AZD0240Oncology
solid tumoursPhase 1
AZD0516Oncology
prostate cancerPhase 1
AZD0754Oncology
prostate cancerPhase 1
AZD2068Oncology
solid tumoursPhase 1
AZD2284Oncology
prostate cancerPhase 1
AZD2962Oncology
haematological malignanciesPhase 1
AZD3632Oncology
haematological malignanciesPhase 1
AZD4360Oncology
solid tumoursPhase 1
AZD4512Oncology
relapsed/refractory B-cell non-Hodgkin lymphomaPhase 1
AZD4956Oncology
solid tumoursPhase 1
AZD5492Oncology / Respiratory & Immunology
haematologyPhase 1systemic lupus erythematosusPhase 1
AZD5863Oncology
solid tumoursPhase 1
AZD6621Oncology
prostate cancerPhase 1
AZD6750Oncology
solid tumoursPhase 1
AZD7003Oncology
hepatocellular carcinoma/squamous non-small cell lung cancerPhase 1
AZD8421Oncology
solid tumoursPhase 1
AZD9750Oncology
prostate cancerPhase 1
AZD9793Oncology
solid tumoursPhase 1
NT-112Oncology
solid tumoursPhase 1
NT-175Oncology
solid tumoursPhase 1
surovatamigOncology / Respiratory & Immunology
r/r B-cell non-Hodgkin lymphomaPhase 1B-cell driven autoimmune diseasePhase 1
surovatamig SOUNDTRACK-EOncology
mature B-cell malignanciesPhase 1
volrustomig + lenvatinibOncology
advanced renal cell carcinomaPhase 1
AZD0120Oncology / Respiratory & Immunology
multiple myelomaPhase 1systemic lupus erythematosusPhase 1autoimmune diseasesPhase 1
AZD0305Oncology
relapsed/refractory multiple myelomaPhase 1
AZD3470Oncology
classic Hodgkin lymphoma, solid tumoursPhase 1
AZD9574Oncology
advanced solid malignanciesPhase 1
camizestrantOncology
HR+ HER2- breast cancerPhase 1
FPI-2265Oncology
prostate cancerPhase 1
IPH5201 + ImfinziOncology
neoadjuvant/adjuvant NSCLCPhase 1
puxitatug samrotecanOncology
solid tumoursPhase 1
rilvegostomig ARTEMIDE-01Oncology
solid tumoursPhase 1
saruparibOncology
solid tumoursPhase 1
sonesitatug vedotinOncology
solid tumoursPhase 1
surovatamig SOUNDTRACK-BOncology
B-cell non-Hodgkin lymphomaPhase 1
surovatamig SYRUSOncology
B-cell acute lymphoblastic leukaemiaPhase 1
tilatamig samrotecanOncology
solid tumoursPhase 1
torvutatug samrotecanOncology
ovarian cancer, solid tumoursPhase 1
volrustomigOncology
solid tumoursPhase 1
volrustomig CANTOROncology
colorectal cancer (mCRC)Phase 1
volrustomig eVOLVE-01Oncology
NSCLCPhase 1
volrustomig eVOLVE-02Oncology
cervical cancer, head and neck squamous cell carcinoma, esophageal squamous cell carcinomaPhase 1
AZD0120 DURGA-4Oncology
relapsed/refractory multiple myelomaPhase 1
camizestrant + CDK4/6i SERENA-6Oncology
1L HR+ HER2- ESR1m advanced breast cancerPhase 1
camizestrant + palbociclib SERENA-4Oncology
1L HR+ HER2- advanced breast cancerPhase 1
camizestrant +/- abemaciclib CAMBRIA-2Oncology
adjuvant HR+ HER2- early breast cancerPhase 1
camizestrant CAMBRIA-1Oncology
adjuvant switch HR+ HER2- early breast cancerPhase 1
Imfinzi + Imjudo HIMALAYAOncology
1L unresectable HCCPhase 1
Imfinzi +/- oleclumab +/- monalizumab PACIFIC-9Oncology
unresectable stage III NSCLCPhase 1
puxitatug samrotecan Bluestar-Endometrial01Oncology
2-3L B7-H4+ endometrial cancerPhase 1
rilvegostomig + bevacizumab +/- Imjudo ARTEMIDE-HCC01Oncology
1L HCCPhase 1
rilvegostomig + CTx ARTEMIDE-Biliary01Oncology
adjuvant biliary tract cancerPhase 1
rilvegostomig + CTx ARTEMIDE-Lung02Oncology
1L PD-L1 TC ≥1% SQ NSCLCPhase 1
rilvegostomig + Enhertu ARTEMIDE-Gastric01Oncology
1L HER2+ gastric cancerPhase 1
rilvegostomig ARTEMIDE-Biliary02Oncology
metastatic biliary tract cancerPhase 1
rilvegostomig ARTEMIDE-Lung03Oncology
1L PD-L1 TC ≥1% NSQ NSCLCPhase 1
rilvegostomig ARTEMIDE-Lung04Oncology
1L PD-L1≥50% NSCLCPhase 1
saruparib + ADT +/- abiraterone EvoPAR-Prostate02Oncology
localised/locally advanced BRCAm prostate cancerPhase 1
saruparib + camizestrant EvoPAR-Breast01Oncology
BRCA/PALB2m HR+ HER2- metastatic breast cancerPhase 1
saruparib + NHA EvoPAR-Prostate01Oncology
HRRm/non-HRRm mCSPCPhase 1
sonesitatug vedotin + CTx +/- rilvegostomig CLARITY-Gastric02Oncology
1L CLDN18.2+ gastric cancerPhase 1
sonesitatug vedotin CLARITY-Gastric01Oncology
2L+ CLDN18.2+ gastric cancerPhase 1
surovatamig SOUNDTRACK-D2Oncology
1L elderly DLBCLPhase 1
surovatamig SOUNDTRACK-F1Oncology
follicular lymphomaPhase 1
torvutatug samrotecan (AZD5335) TREVI-OC-01Oncology
ovarian cancerPhase 1
volrustomig eVOLVE-CervicalOncology
high-risk locally advanced cervical cancerPhase 1
volrustomig eVOLVE-HNSCCOncology
unresected locally advanced head and neck squamous cell carcinomaPhase 1
volrustomig eVOLVE-Lung02Oncology
1L metastatic NSCLCPhase 1
volrustomig eVOLVE-MesoOncology
1L unresectable malignant pleural mesotheliomaPhase 1
Calquence + R-CHOP ESCALADEOncology
1L DLBCLLCM Projects
Calquence + venetoclax + obinutuzumab AMPLIFYOncology
1L CLLLCM Projects
Calquence ECHOOncology
1L MCLLCM Projects
Datroway + Imfinzi + CTx AVANZAROncology
1L NSQ/NSQ TROP2+ NSCLCLCM Projects
Datroway + Imfinzi TROPION-Breast04Oncology
neo/adjuvant TNBC or HR-low/HER2- breast cancerLCM Projects
Datroway + Imfinzi TROPION-Breast05Oncology
1L PD-L1 CPS ≥10 TNBCLCM Projects
Datroway + pembrolizumab TROPION-Lung07Oncology
1L PD-L1 <50% NSQ NSCLCLCM Projects
Datroway + pembrolizumab TROPION-Lung08Oncology
1L PD-L1 TPS ≥50% NSQ NSCLCLCM Projects
Datroway + rilvegostomig TROPION-Lung10Oncology
1L PD-L1 ≥50% NSQ NSCLCLCM Projects
Datroway + Tagrisso TROPION-Lung14Oncology
1L EGFRm NSCLCLCM Projects
Datroway + Tagrisso TROPION-Lung15Oncology
2L EGFRm NSCLCLCM Projects
Datroway +/- Imfinzi TROPION-Breast03Oncology
post-neoadjuvant TNBC with residual diseaseLCM Projects
Datroway TROPION-Breast02Oncology
1L TNBC not candidates for IOLCM Projects
Datroway TROPION-Lung17Oncology
TROP2 QCS NMR+ non-squamous NSCLC 2LLCM Projects
Enhertu (platform) DESTINY-Breast07Oncology
HER2+ breast cancerLCM Projects
Enhertu + pertuzumab DESTINY-Breast09Oncology
1L HER2+ breast cancerLCM Projects
Enhertu + rilvegostomig DESTINY-BTC01Oncology
1L HER2+ biliary tract cancerLCM Projects
Enhertu + rilvegostomig/pembrolizumab DESTINY-Endometrial01Oncology
1L HER2+ pMMR endometrial cancerLCM Projects
Enhertu DESTINY-Breast05Oncology
post-neoadjuvant high-risk HER2+ early breast cancerLCM Projects
Enhertu DESTINY-Endometrial02Oncology
adjuvant endometrial cancerLCM Projects
Enhertu DESTINY-Gastric04Oncology
2L HER2+ gastric cancerLCM Projects
Enhertu DESTINY-Lung04Oncology
1L HER2m NSCLCLCM Projects
Enhertu DESTINY-PanTumor03 (China)Oncology
HER2 expressing solid tumoursLCM Projects
Enhertu followed by THP DESTINY-Breast11Oncology
neoadjuvant high-risk HER2+ early breast cancerLCM Projects
EnhertuDESTINY-PanTumor02Oncology
HER2 expressing solid tumoursLCM Projects
Imfinzi + BCG POTOMACOncology
non-muscle invasive bladder cancerLCM Projects
Imfinzi + CRT KUNLUNOncology
locally advanced ESCCLCM Projects
Imfinzi + domvanalimab following cCRT PACIFIC-8Oncology
unresectable stage III NSCLCLCM Projects
Imfinzi + EV +/- Imjudo VOLGAOncology
muscle invasive bladder cancer (cis-ineligible/refusal)LCM Projects
Imfinzi + FLOT MATTERHORNOncology
resectable early gastric cancerLCM Projects
Imfinzi + Imjudo + SoC NILEOncology
1L urothelial cancerLCM Projects
Imfinzi + Imjudo + TACE +/- lenvatinib EMERALD-3Oncology
locoregional HCCLCM Projects
Imfinzi + SBRT PACIFIC-4Oncology
stage I/II NSCLCLCM Projects
Imfinzi + VEGF + TACE EMERALD-1Oncology
locoregional HCCLCM Projects
Imfinzi +/- bevacizumab EMERALD-2Oncology
adjuvant HCCLCM Projects
Imfinzi +/- Imjudo + CTx POSEIDONOncology
1L NSCLCLCM Projects
Imfinzi combinations BEGONIAOncology
1L TNBCLCM Projects
Imfinzi combinations NeoCOAST-2Oncology
resectable NSCLCLCM Projects
Lynparza MONO-OLA1Oncology
1L BRCAwt ovarian cancerLCM Projects
Tagrisso + Orpathys SAFFRONOncology
advanced EGFRm NSCLCLCM Projects
Tagrisso +/- CTx NeoADAURAOncology
neoadjuvant stage II/III resectable EGFRm NSCLCLCM Projects
Tagrisso ADAURA2Oncology
EGFRm NSCLC stage Ia2-Ia3 following complete tumour resectionLCM Projects
Tagrisso combinations ORCHARDOncology
2L EGFRm osimertinib-resistant NSCLCLCM Projects
Truqap + abiraterone CAPItello-281Oncology
PTEN deficient mHSPCLCM Projects
Truqap + Faslodex + palbociclib CAPItello-292Oncology
1L early relapse/ET resistant advanced HR+ breast cancerLCM Projects
AZD1043Cardiovascular, Renal and Metabolism
obesityPhase 1
AZD1613Cardiovascular, Renal and Metabolism
ADPKDPhase 1
AZD3974Cardiovascular, Renal and Metabolism
cirrhosisPhase 1
AZD4063Cardiovascular, Renal and Metabolism
R14del dilated cardiomyopathyPhase 1
AZD4248Cardiovascular, Renal and Metabolism
cardiorenal diseasePhase 1
AZD4954Cardiovascular, Renal and Metabolism
dyslipidaemiaPhase 1
AZD2389Cardiovascular, Renal and Metabolism
metabolic dysfunction-associated steatohepatitisPhase 1
AZD4144Cardiovascular, Renal and Metabolism
cardiorenal diseasePhase 1
AZD5462Cardiovascular, Renal and Metabolism
heart failurePhase 1
AZD6234Cardiovascular, Renal and Metabolism
chronic weight management in overweight or obesityPhase 1
AZD9550 + AZD6234Cardiovascular, Renal and Metabolism
obesityPhase 1
balcinrenone/dapagliflozinCardiovascular, Renal and Metabolism
CKDPhase 1heart failure with CKDPhase 1
elecoglipronCardiovascular, Renal and Metabolism
T2D/chronic weight managementPhase 1
opemalirsenCardiovascular, Renal and Metabolism
nephropathyPhase 1
baxdrostat BaxHTN Bax24 BaxAsiaCardiovascular, Renal and Metabolism
hypertensionPhase 1
baxdrostat BaxPACardiovascular, Renal and Metabolism
primary aldosteronismPhase 1
baxdrostat/dapagliflozinCardiovascular, Renal and Metabolism
CKDPhase 1prevention of heart failurePhase 1
laroprovstat AZURECardiovascular, Renal and Metabolism
dyslipidaemiaPhase 1
WainuaCardiovascular, Renal and Metabolism
patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)Phase 1patients with hereditary or wild-type transthyretin-mediated amyloid cardiomyopathy (ATTR CM)LCM Projects
zibotentan/dapagliflozinCardiovascular, Renal and Metabolism
CKD with high proteinuriaPhase 1
AZD8965Respiratory & Immunology
idiopathic pulmonary fibrosisPhase 1
AZD1163Respiratory & Immunology
rheumatoid arthritisPhase 1
AZD4604Respiratory & Immunology
asthmaPhase 1
AZD6793Respiratory & Immunology
COPDPhase 1
AZD7798Respiratory & Immunology
Crohn's diseasePhase 1
AZD8630Respiratory & Immunology
asthmaPhase 1
tozorakimabRespiratory & Immunology
asthmaPhase 1
Saphnelo TULIP 1 & TULIP 2 AZALEA (China)Respiratory & Immunology
systemic lupus erythematosusPhase 1
Tezspire NAVIGATOR DIRECTION (China)Respiratory & Immunology
severe uncontrolled asthmaPhase 1
tozorakimab OBERON TITANIA PROSPERO MIRANDARespiratory & Immunology
chronic obstructive pulmonary diseasePhase 1
tozorakimab TILIARespiratory & Immunology
severe viral lower respiratory tract diseasePhase 1
Breztri/Trixeo (PT010) KALOS LOGOSRespiratory & Immunology
asthmaLCM Projects
Breztri/Trixeo THARROSRespiratory & Immunology
cardiopulmonary outcomes trial in COPDLCM Projects
Fasenra MANDARARespiratory & Immunology
eosinophilic granulomatosis with polyangiitisLCM Projects
Fasenra NATRONRespiratory & Immunology
hypereosinophilic syndromeLCM Projects
Saphnelo DAISYRespiratory & Immunology
systemic sclerosisLCM Projects
Saphnelo IRISRespiratory & Immunology
lupus nephritisLCM Projects
Saphnelo JASMINERespiratory & Immunology
myositisLCM Projects
Saphnelo LAVENDERRespiratory & Immunology
cutaneous lupus erythematosusLCM Projects
Saphnelo TULIP-SCRespiratory & Immunology
systemic lupus erythematosus (subcutaneous)LCM Projects
Tezspire CROSSINGRespiratory & Immunology
eosinophilic esophagitisLCM Projects
Tezspire EMBARK, JOURNEYRespiratory & Immunology
chronic obstructive pulmonary diseaseLCM Projects
Tezspire WAYPOINTRespiratory & Immunology
nasal polypsLCM Projects
ALXN2080Rare Disease
healthy participantsPhase 1
ALXN2230Rare Disease
healthy participantsPhase 1
ALXN2350 DCMRestoreRare Disease
BAG3-associated dilated cardiomyopathyPhase 1
AZD0120 ALACRITYRare Disease
amyloid light-chain amyloidosisPhase 1
AZD1390 AGILERare Disease
glioblastomaPhase 1
ALXN1920 AUTUMNRare Disease
primary membranous nephropathyPhase 1
ALXN2030 CONCORDRare Disease
antibody mediated rejectionPhase 1
tarperprumig I TRANSCENDRare Disease
ANCA-associated vasculitisPhase 1
anselamimab CARESRare Disease
amyloid light-chain amyloidosisPhase 1
cliramitug DepleTTR-CMRare Disease
transthyretin amyloid cardiomyopathyPhase 1
efzimfotase alfa Hickory (301), Mulberry (305), Chestnut (303)Rare Disease
hypophosphatasiaPhase 1
eneboparatide CALYPSORare Disease
hypoparathyroidismPhase 1
gefurulimab PREVAILRare Disease
generalised myasthenia gravisPhase 1
Koselugo KOMETRare Disease
neurofibromatosis type 1 adultLCM Projects
UltomirisRare Disease
haematopoietic stem cell transplant–associated thrombotic microangiopathyLCM Projects
Ultomiris AWAKERare Disease
delayed graft functionLCM Projects
Ultomiris I CANRare Disease
immunoglobulin A nephropathyLCM Projects
AZD0292Infectious Disease
bronchiectasisPhase 1
AZD5148Infectious Disease
reduction of C. diff recurrencePhase 1
AZD7760Infectious Disease
prevention of Staph aureus infectionPhase 1
Kavigale SUPERNOVAInfectious Disease
prevention of COVID-19Phase 1

