA (valbenazine) | tardive dyskinesia; chorea associated with Huntington's disease | — |
| BridgeBio Pharma, Inc. | BBIO | Attruby (acoramidis) | transthyretin amyloid cardiomyopathy | Q4 2026 — BridgeBio intends to submit MAA for oral infigratinib in achondroplasia to EMA in fourth quarter of 2026. |
| Summit Therapeutics Inc. | SMMT | ivonescimab (HARMONi) | EGFR-mutated non-squamous NSCLC after third-generation EGFR TKI, in combination with chemotherapy | November 14, 2026 — FDA PDUFA goal action date for BLA |
| Biomarin Pharmaceutical Inc | BMRN | VOXZOGO (vosoritide) | achondroplasia; hypochondroplasia (sNDA) | February 28, 2027 — PDUFA target action date for sNDA (full approval, achondroplasia) |
| Glaukos Corporation | GKOS | iDose TR (travoprost intracameral implant) | Open-angle glaucoma / ocular hypertension (IOP reduction) | — |
| Madrigal Pharmaceuticals, Inc. | MDGL | Rezdiffra (resmetirom) | noncirrhotic MASH with moderate to advanced liver fibrosis | 2027 — MAESTRO-NASH OUTCOMES results |
| Vaxcyte, Inc. | PCVX | VAX-31 (adult 31-valent pneumococcal conjugate vaccine) | prevention of invasive pneumococcal disease and pneumonia in adults 50+ | H1 2027 — OPUS-2 and OPUS-3 Phase 3 data |
| Corcept Therapeutics Inc | CORT | Korlym (mifepristone) | hypercortisolism / Cushing's syndrome | — |
| Krystal Biotech, Inc. | KRYS | VYJUVEK (beremagene geperpavec-svdt, B-VEC) | Dystrophic epidermolysis bullosa | 2026 — IOLITE Phase 3 top-line results |
| ImmunityBio, Inc. | IBRX | ANKTIVA (nogapendekin alfa inbakicept-pmln) plus BCG | BCG-unresponsive non-muscle invasive bladder cancer (CIS with or without Ta/T1) | 2026 — Planned sBLA submission, BCG-naive NMIBC |
| Cytokinetics Inc | CYTK | MYQORZO (aficamten) | symptomatic obstructive hypertrophic cardiomyopathy (oHCM) | Q4 2026 — Company plans to submit a supplemental NDA to FDA for aficamten in symptomatic nHCM in Q4 2026. |
| TG Therapeutics, Inc. | TGTX | BRIUMVI (ublituximab-xiiy) | relapsing forms of multiple sclerosis (adults) | Q4 2026 — Topline data from Phase 3 in RMS |
| Arrowhead Pharmaceuticals, Inc. | ARWR | REDEMPLO (plozasiran) | familial chylomicronemia syndrome (FCS); sHTG in Phase 3 | 2027 — YOSEMITE Phase 3 completion |
| Abivax S.A. | ABVX | obefazimod | moderately to severely active ulcerative colitis | 2026 — selection of follow-on compound |
| Axsome Therapeutics, Inc. | AXSM | AUVELITY (dextromethorphan-bupropion) | major depressive disorder | May 1, 2027 — FDA action on NDA (PDUFA) |
| Alkermes plc | ALKS | alixorexton | narcolepsy type 1 and type 2 | 2027 — Phase 2 ADHD study data expected in 2027. |
| Ionis Pharmaceuticals Inc | IONS | TRYNGOLZA (olezarsen) | severe hypertriglyceridemia (sHTG) and familial chylomicronemia syndrome (FCS) | — |
| Praxis Precision Medicines, Inc. | PRAX | relutrigine | SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) | December 27, 2026 — PDUFA action date (NDA, priority review, extended three months) |
| Lantheus Holdings, Inc. | LNTH | MK-6240 (F-18 tau PET imaging agent) | PET detection of tau neurofibrillary tangle pathology in patients with cognitive impairment evaluated for Alzheimer's disease | Q4 2026 — Commercial availability (rolling geographic launch) |
| Iovance Biotherapeutics, Inc. | IOVA | Amtagvi (lifileucel) | Previously treated advanced (unresectable or metastatic) melanoma | 2026 — UK MHRA marketing authorization decision (expedited review) |
| Rhythm Pharmaceuticals, Inc. | RYTM | IMCIVREE (setmelanotide) | obesity/hyperphagia due to POMC/PCSK1/LEPR deficiency, Bardet-Biedl syndrome, and acquired hypothalamic obesity | 2026 — initiate pivotal Phase 3 trial in acquired HO |
| Kiniksa Pharmaceuticals International, plc | KNSA | ARCALYST (rilonacept) | Recurrent pericarditis | 2026 — Enter Phase 1 |
| Belite Bio, Inc | BLTE | Tinlarebant (LBS-008), oral RBP4 antagonist | Stargardt disease type 1 (STGD1) | February 12, 2027 — FDA PDUFA target action date (NDA with Priority Review) |
| Immunovant, Inc. | IMVT | IMVT-1402 (imeroprubart) | Graves' disease; D2T rheumatoid arthritis; myasthenia gravis; CIDP; Sjogren's disease | H2 2026 — Further program updates |
| Veradermics, Inc | MANE | VDPHL01 | Pattern hair loss (male and female) | H2 2026 — Topline data from Part B of Study '302' (male pattern hair loss) anticipated in the second half of 2026. |
| Kodiak Sciences Inc. | KOD | tarcocimab tedromer (Zenkuda, KSI-301) | wet AMD, diabetic retinopathy, retinal vein occlusion | Q4 2026 — Completion of enrollment, second pivotal cohort (PEAK/PINNACLE) |