NVS

Novartis

novartis.com

62assets

LutatheraAAA601 / Lutathera / SSTR / Radioligand therapy / Oncology: Solid Tumors
Gastroenteropancreatic neuroendocrine tumorsPhase 3Supplementary; filing 2028GEPNET, pediatricsPhase 2
177Lu-NeoBAAA603 / GRPR / Radioligand therapy / Oncology: Solid Tumors
Breast cancerPhase 1Multiple solid tumorsPhase 2Lead; filing >= 2029
AAA614FAP / Radioligand therapy / Oncology: Solid Tumors
Solid tumorsPhase 2Lead
PluvictoAAA617 / Pluvicto / PSMA / Radioligand therapy / Oncology: Solid Tumors
Oligometastatic prostate cancerPhase 3Supplementary; filing >= 2029Metastatic hormone sensitive prostate cancer (mHSPC)RegistrationSupplementary
225Ac-PSMA-617AAA817 / PSMA / Radioligand therapy / Oncology: Solid Tumors
Metastatic castration-resistant prostate cancer (mCRPC)Phase 3Supplementary; filing >= 2029post Lu Metastatic castration-resistant prostate cancer (mCRPC)Phase 3Lead; filing 2028
ScemblixABL001 / Scemblix / BCR-ABL / Inhibitor / Oncology: Hematology
Chronic myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
CosentyxAIN457 / Cosentyx / IL17A / Inhibitor / Immunology
Polymyalgia rheumaticaRegistrationSupplementary; filing 2026
AimovigAMG334 / Aimovig / CGRPR / Antagonist / In-market Brands and Global Health
Migraine, pediatricsPhase 3Supplementary; filing 2028
AMO959DNA PK / Inhibitor / Oncology: Solid Tumors
Prostate cancerPhase 1Lead
MayzentBAF312 / Mayzent / S1P1,5 receptor modulator / Neuroscience
Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2027
VijoyceBYL719 / Vijoyce / PI3K-alpha / Inhibitor / Oncology: Solid Tumors
Lymphatic malformationsPhase 3Supplementary; filing >= 2029
farabursenCYX082 / MIR17 / Inhibitor / Cardiovascular, Renal and Metabolic
Autosomal dominant polycystic kidney diseasePhase 1Lead
pelabresibDAK539 / BET / Inhibitor / Oncology: Hematology
MyelofibrosisPhase 3Lead; filing 2026
DFT383CTNS gene delivery / Neuroscience
CystinosisPhase 1Lead
DII235siRNA targeting Lp(a) MRNA / Cardiovascular, Renal and Metabolic
CVRR-Lp(a)Phase 2Lead; filing >= 2029
del-braxDWH213 / siRNA DUX4 knockdown / Neuroscience
Facioscapulohumeral muscular dystrophyPhase 3Lead; filing 2028
tulmimetostatDZR123 / EZH1, EZH2 / Inhibitor / Oncology: Solid Tumors
Metastatic prostate cancerPhase 1Solid tumors & lymphomasPhase 2Lead
ECI830CDK2 / Inhibitor / Oncology: Solid Tumors
Breast cancerPhase 1Lead
EDI048CpPI(4)K / Inhibitor / In-market Brands and Global Health
CryptosporidiosisPhase 2Lead
EDK060Lead Indication / Neuroscience
Charcot-Marie-Tooth diseasePhase 1
ESP359DLL3 / Radioligand therapy / Oncology: Solid Tumors
Solid tumorsPhase 1Lead
del-desiranEWF980 / siRNA DMPK knockdown / Neuroscience
Myotonic dystrophy type 1Phase 3Lead; filing 2027
EYU688NS4B / Inhibitor / In-market Brands and Global Health
Dengue feverPhase 2Lead
zigakibartFUB523 / Anti-APRIL / Cardiovascular, Renal and Metabolic
IgA nephropathyPhase 3Lead; filing 2027
177Lu-NNS309FXX489 / FAP / Radioligand therapy / Oncology: Solid Tumors
Solid tumorsPhase 1Lead
GCJ904Lead Indication / Oncology: Solid Tumors
Solid tumorsPhase 1
GHZ339Lead Indication / Immunology
Atopic dermatitisPhase 2filing >= 2029
GIA632IL-15 mAb / Immunology
Atopic dermatitisPhase 2Lead; filing >= 2029VitiligoPhase 2Supplementary; filing >= 2029
GXV813M4 / Agonist / Neuroscience
SchizophreniaPhase 2Lead
HJB647Lead Indication / Cardiovascular, Renal and Metabolic
Heart failurePhase 1
votoplamHTT227 / Huntingtin modulator / Neuroscience
Huntington’s diseasePhase 3Lead; filing >= 2029
INE963Plasmodium falciparum / Inhibitor / In-market Brands and Global Health
MalariaPhase 2Lead
IPX643Lead Indication / Immunology
Inflammation-driven diseasesPhase 1
ITU512HbF inducing agent / In-market Brands and Global Health
Sickle cell diseasePhase 1Lead
luxdegalutamideJSB462 / Androgen receptor protein degrader / Oncology: Solid Tumors
Metastatic hormonal sensitive prostate cancerPhase 2Metastatic castration resistant prostate cancerPhase 2Lead; filing >= 2029
cipargaminKAE609 / PfATP4 / Inhibitor / In-market Brands and Global Health
Malaria, uncomplicatedPhase 2Supplementary; filing >= 2029Malaria severePhase 2Lead; filing >= 2029
LeqvioKJX839 / Leqvio / siRNA (regulation of LDL-C) / Cardiovascular, Renal and Metabolic
CVRR (primary prevention)Phase 3Supplementary; filing >= 2029CVRR (secondary prevention)Phase 3Supplementary; filing 2027
Ganaplacide + lumefantrineKLU156 / Non-artemisinin plasmodium falciparum / Inhibitor / In-market Brands and Global Health
Malaria, uncomplicatedPhase 3Lead; filing 2026
del-zotaKPE179 / PMO mediated exon 44 skipping / Neuroscience
Duchenne muscular dystrophyPhase 2Lead; filing 2026
FabhaltaLNP023 / Fabhalta / CFB / Inhibitor / Neuroscience / Cardiovascular, Renal and Metabolic / Oncology: Hematology / Others
Generalized myasthenia gravisPhase 3Supplementary; filing 2027C3 glomerulopathy, pediatricsPhase 3IgAN, pediatricsPhase 3
RhapsidoLOU064 / Rhapsido / BTK / Inhibitor / Immunology
Chronic spontaneous urticaria, pediatricsPhase 3Hidradenitis suppurativaPhase 3Supplementary; filing 2027Chronic inducible urticaria (CINDU)Phase 3Supplementary; filing 2026
remibrutinibLOU064 / BTK / Inhibitor / Neuroscience
Multiple sclerosis, secondary progressivePhase 3Supplementary; filing >= 2029Myasthenia gravisPhase 3Supplementary; filing 2028Multiple sclerosis, relapsing remittingPhase 3Supplementary; filing 2027
LTP001SMURF1 / Inhibitor / Cardiovascular, Renal and Metabolic
Idiopathic pulmonary fibrosisPhase 2Pulmonary arterial hypertensionPhase 2Lead; filing >= 2029
LXE408Proteasome / Inhibitor / In-market Brands and Global Health
ChagasPhase 2Visceral leishmaniosisPhase 2Lead; filing >= 2029
abelacimabMAA868 / FXI / Inhibitor / Cardiovascular, Renal and Metabolic
Atrial fibrillationPhase 3Lead; filing 2028
MAS825IL18 Inhibitor, IL1B / Immunology
Still’s diseasePhase 2Lead
NIO752Tau antisense oligonucleotide / Neuroscience
Alzheimer’s diseasePhase 1Progressive supranuclear palsyPhase 1Lead
OJR520Lead Indication / Cardiovascular, Renal and Metabolic
CKDPhase 1
KesimptaOMB157 / Kesimpta / CD20 / Antagonist / Neuroscience
Multiple sclerosis, new dosing regimenPhase 3Supplementary; filing 2027Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2026
PAC001Anti-IL-6 mAb / Cardiovascular, Renal and Metabolic / Others
ASCVD pacibekitugPhase 2Lead; filing >= 2029Thyroid eye disease (TED) pacibekitugPhase 2
PIT565Anti-CD19, Anti-CD2, Anti-CD3 / Immunology
Systemic lupus erythematosusPhase 1Rheumatoid arthritisPhase 1
RydaptPKC412 / Rydapt / Multi-targeted kinase / Inhibitor / In-market Brands and Global Health
Acute myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
PKN605HDAC6 / Inhibitor / Cardiovascular, Renal and Metabolic
Atrial FibrillationPhase 2Lead
QCZ484Lead Indication / Cardiovascular, Renal and Metabolic
HypertensionPhase 2filing >= 2029
AtecturaQMF149 / Atectura / LABA + ICS / In-market Brands and Global Health
Asthma, pediatricsPhase 3Supplementary; filing >= 2029
AdakveoSEG101 / Adakveo / P-selectin / Inhibitor / In-market Brands and Global Health
Sickle cell disease, pediatricsPhase 3Supplementary; filing >= 2029
TIN816ATP modulator / Cardiovascular, Renal and Metabolic
Acute kidney injuryPhase 2Lead
pelacarsenTQJ230 / ASO targeting Lp(a) / Cardiovascular, Renal and Metabolic
CVRR (secondary prevention) in patients with elevated Lp(a)Phase 3Lead; filing 2026
ianalumabVAY736 / BAFF-R inhibitor, ADCC-mediated B-cell depletor / Immunology / Oncology: Hematology
Lupus NephritisPhase 3Supplementary; filing 2028Systemic lupus erythematosus (SLE)Phase 3Supplementary; filing 2028Systemic sclerosisPhase 2Supplementary; filing 2028
VHB937TREM2 stabilizer and activator / Neuroscience
Amyotrophic lateral sclerosisPhase 2Lead; filing >= 2029Alzheimer's diseasePhase 2Supplementary; filing >= 2029
YMI024Lead Indication / Cardiovascular, Renal and Metabolic
CVRRPhase 1
rapcabtagene autoleucelYTB323 / CD19 CAR-T / Immunology / Neuroscience / Oncology: Hematology
Rheumatoid arthritis and severe, refractory Sjögren’s diseasePhase 1Systemic sclerosisPhase 2Supplementary; filing >= 2029MyositisPhase 2Supplementary; filing >= 2029

ABBV

AbbVie

abbvie.com

42assets

ElezanumabABT-555 / RGMa / Biologic / Neuroscience
Spinal Cord InjuryPhase 2
ABBV-1758ABBV-1758 / Pyroglutamate (3pE) Aβ / Biologic / Neuroscience
Alzheimer's DiseasePhase 1
ABBV-8736ABBV-8736 / TREM1 / Biologic / Immunology
Crohn's DiseasePhase 2
EmraclidineM4 PAM / Small Molecule / Neuroscience
SchizophreniaPhase 2Alzheimer's Disease PsychosisPhase 1
ABBV-525ABBV-525 / MALT1 / Small Molecule / Oncology
Hematologic TumorsPhase 1
AtogepantQULIPTA / CGRP receptor / Small Molecule / Neuroscience
Acute MigraineSubmittedOUSMenstrual MigrainePhase 3OUS
ABBV-319ABBV-319 / CD19 / Biologic ADC / Oncology/Immunology
Hematologic TumorsPhase 1SLE/Sjogren's disease
ABBV-722ABBV-722 / LPAR1 / Small Molecule / Other Specialties
ABBV-141ABBV-141 / LPAR1 / Biologic / Other Specialties
Idiopathic Pulmonary FibrosisPhase 1
Telisotuzumab adizutecanABBV-400 / Temab-A / cMet / Biologic ADC / Oncology
Colorectal CancerPhase 3
ABBV-324ABBV-324 / Biologic ADC / Oncology
Solid TumorsPhase 1
ABBV-295ABBV-295 / Long-Acting Amylin Analog / Large Molecule / Other Specialties
ObesityPhase 1
ABBV-901ABBV-901 / Biologic ADC / Oncology
Ovarian CancerPhase 1
ABBV-706ABBV-706 / SEZ6 / Biologic ADC / Oncology
Small Cell Lung CancerPhase 2Neuroendocrine NeoplasmsPhase 1
IMGN151IMGN151 / FR-alpha / Biologic ADC / Oncology
Gynecologic CancersPhase 2
SurzetoclaxABBV-453 / BCL-2 / Small Molecule / Oncology
Relapsed/Refractory Multiple MyelomaPhase 1
ABBV-2002ABBV-2002 / PAR-2 / Biologic / Neuroscience
MigrainePhase 2
ABBV-1042ABBV-1042 / M4 Receptor / Small Molecule / Neuroscience
Bipolar ManiaPhase 1
UpadacitinibABT-494 / RINVOQ / JAK1 / Small Molecule / Immunology
Alopecia AreataSubmittedEUHidradenitis SuppurativaPhase 3Systemic Lupus ErythematosusPhase 3
ABBV-243ABBV-243 / Integrin β7 (α4β7 and αEβ7) / Biologic / Immunology
Inflammatory Bowel DiseasePhase 1
ABBV-547ABBV-547 / IL-23 / Biologic / Immunology
PsoriasisPhase 1
IcalcaprantABBV-1354 / KOR antagonist / Small Molecule / Neuroscience
Bipolar DepressionPhase 2Major Depressive DisorderPhase 2
EtentamigABBV-383 / CD3, BCMA / Biologic bispecific antibody / Oncology
Relapsed/Refractory Multiple MyelomaPhase 3AL AmyloidosisPhase 2
EpcoritamabEPKINLY / CD3, CD20 / Biologic bispecific antibody / Oncology
Relapsed/Refractory CLLPhase 2First Line Follicular LymphomaPhase 3First Line DLBCLPhase 3
Risankizumab + ABBV-382ABBV-466 / SKYRIZI + ABBV-382 / IL-23 + anti-a4b7 mAb / Biologic combination / Immunology
Crohn's DiseasePhase 2
BudigalimabABBV-181 / PD1 / Biologic / Oncology
Solid and Hematologic TumorsPhase 2
ABBV-701ABBV-701 / TL1A / Biologic / Immunology
Inflammatory Bowel DiseasePhase 1
ABBV-932ABBV-932 / DRD2/DRD3 Modulator / Small Molecule / Neuroscience
Bipolar DepressionPhase 2Generalized Anxiety DisorderPhase 2
Lutikizumab + RavagalimabABBV-9832 / IL-1a/1b + CD40 antagonist / Biologic combination / Immunology
Rheumatoid ArthritisPhase 2
ABBV-6628ABBV-6628 / C5 / Biologic / Eye Care
Geographic AtrophyPhase 1
ABBV-619ABBV-619 / CD19 / Biologic in vivo tLNP CAR-T / Immunology
Autoimmune diseasesPhase 1
LivmoniplimabABBV-151 / GARP-TGF-β1 / Biologic / Oncology
Hepatocellular CarcinomaPhase 2Solid TumorsPhase 2Non-Small Cell Lung CancerPhase 2
BretisilocinSerotonin 5-HT2A receptor / Small Molecule / Neuroscience
Major Depressive DisorderPhase 2
AGN-193408AGN-193408 / PGF2 alpha receptor / Small Molecule / Eye Care
Open Angle GlaucomaPhase 2Ocular HypertensionPhase 2
Surabgene LomparvovecABBV-RGX-314 / Gene Therapy / Eye Care
wet AMD (subretinal)Phase 3
UbrogepantUBRELVY / CGRP receptor / Small Molecule / Neuroscience
Menstrual MigrainePhase 3
ABBV-2001ABBV-2001 / BCMA, CD38, CD3 / Biologic trispecific T-cell engager / Oncology
Relapsed/Refractory Multiple MyelomaPhase 1
Pivekimab SunirinePVEK / CD123 / Biologic ADC / Oncology
BPDCNSubmittedAcute Myeloid LeukemiaPhase 2
ABBV-969ABBV-969 / PSMA, STEAP1 / Biologic ADC / Oncology
mCRPCPhase 1
ABBV-514ABBV-514 / CCR8 / Biologic / Oncology
Solid TumorsPhase 1
Mirvetuximab soravtansineELAHERE / FR-alpha / Biologic ADC / Oncology
Platinum-Sensitive Ovarian CancerPhase 3
Risankizumab + LutikizumabABBV-166 / SKYRIZI + Lutikizumab / IL-23 + IL-1a/1b / Biologic combination / Immunology
Crohn's DiseasePhase 2Psoriatic ArthritisPhase 2

ALNY

Alnylam

alnylam.com

31assets

ONPATTROpatisiran / ONPATTRO / TTR / RNAi therapeutic / Transthyretin Amyloidosis
hATTR amyloidosis with polyneuropathyCommercial
AMVUTTRAvutrisiran / AMVUTTRA / TTR / RNAi therapeutic / Transthyretin Amyloidosis
ATTR amyloidosis with cardiomyopathyCommercialhATTR amyloidosis with polyneuropathyCommercial
NucresiranTTR / RNAi therapeutic / Transthyretin Amyloidosis
ATTR amyloidosis with cardiomyopathyPhase 3hATTR amyloidosis with polyneuropathyPhase 3
Leqvioinclisiran / Leqvio / RNAi therapeutic / Cardiovascular
HypercholesterolemiaCommercialOut-licensed
ZilebesiranRNAi therapeutic / Cardiovascular
HypertensionPhase 3Partnered, Alnylam-led
Zilebesiran + REVERSIRRNAi therapeutic combination / Cardiovascular
HypertensionPhase 1Partnered, Alnylam-led
RapirosiranALN-HSD / HSD / RNAi therapeutic / Metabolic
MASHPhase 2Out-licensed
ALN-ANG3ALN-ANG3 / ANG3 / RNAi therapeutic / Metabolic
Diabetic kidney diseasePhase 2Out-licensed
ALN-4324ALN-4324 / GRB14 / RNAi therapeutic / Metabolic
Type 2 diabetes mellitusPhase 1
ALN-2232ALN-2232 / ACVR1C / RNAi therapeutic / Metabolic
Obesity & weight managementPhase 1
ALN-PNPALN-PNP / PNP / RNAi therapeutic / Metabolic
NAFLDPhase 1Partner-led
ALN-APOC3ALN-APOC3 / APOC3 / RNAi therapeutic / Metabolic
DyslipidemiaPhase 1Out-licensed
ALN-CIDEBALN-CIDEB / CIDEB / RNAi therapeutic / Metabolic
MASHPhase 1Out-licensed
Cemdisiran +/- PozelimabRNAi therapeutic combination / Neuroscience/Other
Myasthenia gravisPhase 3Out-licensedGeographic atrophyPhase 3Out-licensed
MivelsiranALN-APP / APP / RNAi therapeutic / Neuroscience
Cerebral amyloid angiopathyPhase 2Alzheimer's diseasePhase 1
ALN-HTT02ALN-HTT02 / HTT / RNAi therapeutic / Neuroscience
Huntington's diseasePhase 1Partnered, Alnylam-led
ALN-5288ALN-5288 / MAPT / RNAi therapeutic / Neuroscience
Alzheimer's diseasePhase 1Partnered, Alnylam-led
ALN-SODALN-SOD / SOD1 / RNAi therapeutic / Neuroscience
SOD1 ALSPhase 1Partner-led
ALN-SNCAALN-SNCA / SNCA / RNAi therapeutic / Neuroscience
Parkinson's diseasePhase 1Out-licensed
Qfitliafitusiran / Qfitlia / RNAi therapeutic / Hematology
Hemophilia A or BCommercialOut-licensed
Cemdisiran + PozelimabRNAi therapeutic combination / Hematology
Paroxysmal nocturnal hemoglobinuriaPhase 3Out-licensed
ALN-6400ALN-6400 / PLG / RNAi therapeutic / Hematology
Bleeding disordersPhase 2
AG-236ALN-TMP / TMP / RNAi therapeutic / Hematology
Polycythemia veraPhase 1Out-licensed
ALN-CFBALN-CFB / CFB / RNAi therapeutic / Hematology
Paroxysmal nocturnal hemoglobinuriaPhase 1Out-licensed
GIVLAARIgivosiran / GIVLAARI / RNAi therapeutic / Other
Acute hepatic porphyriaCommercial
OXLUMOlumasiran / OXLUMO / RNAi therapeutic / Other
Primary hyperoxaluria type 1Commercial
Elebsiran + TobevibartRNAi therapeutic combination / Other
Hepatitis D virus infectionPhase 2Out-licensed
ALN-BCATALN-BCAT / BCAT / RNAi therapeutic / Other
Hepatocellular carcinomaPhase 1
ALN-4285ALN-4285 / RNAi therapeutic / Other
Healthy volunteersPhase 1
ALN-4915ALN-4915 / RNAi therapeutic / Other
Healthy volunteersPhase 1
ALN-F1202ALN-F1202 / RNAi therapeutic / Other
Healthy volunteersPhase 1Out-licensed

AMGN

Amgen

amgenpipeline.com

30assets

erenumab-aooeAIMOVIG / CGRP receptor / Monoclonal antibody / Neuroscience
Pediatric migraineInvestigational
blinatumomabBLINCYTO / CD19 x CD3 / BiTE molecule / Hematology/Oncology; Inflammation
Newly diagnosed B-cell precursor acute lymphoblastic leukemiaInvestigationalRelapsed/refractory acute lymphoblastic leukemia, subcutaneous administrationPhase 2InvestigationalSystemic lupus erythematosus with and without nephritisInvestigational
dazodalibepCD40L / Fusion protein / Rare Disease
Sjögren’s diseaseInvestigational
romosozumab-aqqgEVENITY / Sclerostin / Monoclonal antibody / Bone
Pediatric osteogenesis imperfectaInvestigational
tarlatamab-dlleIMDELLTRA / DLL3 x CD3 / BiTE molecule / Hematology/Oncology
Small cell lung cancer, limited-stage and extensive-stage settingsInvestigationalSmall cell lung cancer, combination studiesInvestigationalSmall cell lung cancer, subcutaneous administrationPhase 1Investigational
pegloticaseKRYSTEXXA / Uric acid / PEGylated uricase / Rare Disease
Uncontrolled gout, subcutaneous administration with methotrexateInvestigational
sotorasibLUMAKRAS / KRAS G12C / Small molecule / Hematology/Oncology
KRAS G12C-mutated metastatic colorectal cancer, first line combinationInvestigationalPreviously treated advanced KRAS G12C-mutated non-small cell lung cancerInvestigationalNewly diagnosed advanced KRAS G12C-mutated NSCLC, combinationInvestigational
maridebart cafraglutideAMG 133 / MariTide / GLP-1 receptor; GIP receptor / Antibody-peptide conjugate / Cardiometabolic; Diabetes
ObesityInvestigationalCardiovascular risk reduction in ASCVD with obesity or overweightInvestigationalHeart failure events and cardiovascular risk reduction in HFpEF/HFmrEF with obesityInvestigational
romiplostimNPLATE / Thrombopoietin receptor / Peptibody / Hematology/Oncology
Chemotherapy-induced thrombocytopeniaInvestigational
olpasiranAMG 890 / Lp(a) / siRNA / Cardiometabolic
Atherosclerotic cardiovascular diseaseInvestigational
apremilastOTEZLA / PDE4 / Small molecule / Inflammation
Juvenile psoriatic arthritisInvestigationalPediatric Behcet’s diseaseInvestigational
etelcalcetidePARSABIV / Calcium-sensing receptor / Peptide / Nephrology
Pediatric secondary hyperparathyroidism in CKD receiving hemodialysisInvestigational
evolocumabREPATHA / PCSK9 / Monoclonal antibody / Cardiometabolic
Hypercholesterolemia in high cardiovascular risk without prior MI or strokeInvestigational
avacopanTAVNEOS / C5aR1 / Small molecule / Rare Disease
Pediatric ANCA-associated vasculitisInvestigational
teprotumumab-trbwTEPEZZA / IGF-1R / Monoclonal antibody / Rare Disease
Thyroid eye disease, subcutaneous administrationInvestigational
tezepelumab-ekkoTEZSPIRE / TSLP / Monoclonal antibody / Inflammation
Chronic obstructive pulmonary diseaseInvestigationalEosinophilic esophagitisInvestigational
xaluritamigAMG 509 / STEAP1 x CD3 / XmAb bispecific T-cell engager antibody / Hematology/Oncology
Prostate cancerInvestigationalEwing sarcomaInvestigational
ABP 206ABP 206 / PD-1 / Monoclonal antibody biosimilar / Hematology/Oncology
Investigational biosimilar to OPDIVO (nivolumab)Investigational biosimilar
ABP 234ABP 234 / PD-1 / Monoclonal antibody biosimilar / Hematology/Oncology
Investigational biosimilar to KEYTRUDA (pembrolizumab)Investigational biosimilar
ABP 692ABP 692 / CD20 / Monoclonal antibody biosimilar / Inflammation
Investigational biosimilar to OCREVUS (ocrelizumab)Investigational biosimilar
daxdilimabILT7 / Monoclonal antibody / Rare Disease
Dermatomyositis and anti-synthetase inflammatory myositisInvestigationalDiscoid lupus erythematosusInvestigational
inebilizumabUPLIZNA / CD19 / Monoclonal antibody / Inflammation
Systemic lupus erythematosus with nephritisInvestigational
carfilzomibKYPROLIS / Proteasome / Small molecule / Hematology/Oncology
Pediatric acute lymphoblastic leukemiaInvestigational
AMG 104AMG 104 / TSLP / Monoclonal antibody Fab / Inflammation
AsthmaInvestigational
AMG 732AMG 732 / IGF-1R / Monoclonal antibody / Rare Disease
Thyroid eye diseaseInvestigational
AMG 355AMG 355 / CCR8 / Monoclonal antibody / Hematology/Oncology
Advanced solid tumor malignanciesInvestigational
AMG 410AMG 410 / Small molecule / Hematology/Oncology
Solid tumorsInvestigational
AMG 436AMG 436 / Small molecule / Hematology/Oncology
Advanced solid tumor malignanciesInvestigational
AMG 513AMG 513 / Cardiometabolic
ObesityInvestigational
AMG 691AMG 691 / Monoclonal antibody / Inflammation
AsthmaInvestigational