| CG Oncology, Inc. | CGON | cretostimogene grenadenorepvec | BCG-unresponsive high-risk NMIBC with CIS | — |
| Cogent Biosciences, Inc. | COGT | Bezuclastinib (CGT9486) | Systemic mastocytosis (NonAdvSM, AdvSM) and GIST (with sunitinib) | 2026 — IND submission |
| Mirum Pharmaceuticals, Inc. | MIRM | LIVMARLI (maralixibat) | Cholestatic pruritus in Alagille syndrome and PFIC | Q1 2027 — PBC topline data |
| Grifols SA | GRFS | Fibrinogen concentrate (BT524) | Congenital fibrinogen deficiency (US BLA approved 2025); acquired fibrinogen deficiency (EU approved, US Phase III to start 2026) | 2026 — US commercial sales start |
| PTC Therapeutics, Inc. | PTCT | Sephience (sepiapterin) | Phenylketonuria | Q4 2026 — PTC completed the ST-920 acquisition and expects to complete the rolling BLA submission for accelerated approval in Q4 2026. |
| Scholar Rock Holding Corp | SRRK | apitegromab | Spinal muscular atrophy (SMA), non-ambulatory Type 2/3 | H2 2026 — Topline data from Phase 1 healthy-volunteer study |
| Definium Therapeutics, Inc. | DFTX | DT120 ODT (lysergide D-tartrate; formerly MM120) | Generalized anxiety disorder (also MDD and PTSD) | 2026 — Initial Phase 2a data in adult ASD |
| Travere Therapeutics, Inc. | TVTX | FILSPARI (sparsentan) | Primary IgA nephropathy (IgAN); also FSGS | 2026 — Chugai regulatory filing in Japan |
| Dianthus Therapeutics, Inc. | DNTH | claseprubart (DNTH103) | CIDP (CAPTIVATE) and generalized myasthenia gravis (EMERGE) | December 2026 — MoMeNtum Phase 2 topline |
| Relay Therapeutics, Inc. | RLAY | zovegalisib (RLY-2608) | PI3Kalpha-mutated HR+/HER2- locally advanced or metastatic breast cancer (post-CDK4/6 inhibitor) | — |
| Liquidia Corp | LQDA | L606 (liposomal treprostinil, inhaled via next-generation nebulizer) | pulmonary hypertension associated with interstitial lung disease (PH-ILD); also PAH (open-label) | October 2026 — RE-WARM Phase 2a dose-finding study (~75 patients) expected to begin October 2026, primary completion targeted February 2027. |
| Organon & Co. | OGN | Nexplanon (etonogestrel implant) | contraception | H2 2026 — US commercial launch |
| Xenon Pharmaceuticals Inc. | XENE | azetukalner (XEN1101) | focal onset seizures (epilepsy) | H2 2026 — Complete Phase 1 SAD/MAD to support Phase 2 in acute pain |
| Amylyx Pharmaceuticals, Inc. | AMLX | avexitide | post-bariatric hypoglycemia (PBH) | 2027 — IND submission |
| Acadia Pharmaceuticals Inc | ACAD | remlifanserin (ACP-204) | hallucinations and delusions associated with Alzheimer's disease psychosis (ADP); also Lewy Body Dementia Psychosis | November 2026 — Detailed Phase 2 RADIANT results to be presented at CTAD conference in November 2026. |
| IDEAYA Biosciences, Inc. | IDYA | darovasertib (+ crizotinib) | Uveal melanoma (metastatic first line; neoadjuvant; adjuvant) | October 2026 — Updated OptimUM-01 (HLA-A2 positive) data at ESMO |
| Celcuity Inc. | CELC | REVTORPYK (gedatolisib) | HR+/HER2- advanced breast cancer, PIK3CA wild-type, after endocrine therapy | 2028 — Topline data |
| Telix Pharmaceuticals Ltd | TLX | TLX591-Tx (177Lu-rosopatamab tetraxetan) | Metastatic castration-resistant prostate cancer (ProstACT Global) | 2026 — BLA resubmission to FDA |
| Viking Therapeutics, Inc. | VKTX | VK2735 (subcutaneous; VANQUISH-1 and VANQUISH-2) | Obesity | Q4 2026 — Initiate Phase 3 oral dosing studies |
| Dyne Therapeutics, Inc. | DYN | z-rostudirsen (DYNE-251) | Duchenne muscular dystrophy (exon 51 skipping) | January 21, 2027 — FDA action on BLA (PDUFA target date) |
| Disc Medicine, Inc. | IRON | bitopertin | erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) | Q4 2026 — Initial Phase 1b data in sickle cell disease |
| Nurix Therapeutics, Inc. | NRIX | bexobrutideg (NX-5948) | Relapsed/refractory CLL | — |
| Zai Lab Ltd | ZLAB | Zocilurtatug pelitecan (zoci, ZL-1310; DLL3 ADC) | Previously treated extensive-stage small cell lung cancer (also extrapulmonary NECs) | November 30, 2026 — FDA PDUFA action date for accelerated approval BLA in IgAN |
| Viridian Therapeutics, Inc.\ | VRDN | veligrotug (Lumvoa), IV anti-IGF-1R | thyroid eye disease (TED) | H2 2026 — Phase 1 healthy volunteer data |
| HUTCHMED (China) Ltd | HCM | Savolitinib (ORPATHYS) + osimertinib (TAGRISSO) | EGFR-mutated NSCLC with MET overexpression/amplification after EGFR-TKI progression | — |
| Bausch Health Companies Inc. | BHC | Xifaxan (marketed Salix portfolio) | IBS-D; reduction in risk of overt hepatic encephalopathy recurrence; travelers' diarrhea | H1 2027 — FDA approval anticipated following submitted NDA (Phase 3 met all endpoints) |