NVO

Novonordisk

novonordisk.com

30assets

Glucose sensitive insulinNN1845 / Analogue / Diabetes
Type 1 and 2 diabetesPhase 1
Ventus NLRP3iNN6022 / NLRP3 / Inhibitor / Diabetes
CVDPhase 1
siRNA GalXC-GYS2siRNA / Diabetes
siRNA GalXC-GYS2Phase 1
CNPCNP / Analogue / Diabetes
Heart failurePhase 1
Amylin 355Analogue / Obesity
ObesityPhase 1
Amylin 1213Analogue / Obesity
ObesityPhase 1
SLC25A5SLC25A5 / siRNA / Obesity
MASHPhase 1
Inno8Antibody fragment / Rare Blood Disorders
HaemophiliaPhase 1
Subcutaneous ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / Obesity
Subcutaneous ZenagamtidePhase 2ObesityPhase 2
Monlunabant in DKDCB1 receptor / Small molecule / Diabetes
Diabetic Kidney Disease (DKD)Phase 2
Oral ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / Obesity
DiabetesPhase 2ObesityPhase 2
CDR132LmiR-132 / Antisense oligonucleotide / Diabetes
Heart failure with reduced ejection fraction (HFrEF)Phase 2
UBT251 (GGG tri-agonist)Obesity
ObesityPhase 2
TripleGLP1 / GIP / amylin / Tri-agonist / Obesity
ObesityPhase 2
MonlunabantINV-202 / CB1 receptor / Small molecule / Obesity
ObesityPhase 2
NDecEPI01 / Rare Blood Disorders
Sickle cell disease / An oral combination of decitabine and tetrahydrouridine. Project is developed in collaboration with EpiDestiny.Phase 2
EtavopivatRare Blood Disorders
ThalassemiaPhase 2
CagriSema in T2DNN9388 / Analogue / Diabetes
Type 2 diabetesPhase 3
Ziltivekimab ASCVDNN6018 / Diabetes
Atherosclerotic cardiovascular disease (ASCVD) with chronic kidney disease (CKD) and inflammationPhase 3
CoramitugPRX004 / Diabetes
ATTR CardiomyopathyPhase 3
Ziltivekimabin HFpEFDiabetes
Heart failure with preserved ejection fraction (HFpEF)Phase 3
Ziltivekimab AMIDiabetes
Acute Myocardial infarction (AMI)Phase 3
CagrilintideAnalogue / Obesity
ObesityPhase 3
Efruxifermin in MASHFGF21 / Analogue / Obesity
MASHPhase 3
Etavopivat (Sickle Cell Disease)Small molecule / Rare Blood Disorders
Sickle Cell DiseasePhase 3
IcosemaNN1535 / Analogue / Diabetes
Type 2 diabetes / A combination of GLP-1 analogue semaglutide and insulin icodec intended for once-weekly treatment.Filed
Insulin icodecNN1436 / Analogue / Diabetes
DiabetesFiled
CagriSemaNN9898 / Analogue / Obesity
ObesityFiled
Semaglutide 7.2 mgAnalogue / Obesity
ObesityFiled
DenecimigMim8 / Inhibitor / Rare Blood Disorders
Haemophilia A with or without inhibitors / A next generation FVIII mimetic bispecific antibody for subcutaneous prophylaxis of haemophilia A regardless of inhibitor status.Filed

GSK

gsk.com

26assets

ArexvyArexvy / RSV F protein / Recombinant protein, adjuvanted vaccine / Infectious diseases
RSV adults (50-59 years of age at increased risk)Phase III/Registration
gepotidacinGSK2140944 / Bacterial topoisomerase inhibitor / Small molecule / Infectious diseases
Urogenital gonorrhoeaPhase III/Registration
bepirovirsenGSK3228836 / HBsAg / Antisense oligonucleotide / Infectious diseases
Chronic HBV infectionPhase III
BexseroBexsero / MenB / Recombinant protein, OMV vaccine / Infectious diseases
Meningitis B (infants US)Phase III/RegistrationUS
MenABCWY vaccineGSK3536819 / Meningococcal ABCWY / Recombinant protein, OMV, conjugated vaccine / Infectious diseases
Meningococcal diseasePhase III/Registration
Nucala (mepolizumab)Nucala / IL-5 / Monoclonal antibody / Respiratory/Immunology
COPD with eosinophilic phenotypePhase III/Registration
depemokimabGSK3511294 / IL-5 / Monoclonal antibody / Respiratory/Immunology
Asthma with type 2 inflammationPhase III/RegistrationChronic rhinosinusitis with nasal polypsPhase III/RegistrationEGPAPhase III
camlipixantGSK4523199 / P2X3 / Small molecule antagonist / Respiratory/Immunology
Refractory chronic coughPhase III
linerixibatGSK2330672 / IBAT / Small molecule / Respiratory/Immunology
Cholestatic pruritus in primary biliary cholangitisPhase III
Blenrep (belantamab mafodotin)GSK2857916 / Blenrep / BCMA / Antibody-drug conjugate / Oncology
Relapsed/refractory multiple myelomaPhase III/Registration
Jemperli (dostarlimab)Jemperli / PD-1 / Monoclonal antibody / Oncology
Endometrial cancerPhase III/RegistrationRectal cancer (dMMR)Phase IINon-small cell lung cancerPhase III
Zejula (niraparib)Zejula / PARP / Small molecule / Oncology
Ovarian cancerPhase III/Registration
belrestotugGSK4428859 / TIGIT / Monoclonal antibody / Oncology
NSCLC and other solid tumorsPhase III
GSK5764227GSK5764227 / B7-H3 / Antibody-drug conjugate / Oncology
Extensive-stage small-cell lung cancerPhase IIIAdvanced GI solid tumorsPhase I/II
cobolimabGSK4069889 / TIM-3 / Monoclonal antibody / Oncology
Non-small cell lung cancerPhase II/III
GSK4532990GSK4532990 / HSD17B13 / RNA interference / Opportunity driven
NASH/MASHPhase II
GSK4172239GSK4172239 / DNMT1 / Small molecule inhibitor / Opportunity driven
Sickle cell diseasePhase I
GSK3882347GSK3882347 / FimH / Small molecule antagonist / Infectious diseases
Uncomplicated UTIPhase I
GSK3923868GSK3923868 / PI4K beta / Small molecule inhibitor / Infectious diseases
Rhinovirus diseasePhase I
GSK3965193GSK3965193 / PAPD5/PAPD7 / Small molecule inhibitor / Infectious diseases
Chronic HBV infectionPhase I
GSK2556286GSK2556286 / Mtb cholesterol-dependent / Small molecule inhibitor / Infectious diseases
TuberculosisPhase I
GSK3772701GSK3772701 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseases
MalariaPhase I
GSK4024484GSK4024484 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseases
MalariaPhase I
GSK3536867GSK3536867 / Salmonella typhoid + paratyphoid A / Bivalent conjugate vaccine / Infectious diseases
Salmonella (typhoid + paratyphoid A)Phase I
latiglutenase / AMG 133-like programs
AGN-151586 / progranulin program (latozinemab)AL001 / Progranulin/sortilin / Monoclonal antibody / Opportunity driven
Frontotemporal dementia with progranulin gene mutationPhase III

ONC

Beone Medicines

beonemedicines.com

26assets

ZanubrutinibBTK / Inhibitor / Hematologic Malignancies / Immunology & Inflammation
Treatment-Naive Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Waldenström Macroglobulinemia and Treatment-Naïve Waldenström MacroglobulinemiaApproved
SonrotoclaxBCL2 / Inhibitor / Hematologic Malignancies
Treatment-Naive Chronic Lymphocytic LeukemiaPhase 3Treatment-Naive Chronic Lymphocytic LeukemiaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaApproved
TislelizumabPD-1 / Monoclonal Antibody / Lung Cancers / Gastrointestinal Cancers / Other Cancers
First-Line Extensive-Stage Small Cell Lung CancerApprovedFirst-Line Non-Squamous Non-Small Cell Lung CancerApprovedFirst-Line Squamous Non-Small Cell Lung CancerApproved
BGB-16673BTK Chimeric Degradation Activation Compound [CDAC] / Hematologic Malignancies / Immunology & Inflammation
Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Mantle Cell LymphomaPhase 2
BlinatumomabCD19 x CD3 / BiTE® [Bispecific T-Cell Engager] / Hematologic Malignancies
Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (subcutaneous formulation)Phase 2
TarlatamabDLL3 x CD3 / BiTE® [Bispecific T-cell Engager] / Lung Cancers
First-Line Extensive-Stage Small Cell Lung CancerPhase 3First-Line Extensive-Stage Small Cell Lung CancerPhase 3Second-Line Small Cell Lung CancerPhase 3
ZanidatamabHER2 Bispecific / Antibody / Gastrointestinal Cancers
First-Line HER2 Positive Gastroesophageal AdenocarcinomaPhase 3Second-Line HER2 Positive Biliary Tract CancerApprovedFirst-Line HER2 positive Biliary Tract CancerPhase 3
BG-68501CDK2 / Inhibitor / Breast/Gynecologic Cancers
Breast Cancer and Solid TumorsPhase 1
BGB-21447BCL2 / Inhibitor / Breast/Gynecologic Cancers
Hormone Receptor Positive Breast CancerPhase 1
BGB-43395CDK4 / Inhibitor / Breast/Gynecologic Cancers
Breast Cancer and Solid TumorsPhase 1
BGB-B455Claudin 6 x CD3 Bispecific / Antibody / Breast/Gynecologic Cancers
Gynecologic and Solid TumorsPhase 1
BG-C9074B7-H4 Antibody Drug Conjugate / Breast/Gynecologic Cancers
Breast Cancer and Solid TumorsPhase 1
BG-75202KAT6A/B / Inhibitor / Breast/Gynecologic Cancers
Breast CancerPhase 1
BG-75098CDK2 Chimeric Degradation Activation Compound [CDAC] / Breast/Gynecologic Cancers
Breast CancerPhase 1
BGB-B2033GPC3 x 4-1BB Bispecific / Antibody / Gastrointestinal Cancers
Solid TumorsPhase 1
BG-C137FGFR2b Antibody Drug Conjugate / Gastrointestinal Cancers
Gastrointestinal CancersPhase 1
BGB-53038Pan-KRAS / Inhibitor / Gastrointestinal Cancers
Lung Cancers and GI CancersPhase 1
BG-C477CEA Antibody Drug Conjugate / Gastrointestinal Cancers
Lung Cancers and GI CancersPhase 1
BG-C0902EGFR x MET x MET Antibody Drug Conjugate / Lung Cancers
Lung Cancers and GI CancersPhase 1
BG-T187EGFR x MET x MET Trispecific / Antibody / Lung Cancers
Lung Cancers and GI CancersPhase 1
BGB-58067MTA Cooperative PRMT5 / Inhibitor / Lung Cancers
Lung Cancers and GI CancersPhase 1
BG-89894MAT2A / Inhibitor / Lung Cancers
Lung Cancers and Solid TumorsPhase 1
BG-60366EGFR Chimeric Degradation Activation Compound [CDAC] / Lung Cancers
Lung CancersPhase 1
BGB-26808HPK1 / Inhibitor / Gastrointestinal Cancers
Solid Tumors/Pan- TumorPhase 1
XaluritamigSTEAP1 x CD3 Bispecific T-cell Engager (XmAb®) / Other Cancers
Metastatic Castration-Resistant Prostate CancerPhase 1
BGB-45035IRAK4 Chimeric Degradation Activation Compound [CDAC] / Immunology & Inflammation
Rheumatoid ArthritisPhase 2Skin DiseasesPhase 1

BNTX

Biontech

biontech.com

21assets

PumitamigBNT327/PM8002 / PD-L1 x VEGF-A / bispecific antibody / Oncology
Multiple solid tumorsPhase 1active2L ES-SCLCactiveChina2L+ EGFRm NSCLCactiveChina
BNT325/DB-1305BNT325/DB-1305 / targeted therapy / ADC / Oncology
Multiple solid tumorsactive
BNT324/DB-1311BNT324/DB-1311 / targeted therapy / ADC / Oncology
Multiple solid tumorsPhase 1/2active
BNT116BNT116 / mRNA cancer immunotherapy / Oncology
Advanced NSCLCPhase 1active1L advanced NSCLCPhase 2active
Autogene cevumeranBNT122/RO7198457 / individualized neoantigens / mRNA cancer immunotherapy / Oncology
Adjuvant CRCPhase 2/3activeAdjuvant PDACPhase 2active
BNT113BNT113 / HPV16 / mRNA cancer immunotherapy / Oncology
1L HPV16+ HNSCCPhase 2active
GotistobartBNT316/ONC-392 / CTLA-4 / immunomodulator antibody / Oncology
Metastatic NSCLCactiveMetastatic CRPCactiveMultiple solid tumorsactive
BNT211BNT211 / CLDN6 / CAR-T cell therapy plus CARVac / Oncology
Multiple solid tumorsPhase 1/2active
BNT314/GEN1059BNT314/GEN1059 / immunomodulator antibody / Oncology
Multiple solid tumorsactiveMetastatic CRCactive
BNT329BNT329 / next-generation immunomodulator / Oncology
Multiple solid tumorsactive
BNT326/YL202BNT326/YL202 / targeted therapy / ADC / Oncology
Multiple solid tumorsactiveAdvanced/metastatic breast canceractiveChina
BNT317BNT317 / immunomodulator / Oncology
Multiple solid tumorsactive
Trastuzumab pamirtecanBNT323/DB-1303 / HER2 / targeted therapy / ADC / Oncology
Metastatic breast canceractive2L endometrial canceractiveMultiple solid tumorsactive
BNT321BNT321 / targeted therapy / Oncology
Multiple solid tumorsactive
BNT162 + BNT161BNT162 + BNT161 / SARS-CoV-2; influenza / mRNA vaccine combination / Infectious Diseases
COVID-19-influenza combinationPhase 1active
BNT163BNT163 / HSV / mRNA vaccine / Infectious Diseases
HSVPhase 1active
BNT166BNT166 / Mpox / mRNA vaccine / Infectious Diseases
MpoxPhase 1/2active
BNT162BNT162 / COMIRNATY / SARS-CoV-2 / mRNA vaccine / Infectious Diseases
COVID-19Commercialmarketed / active development
BNT351BNT351 / HIV / mRNA vaccine / Infectious Diseases
HIVPhase 1/2active
BNT164BNT164 / Tuberculosis / mRNA vaccine / Infectious Diseases
TuberculosisPhase 1/2active
BNT165BNT165 / Malaria / mRNA vaccine / Infectious Diseases
MalariaPhase 1/2active

ARWR

Arrowhead Pharma

arrowheadpharma.com

19assets

PlozasiranARO-APOC3 / REDEMPLO / APOC3 / RNAi therapeutic / Cardiometabolic
Familial chylomicronemia syndromeIn MarketSevere hypertriglyceridemiaPhase 3ActiveHypertriglyceridemiaPhase 3Active
ZodasiranARO-ANG3 / ANGPTL3 / RNAi therapeutic / Cardiometabolic
Homozygous familial hypercholesterolemiaPhase 3Recruiting
OlpasiranAMG 890; ARO-LPA / Apolipoprotein(a) / RNAi therapeutic / Cardiovascular disease
Cardiovascular disease with elevated lipoprotein(a)Phase 3Recruiting/Active
GSK4532990ARO-HSD / HSD17B13 / RNAi therapeutic / Liver disease
MASH/NASHPhase 2Recruiting
ARO-DIMER-PAPCSK9; APOC3 / dual-functional RNAi molecule / Cardiometabolic
Mixed hyperlipidemiaPhase 1Recruiting
ARO-PNPLA3JNJ-75220795 / PNPLA3 / RNAi therapeutic / Liver disease
MASH/NASHPhase 1
ARO-INHBEINHBE / RNAi therapeutic / Obesity
ObesityPhase 1Recruiting
ARO-ALK7ACVR1C/ALK7 / RNAi therapeutic / Obesity
ObesityPhase 1Recruiting
ARO-MAPTMAPT / RNAi therapeutic / Central nervous system
Alzheimer's diseasePhase 1Recruiting
ARO-ATXN2ATXN2 / RNAi therapeutic / Central nervous system
Spinocerebellar ataxia 2Phase 1Recruiting
SRP-1005ARO-HTT / HTT / siRNA therapeutic / Central nervous system
Huntington's diseasePhase 1
ARO-RAGERAGE / RNAi therapeutic / Pulmonary
Inflammatory pulmonary diseasesPhase 2Active
ARO-MMP7MMP7 / RNAi therapeutic / Pulmonary
Idiopathic pulmonary fibrosisPhase 1Complete
FazirsiranARO-AAT; TAK-999 / Z-AAT / RNAi therapeutic / Liver disease
Alpha-1 antitrypsin deficiency-associated liver diseasePhase 3Recruiting/Active
ARO-HBVJNJ-73763989 / HBV gene products / RNAi therapeutic / Infectious disease
Chronic hepatitis BPhase 2Complete
ARO-DUX4DUX4 / RNAi conjugate / Muscular disease
Facioscapulohumeral muscular dystrophy type 1Phase 2Recruiting
ARO-DM1DMPK / RNAi conjugate / Muscular disease
Myotonic dystrophy type 1Phase 2Recruiting
ARO-C3Complement component 3 / RNAi therapeutic / Complement mediated disease
C3 glomerulopathy; IgA nephropathyPhase 1Recruiting
ARO-CFBComplement factor B / RNAi therapeutic / Complement mediated disease
Complement mediated diseasePhase 1

GILD

Gilead

gilead.com

19assets

LenacapavirHIV capsid inhibitor / Long-acting injectable / Virology
HIV Prevention (PrEP, PURPOSE 365)Phase 3HIV Treatment (in combinations)Phase 3HIV Treatment (with bNAbs)Phase 2
Bictegravir/lenacapavir oral combinationINSTI + capsid inhibitor / Oral / Virology
HIV Treatment (virologically suppressed, ARTISTRY-1 & ARTISTRY-2)Phase 3
Islatravir/lenacapavir oral combinationLong-acting oral / Virology
HIV Treatment (virologically suppressed, ISLEND-1 & ISLEND-2)Phase 3
Lenacapavir + teropavimab + zinlirvimabCapsid + bNAbs / Long-acting injectable / Virology
HIV Treatment (virologically suppressed)Phase 2
GS-1720/GS-4182GS-1720/GS-4182 / INSTI/capsid inhibitor / Long-acting oral / Virology
HIV Treatment (WONDERS-1 & -2)Phase 2
Teropavimab + zinlirvimabbNAbs / Broadly neutralizing antibodies / Virology
HIV CurePhase 2
VesatolimodVirology
HIV Cure (FRESH)Phase 2
SelgantolimodVirology
HBV CurePhase 2
HepcludexHepcludex / Virology
Chronic HDV with compensated liver disease (MYR301)FiledBLA pending (US); Approved (EU)USEU
EdecesertibInflammation
Lupus (COSMIC)Phase 2
Tilpisertib fosmecarbilInflammation
Inflammatory bowel disease (PALEKONA)Phase 2
α4β7 inhibitorα4β7 / Inflammation
Inflammatory Bowel Disease (SWIFT)Phase 2
Sacituzumab govitecan-hziyAntibody-drug conjugate / Oncology
1L metastatic triple-negative breast cancer (PD-L1-, ASCENT-03)FiledsBLA pendingUS1L metastatic triple-negative breast cancer (PD-L1+, with pembrolizumab, ASCENT-04)FiledsBLA pendingUSHigh risk adjuvant triple-negative breast cancer (with pembrolizumab, ASCENT-05)Phase 3
Axicabtagene ciloleucelCD19 CAR / Cell therapy / Oncology
2L+ high-risk follicular lymphoma (ZUMA-22)Phase 31L high-risk large B-cell lymphoma (ZUMA-23)Phase 3
Brexucabtagene autoleucelCD19 CAR / Cell therapy / Oncology
Pediatric acute lymphocytic leukemia/non-Hodgkin lymphoma (ZUMA-4)Phase 2
Anitocabtagene autoleucelCell therapy / Oncology
2-4L relapsed/refractory multiple myeloma (iMMagine-3)Phase 34L+ relapsed/refractory multiple myeloma (iMMagine-1)FiledBLA pendingUS
KITE-363KITE-363 / CD19/CD20 bicistronic / Cell therapy / Oncology
Relapsed/refractory diffuse large B-cell lymphomaPhase 1RMAT designationRheumatology (autoimmune)Phase 1
DenikitugGS-1811 / Oncology
Advanced cancersPhase 1
Domvanalimab + zimberelimabAnti-TIGIT + anti-PD1 / Combination therapy / Oncology
1L metastatic non-small cell lung cancer (with chemo, STAR-121)Phase 31L head and neck squamous cell carcinoma (VELOCITY-HNSCC)Phase 2

IONS

Ionis

ionis.com

19assets

ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / Neurology
Alexander DiseasePhase 1/3ActiveGlobal
ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / Neurology
Angelman SyndromeClinicalActive
ION269ION269 / APP / RNA-Targeted Therapeutic / Neurology
Alzheimer's DiseaseClinicalActive
ION717ION717 / PRNP / RNA-Targeted Therapeutic / Neurology
Prion DiseaseClinicalActive
ION356ION356 / PLP1 / RNA-Targeted Therapeutic / Neurology
Pelizaeus-Merzbacher DiseaseClinicalActive
ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / Neurology
Parkinson's DiseaseClinicalActive
ION464ION464 / SNCA / RNA-Targeted Therapeutic / Neurology
Multiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
ION440ION440 / MECP2 / RNA-Targeted Therapeutic / Neurology
MECP2 Duplication SyndromeClinicalActive
OlezarsenApoC-III / RNA-Targeted Therapeutic / Cardiology
Severe HypertriglyceridemiaClinicalActive
DonidalorsenPKK / RNA-Targeted Therapeutic / Specialty/Rare
Hereditary AngioedemaClinicalActive
SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/Rare
Polycythemia VeraClinicalActive
UlefnersenFUS / RNA-Targeted Therapeutic / Neurology
Amyotrophic Lateral SclerosisClinicalActive
TofersenSOD1 / RNA-Targeted Therapeutic / Neurology
Amyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / Neurology
Alzheimer's DiseaseClinicalActive
TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / Neurology
Huntington's DiseasePhase 2Active
ION306ION306 / SMN2 / RNA-Targeted Therapeutic / Neurology
Spinal Muscular AtrophyClinicalActive
EplontersenTTR / RNA-Targeted Therapeutic / Cardiology
Transthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / Cardiology
Treatment-Resistant HypertensionClinicalActive
BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other Medicines
Hepatitis B Virus InfectionPhase 3Active

ALPMY

Astellas

astellas.com

18assets

zolbetuximabIMAB362 / VYLOY / Claudin 18.2 / Antibody / Oncology
Gastric and gastroesophageal junction adenocarcinoma, combo with pembrolizumab and chemotherapyFiledEuropeJapan
gilteritinibASP2215 / XOSPATA / FLT3 / Small molecule / Oncology
Newly diagnosed acute myeloid leukemia with high intensity induction chemotherapyPhase 3Post-chemotherapy maintenance acute myeloid leukemiaPhase 3Post-hematopoietic stem cell transplant maintenance acute myeloid leukemiaPhase 3
enfortumab vedotinASG-22ME / PADCEV / Nectin-4 / Antibody-drug conjugate (ADC) / Oncology
Cisplatin-eligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeUSCisplatin-ineligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeJapanBladder-sparing muscle-invasive bladder cancer, combo with pembrolizumabPhase 2
fezolinetantESN364 / VEOZAH / NK3 receptor / Small molecule / Women's Health
Vasomotor symptoms due to menopausePhase 3ChinaVasomotor symptoms in breast cancer patients on adjuvant endocrine therapyPhase 2
zocaglusagene nuzaparvovecAT845 / GAA gene replacement / Gene therapy (AAV-based gene therapy) / Genetic Regulation
Pompe diseasePhase 1
ASP2957ASP2957 / MTM1 gene replacement / Gene therapy (AAV-based gene therapy) / Genetic Regulation
X-linked myotubular myopathyPhase 1
setidegrasibKRAS G12D / Small molecule degrader / Targeted Protein Degradation
Pancreatic ductal adenocarcinomaPhase 3
ASP7317ASP7317 / Retinal pigment epithelial cells / Cell therapy / Ophthalmology
Geographic atrophy secondary to age-related macular degenerationPhase 1
ASP1002ASP1002 / Claudin 4/CD137 / T-cell engager bispecific antibody / Oncology
Gastric and gastroesophageal junction adenocarcinoma; pancreatic adenocarcinomaPhase 1
ASP5834ASP5834 / Pan-KRAS / Small molecule degrader / Oncology
CancerPhase 1
VIR-5500VIR-5500 / PSMA/CD3 / T-cell engager / Oncology
Prostate cancerPhase 1
ASP2998ASP2998 / TROP2 / Antibody-drug conjugate (ADC) / Oncology
CancerPhase 1
ASP2138ASP2138 / Claudin 18.2/CD3 / T-cell engager bispecific antibody / Oncology
CancerPhase 2
ASP546CASP546C / Claudin 18.2 / Antibody-drug conjugate (ADC) / Oncology
CancerPhase 2Europe
abiraterone decanoateASP5541/PRL-02 / CYP17 lyase / Small molecule / Oncology
Prostate cancerPhase 2
mirabegronYM178 / Beta-3 receptor / Small molecule / Urology
Neurogenic detrusor overactivity in pediatric patients aged 6 months to less than 3 yearsPhase 3Europe
roxadustatASP1517/FG-4592 / HIF-PH / Small molecule / Nephrology
Anemia associated with chronic kidney disease in pediatric patientsPhase 3Europe
pudexacianinium chlorideASP5354 / Drug-device combination / Other
Intraoperative ureter visualization for minimally invasive and open abdominopelvic surgeriesPhase 3

AMRX

Amneal

amneal.com

18assets

Mesalamine DRAsacol HD / generic small molecule / Gastroenterology
EverolimusAfinitor / mTOR / generic small molecule / Oncology
Prednisolone acetatePred-Forte / generic small molecule / Ophthalmology
EltrombopagPromacta / TPO receptor agonist / generic small molecule / Hematology
EpinephrineAdrenalin / adrenergic receptors / generic small molecule / Emergency/Critical Care
LenalidomideRevlimid / generic small molecule / Oncology
Risperidone ERRisperdal Consta / dopamine/serotonin receptors / generic long-acting injectable / Psychiatry
Romidepsin injectionRomidepsin / HDAC / generic small molecule / Oncology
CyclosporineRestasis / calcineurin / generic small molecule / Ophthalmology
Beclomethasone dipropionateQVAR / glucocorticoid receptor / generic inhaled corticosteroid / Respiratory
Sodium BicarbonateNeut / generic injectable / Critical Care
Lanreotide injectionSomatuline Depot / somatostatin receptors / generic peptide injectable / Endocrinology/Oncology
RifaximinXifaxan / generic small molecule / Gastroenterology
Omalizumab biosimilarXOLAIR / IgE / biosimilar monoclonal antibody / Immunology/Allergy
Sodium oxybateXyrem / generic small molecule / Neurology
BimatoprostLumigan / prostaglandin receptor / generic small molecule / Ophthalmology
Denosumab biosimilarsPROLIA & XGEVA / RANKL / biosimilar monoclonal antibody / Osteoporosis/Bone Cancer
Albuterol sulfateProAir HFA / beta-2 adrenergic receptor / generic inhaled small molecule / Respiratory

BMY

Bristol Myers Squibb

bms.com

18assets

AR LDDandrogen receptor / ligand-directed degrader / Oncology
Metastatic castration-resistant prostate cancerPhase III
atigotatug + nivolumabantibody combination / Oncology
1L extensive-stage small cell lung cancerPhase III
KRAZATIKRAZATI / KRAS G12C / small molecule / Oncology
1L non-small cell lung cancerPhase III1L non-small cell lung cancer PD-L1≥50%Phase III
nivolumab + relatlimab HDPD-1 + LAG-3 / antibody combination / Oncology
1L non-small cell lung cancer PD-L1≥1%Phase III
OPDIVOnivolumab / OPDIVO / PD-1 / monoclonal antibody / Oncology
Adjuvant hepatocellular carcinomaPhase III1L classical Hodgkin lymphomaRegistrationRegulatory submissionEU
pumitamigBNT327/BMS-986545 / PD-L1 + VEGF-A / bispecific antibody / Oncology
1L extensive-stage small cell lung cancerPhase IIIPartner-run study1L non-small cell lung cancerPhase IIIPartner-run study1L non-small cell lung cancer PD-L1≥50%Phase III
RYZ101SSTR2 / radiopharmaceutical / Oncology
2L+ SSTR2+ gastroenteropancreatic neuroendocrine tumorsPhase III
arlo-celcell therapy / Hematology
2-4L multiple myelomaPhase III
golcadomideCELMoD / Hematology
2L+ follicular lymphomaPhase IIIHigh risk 1L large B-cell lymphomaPhase III
iberdomideCELMoD / Hematology
2L+ multiple myelomaPhase IIIPost-ASCT maintenance newly diagnosed multiple myelomaPhase III2L+ multiple myeloma [MRD]RegistrationRegulatory submissionUS
mezigdomideCELMoD / Hematology
2L+ multiple myeloma KdPhase III2L+ multiple myeloma VdPhase III
REBLOZYLluspatercept / REBLOZYL / biologic / Hematology
1L NTD myelodysplastic syndrome associated anemiaPhase III1L TD myelofibrosis associated anemiaPhase III
milvexianFactor XIa / small molecule / Cardiovascular
Atrial fibrillationPhase IIIPartner-run studySecondary stroke preventionPhase IIIPartner-run study
admilparantLPA1 / small molecule / Immunology/Fibrosis
Idiopathic pulmonary fibrosisPhase IIIProgressive pulmonary fibrosisPhase III
obexelimabCD19/FcγRIIb / bispecific antibody / Immunology
IgG4-related diseasePhase III
SOTYKTUdeucravacitinib / SOTYKTU / TYK2 / small molecule / Immunology
Sjögren's diseasePhase IIISystemic lupus erythematosusPhase IIIPsoriatic arthritisRegistrationRegulatory submissionEUJP
zola-celcell therapy / Immunology
Systemic sclerosisPhase III
COBENFYKarXT / COBENFY / muscarinic receptors / small molecule / Neuroscience
Adjunctive bipolar-I maniaPhase IIIAgitation in Alzheimer's diseasePhase IIIAlzheimer's disease cognitionPhase III

XOMA

Xoma

xoma.com

18assets

Vabysmofaricimab-svoa / VABYSMO / VEGF/Ang-2 / Bispecific antibody / Ophthalmology
Neovascular age-related macular degeneration (nAMD) and Diabetic macular edema (DME)Approvedapproved
Ojemdatovorafenib / OJEMDA / Oncology
Oncology (undisclosed approved indication)Approvedapproved
Miplyffaarimoclomol / MIPLYFFA / Rare Diseases / Neurology
Niemann-Pick disease type C (NPC)ApprovedapprovedUS
Xaciatoclindamycin phosphate vaginal gel 2% / XACIATO / Small molecule / Gynecology / Infectious Disease
Bacterial VaginosisApprovedapproved
Ixinitycoagulation factor IX (recombinant) / IXINITY / Factor IX / Recombinant protein / Hematology
Hemophilia BApprovedapproved
Dsuviasufentanil sublingual tablet / DSUVIA / Opioid receptor / Small molecule / Pain Management
PainApprovedapproved
Dare to Playsildenafil cream / DARE to PLAY / PDE5 / Small molecule / Gynecology / Sexual Health
Female Sexual Arousal DisorderApprovedapproved
ersodetugRZ358 / INSR / Antibody / Metabolic Disorders
Hypoglycemia due to congenital hyperinsulinism (cHI)Phase 3ongoingHypoglycemia due to tumor hyperinsulinismPhase 3ongoing
RZ402RZ402 / Ophthalmology
Diabetic macular edema (DME)Phase 2ongoing
D-FiFCX-007 / Gene Therapy / Dermatology / Rare Diseases
Dystrophic epidermolysis bullosa (DEB)Phase 3ongoing
seralutinibGB002 / PDGFR, CSF1R, and c-KIT / Inhaled tyrosine kinase inhibitor / Respiratory / Cardiovascular
Pulmonary arterial hypertension (PAH)Phase 3ongoingPulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)Phase 2ongoing
cetrelimabJNJ-63723283 / PD-1 / Antibody / Oncology / Infectious Disease
Muscle-invasive bladder cancer (MIBC)Phase 2ongoingChronic Hepatitis B Virus InfectionPhase 2ongoing
volixibatLUM-002 / Ileal bile acid transporter (IBAT) / Small molecule / Gastroenterology
Primary sclerosing cholangitis (PSC)Phase 2bongoingPrimary biliary cholangitis (PBC)Phase 2ongoing
osavampatorNeurology / Psychiatry
Major depressive disorderPhase 3ongoing
OHB-607OHB-607 / Recombinant human IGF-I/IGFBP-3 / Recombinant protein / Respiratory / Neonatology
Prevention of bronchopulmonary dysplasia in extremely premature infantsPhase 2ongoing
REC-4881REC-4881 / Allosteric MEK1/2 / Small molecule / Oncology
Familial adenomatous polyposisPhase 2ongoing
NIS793NIS793 / Antibody / Oncology
Solid tumorsPhase 3ongoing
QTORIN rapamycinQTORIN / mTOR / Small molecule / Dermatology / Rare Diseases
Cutaneous venous malformationsPhase 3ongoingClinically significant angiokeratomasPhase 2ongoing

NBIX

Neurocrine

neurocrine.com

17assets

direclidineM4 / Small Molecule / Psychiatry
SchizophreniaPhase 3Bipolar ManiaPhase 2
NBI-570M1/M4 / Small Molecule / Psychiatry
SchizophreniaPhase 2
osavampatorAMPA / Small Molecule / Psychiatry
Major Depressive DisorderPhase 3
NBI-986M4 / Small Molecule / Neurology
Movement DisordersPhase 1
NBI-890VMAT2 / Small Molecule / Psychiatry
Tardive DyskinesiaPhase 2
NBI-569M1/M4 / Small Molecule / Psychiatry
Alzheimer's PsychosisPhase 1
NBI-567M1 / Small Molecule / Neurology
Alzheimer's CognitionPhase 1
NBI-355Nav1.2/1.6 / Small Molecule / Neurology
EpilepsyPhase 1
NBIP-1435CRF1 / Peptide / Endocrinology
Classic Congenital Adrenal HyperplasiaPhase 1
NBIP-2118CRF2 / Peptide / Endocrinology
ObesityPhase 1
NBIP-1968TG + NBIP-2118 Combo / Peptide / Endocrinology
ObesityPhase 4
NBIB-223Frataxin / Gene Therapy / Neurology
Friedreich's AtaxiaPhase 4
NBI-675VMAT2 / Small Molecule / Psychiatry
CNS IndicationsPhase 1
NBI-188CRF1 / Small Molecule / Endocrinology
Women's HealthPhase 4
NBIM-1748Immunology
UndisclosedPhase 4
NBIM-1008Immunology
UndisclosedPhase 4
NBIM-1112Immunology
UndisclosedPhase 1

REGN

Regeneron

regeneron.com

17assets

FianlimabLAG3 / Monoclonal Antibody / Oncology
1L metastatic melanoma, adjuvant melanomaPhase 3ActiveGlobal
CemiplimabLibtayo / PD-1 / Monoclonal Antibody / Oncology
CancerPhase 2/3ActiveGlobal
DavutamigREGN5093 / METxMET / Bispecific Antibody / Oncology
CancerPhase 2ActiveGlobal
ItepekimabIL-33 / Monoclonal Antibody / Immunology & Inflammation / Respiratory
COPD, CRSwNPPhase 3ActiveGlobal
LinvoseltamabLynozyfic / BCMAxCD3 / Bispecific Antibody / Oncology
Multiple myelomaPhase 2/3ActiveGlobal
MarlotamigREGN7075 / EGFRxCD28 / Bispecific Antibody / Oncology
Solid TumorsPhase 2ActiveGlobal
OdronextamabOrdspono / CD20xCD3 / Bispecific Antibody / Oncology
LymphomaPhase 2ActiveGlobal
UbamatamabREGN4018 / MUC16xCD3 / Bispecific Antibody / Oncology
Ovarian CancerPhase 2ActiveGlobal
NezastomigREGN5678 / PSMAxCD28 / Bispecific Antibody / Oncology
Prostate CancerPhase 2ActiveGlobal
RapirosiranALN-HSD / HSD17B13 / siRNA / Cardiovascular & Metabolic
NASHPhase 2ActiveGlobal
ALN-ANG3ALN-ANG3 / ANGPTL3 / siRNA / Cardiovascular & Metabolic
DyslipidemiaPhase 2ActiveGlobal
CemdisiranC5 / siRNA / Ophthalmology / Immunology
Geographic atrophyClinicalActiveGlobal
PozelimabC5 / Monoclonal Antibody / Ophthalmology / Immunology
Geographic atrophyClinicalActiveGlobal
BetV1REGN5713-5715 / BetV1 / Monoclonal Antibody / Immunology & Inflammation
Birch AllergyClinicalActiveGlobal
DB-OTOOtarmeni / Otoferlin / Gene Therapy / Neurology / Hearing Loss
Otoferlin-related hearing lossPhase 1/2ActiveGlobal
MibavademabUnknown / Biologic / Oncology
CancerPhase 2ActiveGlobal
TrevogrumabREGN1033 / Myostatin / Monoclonal Antibody / Metabolic
Muscle weakness/wastingPhase 2ActiveGlobal