| Arcus Biosciences, Inc. | RCUS | casdatifan (HIF-2a inhibitor) | clear cell renal cell carcinoma (ccRCC); PEAK-1 with cabozantinib post-immunotherapy | 2026 — Initiation of second Phase 3 trial in first-line ccRCC (TKI-free regimen) |
| NewAmsterdam Pharma Company N.V. | NAMS | obicetrapib | Hypercholesterolemia / ASCVD | Q4 2026 — DSMB-conducted interim analysis |
| Sellas Life Sciences Group, Inc. | SLSN | Galinpepimut-S (GPS) | Acute myeloid leukemia (AML) in maintenance setting after second complete remission | Q4 2026 — Topline data from Phase 2 trial of SLS009 in newly diagnosed first-line AML expected in Q4 2026. |
| Celldex Therapeutics, Inc. | CLDX | barzolvolimab (CDX-0159) | chronic spontaneous urticaria (CSU) | 2027 — BLA filing (assuming positive Phase 3) |
| Agios Pharmaceuticals, Inc. | AGIO | mitapivat (PYRUKYND/AQVESME) | PK deficiency hemolytic anemia; alpha/beta-thalassemia anemia (sickle cell disease sNDA under review) | H2 2026 — confirm proof of mechanism in PKU patients |
| Pharvaris N.V. | PHVS | Deucrictibant IR capsule (on-demand) | On-demand treatment of hereditary angioedema (HAE) attacks | Q1 2027 — Topline data from Part 1 of CREAATE in AAE-C1INH |
| Vericel Corp | VCEL | MACI (autologous cultured chondrocytes on scaffold) | symptomatic knee cartilage defects | 2027 — UK MHRA authorization and commercialization |
| Immunome Inc. | IMNM | varegacestat (AL102) | progressing desmoid tumors | Q4 2026 — IND submission |
| Harmony Biosciences Holdings, Inc. | HRMY | Pitolisant GR (gastro-resistant formulation) | Narcolepsy | Q4 2026 — Phase 1 MAD topline data |
| Biohaven Ltd. | BHVN | Opakalim (BHV-7000) | Refractory focal epilepsy (adults) | H2 2026 — RISE 3 topline results |
| Biocryst Pharmaceuticals Inc | BCRX | ORLADEYO (berotralstat) | prevention of hereditary angioedema (HAE) attacks | — |
| Braveheart Bio, Inc. | BRVE | BHB-1893 | Obstructive hypertrophic cardiomyopathy (oHCM) and non-obstructive HCM (nHCM) | H1 2027 — Initiate global Phase 3 trial in nHCM |
| ADARx Pharmaceuticals, Inc. | ADRX | onvuzosiran | Prevention of hereditary angioedema (HAE) attacks | Q4 2027 — STOP-HAE Phase 3 topline data |
| Sarepta Therapeutics, Inc. | SRPT | ELEVIDYS (delandistrogene moxeparvovec-rokl) and PMO products EXONDYS 51, VYONDYS 53, AMONDYS 45 | Duchenne muscular dystrophy | H2 2026 — Further Phase 1/2 data |
| Xencor, Inc. | XNCR | XmAb819 | clear cell renal cell carcinoma (ccRCC) | H2 2026 — Planned Phase 3 pivotal study for advanced ccRCC |
| Mineralys Therapeutics, Inc. | MLYS | Lorundrostat | Hypertension (uncontrolled and resistant) with and without CKD | — |
| Vir Biotechnology, Inc. | VIR | tobevibart + elebsiran (ECLIPSE program) | chronic hepatitis delta (CHD) | H2 2026 — Updated Phase 1 dose-escalation data |
| Palvella Therapeutics, Inc. | PVLA | QTORIN 3.9% rapamycin anhydrous gel | Microcystic lymphatic malformations | H2 2026 — Phase 2 trial initiation |
| Trevi Therapeutics, Inc. | TRVI | Haduvio (oral nalbuphine ER) | Chronic cough in idiopathic pulmonary fibrosis (IPF) | Q4 2026 — Phase 2b LAKE sample size re-estimation |
| Zenas BioPharma, Inc. | ZBIO | obexelimab | IgG4-related disease | Q4 2026 — Topline results from the Phase 2 SunStone trial in Systemic Lupus Erythematosus expected in 4Q 2026. |
| UroGen Pharma Ltd. | URGN | Zusduri (mitomycin intravesical solution) and Jelmyto (mitomycin pyelocalyceal solution) | Recurrent low-grade intermediate risk NMIBC; low-grade UTUC | Q4 2026 — Begin Phase 1 trial in NMIBC |
| Kailera Therapeutics, Inc. | KLRA | ribupatide injection (KAI-9531) | obesity / overweight | H1 2027 — Initiate global Phase 3 trials |
| Stoke Therapeutics, Inc. | STOK | zorevunersen (STK-001) | Dravet syndrome | H2 2026 — Pre-NDA meeting with FDA |
| Novavax Inc | NVAX | COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates (Matrix-M adjuvanted) | COVID-19 and influenza prevention in adults 65 and older | 2027 — Manufacturing technology transfer to Sanofi complete ($75M milestone) |
| Mesoblast Ltd | MESO | rexlemestrocel-L | chronic low back pain associated with degenerative disc disease | — |
| Immunocore Holdings plc | IMCR | KIMMTRAK (tebentafusp) | unresectable or metastatic uveal melanoma | Q4 2026 — Phase 3 topline data |
| Generate Biomedicines, Inc. | GENB | GB-0895 | Severe asthma (long-acting anti-TSLP, Q26W dosing) | 2026 — Phase 1b data readout |