JNJ

Johnson & Johnson

investor.jnj.com

16assets

guselkumabTREMFYA / IL-23 / Monoclonal Antibody / Immunology
PsoriasisPsoriatic ArthritisCrohn's Disease
ibrutinibIMBRUVICA / BTK / Small Molecule / Oncology
Mantle Cell LymphomaOther B-cell malignancies
icotrokinraJNJ-2113 / IL-23R / Targeted Oral Peptide / Immunology
PsoriasisPsoriatic Arthritis
JNJ-4804JNJ-4804 / Co-antibody / Co-antibody Therapy / Immunology
Ulcerative Colitis
nipocalimabIMAAVY / FcRn / Monoclonal Antibody / Immunology / Neuroscience
Generalized Myasthenia GravisRheumatoid ArthritisWarm Autoimmune Hemolytic Anemia
aticaprantKOR antagonist / Small Molecule / Neuroscience
Adjunctive Treatment for Major Depressive Disorder with Anhedonia
RPGR Gene TherapyRPGR / Gene Therapy / Vision / Ophthalmology
Retinitis Pigmentosa
teclistamabTECVAYLI / BCMA x CD3 / Bispecific Antibody / Oncology
Multiple Myeloma
sCD59JNJ-1887 / sCD59 / Gene Therapy / Vision / Ophthalmology
Geographic Atrophy
gemcitabine intravesical delivery systemTAR-200 / INLEXZO / DNA synthesis / Drug Delivery System / Oncology
High Risk Non-Muscle Invasive Bladder Cancer
niraparib / abirateroneAKEEGA / PARP / CYP17 / Small Molecule Combination / Oncology
Metastatic Castration-Sensitive Prostate CancerMetastatic Castration-Resistant Prostate Cancer
daratumumabDARZALEX / CD38 / Monoclonal Antibody / Oncology
Multiple Myeloma
erdafitinibBALVERSA / FGFR / Small Molecule / Oncology
Urothelial Carcinoma
apalutamideERLEADA / Androgen Receptor / Small Molecule / Oncology
Prostate Cancer
ciltacabtagene autoleucelCARVYKTI / BCMA / CAR-T Cell Therapy / Oncology
Multiple Myeloma
Filovirus multivalent vaccineVAC69120 / MVA-BN-Filo / Ebola Virus / Vaccine / Infectious Diseases
Ebola

ZLAB

Zailaboratory

zailaboratory.com

16assets

NiraparibPARP / Inhibitor / Oncology
Ovarian Cancer (1st line maintenance)ApprovedOvarian Cancer (Platinum sensitive relapsed maintenance)Approved
Tumor Treating FieldsOncology
Glioblastoma (GBM)ApprovedPancreatic Cancer (1st line)Phase 3 / Pivotal
RipretinibTKI / Oncology
Gastrointestinal Stromal Tumors (GIST) (4th line)Approved
RepotrectinibROS1 / TRK / Oncology
ROS1+ NSCLCApprovedNTRK+ solid tumorsApproved
Tisotumab vedotinTF / ADC / Oncology
Cervical Cancer (2nd line+ r/m)Phase 3 / PivotalCervical Cancer (1st line r/m, combo)Phase 2 / POC
Zocilurtatug pelitecanZL-1310 / Zoci / DLL3 / ADC / Oncology
ES-SCLCPhase 3 / PivotalOther NECsPhase 2 / POC
ZL-6201LRRC15 / ADC / Oncology
Sarcoma & CAF+ Solid TumorPhase 1
ZL-1311MUC17xCD3 / Oncology
GI TumorsPre-clinical
ZL-1222PD-1/IL-12 / Oncology
Solid TumorsPre-clinical
EfgartigimodFcRn / Immunology
Generalized Myasthenia Gravis (gMG)ApprovedChronic Inflammatory Demyelinating Polyneuropathy (CIDP)ApprovedMyositisPhase 3 / Pivotal
PovetaciceptBAFF/APRIL / Immunology
IgA NephropathyPhase 3 / PivotalPrimary Membranous NephropathyPhase 2 / POC
ElegrobartIGF-1R / Immunology
TEDPhase 3 / Pivotal
ZL-1503IL13/IL31R / Immunology
Mod-to-Sev ADPhase 1
Xanomeline-TrospiumKarXT / Neuroscience
SchizophreniaApproved
OmadacyclineInfectious Disease
Acute Bacterial Skin and Skin Structure Infection (ABSSSI)ApprovedCommunity-Acquired Bacterial Pneumonia (CABP)Approved
Sulbactam-DurlobactamInfectious Disease
HABP/VABP caused by Susceptible Isolates of Acinetobacter Baumannii-calcoaceticus ComplexApproved

TAK

Takeda

takeda.com

15assets

ALVO

Alvotech

alvotech.com

14assets

High-concentration, low-volume adalimumabAVT02 / HUMIRA® / biosimilar biologic / Immunology
ImmunologyApprovalApprovedU.S.Europe (EEA, UK, Switzerland, Balkans)CanadaAustraliaSaudi ArabiaIsraelEgyptSouth AfricaMoroccoPeruBolivia
ustekinumabAVT04 / STELARA® / biosimilar biologic / Immunology
ImmunologyApprovalApprovedU.S.JapanCanadaEU/EEA
denosumabAVT03 / PROLIA®/XGEVA® / biosimilar biologic / Bone Disease
Bone DiseaseApprovalApprovedJapanEU/EEA
golimumabAVT05 / SIMPONI®/SIMPONI ARIA® / biosimilar biologic / Immunology
ImmunologyApprovalApprovedJapanEU/EEAUnited Kingdom
afliberceptAVT06 / EYLEA® / biosimilar biologic / Ophthalmology
OphthalmologyApprovalApprovedEU/EEAJapanUnited Kingdom
omalizumabAVT23 / XOLAIR® / biosimilar biologic / Respiratory
vedolizumabAVT16 / ENTYVIO® / biosimilar biologic / Immunology
pembrolizumabAVT33 / KEYTRUDA® / biosimilar biologic / Oncology
certolizumab pegolAVT10 / CIMZIA® / biosimilar biologic / Immunology
ixekizumabAVT28 / TALTZ® / biosimilar biologic / Immunology
canakinumabAVT48 / ILARIS® / biosimilar biologic / Immunology
guselkumabAVT41 / TREMFYA® / biosimilar biologic / Immunology
ofatumumabAVT65 / KESIMPTA® / biosimilar biologic / Immunology
dupilumabAVT19 / DUPIXENT® / biosimilar biologic / Immunology

AMPH

Amphastar

amphastar.com

14assets

AMP-105AMP-105 / peptide/NCE / Oncology
HNSCC; HCC; lymphoma; multiple myeloma; solid tumorsPreclinicalDevelopmentUS
AMP-107AMP-107 / VEGFR; integrin αvβ3 / peptide/NCE eye drop / Ophthalmology
wet AMD; diabetic macular edemaPreclinicalDevelopmentUS
AMP-109AMP-109 / peptide-coupled docetaxel / Oncology
NSCLC; colorectal; gastric; pancreatic cancersPreclinicalDevelopmentUS
AMP-110AMP-110 / ACTH receptor / synthetic human corticotropin analog / Rheumatology; Ophthalmology; Neurology
acute MS/RA exacerbations; gouty arthritis flares; ocular inflammationPhase IClinical developmentUS
AMP-018AMP-018 / GLP-1 / generic injectable / Diabetes/Metabolic
GLP-1 productCommercial launch expected in 2027US
AMP-029AMP-029 / generic injectable
UndisclosedDevelopmentUS
AMP-017AMP-017 / generic inhalation
UndisclosedPlanned filing 1H 2027US
AMP-023AMP-023 / generic inhalation
UndisclosedDevelopmentUS
AMP-004AMP-004 / Insulin aspart / interchangeable biosimilar insulin / Diabetes
Diabetes mellitusCommercial launch expected in 2027US
AMP-005AMP-005 / Recombinant human insulin / interchangeable biosimilar insulin / Diabetes
Diabetes mellitusPlanned filing 1H 2027US
AMP-028AMP-028 / biosimilar
UndisclosedDevelopmentUS
Primatene MIST with Green propellantPrimatene MIST / Epinephrine / OTC metered-dose inhaler / Respiratory
AsthmaDeveloping new green-propellant formulationUS
Intranasal epinephrineEpinephrine / intranasal formulation / Allergy/Emergency medicine
UndisclosedPipelineUS
AMP-007AMP-007 / Ipratropium bromide / generic inhalation aerosol / Respiratory
Bronchospasm/COPDApproved Feb 2026; launch planned early Q2 2026US

TLX

Telix Pharma

telixpharma.com

14assets

177Lu rosopatamab tetraxetanTLX591-Tx / PSMA / Antibody / Urologic Oncology
Prostate Cancer
177Lu-DOTA-PSMATLX597-Tx / PSMA / Small molecule / Urologic Oncology
Prostate Cancer
177Lu-girentuximabTLX250-Tx / CAIX / Antibody / Urologic Oncology
Kidney Cancer / ccRCC
131I-IPATLX101-Tx / LAT / Small molecule / Neuro-oncology
Brain Cancer / Glioma
90Y-besilesomabTLX66-Tx / CD66 / Antibody / Hematology
Bone-marrow conditioning
225Ac-RADmAbTLX592-Tx / PSMA / Antibody / Urologic Oncology
Prostate Cancer
225Ac-girentuximabTLX252-Tx / CAIX / Antibody / Urologic Oncology
Kidney Cancer
UndisclosedTLX400-Tx / FAP / Small molecule / Urologic Oncology
Bladder Cancer
211At-APATLX102-Tx / LAT / Small molecule / Neuro-oncology
Brain Cancer
-olaratumab conjugateTLX300-Tx / PDGFRα / Antibody / Musculoskeletal Oncology
Soft Tissue Sarcoma
153Sm-DOTMPTLX090-Tx / Small molecule / Musculoskeletal Oncology
Bone metastasesPain palliation
68Ga-PSMA-11TLX591-CDx / Illuccix / Gozellix / PSMA / Diagnostic Radiopharmaceutical / Oncology
Prostate Cancer Imaging
18F-floretyrosineTLX101-CDx / Pixclara / Pixlumi / LAT / Diagnostic Radiopharmaceutical / Neuro-oncology
Glioma Imaging
89Zr-DFO-girentuximabTLX250-CDx / Zircaix / CAIX / Diagnostic Radiopharmaceutical / Oncology
Clear Cell Renal Cell Carcinoma (ccRCC) Imaging

LNTH

Lantheus

lantheus.com

13assets

AGEN

Agenus Bio

agenusbio.com

12assets

BotensilimabAGEN1181 / CTLA-4 / Fc-enhanced anti-CTLA-4 antibody / Oncology
Non MSI-H 4L+ metastatic colorectal cancerPhase 3Sites activeNon MSI-H metastatic colorectal cancer NLMPhase 2Enrollment completePD-1 R/R melanomaPhase 2
BalstilimabAGEN2034 / PD-1 / Anti-PD-1 antibody / Oncology
Non MSI-H 4L+ metastatic colorectal cancer with botensilimabPhase 3Sites activeNon MSI-H metastatic colorectal cancer NLM with botensilimabPhase 2Enrollment complete
AGEN2373AGEN2373 / CD137 / CD137 agonist antibody / Oncology
Solid tumorsPhase 1PD-1 R/R melanoma with botensilimabPhase 2
AGEN1571AGEN1571 / ILT-2 / Anti-ILT-2 antibody / Oncology
Solid tumorsPhase 1
AGEN1777AGEN1777 / TIGIT x CD96 / Bispecific antibody / Oncology
Solid tumors with balstilimabPhase 1
AGEN1423AGEN1423 / CD73 x TGFB / Bispecific antibody / Oncology
Solid tumorsPhase 1
INCAGN1876INCAGN1876 / GITR / Anti-GITR antibody / Oncology
Solid tumorsPhase 1
AGEN1949AGEN1949 / OX40 / OX40 agonist antibody / Oncology
Solid tumorsPhase 1
MK-4830MK-4830 / ILT4 / Anti-ILT4 antibody / Oncology
Neoadjuvant ovarian cancerPhase 2Ongoing
CR6086 + BalstilimabCR6086; AGEN2034 / EP4 + PD-1 / Small molecule + antibody combination / Oncology
Non-MSI-H colorectal cancerPhase 1/2Ongoing
NLM001 + ZalifrelimabNLM-001 / Hedgehog + CTLA-4 / Small molecule + antibody combination / Oncology
Pancreatic cancerPhase 1/2Ongoing
OBT076 + BalstilimabOBT076; AGEN2034 / CD205 + PD-1 / Antibody-drug conjugate + antibody combination / Oncology
Solid tumorsClinical trial

ARGX

Argenx

argenx.com

12assets

EfgartigimodVYVGART; VYVGART Hytrulo / FcRn / antibody fragment / Autoimmune
Generalized Myasthenia Gravis (gMG)CommercialChronic Inflammatory Demyelinating Polyneuropathy (CIDP)CommercialPrimary Immune Thrombocytopenia (ITP)CommercialJapan
EmpasiprubartC2 / monoclonal antibody / Autoimmune
Multifocal Motor Neuropathy (MMN)RegistrationalDelayed Graft Function (DGF)Proof of ConceptChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Registrational
AdimanebartMuSK / agonist antibody / Neurology
Congenital Myasthenic Syndromes (CMS)Proof of ConceptSpinal Muscular Atrophy (SMA)Proof of Concept
ARGX-109ARGX-109 / IL-6 / antibody / Undisclosed
UndisclosedPhase 1
ARGX-121ARGX-121 / IgA / antibody / Undisclosed
UndisclosedPhase 1
ARGX-213ARGX-213 / FcRn / antibody / Undisclosed
UndisclosedPhase 1
ARGX-118ARGX-118 / Galectin-10 / Airway Inflammation
Airway InflammationPreclinical
ARGX-125ARGX-125 / bispecific antibody / Undisclosed
UndisclosedPreclinical
TSP-101TSP-101 / Fn14 / Undisclosed
UndisclosedPreclinical
LP0145ARGX-112 / IL-22R / SIMPLE Antibody / Dermatology
Atopic Dermatitispartnered
AGMB-101ARGX-114 / MET / HGF-mimetic agonist antibody
ABBV-151ARGX-115 / GARP-TGF-beta1 / SIMPLE Antibody / Oncology
Tumorspartnered

BHVN

Biohaven

biohaven.com

12assets

Taldefgrobep AlfaBHV-2000 / Myostatin / recombinant/fusion protein; myostatin inhibitor / Neuromuscular; Metabolic
Spinal Muscular AtrophyPhase 3ObesityPhase 1
OpakalimBHV-7000 / Kv7.2/7.3 potassium channels / Kv7 activator / Neurology; Psychiatry
Focal EpilepsyPhase 3Generalized EpilepsyPhase 3Major Depressive DisorderPhase 2
BHV-2100BHV-2100 / TRPM3 / TRPM3 antagonist / Pain
Pain DisordersPhase 2
BHV-8000BHV-8000 / TYK2/JAK1 / brain-penetrant TYK2/JAK1 inhibitor / Neurology; Neuroinflammation
Parkinson’s DiseasePhase 3Prevention of Amyloid Therapy Induced ARIAPhase 1Alzheimer’s DiseasePhase 1
BHV-1300BHV-1300 / IgG / IgG degrader; MoDE bispecific molecule / Inflammation & Immunology
Common Disease (Graves’, RA)Phase 1
BHV-1310BHV-1310 / IgG / IgG degrader; MoDE bispecific molecule / Inflammation & Immunology
Rare Disease (Myasthenia Gravis)Preclinical
BHV-1400BHV-1400 / Gd-IgA1 / TRAP degrader / Inflammation & Immunology; Nephrology
IgA NephropathyPhase 1
BHV-1600BHV-1600 / β1AR autoantibodies / autoantibody degrader; bifunctional MoDE / Cardiovascular; Immunology
Peripartum CardiomyopathyPhase 1
BHV-1510BHV-1510 / Trop2 / antibody-drug conjugate +/- PD1 / Oncology
Advanced or Metastatic Epithelial TumorsPhase 1
BHV-1530BHV-1530 / FGFR3 / antibody-drug conjugate / Oncology
Urothelial Cancer & Other TumorsPhase 1
BHV-1500BHV-1500 / CD30 / antibody-drug conjugate / Oncology
Hodgkin LymphomaPreclinical
Undisclosed TargetsUndisclosed
Merus and GeneQuantum CollaborationsPreclinical

BIIB

Biogen

biogen.com

12assets

Dapirolizumab pegolCD40L / PEGylated Fab antibody fragment / Immunology
Systemic lupus erythematosusPhase 3PHOENYCS FLY; registrational
LitifilimabBDCA2 / Monoclonal antibody / Immunology
Systemic lupus erythematosusPhase 3TOPAZ-1 and TOPAZ-2Cutaneous lupus erythematosusPhase 3AMETHYST
FelzartamabCD38 / Monoclonal antibody / Immunology / Nephrology
IgA nephropathyPhase 3PREVAILLate antibody-mediated rejectionPhase 3TRANSCENDPrimary membranous nephropathyPhase 3PROMINENT
ZorevunersenSCN1A / Antisense oligonucleotide / Rare disease / Neurology
Dravet syndromePhase 3EMPEROR
SalanersenSMN2 / Antisense oligonucleotide / Rare disease / Neurology
Spinal muscular atrophyPhase 3 readySTELLAR-1 planned
Lecanemab-irmbLEQEMBI / Amyloid beta / Monoclonal antibody / Neurology
Preclinical Alzheimer's diseasePhase 3AHEAD 3-45Alzheimer's disease treatment initiationsBLA submittedSubcutaneous autoinjector for treatment initiation; submitted November 2025US
OmaveloxoloneSKYCLARYS / Nrf2 activator / Small molecule / Rare disease / Neurology
Friedreich ataxia, pediatricPhase not specifiedBRAVE pediatric program
BIIB080BIIB080 / Tau / Antisense oligonucleotide / Neurology
Alzheimer's diseasePhase 2early AD; anti-tau ASO
BIIB122BIIB122 / LRRK2 / Small molecule inhibitor / Neurology
Parkinson's diseasePhase 2oral LRRK2 inhibitor
BIIB091BIIB091 / BTK / Small molecule inhibitor / Neurology / Immunology
Multiple sclerosisPhase 2peripheral BTKi
IRAK4 degraderIRAK4 / Targeted protein degrader / Immunology
Not disclosedPhase 1new Phase 1 program
BTK degraderBTK / Targeted protein degrader / Immunology
Not disclosedPhase 1new Phase 1 program

CVAC

Curevac

curevac.com

12assets

CVGBMCVGBM / Eight epitopes from four glioblastoma tumor-associated antigens / Unmodified mRNA multi-epitope cancer vaccine; LNP-formulated / Oncology
Resected glioblastomaPhase 1Ongoing proof-of-principle studyGermanyBelgiumNetherlands
CVHNLCCVHNLC / Proprietary tumor epitopes / mRNA-based precision immunotherapy / cancer vaccine / Oncology
Squamous non-small cell lung cancerPhase 1IND clearedUnited States
Off-the-shelf cancer vaccine candidatesUndisclosed tumor antigens / mRNA cancer vaccine / Oncology
Undisclosed cancer indicationsIn development
Personalized cancer vaccine candidatesPatient-specific tumor genomic profile / Personalized mRNA cancer vaccine / Oncology
Undisclosed cancer indicationsIn development
UPEC vaccine candidateUropathogenic Escherichia coli / mRNA prophylactic vaccine / Infectious disease
Urinary tract infections caused by UPECPipeline program
Seasonal influenza vaccine candidate (influenza B strain optimization)WHO-recommended influenza strains / Modified multivalent mRNA prophylactic vaccine / Infectious disease
Seasonal influenzaPhase 2Licensed to GSK
Seasonal influenza vaccine candidateFormerly four WHO-recommended influenza strains / Modified multivalent mRNA prophylactic vaccine / Infectious disease
Seasonal influenzaPhase 1/2Licensed to GSK; Phase 2 dose-confirmation part
Avian influenza A (H5N1) vaccine candidateInfluenza A H5N1 / Modified monovalent mRNA prophylactic vaccine / Infectious disease
Avian influenza / pre-pandemic H5N1 influenzaPhase 1/2Licensed to GSKUnited States
CV0601CV0601 / Omicron BA.4-5 spike protein / Monovalent mRNA prophylactic vaccine / Infectious disease
COVID-19 boosterPhase 2Licensed to GSK; fully recruited studyAustralia
CV0701CV0701 / Omicron BA.4-5 spike protein and original SARS-CoV-2 spike protein / Bivalent mRNA prophylactic vaccine / Infectious disease
COVID-19 boosterPhase 2Licensed to GSK; fully recruited studyAustralia
Seasonal influenza and COVID-19 combination vaccine candidateSeasonal influenza and SARS-CoV-2 antigens / mRNA prophylactic vaccine / Infectious disease
Seasonal influenza and COVID-19Licensed to GSK
Cas9 gene editingCas9 / mRNA-based molecular therapy / gene editing / Molecular therapies
Undisclosed indicationsPipeline program

RCUS

Arcus Bio

arcusbio.com

12assets

CasdatifanAB521 / HIF-2α Inhibitor
QuemliclustatCD73 Inhibitor
DomvanalimabAnti-TIGIT Antibody
ZimberelimabAnti-PD-1 Antibody
EtrumadenantA2a/A2b Receptor Antagonist
AB598Anti-CD39 Antibody
MRGPRX2 Inhibitor
TNF Inhibitor
CCR6 Inhibitor
CD89 Monoclonal Antibody
CD40L Inhibitor
AXL Inhibitor