| Intellia Therapeutics, Inc. | NTLA | lonvoguran ziclumeran (lonvo-z; NTLA-2002) | hereditary angioedema (HAE) | H1 2027 — US commercial launch if approved |
| COMPASS Pathways plc | CMPS | COMP360 psilocybin treatment | Treatment-resistant depression (TRD) | 2027 — Potential FDA approval and first product revenue |
| Ultragenyx Pharmaceutical Inc. | RARE | Dojolvi (triheptanoin); portfolio also Crysvita, Mepsevii | long-chain fatty acid oxidation disorders (LC-FAOD) | H2 2026 — Complete enrollment in Phase 2/3 Aurora study |
| Structure Therapeutics Inc. | GPCR | aleniglipron (GSBR-1290) | Chronic weight management (obesity/overweight with and without T2DM) | Q4 2026 — Body composition trial topline |
| Nanobiotix S.A. | NBTX | JNJ-1900 (NBTXR3) | Locally advanced platinum-ineligible head and neck squamous cell cancer (NANORAY-312); also Stage III unresectable NSCLC and recurrent NSCLC | — |
| Ascentage Pharma Group International | AAPG | Olverembatinib (HQP1351) | Ph+ ALL (POLARIS-1), previously treated CML-CP (POLARIS-2), SDH-deficient GIST (POLARIS-3) | 2026 — Pursue inclusion in China NRDL |
| Ocular Therapeutix, Inc. | OCUL | AXPAXLI (axitinib intravitreal implant) | neovascular (wet) age-related macular degeneration | Q4 2026 — Submit NDA for wet AMD |
| Inhibrx Biosciences, Inc. | INBX | ozekibart (INBRX-109) | conventional chondrosarcoma (BLA); also colorectal cancer, Ewing sarcoma | Q4 2026 — FDA meeting on first-line registrational CRC trial and potential accelerated pathway in 4L CRC |
| Oric Pharmaceuticals, Inc. | ORIC | rinzimetostat (ORIC-944) | metastatic castration-resistant prostate cancer (post-abiraterone), in combination with darolutamide | H2 2026 — Phase 1b data in 1L NSCLC EGFR exon 20 (monotherapy and with SC amivantamab) and 1L EGFR atypical mutations (monotherapy) |
| Nektar Therapeutics | NKTR | rezpegaldesleukin (REZPEG) | moderate-to-severe atopic dermatitis | Q1 2027 — Initiate global registrational ZENITH-AA-1 trial in alopecia areata |
| LB Pharmaceuticals Inc | LBRX | LB-102 | acute schizophrenia (pivotal Phase 3 NOVA-2) | H2 2027 — Initiation of trial in negative symptoms of schizophrenia |
| Rapport Therapeutics, Inc. | RAPP | RAP-219 | drug-resistant focal onset seizures | H2 2027 — FDA meeting planned Q1 2027 on bipolar mania registrational design; two Phase 3 trials expected to begin 2H 2027. |
| Kardigan, Inc. | KARD | Danicamtiv | Genetic dilated cardiomyopathy (MYH7 and TTN variants) | — |
| Syndax Pharmaceuticals Inc | SNDX | Revuforj (revumenib) | relapsed/refractory acute leukemia with KMT2A translocation; R/R NPM1-mutant AML | Q4 2026 — Phase 2 topline data |
| Bicara Therapeutics Inc. | BCAX | ficerafusp alfa | 1L recurrent/metastatic HPV-negative head and neck squamous cell carcinoma (FORTIFI-HN01) | H2 2026 — Data presentation from cohorts |
| Immatics N.V. | IMTX | anzu-cel (anzutresgene autoleucel, IMA203) | second-or-later-line advanced cutaneous melanoma (PRAME-positive); also metastatic uveal melanoma (Phase 2) | — |
| Vor Biopharma Inc. | VOR | Telitacicept (licensed from RemeGen, ex-Greater China rights) | Generalized myasthenia gravis (global Phase 3); Sjögren's disease (global Phase 3) | — |
| Replimune Group, Inc. | REPL | TUDRIQEV (vusolimogene oderparepvec-wtpg; RP1) + nivolumab | unresectable advanced cutaneous melanoma after anti-PD-1 progression | 2026 — Preliminary HCC data release |
| ArriVent BioPharma, Inc. | AVBP | firmonertinib | first-line EGFR exon 20 insertion NSCLC (FURVENT) and PACC-mutant NSCLC (ALPACCA) | H2 2026 — First patient dosed in Phase 1 trial |
| MoonLake Immunotherapeutics | MLTX | sonelokimab (SLK, M1095) | moderate-to-severe hidradenitis suppurativa (HS) | H2 2026 — Final topline results |
| Not specified in extract | Not specified in extract | onzotrigine | moderate to severe acute pain, including postoperative pain | H2 2026 — Initiate placebo-controlled Phase 3 trial in bunionectomy and open-label Phase 3 safety trial |
| SpyGlass Pharma, Inc. | SGP | Bimatoprost Drug Pad-IOL System (BIM-IOL System) | Open-angle glaucoma and ocular hypertension (IOP reduction at time of cataract surgery) | 2027 — Complete enrollment in two registrational Phase 3 trials |
| Cullinan Therapeutics, Inc. | CGEM | zipalertinib (CLN-081/TAS6417) | EGFR exon 20 insertion NSCLC, previously treated | Q4 2026 — Clinical data update from dose escalation |
| Corvus Pharmaceuticals, Inc. | CRVS | soquelitinib (CPI-818, ITK inhibitor) | relapsed/refractory peripheral T cell lymphoma (PTCL) | 2026 — Initiate Phase 1b in hidradenitis suppurativa and Phase 2 in asthma |
| MeiraGTx Holdings plc | MGTX | Botaretigene sparoparvovec (bota-vec) | X-linked retinitis pigmentosa (RPGR) | Q2 2027 — Pivotal Phase 2 AQUAx2 12-month data readout |
| Alumis Inc. | ALMS | envudeucitinib (envu, formerly ESK-001) | moderate-to-severe plaque psoriasis | H1 2027 — Initiate Phase 2 biomarker trial in Parkinson's disease |
| Inventiva S.A. | IVA | lanifibranor | MASH (metabolic dysfunction-associated steatohepatitis) | — |
| Savara Inc | SVRA | MOLBREEVI (molgramostim inhalation solution) | autoimmune pulmonary alveolar proteinosis (autoimmune PAP) | November 22, 2026 — FDA PDUFA target action date for BLA (extended after a major amendment) |
| Kura Oncology, Inc. | KURA | KOMZIFTI (ziftomenib) | relapsed/refractory NPM1-mutated AML | H2 2026 — KOMET-007 data |
| Pharming Group N.V. | PHAR | Joenja (leniolisib) | Activated PI3K delta syndrome (APDS) | November 2026 — Phase II topline results |
| Apnimed, Inc. | APMD | AD109 (Oxnimbi) | Obstructive Sleep Apnea (OSA) | February 2027 — PDUFA goal date for NDA review |
| Ardelyx, Inc. | ARDX | tenapanor (IBSRELA) in chronic idiopathic constipation (ACCEL) | chronic idiopathic constipation (CIC) | 2026 — Complete enrollment in Phase 3 ACCEL |
| Helus Pharma Inc. | HELP | HLP003 (deuterated psilocin, oral) | Adjunctive treatment of major depressive disorder (MDD) | Q4 2026 — Topline data readout from APPROACH Phase 3 study of HLP003 for adjunctive treatment of major depressive disorder on track for Q4 2026. |
| Geron Corporation | GERN | imetelstat (RYTELO) IMpactMF | intermediate-2 or high-risk relapsed/refractory myelofibrosis | H2 2026 — Interim overall survival analysis |
| Annexon, Inc. | ANNX | tanruprubart (ANX005) | Guillain-Barre Syndrome (GBS) | Q4 2026 — ARCHER II Phase 3 Month 15 primary endpoint readout |
| Achieve Life Sciences, Inc. | ACHV | cytisinicline | smoking cessation (nicotine dependence); also vaping cessation | H1 2027 — Potential FDA approval |
| Nuvectis Pharma, Inc. | NVCT | NXP100 (ciprocopan / HSK39297) | paroxysmal nocturnal hemoglobinuria; IgA nephropathy; lupus nephritis | date not stated — Start of combination study with ALK inhibitor lorlatonib |
| Aura Biosciences, Inc. | AURA | bel-sar (belzupacap sarotalocan) | Early choroidal melanoma | — |
| Gyre Therapeutics, Inc. | GYRE | F351 (hydronidone) | Chronic hepatitis B-associated liver fibrosis (PRC); MASH-associated liver fibrosis (US, pre-Phase 2) | 2026 — NMPA IND submission |
| Sinovac Biotech Ltd | SVA | Bivalent enterovirus vaccine (HFMD2) | hand, foot and mouth disease (EV71/CA16) | date not stated — WHO prequalification application submission |
| Solid Biosciences Inc. | SLDB | SGT-003 | Duchenne muscular dystrophy | Q4 2026 — FDA meeting to discuss INSPIRE DUCHENNE data package and potential accelerated approval pathway |
| Lexicon Pharmaceuticals, Inc. | LXRX | INPEFA (sotagliflozin) | reduce CV death, HF hospitalization and urgent HF visits in adults with heart failure or T2DM, CKD and other CV risk factors | — |
| Candel Therapeutics, Inc. | CADL | aglatimagene besadenovec (CAN-2409) plus valacyclovir | Localized intermediate/high-risk prostate cancer (completed Phase 3, positive topline); metastatic non-squamous NSCLC (Phase 3 AURORA enrolling) | Q4 2026 — Presentation of data on potential long-term survivors |
| CorMedix Inc. | CRMD | DefenCath (taurolidine and heparin) | Reduction of catheter-related bloodstream infections in adult hemodialysis patients with a central venous catheter | H2 2027 — CorMedix is targeting to begin promotional efforts for REZZAYO in the new indication in H2 2027. |
| 4D Molecular Therapeutics, Inc. | FDMT | 4D-150 | Wet age-related macular degeneration (wet AMD); DME in preparation | Q2 2027 — 4FRONT-1 Phase 3 topline data |
| Zevra Therapeutics, Inc. | ZVRA | MIPLYFFA (arimoclomol) | neurological manifestations of Niemann-Pick disease type C (NPC), in combination with miglustat | H2 2026 — Follow-up FDA meeting on regulatory options to potentially accelerate development program (after Type C meeting in 2026-Q1) |
| DBV Technologies S.A. | DBVT | Viaskin Peanut patch | Peanut allergy in children (4 through 7 years; also ages 1 through 3) | — |
| Ironwood Pharmaceuticals Inc | IRWD | apraglutide | Short bowel syndrome dependent on parenteral support | — |
| Olema Pharmaceuticals, Inc. | OLMA | Palazestrant (OP-1250) | ER+/HER2- metastatic breast cancer | Q4 2026 — Initiate combination arm with darolutamide in mCRPC (Bayer collaboration) |