DNLI

Denali Therapeutics

denalitherapeutics.com

11assets

tividenofusp alfa-eknmDNL310; ETV:IDS / AVLAYAH / IDS enzyme replacement; TfR TransportVehicle-enabled delivery / Enzyme TransportVehicle biologic / Lysosomal storage disorders
Hunter syndrome (MPS II)Approved; Phase 2/3 confirmatory study ongoingU.S. accelerated approval; global Phase 2/3 COMPASS ongoingUSGlobal
DNL126ETV:SGSH / SGSH enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
Sanfilippo syndrome Type A (MPS IIIA)Phase 1/2; Phase 3 plannedOngoing Phase 1/2; global Phase 3 confirmatory study plannedGlobal
DNL593TAK-594/DNL593; PTV:PGRN / Progranulin replacement / Protein TransportVehicle biologic / Neurodegenerative disease
GRN-related frontotemporal dementia (FTD-GRN)Phase 1/2Ongoing; Denali received notice that Takeda will terminate collaboration, with Denali planning to continue developmentGlobal
DNL628OTV:MAPT / MAPT / tau / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative disease
Alzheimer's diseasePhase 1bCTA approved; study start-up underway
DNL952ETV:GAA / GAA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
Pompe diseasePhase 1IND clinical hold lifted; proceeding to Phase 1US
BIIB122/DNL151DNL151; BIIB122 / LRRK2 / Small molecule inhibitor / Neurodegenerative disease
Parkinson's diseasePhase 2bLUMA enrollment completed; readout expected 2026GlobalLRRK2-associated Parkinson's diseasePhase 2aBEACON ongoing
eclitasertibSAR443122/DNL758 / RIPK1 / Small molecule inhibitor / Inflammatory disease
Ulcerative colitisPhase 2Developed by Sanofi; Phase 2 results expected in 1H 2026
DNL921ATV:Abeta / Amyloid beta / Antibody TransportVehicle therapeutic / Neurodegenerative disease
Alzheimer's diseaseIND-enablingPhase 1/1b initiation planned
DNL111ETV:GCase / GCase / Enzyme TransportVehicle biologic / Neurodegenerative disease; lysosomal storage disorders
Parkinson's diseaseIND-enablingIND-enablingGaucher diseaseIND-enablingIND-enabling
DNL622ETV:IDUA / IDUA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
MPS IIND-enablingIND-enabling
DNL422OTV:SNCA / SNCA / alpha-synuclein / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative disease
Parkinson's diseaseIND-enablingIND-enabling

INCY

Incyte

incyte.com

11assets

INCA033989INCA033989 / mutCALR / Hematology
CALR-mutated essential thrombocythemia (2L)Phase 3Initiation mid-2026USCALR-mutated myelofibrosis (1L)Phase 1Data expected 2H 2026CALR-mutated myelofibrosis (2L)Phase 3Initiation 2H 2026
INCB161734INCB161734 / KRAS G12D / Oncology
1L G12D-mutated pancreatic ductal adenocarcinoma (PDAC)Phase 3Ongoing (in combo with SOC chemo)KRAS G12D-mutated solid tumorsPhase 1Data expected 2H 2026
INCA33890INCA33890 / TGFβR2 × PD-1 / bispecific / Oncology
1L microsatellite-stable colorectal cancer (MSS CRC)Phase 3Ongoing1L MSS PDAC (combo with SOC)Phase 1Data expected 2H 2026
INCB123667INCB123667 / CDK2 / Oncology
Platinum-resistant ovarian cancer (PROC)PivotalOngoing1L maintenance ovarian cancer (CCNE1+)Phase 3Initiation planned 2H 2026
povorcitinibJAK1 / small molecule / Inflammation and Autoimmunity
Moderate-to-severe hidradenitis suppurativa (HS)Phase 3NDA accepted; approval anticipated 1Q 2027 (US), late 2026 (EU)USEUNonsegmental vitiligoPhase 3Positive data; sNDA submission 1H 2027Moderate-to-severe prurigo nodularis (PN)Phase 3Ongoing; topline data 4Q 2026
ruxolitinib creamOpzelura / JAK1/JAK2 / topical / Inflammation and Autoimmunity
Moderate atopic dermatitisApprovedApproval and launch (ex-US)EUMild-to-moderate hidradenitis suppurativa (HS)Phase 3Ongoing; topline data 4Q 2026
axatilimabNiktimvo / CSF-1R / Hematology
1L chronic GVHD (+ ruxolitinib)PivotalData 2H 20261L chronic GVHD (+ steroids)Data early 20283L+ chronic GVHDApprovedApprovedUS
INCB160058INCB160058 / JAK2V617F / Hematology
JAK2 V617F-mutated myeloproliferative neoplasms (MPNs)Phase 1Data 2H 2026 (ASD formulation)
tafasitamabMonjuvi / CD19 / Hematology
1L diffuse large B-cell lymphoma (DLBCL)Phase 3sBLA submission 1H 2026USRelapsed/refractory DLBCLApprovedApprovedUSEUJapan
ruxolitinib XRJakafi XR / JAK1/JAK2 / Hematology
Myelofibrosis, polycythemia vera, chronic GVHDRegistrationFDA resubmissionUS
retifanlimabZynyz / PD-1 / Oncology
Squamous cell carcinoma of the anal canal (SCAC)ApprovedApproved (EC approval noted)USEUMerkel cell carcinomaApprovedApprovedUSEUJapan

KRYS

Krystal Bio

krystalbio.com

11assets

B-VECB-VEC / VYJUVEK / Type VII collagen (COL7A1) / Gene Therapy (HSV-1 vector) / Dermatology
Dystrophic Epidermolysis Bullosa (DEB)CommercialApprovedGlobal
KB105KB105 / Transglutaminase 1 (TGM1) / Gene Therapy / Dermatology
TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)Phase 1/2ActiveGlobal
KB104KB104 / Serine Peptidase Inhibitor Kazal Type 5 (SPINK5) / Gene Therapy / Dermatology
Netherton SyndromePreclinicalActiveGlobal
KB407KB407 / Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) / Gene Therapy / Respiratory
Cystic FibrosisPhase 1ActiveGlobal
KB408KB408 / Alpha-1 antitrypsin / Gene Therapy / Respiratory
Alpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
KB707KB707 / IL-12 and IL-2 / Gene Therapy (HSV-1 vector) / Oncology
Locally advanced or metastatic solid tumor malignanciesPhase 1ActiveGlobal
KB301KB301 / Type III collagen / Gene Therapy / Aesthetics
Aesthetic Skin ConditionsPhase 1/2ActiveGlobal
KB302KB302 / Type I collagen / Gene Therapy / Aesthetics
Aesthetic Skin ConditionsPreclinicalActiveGlobal
KB303KB303 / Elastin / Gene Therapy / Aesthetics
Aesthetic Skin ConditionsPreclinicalActiveGlobal
KB304KB304 / Type III collagen and Elastin / Gene Therapy / Aesthetics
Aesthetic Skin ConditionsPreclinicalActiveGlobal
KB305KB305 / Type IV collagen / Gene Therapy / Aesthetics
Aesthetic Skin ConditionsPreclinicalActiveGlobal

SNY

Sanofi

sanofi.com

11assets

amlitelimabOX40L / Monoclonal antibody / Immunology
Atopic dermatitisPhase 3ActiveGlobal
tolebrutinibBTK / Small molecule / Neurology
Multiple sclerosisRegistrationActiveGlobalUS
lunsekimigBiologic / Immunology and Inflammation
Asthma / Respiratory diseasesPhase 2ActiveGlobal
rilzabrutinibBTK / Small molecule / Rare Blood Disorders / Immunology
Sickle cell diseasePhase 2/3ActiveGlobalIgG4-related diseasePhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
venglustatSmall molecule / Rare Diseases
Gaucher disease type 3 (GD3)Phase 3ActiveGlobal
frexalimabCD40L / Monoclonal antibody / Immunology
Multiple sclerosisPhase 3ActiveGlobal
SAR441566SAR441566 / TNFR1si / Small molecule / Immunology
Immunological diseasesPhase 2ActiveGlobal
duvakitugTL1A / Monoclonal antibody / Immunology
Ulcerative colitisPhase 2ActiveGlobalCrohn's diseasePhase 2ActiveGlobal
itepekimabIL-33 / Monoclonal antibody / Immunology and Inflammation
COPDPhase 3ActiveGlobal
isatuximabSarclisa / CD38 / Monoclonal antibody / Oncology
Multiple myeloma (subcutaneous)RegistrationActiveEU
acoziboroleSmall molecule / Infectious Diseases
Sleeping sicknessRegistrationActiveAfricaEU

BCAB

Bioatla

bioatla.com

10assets

Ozuriftamab vedotinBA3021 / ROR2 / CAB antibody-drug conjugate / Oncology
OPSCCPhase 3
Mecbotamab vedotinBA3011 / AXL / CAB antibody-drug conjugate / Oncology
Sarcoma (soft tissue and bone)Phase 2mKRAS NSCLCPhase 2
EvalstotugBA3071 / CTLA-4 / CAB antibody / Oncology
Unresectable and/or metastatic cutaneous melanomaPhase 2
BA3182BA3182 / EpCAM x CD3 / Dual CAB T-cell engager / Oncology
AdenocarcinomaPhase 1ongoing
BA3361BA3361 / Nectin-4 / CAB antibody-drug conjugate / Oncology
Solid tumorsPreclinicalIND-approved
BA3151BA3151 / B7-H4 / CAB antibody-drug conjugate / Oncology
Breast cancerPreclinical
BA3142BA3142 / B7-H3 x CD3 / Dual CAB T-cell engager / Oncology
Solid tumorsPreclinicalIND-ready
BA3311BA3311 / EGFR x CD3 / T-cell engager / Oncology
Solid tumorsPreclinical
BA3241BA3241 / Trop2 x CD3 / Dual CAB T-cell engager / Oncology
Solid tumorsPreclinical
BA3362BA3362 / Nectin-4 x CD3 / Dual CAB T-cell engager / Oncology

HCM

Hutch Med

hutch-med.com

10assets

FruquintinibHMPL-013 / ELUNATE / FRUZAQLA / VEGFR 1/2/3 / Small molecule / Oncology
Colorectal cancerApproved/MarketedApprovedChinaUSEuropeJapanGastric cancerPhase IIIActiveGlobalOther solid tumors (combination studies)ClinicalActiveGlobal
SavolitinibHMPL-504 / AZD6094 / ORPATHYS / c-MET / Small molecule / Oncology
Non-small cell lung cancer (MET exon 14)Approved/MarketedApprovedChinaNSCLC combinations (with immunotherapy/targeted therapy)Phase IIIActiveGlobal
SurufatinibHMPL-012 / SULANDA / VEGFR/FGFR/CSF-1R / Small molecule / Oncology
Neuroendocrine tumorsApproved/MarketedApprovedChina
SovleplenibHMPL-523 / Syk / Small molecule / Hematology/Immunology
Immune thrombocytopenia (ITP)NDA/Regulatory reviewUnder regulatory reviewChina
FanregratinibHMPL-453 / FGFR / Small molecule / Oncology
Cholangiocarcinoma (FGFR2 fusion)NDA/Regulatory reviewUnder regulatory reviewChina
HMPL-760HMPL-760 / BTK (non-covalent) / Small molecule / Oncology/Hematology
Relapsed/refractory diffuse large B-cell lymphoma (DLBCL)Phase IIIInitiatedChina
HMPL-A251HMPL-A251 / HER2 (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
HER2-positive/HER2-low solid tumorsPhase IActiveChinaUS
HMPL-A580HMPL-A580 / EGFR (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
Solid tumorsPhase IInitiatedGlobal
HMPL-A830HMPL-A830 / ATTC (undisclosed) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
Solid tumorsPreclinical/Phase I plannedPlannedGlobal
TazemetostatTAZVERIK / EZH2 / Small molecule / Oncology
Epithelioid sarcoma / follicular lymphomaLicensed/CommercialLicensed product updateChina

LLY

Lilly

lilly.com

10assets

PFE

Pfizer

pfizer.com

10assets

BerobenatidePF-06954522 / GLP-1/GIP / Small Molecule / Internal Medicine
Chronic Weight ManagementPhase 2bOngoingGlobal
elranatamabPF-06863135 / ELREXFIO / BCMA x CD3 / Bispecific Antibody / Oncology
Double-class Exposed Relapsed / Refractory Multiple MyelomaPhase 3OngoingGlobal
ritlecitinibPF-06651600 / LITFULO / JAK3/TEC / Small Molecule / Inflammation and Immunology
VitiligoPhase 3OngoingGlobalModerate Alopecia AreataPhase 3OngoingGlobal
marstacimabPF-06741086 / HYMPAVZI / TFPI / Monoclonal Antibody / Rare Disease
Hemophilia A/B with InhibitorsRegistrationOngoingGlobal
MevrometostatPF-06821497 / EZH2 / Small Molecule / Oncology
1-2L Metastatic Castration-resistant Prostate Cancer Post-abirateronePhase 3OngoingGlobal
Sigvotatug vedotinPF-08046047 / Integrin beta-6 / Antibody-Drug Conjugate (ADC) / Oncology
Non-Small Cell Lung Cancer (NSCLC)Phase 3OngoingGlobal
VepdegestrantARV-471 / Estrogen Receptor (ER) / PROTAC Protein Degrader / Oncology
ER+/HER2- Metastatic Breast CancerPhase 3OngoingGlobal
PCV25PF-07872412 / Pneumococcus / Vaccine / Vaccines
Pneumococcal InfectionPhase 3OngoingGlobal
enfortumab vedotinPADCEV / Nectin-4 / Antibody-Drug Conjugate (ADC) / Oncology
Muscle-invasive Bladder Cancer (Bladder Sparing)Phase 3OngoingGlobal
Lyme Disease Vaccine CandidatePF-07307405 / Borrelia burgdorferi OspA / Vaccine / Vaccines
Lyme Disease InfectionPhase 3OngoingGlobal

ADAG

Adagene

adagene.com

9assets

muzastotugADG126 / CTLA-4 / fully human anti-CTLA-4 SAFEbody / Oncology
Advanced/metastatic solid tumorsPhase 1b/2dose expansion ongoing with anti-PD-1 therapies
ADG116ADG116 / CTLA-4 / fully human anti-CTLA-4 NEObody mAb / Oncology
Advanced/metastatic solid tumorsPhase 1b/2ongoing
ADG206ADG206 / CD137 / masked Fc-engineered anti-CD137 POWERbody / Oncology
Advanced/metastatic tumorsPhase 1ongoing
ADG106ADG106 / CD137 / fully human ligand-blocking agonistic anti-CD137 IgG4 mAb / Oncology
Advanced/metastatic solid tumorsPhase 1b/2ongoingRelapsed/refractory non-Hodgkin’s lymphomaPhase 1b/2ongoing
ADG153ADG153 / CD47 / masked anti-CD47 IgG1 SAFEbody / Oncology
Hematologic and solid tumorsIND-enablingpreclinical
ADG138ADG138 / HER2 x CD3 / masked T-cell engager POWERbody / Oncology
HER2-expressing solid tumorsIND-enablingpreclinical
ADG152ADG152 / CD20 x CD3 / bispecific T-cell engager POWERbody / Oncology
B-cell malignanciesIND-enablingpreclinical
ADG104ADG104 / PD-L1 / anti-PD-L1 antibody / Oncology
CancerPhase 2in development by Sanjin
ADG125ADG125 / CSF-1R / antibody / Oncology
CancerPhase 1in development by Dragon Boat Biopharmaceutical

CLLS

Cellectis

cellectis.com

9assets

lasme-celUCART22 / CD22 / Allogeneic gene-edited CAR T-cell therapy / Oncology / hematologic malignancies
Relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)Pivotal Phase 2BALLI-01; enrolling/activeUnited StatesEuropean Union
eti-celUCART20x22 / CD20 and CD22 / Allogeneic gene-edited CAR T-cell therapy / Oncology / hematologic malignancies
Relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL)Phase 1/2aNATHALI-01; enrolling/activeUnited States
CLLS52CLLS52 / CD52 / Anti-CD52 monoclonal antibody (alemtuzumab) / Oncology / lymphodepletion regimen
Acute lymphoblastic leukemia (ALL)Investigational medicinal product used in BALLI-01 and NATHALI-01 protocols; FDA orphan drug designationUnited StatesEuropean Union
UCARTFAPUCARTFAP / FAP / Allogeneic gene-edited CAR T-cell therapy / Oncology / solid tumors
Solid tumorsPreclinicalPreclinical
UCARTMUC1UCARTMUC1 / MUC1 / Allogeneic gene-edited CAR T-cell therapy / Oncology / solid tumors
Solid tumorsPreclinicalPreclinical
cemacabtagene ansegedleucelALLO-501A / CD19 / Allogeneic gene-edited CAR T-cell therapy / Oncology / hematologic malignancies
Large B-cell lymphoma (LBCL)Pivotal Phase 2ALPHA3; activeUnited StatesEuropean UnionUnited Kingdom
ALLO-316ALLO-316 / CD70 / Allogeneic gene-edited CAR T-cell therapy / Oncology / solid tumors
Renal cell carcinoma (RCC)Phase 1TRAVERSE; activeUnited States
ALLO-213ALLO-213 / DLL3 / Allogeneic gene-edited CAR T-cell therapy / Oncology / solid tumors
Small cell lung cancer and neuroendocrine tumorsPreclinicalPreclinical
ALLO-182ALLO-182 / Claudin 18.2 / Allogeneic gene-edited CAR T-cell therapy / Oncology / solid tumors
Gastric cancer and pancreatic cancerPreclinicalPreclinical

CRSP

Crisprtx

ir.crisprtx.com

9assets

CASGEVYexa-cel / CASGEVY / BCL11A erythroid-specific enhancer / Non-viral ex vivo CRISPR/Cas9 gene-edited autologous cell therapy / Hemoglobinopathies
Sickle cell disease, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobalTransfusion-dependent beta thalassemia, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobal
CTX310CTX310 / ANGPTL3 / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular/metabolic
Severe hypertriglyceridemiaPhase 1bActive; update expected H2 2026Refractory hypercholesterolemiaPhase 1bActive; update expected H2 2026Heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias or severe hypertriglyceridemiaPhase 1Developed/being evaluated
CTX320CTX320 / LPA / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
Elevated lipoprotein(a)Clinical trial / dose escalationActive; Lp(a) program update expected in 2026
CTX321CTX321 / LPA / Next-generation in vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
Elevated lipoprotein(a)IND/CTA-enablingActive
CTX460CTX460 / SERPINA1 / In vivo SyNTase gene correction; LNP delivery / Rare disease
Alpha-1 antitrypsin deficiencyPreclinical; clinical trial expected mid-2026Active
CTX340CTX340 / AGT / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
Refractory hypertensionIND/CTA-enabling; clinical trial expected H1 2026Active
CTX611SRSD107 / Factor XI / FXI mRNA / Long-acting double-stranded siRNA / Thromboembolic/cardiovascular diseases
Venous thromboembolism prevention after total knee arthroplastyPhase 2Ongoing; top-line data expected H2 2026EuropeAtrial fibrillation, venous thromboembolism, ischemic stroke, cancer-associated thrombosis, chronic kidney disease, peripheral vascular disease, chronic coronary artery diseaseTarget indications disclosed
Zugocabtagene geleucelzugo-cel; formerly CTX112 / CD19 / Allogeneic CAR-T cell therapy; CRISPR Cas9 engineered / Autoimmune disease; oncology
Systemic lupus erythematosusPhase 1Ongoing; update expected H2 2026Systemic sclerosisPhase 1Ongoing; update expected H2 2026Inflammatory myositisPhase 1Ongoing; update expected H2 2026
CTX213CTX213 / iPSC-derived allogeneic gene-edited beta islet cell precursor therapy / Regenerative medicine / diabetes
Type 1 diabetesPreclinical; progressing toward clinicActive

EVO

Evotec

evotec.com

9assets

EVT8683BMS-986419 / eIF2b / Neuroscience
Neurodegeneration
CNTX 6016CB2 / Pain
Pain
EVT801VEGFR3 / Oncology
Oncology
EVT401P2X7 / Immunology
InflammationAutoimmune
EVT894Infectious Diseases
Chikungunya
EXS21546A2a / Oncology
Oncology
DSP-11815-HT1A / Neuroscience
Obsessive Compulsive Disorder (OCD)
CT7001CDK7 / Oncology
Oncology
TPM203Immunology
Pemphigus Vulgaris