| Aquestive Therapeutics, Inc. | AQST | Anaphylm (dibutepinephrine) sublingual film | Severe allergic reactions including anaphylaxis | H2 2026 — Additional preclinical and clinical studies |
| Valneva SE | VALN | LB6V (VLA15) | Prevention of Lyme disease | 2026 — VLA1553-303 five-year seroresponse data |
| Oculis Holding AG | OCS | OCS-01 (OPTIREACH dexamethasone eye drop) | Diabetic macular edema (DME) | Q4 2026 — PREDICT-1 registrational Phase 2/3 topline results |
| Monopar Therapeutics | MNPR | ALXN1840 (tiomolybdate choline) | Wilson disease | 2026 — Complete rolling NDA submission (FDA filing acceptance decision to follow) |
| Kamada Ltd | KMDA | KEDRAB (rabies immune globulin) | rabies post-exposure prophylaxis | H2 2026 — start of NSP sales |
| REGENXBIO Inc. | RGNX | RGX-121 (clemidsogene lanparvovec) | Mucopolysaccharidosis type II (MPS II, Hunter syndrome) | Q4 2026 — Topline data from ATMOSPHERE and ASCENT pivotal trials |
| Prokidney Corp. | PROK | rilparencel (autologous Selected Renal Cells) | advanced chronic kidney disease with type 2 diabetes | Q2 2027 — PROACT 1 topline readout of surrogate endpoint (eGFR slope) supporting potential accelerated approval |
| Altimmune, Inc. | ALT | pemvidutide | MASH | 2029 — 52-week PERFORMA Phase 3 readout |
| Rezolute, Inc. | RZLT | Ersodetug (RZ358) | Hypoglycemia due to tumor hyperinsulinism (upLIFT); congenital hyperinsulinism (sunRIZE) | — |
| Fennec Pharmaceuticals Inc. | FENC | PEDMARK (sodium thiosulfate injection) | reduce risk of cisplatin-induced ototoxicity in pediatric patients with localized, non-metastatic solid tumors | 2026 — Norgine launches in additional European markets |
| Capricor Therapeutics, Inc. | CAPR | deramiocel | Duchenne muscular dystrophy cardiomyopathy | 2026 — Final Phase 1 results |
| Kyverna Therapeutics, Inc. | KYTX | miv-cel (KYV-101) | Stiff person syndrome | Q4 2026 — Complete rolling BLA submission |
| Atea Pharmaceuticals, Inc. | AVIR | bemnifosbuvir/ruzasvir (BEM/RZR) | Chronic hepatitis C | Q1 2027 — C-FORWARD Phase 3 topline results |
| X4 Pharmaceuticals, Inc | XFOR | Mavorixafor (4WARD trial) | chronic neutropenia (congenital, acquired primary autoimmune, idiopathic) with recurrent/serious infections | H2 2027 — Top-line data |
| Opus Genetics, Inc. | IRD | Phentolamine Ophthalmic Solution 0.75% (PS) - presbyopia sNDA and LYNX-3 (mesopic vision); OPGx-LCA5 is co-lead registrational gene therapy | Presbyopia (sNDA under FDA review, PDUFA 2026-10-17); decreased mesopic vision after keratorefractive surgery (LYNX-3 Phase 3) | October 2026 — Present data at EURETINA Congress |
| Ocugen, Inc. | OCGN | OCU400 | Retinitis pigmentosa (gene-agnostic, early to late stage) | 2028 — OCU410 BLA submission targeted for 2028 for geographic atrophy. |
| Vanda Pharmaceuticals Inc. | VNDA | Quimilza (imsidolimab) | Generalized pustular psoriasis (GPP) | December 12, 2026 — FDA PDUFA action date for BLA |
| Unknown | Unknown | atebimetinib (IMM-1-104) | first-line metastatic pancreatic ductal adenocarcinoma (PDAC) | — |
| Greenwich LifeSciences, Inc. | GLSI | GLSI-100 (GP2 + GM-CSF) | Prevention of breast cancer recurrences in HER2/neu positive patients with residual disease or high-risk pathologic complete response | — |
| Lyell Immunopharma, Inc. | LYEL | rondecabtagene autoleucel (ronde-cel, LYL314) | relapsed/refractory large B-cell lymphoma (2L Phase 3 head-to-head; 3L+ pivotal single-arm) | H2 2026 — Additional data from pivotal single-arm trial |
| Sol-Gel Technologies Ltd. | SLGL | SGT-610 (patidegib gel 2%) | chronic use and prevention of new basal cell carcinomas in Gorlin syndrome | — |
| Inhibikase Therapeutics, Inc. | IKT | IKT-001 (imatinib prodrug), IMPROVE-PAH | Pulmonary arterial hypertension | date not stated — Topline data readout in Part B of IMPROVE-PAH Phase 3 |
| Zentalis Pharmaceuticals, Inc. | ZNTL | Azenosertib (ZN-c3) | Cyclin E1-positive platinum-resistant ovarian cancer | H1 2027 — DENALI Part 2 topline readout |
| Cartesian Therapeutics, Inc. | RNAC | Descartes-08 | Myasthenia gravis | Q1 2027 — Topline data from Phase 3 AURORA trial of Descartes-08 in myasthenia gravis expected in first quarter of 2027. |
| ADC Therapeutics SA | ADCT | ZYNLONTA (loncastuximab tesirine-lpyl) | relapsed/refractory DLBCL after two or more lines of systemic therapy | November 2026 — Company announces LOTIS-7 and MZL data abstracts expected in early November with presentations in December at ASH. |
| Invivyd, Inc. | IVVD | VYD2311 | COVID-19 prevention/treatment (DECLARATION and LIBERTY Phase 3) | — |