IDYA

Ideaya Bio

ideayabio.com

9assets

DarovasertibIDE196 / PKC / Small Molecule / Oncology
1L metastatic uveal melanoma (HLA-A*02:01 negative)Phase 2/3Positive topline; NDA initiationGlobalNeoadjuvant primary uveal melanomaPhase 2/3OngoingUS
IDE397IDE397 / MAT2A / Small Molecule / Oncology
MTAP-deleted solid tumorsPhase 1OngoingUS
IDE849IDE849 / DLL3 / ADC / Oncology
DLL3-expressing solid tumors (SCLC, NEC)Phase 1OngoingUS
IDE161IDE161 / PARG / Small Molecule / Oncology
HRD solid tumorsPhase 1RecruitingUS
IDE034IDE034 / PTK7/B7H3 / Bispecific ADC / Oncology
B7H3/PTK7 solid tumors (NSCLC, CRC, breast, ovarian)Phase 1OngoingUS
IDE892IDE892 / PRMT5 / Small Molecule / Oncology
MTAP-deleted solid tumorsPhase 1OngoingUS
IDE574IDE574 / KAT6/7 / Small Molecule / Oncology
Solid tumors (breast, prostate, CRC, lung)Phase 1OngoingUS
IDE275IDE275 / Werner Helicase (WRN) / Small Molecule / Oncology
MSI-high solid tumorsPhase 1OngoingUS
IDE705IDE705 / Pol Theta (POLQ) / Small Molecule / Oncology
HRD solid tumorsPhase 1OngoingUS

INSM

Insmed

insmed.com

9assets

BrensocatibBRINSUPRI / DPP1 / Small Molecule / Respiratory / Immunology
Non-Cystic Fibrosis Bronchiectasis (NCFB)MarketedActiveUSEUHidradenitis SuppurativaPhase 2ActiveGlobal
Amikacin Liposome Inhalation SuspensionARIKAYCE / Bacterial 30S ribosomal subunit / Liposomal Inhalation Suspension / Respiratory / Infectious Disease
Refractory Mycobacterium avium complex (MAC) Lung DiseaseMarketedActiveUSEUJPMAC Lung Disease (Label Expansion)Phase 3ActiveGlobal
Treprostinil Palmitil Inhalation PowderTPIP / Prostacyclin receptor / Inhalation Powder / Respiratory / Pulmonary
Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 3ActiveGlobalPulmonary Arterial Hypertension (PAH)Phase 3ActiveGlobalProgressive Pulmonary Fibrosis (PPF)Phase 3 PlannedActiveGlobal
INS1148INS1148 / Stem Cell Factor SCF248 / Monoclonal Antibody / Respiratory / Immunology
Interstitial Lung Disease (ILD)Phase 2ActiveGlobalModerate-to-severe asthmaPhase 2ActiveGlobal
INS1033INS1033 / Unknown / Unknown / Immunology & Inflammation
Inflammatory diseasesPreclinicalActiveGlobal
Intrathecal gene therapy for DMDDystrophin / Gene Therapy / Neurology / Rare Disease
Duchenne Muscular Dystrophy (DMD)Phase 1ActiveGlobal
INS1202INS1202 / Unknown / Unknown / Neurology / Rare Disease
Amyotrophic Lateral Sclerosis (ALS)Phase 1ActiveGlobal
Stargardt Disease Gene TherapyUnknown / Gene Therapy / Ophthalmology / Rare Disease
Stargardt DiseasePreclinicalActiveGlobal
Next-Gen DPP1 InhibitorsDPP1 / Small Molecule / Immunology & Inflammation
UndisclosedPreclinicalActiveGlobal

JAZZ

Jazz Pharmaceuticals

jazzpharmaceuticals.com

9assets

ZanidatamabHER2 / Bispecific Antibody / Oncology
Biliary Tract Cancers (BTC)Gastroesophageal Adenocarcinoma (GEA)Colorectal Cancer (CRC)
LurbinectedinZepzelca / DNA alkylating / Small Molecule / Oncology
Small Cell Lung Cancer (SCLC)Ewing Sarcoma
DordaviproneModeyso / ClpP agonist / DRD2 antagonist / Small Molecule / Oncology
H3 K27M-mutant diffuse glioma
Daunorubicin and cytarabineJZP351 / Vyxeos / Vyxeos Liposomal / Topoisomerase inhibitor / nucleoside metabolic inhibitor / Liposomal Combination / Oncology
Myelodysplastic Syndromes (MDS)Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
JZP898JZP898 / IFNα2b cytokine pro-drug / Biologic / Oncology
Solid Tumors
JZP3507JZP3507 / ClpP agonist / DRD2 antagonist / Small Molecule / Oncology
Oncology
JZP815JZP815 / pan-RAF / Small Molecule / Oncology
Solid TumorsHematologic Malignancies
CannabidiolEpidyolex / Cannabinoid Receptors / Small Molecule / Neuroscience
Lennox-Gastaut Syndrome (LGS)Dravet Syndrome (DS)Tuberous Sclerosis Complex (TSC)
JZP441JZP441 / Orexin-2 receptor agonist / Small Molecule / Neuroscience
Narcolepsy Type 1Idiopathic Hypersomnia

MRK

Merck

merck.com

9assets

pembrolizumabMK-3475 / Keytruda
belzutifanMK-6482 / Welireg
sotaterceptMK-7962
sacituzumab tirumotecanMK-2870
efinopegdutideMK-6024
gocatamigMK-6070
MK-1084
MK-0616
V116

MRNA

Modernatx

modernatx.com

9assets

SpikevaxmRNA-1273 / COVID-19 Vaccine
mRESVIAmRNA-1345 / RSV Vaccine
mNEXSPIKECOVID-19 Vaccine
mCOMBRIAXFlu/COVID Combination Vaccine
mRNA-1010Flu Vaccine
mRNA-4157Individualized Neoantigen Therapy (INT)
mRNA-1647CMV Vaccine
mRNA-1403Norovirus Vaccine
mRNA-3927Propionic Acidemia (PA)

SGMO

Sangamo

sangamo.com

9assets

Isaralgagene civaparvovecST-920 / alpha-galactosidase A (GLA) / Gene Therapy (AAV) / Metabolic Diseases
Fabry DiseaseBLA/FilingActiveUS
Giroctogene fitelparvovecSB-525 / Factor VIII / Gene Therapy (AAV) / Hematology
Hemophilia APhase 3ActiveUS
ST-503ST-503 / Nav1.7 (SCN9A) / Gene Therapy (Zinc Finger Repressor, AAV9) / Neurology
Small Fiber Neuropathy (Chronic Neuropathic Pain)Phase 1/2ActiveUS
ST-506ST-506 / Prion protein (PRNP) / Gene Therapy (Zinc Finger Repressor, STAC-BBB AAV) / Neurology
Prion DiseasePreclinicalActiveUS
TX200TX200 / HLA-A2 / Cell Therapy (Autologous CAR-Treg) / Transplantation
Prevention of Immune-Mediated Rejection in HLA-A2 Mismatched Kidney TransplantationPhase 1/2ActiveUSEurope
IL23R CAR-TregIL23R / Cell Therapy (CAR-Treg) / Autoimmunity
Inflammatory Bowel Disease (Crohn's Disease)PreclinicalActiveGlobal
Genentech Tau ProgramTau / Gene Therapy (Zinc Finger Epigenetic Regulator) / Neurology
Tauopathies / Alzheimer's DiseasePreclinicalActiveGlobal
Takeda Huntington's ProgramHTT / Gene Therapy (Zinc Finger Epigenetic Regulator) / Neurology
Huntington's DiseasePreclinicalActiveGlobal
Alexion C9ORF72 ProgramC9ORF72 / Gene Therapy (Zinc Finger Transcription Factor) / Neurology
Amyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobalFrontotemporal Lobar Degeneration (FTLD)PreclinicalActiveGlobal

TEVA

Tevapharm

ir.tevapharm.com

9assets

VRTX

Vrtx

vrtx.com

9assets

SuzetrigineVX-548 / Journavx / NaV1.8 / Small Molecule / Pain
Moderate-to-Severe Acute PainPainful Diabetic Peripheral Neuropathy
VX-993VX-993 / NaV1.8 / Small Molecule / Pain
Painful Diabetic Peripheral NeuropathyModerate-to-Severe Acute Pain
Vanzacaftor/tezacaftor/deutivacaftorCFTR / Small Molecule / Cystic Fibrosis
Cystic Fibrosis
VX-522VX-522 / CFTR / mRNA / Cystic Fibrosis
Cystic Fibrosis (non-responsive to modulators)
InaxaplinVX-147 / APOL1 / Small Molecule / Kidney Disease
APOL1-mediated Kidney Disease (AMKD)
VX-407VX-407 / PC1 / Small Molecule / Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
VX-880VX-880 / Islet Cell / Cell Therapy / Type 1 Diabetes
Type 1 Diabetes
VX-264VX-264 / Islet Cell / Cell Therapy / Type 1 Diabetes
Type 1 Diabetes
PovetaciceptALPN-303 / BAFF/APRIL / Biologic / Autoimmune / Kidney Disease
IgA Nephropathy

XNCR

Xencor

xencor.com

9assets

XmAb819XmAb819 / ENPP3 x CD3 / Bispecific Antibody / Oncology
Clear Cell Renal Cell Carcinoma (ccRCC)Phase 1ActiveGlobalColorectal Cancer (CRC)Phase 1ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)Phase 1ActiveGlobal
XmAb541XmAb541 / CLDN6 x CD3 / Bispecific Antibody / Oncology
Ovarian CancerPhase 1ActiveGlobalGerm Cell Tumors (GCT)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
XmAb808XmAb808 / B7-H3 x CD28 / Bispecific Antibody / Oncology
Prostate Cancer / Solid TumorsPhase 1ActiveGlobal
XmAb942XmAb942 / TL1A / Monoclonal Antibody / Immunology
Ulcerative Colitis (UC)Phase 2ActiveGlobalInflammatory Bowel Disease (IBD)Phase 2ActiveGlobal
XmAb412XmAb412 / TL1A x IL23p19 / Bispecific Antibody / Immunology
Inflammatory Bowel Disease (IBD)Phase 1ActiveGlobal
PlamotamabCD20 x CD3 / Bispecific Antibody / Immunology
Rheumatoid ArthritisPhase 1ActiveGlobal
XmAb657XmAb657 / CD19 x CD3 / Bispecific Antibody / Immunology
Idiopathic Inflammatory MyositisPhase 1ActiveGlobal
ObexelimabCD19 x FcγRIIb / Monoclonal Antibody / Immunology
Autoimmune DiseasesPhase 3ActiveGlobal
TobevibartHepatitis B Virus / Monoclonal Antibody / Infectious Disease
Chronic Hepatitis B / Hepatitis DPhase 3ActiveGlobal

DYN

Dyne Tx

dyne-tx.com

8assets

DYNE-251 (zeleciment rostudirsen)DMD Exon 51
DYNE-101 (zeleciment basivarsen)DM1
DYNE-302FSHD
DYNE-401Pompe Disease
DYNE-253DMD Exon 53
DYNE-245DMD Exon 45
DYNE-244DMD Exon 44
DYNE-255DMD Exon 55

EVAX

Evaxion

evaxion.ai

8assets

EVX-01EVX-01 / Personalized neoantigens / Liposomal/peptide personalized cancer vaccine / Cancer
Advanced melanomaPhase 2
EVX-03EVX-03 / Personalized neoantigens and endogenous retrovirus antigens / Targeted DNA personalized cancer vaccine / Cancer
Solid tumorsPreclinicalCTA/IND ready
EVX-04EVX-04 / Tumor-specific antigens derived from endogenous retrovirus elements / DNA off-the-shelf therapeutic cancer vaccine / Cancer
Acute myeloid leukemiaPreclinicalPlanned first-in-human study
EVX-B1EVX-B1 / S. aureus toxins and virulence factors / Protein multi-component prophylactic vaccine / Infectious diseases
S. aureus skin and soft tissue infectionsPreclinical
EVX-B2EVX-B2 / N. gonorrhoeae antigens / Protein multi-component prophylactic vaccine / Infectious diseases
GonorrheaPreclinical
EVX-B2 mRNAEVX-B2 / N. gonorrhoeae antigens / mRNA multi-component prophylactic vaccine / Infectious diseases
GonorrheaPreclinical
EVX-B4EVX-B4 / Group A Streptococcus antigens / Prophylactic vaccine / Infectious diseases
Group A Streptococcus infectionTarget discovery
EVX-V1EVX-V1 / CMV B- and T-cell antigens; pre-fusion glycoprotein B construct / Multi-component prophylactic vaccine / Infectious diseases
Cytomegalovirus infectionPreclinical

EXEL

Exelixis

exelixis.com

8assets

ZanzalintinibXL092 / MET/VEGFR/AXL/MER / Next-generation TKI / Oncology
Colorectal cancerPivotal / Phase 3STELLAR-303: zanzalintinib + atezolizumab vs. regorafenibNon-clear cell renal cell carcinomaPivotal / Phase 3STELLAR-304: zanzalintinib + nivolumab vs. sunitinibNeuroendocrine tumorsPivotal / Phase 2/3STELLAR-311: zanzalintinib vs. everolimus
XL309XL309 / USP1 / USP1 inhibitor / Oncology
Multiple solid tumorsPhase 1XL309 or XL309 + olaparib
XB010XB010 / 5T4 / Next-generation 5T4-MMAE ADC / Oncology
Multiple solid tumorsPhase 1XB010 or XB010 + pembrolizumab
XB628XB628 / PD-L1 + NKG2A / Bispecific antibody / Oncology
Multiple solid tumorsPhase 1Ongoing clinical study
XB371XB371 / Tissue factor / Next-generation TF-TOPOi ADC / Oncology
Multiple solid tumorsPhase 1Ongoing clinical study
ADU-1805ADU-1805 / SIRPα / SIRPα-targeting mAb / Oncology
Multiple solid tumorsPhase 1ADU-1805 or ADU-1805 + pembrolizumab
XB773XB773 / DLL3 / DLL3-TOPOi ADC / Oncology
Not disclosedDiscovery/PreclinicalDiscovery/Preclinical
SSTR2 AgonistSomatostatin receptor subtype 2 / SSTR2 agonist program development candidate / Oncology
Not disclosedDiscovery/PreclinicalDiscovery/Preclinical

IMCR

Immunocore

immunocore.com

8assets

TebentafuspKIMMTRAK / gp100 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
metastatic uveal melanomaApprovedmarketedUSEUadjuvant uveal melanomaPhase 3ongoing (ATOM, EORTC-sponsored)2L+ advanced cutaneous melanomaPhase 3ongoing (TEBE-AM)
BrenetafuspIMC-F106C / PRAME (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
1L advanced cutaneous melanomaPhase 3ongoing (PRISM-MEL-301)ovarian cancerPhase 1/2ongoing (combinations)NSCLCPhase 1/2ongoing (monotherapy and combinations)
IMC-P115CIMC-P115C / PRAME (HLA-A*02 HLE) / ImmTAC (TCR bispecific) / Oncology
multiple solid tumorsPhase 1ongoing
IMC-R117CIMC-R117C / PIWIL1 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
colorectal and GI cancersPhase 1/2ongoing
IMC-M113VIMC-M113V / Gag (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious Disease
HIVPhase 1ongoing
IMC-I109VIMC-I109V / Envelope (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious Disease
HBVclinical development
IMC-S118AIIMC-S118AI / PPI x PD1 (HLA-A*02) / ImmTAAI (TCR bispecific) / Autoimmune Disease
type 1 diabetesPhase 1ongoing
IMC-U120AIIMC-U120AI / CD1a x PD1 (non-HLA restricted) / ImmTAAI (TCR bispecific) / Autoimmune Disease
atopic dermatitisIND-enabling

IPHA

Innate Pharma

innate-pharma.com

8assets

LacutamabKIR3DL2 / Monoclonal antibody / Oncology
Sézary syndromeMycosis fungoidesCutaneous T cell lymphomas
IPH4502IPH4502 / Nectin-4 / Antibody-drug conjugate (ADC) / Oncology
Solid tumors
MonalizumabNKG2A / Monoclonal antibody / Oncology
Non-small cell lung cancer (NSCLC)
IPH6501IPH6501 / CD20 / NK cell engager (ANKET) / Oncology
B-cell Non-Hodgkin's Lymphoma
IPH6101IPH6101 / CD123 / NK cell engager (ANKET) / Oncology / Autoimmune
Hematologic malignanciesAutoimmune indications
IPH6401IPH6401 / BCMA / NK cell engager (ANKET) / Oncology
Multiple myeloma
IPH5201IPH5201 / CD39 / Monoclonal antibody / Oncology
Solid tumors
IPH5301IPH5301 / CD73 / Monoclonal antibody / Oncology
Solid tumors

IRD

Opusgtx

opusgtx.com

8assets

OPGx-LCA5OPGx-LCA5 / LCA5 / AAV Gene Therapy / Ophthalmology
Leber Congenital Amaurosis Type 5 (LCA5)
OPGx-BEST1OPGx-BEST1 / BEST1 / AAV Gene Therapy / Ophthalmology
Best Vitelliform Macular Dystrophy (BVMD)Autosomal Recessive Bestrophinopathy (ARB)
OPGx-RDH12OPGx-RDH12 / RDH12 / AAV Gene Therapy / Ophthalmology
RDH12-associated Leber Congenital Amaurosis (RDH12-LCA)
OPGx-MERTKOPGx-MERTK / MERTK / AAV Gene Therapy / Ophthalmology
MERTK-associated Retinitis Pigmentosa
OPGx-RHOOPGx-RHO / RHO / AAV Gene Therapy / Ophthalmology
Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
OPGx-CNGB1OPGx-CNGB1 / CNGB1 / AAV Gene Therapy / Ophthalmology
CNGB1-associated Inherited Retinal Diseases
OPGx-NMNAT1OPGx-NMNAT1 / NMNAT1 / AAV Gene Therapy / Ophthalmology
NMNAT1-associated Inherited Retinal Diseases
Phentolamine Ophthalmic Solution 0.75%Small Molecule / Ophthalmology
Pharmacologically induced mydriasisPresbyopiaLow-light visual disturbances following keratorefractive surgery

KYMR

Kymeratx

kymeratx.com

8assets

KT-621KT-621 / STAT6 / Targeted Protein Degrader / Immunology / Respiratory
Atopic DermatitisPhase 2bOngoingGlobalAsthmaPhase 2bOngoingGlobal
KT-579KT-579 / IRF5 / Targeted Protein Degrader / Immunology
Autoimmune Diseases (Lupus, Rheumatoid Arthritis, IBD)Phase 1PlannedGlobal
KT-295KT-295 / TYK2 / Targeted Protein Degrader / Immunology
Inflammatory Bowel Disease (IBD), PsoriasisPhase 1OngoingGlobal
KT-485SAR447971 / IRAK4 / Targeted Protein Degrader / Immunology
Immuno-inflammatory diseasesPreclinicalOngoingGlobal
KT-200KT-200 / CDK2 / Molecular Glue Degrader / Oncology
Breast Cancer and other Solid TumorsPreclinicalOngoingGlobal
KT-333KT-333 / STAT3 / Targeted Protein Degrader / Oncology
Hematological Malignancies and Solid TumorsPhase 1OngoingGlobal
KT-253KT-253 / MDM2 / Targeted Protein Degrader / Oncology
High-Grade Myeloid Malignancies and Solid TumorsPhase 1OngoingGlobal
KT-474SAR444656 / IRAK4 / Targeted Protein Degrader / Immunology
Hidradenitis Suppurativa (HS), Atopic DermatitisPhase 2bDiscontinuedGlobal

PTCT

Ptc Bio

ptcbio.com

8assets

sepiapterinSephience
atalurenTranslarna
deflazacortEmflaza
eladocagene exuparvovecUpstaza / Kebilidi
votoplamPTC518
vatiquinone
utreloxastatPTC857
risdiplamEvrysdi

RHHBY

Roche

roche.com

8assets

fenebrutinibBTK / Small molecule / Neuroscience
Multiple Sclerosis
petrelintideAmylin / Peptide / Cardiovascular, Renal & Metabolism
Obesity
CT-996CT-996 / GLP-1 / Small molecule / Cardiovascular, Renal & Metabolism
Obesity
giredestrantEstrogen receptor (SERD) / Small molecule / Oncology
Early and metastatic breast cancer
trontinemabAmyloid beta / TfR / Bispecific antibody / Neuroscience
Alzheimer's Disease
NXT007NXT007 / Factor IX/X / Bispecific antibody / Hematology
Hemophilia A
afimkibartBiologic / Immunology
Immunological disorders
RVT-3101RVT-3101 / TL1A / Monoclonal antibody / Immunology
Inflammatory Bowel Disease