| MediWound Ltd. | MDWD | NexoBrid | enzymatic eschar removal in deep partial- and full-thickness thermal burns | H2 2026 — Initiate Phase II study in diabetic foot ulcers |
| TuHURA Biosciences, Inc. | HURA | IFx-2.0 | first-line advanced/metastatic Merkel cell carcinoma, with pembrolizumab | date not stated — Phase 1b/2 trial with menin inhibitor in mutNPM1 AML (planned) |
| Compass Therapeutics, Inc. | CMPX | Tovecimig (CTX-009) | Biliary tract cancer (second-line) | Q4 2026 — Clinical data |
| Cardiol Therapeutics Inc. | CRDL | CardiolRx (MAVERIC) | recurrent pericarditis | H1 2027 — Cardiol expands Phase III MAVERIC trial enrollment to up to 150 patients and pushes topline results from Q1 2027 to mid-2027. |
| Jyong Biotech Ltd. | JYNG | Botreso® | urinary system diseases | — |
| Organogenesis Holdings Inc. | ORGO | ReNu | wound healing (type not specified) | April 24, 2027 — PDUFA target action date |
| Humacyte, Inc. | HUMA | Symvess (acellular tissue engineered vessel, ATEV) | vascular trauma; AV access for hemodialysis (supplemental BLA planned) | — |
| Minerva Neurosciences, Inc. | NERV | roluperidone | negative symptoms of schizophrenia | H2 2027 — C19 Phase 3 topline efficacy results (12-week primary endpoint) |
| Unicycive Therapeutics, Inc. | UNCY | Oxylanthanum carbonate (OLC) | Hyperphosphatemia in chronic kidney disease patients on dialysis | — |
| NRx Pharmaceuticals, Inc. | NRXP | NRX-100 (preservative-free IV ketamine) / NRX-101 (D-cycloserine + lurasidone) | Suicidal ideation in depression incl. bipolar depression (NRX-100); suicidal bipolar depression (NRX-101) | Q4 2026 — Commercial launch following ANDA approval |
| PolyPid Ltd. | PYPD | D-PLEX100 | Prevention of surgical site infections (colorectal and abdominal surgery) | November 2026 — D-PLEX100 NDA under FDA Priority Review with November 28, 2026 PDUFA goal date. |
| Immunic, Inc. | IMUX | vidofludimus calcium (IMU-838) | relapsing multiple sclerosis | 2026 — ENSURE-1 and ENSURE-2 topline data |
| vTv Therapeutics Inc. | VTVT | Cadisegliatin (TTP399), CATT1 | Adjunctive therapy to insulin in type 1 diabetes | Q4 2026 — Initiate Phase 2 study in T1D patients using hybrid closed loop systems |
| Inovio Pharmaceuticals, Inc. | INO | INO-3107 | recurrent respiratory papillomatosis (RRP) in adults | October 30, 2026 — FDA PDUFA action date for BLA (accelerated approval review) |
| Genelux Corp | GNLX | Olvi-Vec (olvimulogene nanivacirepvec) | platinum-resistant/refractory ovarian cancer (OnPrime/GOG-3076) | — |
| Atara Biotherapeutics, Inc. | ATRA | Tabelecleucel (tab-cel, Ebvallo) | EBV+ post-transplant lymphoproliferative disease (EBV+ PTLD) after rituximab +/- chemotherapy | — |
| OKYO Pharma Ltd | OKYO | urcosimod | Neuropathic corneal pain | — |
| Cingulate Inc. | CING | CTx-1301 (dexmethylphenidate, PTR platform) | ADHD | date not stated — NDA resubmission following completion of requested CMC work |
| MAIA Biotechnology, Inc. | MAIA | ateganosine (THIO) | third-line non-small cell lung cancer (NSCLC), CPI- and chemotherapy-resistant | 2026 — Filing for accelerated approval in advanced NSCLC |
| Rafael Holdings, Inc. | RFL | Trappsol Cyclo (hydroxypropyl beta cyclodextrin) | Niemann-Pick Disease Type C1 (NPC1) | Q4 2026 — Rafael Holdings remains on track to submit an NDA for Trappsol Cyclo in the fourth quarter of 2026. |
| Tenax Therapeutics, Inc. | TENX | TNX-103 (oral levosimendan) | pulmonary hypertension in heart failure with preserved ejection fraction (PH-HFpEF) | 2026 — Public release of 12-week randomized treatment period results |
| Verrica Pharmaceuticals Inc. | VRCA | YCANTH (VP-102, cantharidin) | Molluscum contagiosum | 2027 — Phase 3 top-line data |
| Gossamer Bio, Inc. | GOSS | seralutinib | pulmonary arterial hypertension (PAH); PH-ILD | Q3 2027 — Potential FDA approval decision if NDA accepted |
| RenovoRx, Inc. | RNXT | IAG (intra-arterial gemcitabine delivered via RenovoCath, TAMP platform) | locally advanced pancreatic cancer (LAPC) | H1 2027 — Completion of Phase III trial after 86 events |
| Talphera, Inc. | TLPH | Niyad | Regional anticoagulant for the dialysis circuit | 2026 — Enrollment completion |
| LeonaBio, Inc. | LONA | Lasofoxifene | ER+/HER2- metastatic breast cancer with ESR1 mutations | date not stated — Topline results of the ELAINE-3 trial |
| Mereo BioPharma Group plc | MREO | setrusumab (BPS-804/UX143) | osteogenesis imperfecta (OI) | — |
| Annovis Bio, Inc. | ANVS | Buntanetap | Early Alzheimer's disease | October 2026 — AD OLE start |
| Grace Therapeutics, Inc. | GRCE | GTx-104 (IV nimodipine) | Aneurysmal subarachnoid hemorrhage (aSAH) | date not stated — NDA resubmission following CRL |
| Inmune Bio, Inc. | INMB | CORDStrom | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | December 2026 — US BLA submission (10-K target; undated in later 10-Q) |
| Aldeyra Therapeutics, Inc. | ALDX | reproxalap | dry eye disease (also allergic conjunctivitis) | 2027 — ADX-2191 Phase 3 PVRL trial initiation now expected in 2027 (previously H1 2026). |
| Edesa Biotech, Inc. | EDSA | EB05 (paridiprubart) | Acute Respiratory Distress Syndrome (ARDS) | date not stated — Start of Phase 2 proof-of-concept enrollment in vitiligo (Health Canada approved; FDA IND CMC information being provided) |
| Aardvark Therapeutics, Inc. | AARD | ARD-101 | Hyperphagia associated with Prader-Willi Syndrome | — |
| BriaCell Therapeutics Corp. | BCTX | Bria-IMT (SV-BR-1-GM) plus checkpoint inhibitor | advanced metastatic breast cancer (pivotal Phase 3, Bria-ABC, NCT06072612) | date not stated — Initiate Phase 1/2a study in metastatic breast cancer |
| CEL-SCI Corp | CVM | Multikine (Leukocyte Interleukin, Injection) | newly diagnosed resectable locally advanced primary squamous cell carcinoma of the head and neck (no lymph node involvement, low PD-L1) | date not stated — Start of 212-patient confirmatory registration study, contingent on raising capital; full enrollment 18-24 months after start |
| Scilex Holding Co | SCLX | ZTlido (lidocaine topical system) 1.8% | neuropathic pain associated with postherpetic neuralgia | — |
| BeyondSpring Inc. | BYSI | Plinabulin | 2L/3L EGFR wild-type non-squamous NSCLC after progression on immune checkpoint inhibitors (with docetaxel) | date not stated — Launch of confirmatory global Phase 3 study DUBLIN-4 (plinabulin + docetaxel vs docetaxel) |
| Jaguar Health, Inc. | JAGX | Mytesi (crofelemer 125 mg delayed-release tablets) | Symptomatic relief of non-infectious diarrhea in adults with HIV/AIDS on antiretroviral therapy | H2 2026 — Submit final clinical study report to FDA |
| BioCardia, Inc. | BCDA | CardiAMP Cell Therapy (BCDA-01) | Ischemic heart failure with reduced ejection fraction (HFrEF) | date not stated — Submission for approval in Japan (PMDA) |
| TScan Therapeutics, Inc. | TCRX | TSC-101 | AML and MDS patients undergoing allogeneic HCT (prevention of relapse; HLA-A*02:01) | — |
| Dare Bioscience, Inc. | DARE | Ovaprene | hormone-free monthly intravaginal contraception | 2027 — Top-line data from pivotal Phase 3 study |
| Lipocine Inc. | LPCN | LPCN 1154 | Postpartum depression | — |
| Karyopharm Therapeutics Inc. | KPTI | XPOVIO (selinexor) | Multiple myeloma (approved); myelofibrosis in development | H2 2026 — Topline data from event-driven Phase 3 (European Myeloma Network sponsored) |
| Biofrontera Inc. | BFRI | Ameluz (aminolevulinic acid gel) with RhodoLED lamps, photodynamic therapy | Actinic keratosis (face and scalp); label-expansion programs in superficial basal cell carcinoma, AK trunk/extremities, acne | Q4 2026 — Biofrontera plans to officially launch the sBCC indication late in the fourth quarter of 2026 through the first quarter of 2027. |
| Citius Pharmaceuticals, Inc. | CTXR | LYMPHIR (denileukin diftitox-cdxl) | Persistent or recurrent cutaneous T-cell lymphoma (CTCL) | — |
| Intensity Therapeutics, Inc. | INTS | INT230-6 | second- and third-line soft tissue sarcoma (INVINCIBLE-3) | 2027 — Intensity is targeting complete enrollment of the INVINCIBLE-4 Study by the end of 2027. |
| Ensysce Biosciences, Inc. | ENSC | PF614 | Severe/chronic pain (abuse-resistant TAAP oxycodone prodrug) | — |
| Vistagen Therapeutics, Inc. | VTGN | fasedienol (PH94B) nasal spray | acute treatment of anxiety in adults with social anxiety disorder | — |
| Imunon, Inc. | IMNN | IMNN-001 (TheraPlas IL-12 DNA immunotherapy) | Newly diagnosed advanced (stage III/IV) ovarian cancer, with neoadjuvant/adjuvant chemotherapy | H1 2029 — Complete ITT enrollment in Phase 3 OVATION 3 |
| Can-Fite BioPharma Ltd. | CANF | Namodenoson (CF102) | Advanced hepatocellular carcinoma (Child-Pugh B7) | Q4 2026 — Initiation of Lowe syndrome Phase 2 |
| Biodexa Pharmaceuticals Plc | BDRX | eRapa | Familial adenomatous polyposis (FAP) | H1 2027 — Biodexa announces the pre-specified futility analysis for its Serenta Phase 3 trial of eRapa in FAP could take place early next year with database lock potentially occurring around the end of next year. |
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.