RPRX

Royalty Pharma

royaltypharma.com

8assets

ecopipamNeurology
Tourette's syndrome
TEV-'749TEV-'749 / Neurology
Schizophrenia
deucrictibantRare Disease
Hereditary angioedema
daraxonrasibOncology
Pancreatic cancer
litifilimabImmunology
Lupus
pelacarsenCardiovascular
Cardiovascular disease
zidesamtinibROS1 / Oncology
ROS1 mutation-positive non-small cell lung cancer
JNJ-4804JNJ-4804 / Immunology
Autoimmune diseases

SION

Sionnatx

sionnatx.com

8assets

SION-719SION-719 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 2aActive
SION-451SION-451 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 1Active
SION-638SION-638 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 1Active
SION-109SION-109 / ICL4 (Intracellular Loop 4 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 1Active
GalicaftorABBV-2222 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 2Active
NavocaftorABBV-3067 / CFTR potentiator / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 2Active
ABBV-2851ABBV-2851 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPhase 1Active
SION-676SION-676 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
Cystic FibrosisPreclinicalActive

SRPT

Sarepta

sarepta.com

8assets

SRP-1001SRP-1001 / FSHD1 / siRNA / Neuromuscular
Facioscapulohumeral muscular dystrophy, Type 1 (FSHD1)
SRP-1003SRP-1003 / DM1 / siRNA / Neuromuscular
Myotonic dystrophy, Type 1 (DM1)
SRP-1002SRP-1002 / siRNA / Pulmonary
Idiopathic pulmonary fibrosis (IPF)
SRP-1004SRP-1004 / siRNA / Neurology
Spinocerebellar ataxia type 2 (SCA2)
SRP-1005SRP-1005 / siRNA / Neurology
Huntington's disease (HD)
SRP-1007SRP-1007 / siRNA / Neurology
Spinocerebellar ataxia type 1 (SCA1)
SRP-1006SRP-1006 / siRNA / Neurology
Spinocerebellar ataxia type 3 (SCA3)
bidridistrogene xeboparvovecSRP-9003 / LGMD2E/R4 β-sarcoglycan / Gene Therapy / Neuromuscular
Limb-girdle Muscular Dystrophy

ZBIO

Zenas Bio

zenasbio.com

8assets

ZYME

Zymeworks

zymeworks.com

8assets

ZanidatamabZW25 / Ziihera / HER2 / Bispecific Antibody / Oncology
Biliary Tract Cancer (BTC)ApprovedActiveUSEuropeGastroesophageal Adenocarcinoma (GEA)Phase 3ActiveGlobalBreast Cancer (Neoadjuvant and Adjuvant)Phase 2ActiveGlobal
ZW191ZW191 / FRα / Antibody-Drug Conjugate (ADC) / Oncology
Advanced Solid Tumors (Ovarian/Gynecological and Lung/Thoracic)Phase 1ActiveGlobal
ZW251ZW251 / GPC3 / Antibody-Drug Conjugate (ADC) / Oncology
Hepatocellular Carcinoma (HCC) / Solid TumorsPhase 1ActiveGlobal
ZW220ZW220 / NaPi2b / Antibody-Drug Conjugate (ADC) / Oncology
Gynecological and Thoracic CancersPreclinicalActiveGlobal
ZW209ZW209 / DLL3 x CD3 x CD28 / Trispecific T-Cell Engager / Oncology
Thoracic CancersPreclinicalActiveGlobal
ZW1528ZW1528 / IL-4Rα x IL-33 / Bispecific Antibody / Autoimmune / Inflammatory Diseases
Respiratory InflammationPreclinicalActiveGlobal
PasritamigJNJ-78278343 / CD3 / Prostate Target / Bispecific Antibody / Oncology
Solid TumorsClinicalActiveGlobal
DS-2243DS-2243 / T-Cell Engager / Bispecific T-Cell Engager / Oncology
Advanced Solid TumorsPhase 1ActiveGlobal

AAPG

Ascentage Pharma

ascentage.com

7assets

OlverembatinibHQP1351 / BCR-ABL1/KIT / small molecule TKI / Oncology
CML-CP/CML-APMarketedapproved in China; additional global Phase III in CMLChinaGlobalFirst-line Ph+ ALLPhase IIIongoing registrationalGlobalSDH-deficient GISTPhase IIIongoing registrationalMultinational
LisaftoclaxAPG-2575 / Bcl-2 / small molecule inhibitor / Oncology
CLL/SLL after systemic therapy including BTK inhibitorsMarketedapproved in China 2025ChinaCLL/SLL sub-optimal BTKi responsePhase IIIongoing registrationalGlobalFirst-line CLL/SLLPhase IIIongoing registrationalGlobal
APG-2449APG-2449 / FAK/ALK/ROS1 / small molecule TKI / Oncology
ALK-positive advanced/locally advanced NSCLCPhase IIIongoing registrationalChinaNSCLC resistant/intolerant to 2nd-generation ALK TKIsPhase IIIongoing registrationalChinaPlatinum-resistant ovarian cancerPhase Ib/IIongoing
AlrizomadlinAPG-115 / MDM2-p53 / small molecule inhibitor / Oncology
Unresectable/metastatic melanoma or advanced solid tumorsPhase Ib/IIongoingUnited StatesAustraliaR/R T-PLL or NHLPhase IIaongoingUnited StatesAustraliaSalivary gland cancerPhase IIongoing
PelcitoclaxAPG-1252 / Bcl-2/Bcl-xL / small molecule inhibitor / Oncology
EGFR-mutant NSCLCPhase Ibongoing with osimertinibChinaR/R NHLPhase Ib/IIongoingChinaRecurrent ovarian and endometrial cancersPhase Iongoing with cobimetinib
APG-5918APG-5918 / EED/PRC2 / small molecule inhibitor / Oncology; Hematology
Advanced solid tumors and hematologic malignanciesPhase IongoingChinaUnited StatesAnemia-related indicationsPhase IongoingChina
APG-3288APG-3288 / BTK / PROTAC degrader / Oncology
R/R B-cell malignanciesPhase IIND cleared; study planned/initiating in 2026United StatesChina

ANRO

Altoneuroscience

altoneuroscience.com

7assets

ALTO-207ALTO-207 / D3/D2 & 5-HT3 / fixed-dose combination of pramipexole and ondansetron / neuropsychiatric disorders
treatment-resistant depressionPhase 2b plannedon track to initiate in 1H 2026U.S.U.K.
ALTO-300ALTO-300 / MT1/2 & 5-HT2C / small molecule melatonergic agonist and serotonergic antagonist / neuropsychiatric disorders
major depressive disorderPhase 2bongoingUnited States
ALTO-100ALTO-100 / BDNF / oral small molecule / neuropsychiatric disorders
bipolar depressionPhase 2bongoing
ALTO-101ALTO-101 / PDE4 / PDE4 inhibitor; transdermal and modified-release oral formulations / neuropsychiatric disorders
cognitive impairment associated with schizophreniaPhase 2 proof-of-concept completednot independently advancing; seeking partnering opportunities
ALTO-203ALTO-203 / H3 / oral small molecule histamine H3 receptor inverse agonist / neuropsychiatric disorders
major depressive disorder with higher anhedoniaPhase 2aproof-of-concept study completed
ALTO-202ALTO-202 / NMDA NR2B / oral small molecule negative allosteric modulator / neuropsychiatric disorders
major depressive disorder
ALTO-208ALTO-208 / D3/D2 & NK-1 / fixed-dose combination of pramipexole and aprepitant / neurology
Parkinson's diseasein development

ARVN

Arvinas

arvinas.com

7assets

ARV-102ARV-102 / LRRK2 / oral brain-penetrant PROTAC degrader / Neurology
Parkinson’s diseasePhase 1/1bunder investigationProgressive supranuclear palsyunder investigation
ARV-027ARV-027 / polyQ-AR / oral peripherally restricted PROTAC degrader / Neurology
Spinal-bulbar muscular atrophyPhase 1/1bunder investigation
ARV-806ARV-806 / KRAS G12D / PROTAC degrader / Oncology
Pancreatic cancerPhase 1/1bunder investigationColorectal cancerPhase 1/1bunder investigationNon-small cell lung cancerPhase 1/1bunder investigation
ARV-393ARV-393 / BCL6 / oral PROTAC degrader / Oncology
Non-Hodgkin lymphoma / B-cell malignanciesPhase 1/1bunder investigation
ARV-6723ARV-6723 / HPK1 / oral PROTAC degrader / Oncology
Advanced solid tumorsPreclinicalunder investigation
VepdegestrantARV-471 / ER / oral PROTAC estrogen receptor degrader / Oncology
ER+/HER2- locally advanced or metastatic breast cancerPhase 3under investigation
LuxdegalutamideARV-766; JSB462 / AR / oral PROTAC androgen receptor degrader / Oncology
Prostate cancerPhase 2under investigation

CLSD

Clearside Bio

clearsidebio.com

7assets

CLS-AX (axitinib)CLS-AX / VEGF receptors-1, -2, and -3 / Tyrosine kinase inhibitor; injectable suspension / Ophthalmology
Wet AMDPhase 3 readyFDA End-of-Phase 2 / Phase 3 ready
Undisclosed small moleculeImprove choroidal perfusion / Small molecule / Ophthalmology
Geographic Atrophy (GA)Undisclosed
Undisclosed small moleculeModulate pro-inflammatory cells / Small molecule / Ophthalmology
Geographic Atrophy (GA)Undisclosed
XIPERE / ARCATUS (triamcinolone acetonide injectable suspension)ARVN001 / XIPERE / ARCATUS / Corticosteroid injectable suspension / Ophthalmology
Uveitic Macular EdemaCurrent status not text-disclosed on pipeline pageAustraliaSingaporeChinaDiabetic Macular EdemaCurrent status not text-disclosed on pipeline pageAsia Pacific (ex-Japan)
Bel-SarViral-like drug conjugate / Ocular oncology
Choroidal MelanomaPhase 3CoMpass trial
ABBV-RGX-314ABBV-RGX-314 / Anti-VEGF / AAV gene therapy / Ophthalmology
Diabetic RetinopathyPhase 2/3ALTITUDE / Phase 3 planningWet AMDPhase 2AAVIATE
AvoralstatPlasma kallikrein / Plasma kallikrein inhibitor / Ophthalmology
Diabetic Macular EdemaIND-enablingClinical trial authorization disclosed by partnerAustralia

GMAB

Genmab

genmab.com

7assets

epcoritamabGEN3013 / EPKINLY/TEPKINLY / CD20xCD3 / Bispecific antibody / Oncology
Relapsed or refractory diffuse large B-cell lymphoma (DLBCL)ApprovedMarketedUSEUJapanRelapsed or refractory follicular lymphoma (FL)ApprovedMarketedUSEURelapsed or refractory FL in combination with rituximab and lenalidomide (R2)ApprovedMarketedUS
rinatabart sesutecanRina-S / Rina-S / FRα (folate receptor alpha) / Antibody-drug conjugate (ADC) / Oncology
Platinum-resistant ovarian cancerPhase 3ActiveGlobalAdvanced endometrial cancerPhase 1/2ActiveGlobal
petosemtamabMCLA-158 / EGFRxLGR5 / Bispecific antibody / Oncology
First-line head and neck squamous cell carcinoma (HNSCC)Phase 3ActiveGlobalSecond/third-line head and neck squamous cell carcinoma (HNSCC)Phase 3ActiveGlobal
daratumumabDARZALEX/DARZALEX FASPRO / CD38 / Monoclonal antibody / Oncology
Multiple myeloma (multiple lines)ApprovedMarketedGlobal
ofatumumabKesimpta/Arzerra / CD20 / Monoclonal antibody / Neurology/Oncology
Relapsing multiple sclerosis (Kesimpta)ApprovedMarketedGlobalChronic lymphocytic leukemia (Arzerra)ApprovedMarketedUSEU
tisotumab vedotinTivdak / Tissue factor / Antibody-drug conjugate (ADC) / Oncology
Recurrent or metastatic cervical cancerApprovedMarketedUSEUJapan
HexaBody-CD38GEN3014 / CD38 / HexaBody monoclonal antibody / Oncology
Multiple myelomaPhase 1/2ActiveGlobal

GPCR

Structuretx

ir.structuretx.com

7assets

AleniglipronGSBR-1290 / GLP-1R / Oral small molecule agonist / Metabolic / Obesity
Obesity / overweight with weight-related comorbidityPhase 2 (ACCESS / ACCESS II); Phase 3 planned 2H 2026Active; End-of-Phase 2 FDA meeting Q2 2026GlobalType 2 diabetes mellitus with obesity/overweightPhase 2Enrolling (38-week study)Global
ACCG-2671ACCG-2671 / Amylin/calcitonin receptor (DACRA) / Oral small molecule agonist / Metabolic / Obesity
ObesityPhase 1Active (SAD study initiated Dec 2025)Global
ACCG-3535ACCG-3535 / Amylin/calcitonin receptor (DACRA) / Oral small molecule agonist / Metabolic / Obesity
ObesityPreclinical / IND-enablingPhase 1 initiation expected 2H 2026Global
ANPA-0073ANPA-0073 / Apelin (APJ) receptor / Oral small molecule biased agonist / Metabolic / Obesity
Obesity (selective/muscle-sparing weight loss)Phase 2 readyPhase 1 SAD/MAD completedGlobal
LTSE-2578LTSE-2578 / LPA1R (lysophosphatidic acid 1 receptor) / Oral small molecule antagonist / Pulmonary / Fibrosis
Idiopathic pulmonary fibrosis (IPF)Phase 1Phase 1 SAD/MAD completedGlobal
GIPR ProgramGIPR (and GLP-1R/GIPR) / Oral small molecule (agonist/antagonist) / Metabolic / Obesity
Obesity and related diseasesDiscovery / PreclinicalActiveGlobal
GCGR ProgramGCGR (and GLP-1R/GCGR) / Oral small molecule agonist / Metabolic / Obesity
Obesity and related diseasesDiscovery / PreclinicalActiveGlobal

HRMY

Harmony Biosciences

harmonybiosciences.com

7assets

PitolisantWAKIX / histamine-3 receptor (H3R) antagonist/inverse agonist / small molecule / Rare neurological disorders
Prader-Willi syndromePhase 3USNarcolepsy (GR formulation)Phase 3USNarcolepsy (HD formulation)Phase 3US
BP1.15205orexin-2 receptor (OX2R) agonist / small molecule / Sleep/wake disorders
Narcolepsy and other sleep/wake disordersPhase 1USLatin America
EPX-1005-HT (serotonin) receptor agonist / small molecule / Rare epilepsies
Dravet syndromePhase 3USLennox-Gastaut syndromePhase 3US
EPX-200selective 5HT2C agonist / small molecule / Rare epilepsies
Developmental and epileptic encephalopathiesPreclinicalIND-enablingUS
CBS-105cell therapy / Rare neurological disorders
Treatment-resistant narcolepsyPreclinicalUS
HBS-102MCHR1 antagonist / small molecule / Rare neurological disorders
Prader-Willi syndromePreclinicalUS
CBS-104cell therapy / Rare epilepsies
Refractory epilepsyPreclinicalUS

INVA

Inva

inva.com

7assets

IOVA

Iovance

iovance.com

7assets

LifileucelLN-144 / Amtagvi / Polyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
MelanomaNon-Small Cell Lung Cancer (NSCLC)
TIL CombinationsPolyclonal Neoantigens / Cell Therapy Combination / Oncology
Solid Tumors
PD-1 Selected TILPolyclonal Neoantigens / PD-1+ / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
Solid Tumors
Third Generation (Gen 3) TILPolyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
Solid Tumors
PBL TherapyPolyclonal Neoantigens / Peripheral Blood Lymphocyte Therapy / Oncology
Solid Tumors
PD-1 Inactivated TILIOV-4001 / PD-1 / Genetically Modified Cell Therapy / Oncology
Solid Tumors
Modified IL-2 Fusion ProteinIOV-3001 / IL-2 Receptor / Antibody Cytokine Engrafted Protein / Oncology
MelanomaSolid Tumors

JANX

Januxrx

januxrx.com

7assets

JANX007PSMA-TRACTr
JANX008EGFR-TRACTr
JANX009PD-L1/CD28-TRACIr
JANX011CD19-ARM
JANX013PSMA-TRACIr
JANX014PSMA-directed TCE
TROP2-TRACTr

LEGN

Legend Biotech

legendbiotech.com

7assets

ciltacabtagene autoleucelcilta-cel / CARVYKTI
LUCAR-G39D
LB1909
LB1905
CD19 x GPRC5D CAR-T
LCAR-AIO
CD20/CD19 dual-targeting in vivo CAR-T

LXEO

Lexeotx

lexeotx.com

7assets

LX2006LX2006 / FXN / AAV gene therapy / Cardiovascular
Friedreich's ataxia (FA) cardiomyopathy
LX2020LX2020 / PKP2 / AAV gene therapy / Cardiovascular
PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)
LX2021LX2021 / Cx43 / AAV gene therapy / Cardiovascular
DSP (Desmoplakin) cardiomyopathyDilated cardiomyopathy
LX2022LX2022 / TNNI3 / AAV gene therapy / Cardiovascular
Hypertrophic cardiomyopathy (HCM)
LX1001LX1001 / APOE2 / AAV gene therapy / Neurology
APOE4-associated Alzheimer's disease
LX1021LX1021 / Christchurch-modified APOE2 / AAV gene therapy / Neurology
APOE4-associated Alzheimer's disease
LX1020LX1020 / APOE2 / APOE4 (miRNA) / AAV gene therapy / Neurology
APOE4-associated Alzheimer's disease

MNKD

Mannkindcorp

mannkindcorp.com

7assets

insulin humanAfrezza / Insulin receptor / Recombinant Protein / Endocrine
Type 1 and Type 2 Diabetes MellitusMarketedApprovedUSBrazilPediatric Diabetes MellitusPhase 3OngoingUS
treprostinilTyvaso DPI / Prostacyclin receptor / Small Molecule / Orphan Lung Diseases
Pulmonary Arterial Hypertension (PAH)MarketedApprovedUSPulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)MarketedApprovedUS
clofazimine inhalation suspensionMNKD-101 / Bacterial DNA / Mycobacterial growth / Small Molecule / Orphan Lung Diseases
Nontuberculous Mycobacterial (NTM) Lung DiseasePhase 3DiscontinuedUS
clofazimine DPIMNKD-102 / Bacterial DNA / Mycobacterial growth / Small Molecule / Orphan Lung Diseases
Nontuberculous Mycobacterial (NTM) Lung DiseasePreclinicalActiveUS
nintedanib DPIMNKD-201 / Tyrosine kinase / Small Molecule / Orphan Lung Diseases
Idiopathic Pulmonary Fibrosis (IPF)Phase 1CompletedUS
dornase alfa DPIMNKD-301 / DNase I / Recombinant Protein / Orphan Lung Diseases
Cystic FibrosisPreclinicalActiveUS
FBM5712 DPIMNKD-501 / ALK-5 kinase / TGF-beta / Small Molecule / Orphan Lung Diseases
Pulmonary FibrosisPreclinicalActiveUS

OPK

Opko

opko.com

7assets

SomatrogonhGH-CTP / NGENLA / Growth hormone receptor / Biologic / Endocrinology
Growth Hormone Deficiency (Pediatric)ApprovedactiveUSEUJapanGlobalGrowth Hormone Deficiency (Adults)Phase 3activeGlobal
Calcifediol extended-releaseRayaldee / Rayaldee / Vitamin D receptor / Small Molecule / Nephrology
Secondary Hyperparathyroidism in Stage 5 Chronic Kidney Disease (Hemodialysis)Phase 2activeUS
MDX2004MDX2004 / CD3 x CD28 x 4-1BB / Multispecific Antibody / Oncology
Advanced CancersPhase 1/2aactiveUS
MDX2003MDX2003 / T-cell engager / Multispecific Antibody / Oncology
LymphomaPhase 1activeUS
ModeX COVID-19 Multispecific AntibodySARS-CoV-2 / Multispecific Antibody / Infectious Disease
COVID-19PreclinicalactiveUS
ModeX Influenza Multispecific AntibodyInfluenza virus / Multispecific Antibody / Infectious Disease
InfluenzaPreclinicalactiveUS
Oral GLP-2 AgonistGLP-2 / Peptide / Gastroenterology
Short Bowel SyndromePreclinicalactiveGlobal

RADX

Radiopharmtheranostics

radiopharmtheranostics.com

7assets

18F-PivalateRAD 101 / FASN (Fatty acid synthase) / Small Molecule Radiopharmaceutical / Diagnostics
Brain metastases from solid tumors (Imaging)Phase 2bEnrollingUS
177Lu-RAD202RAD 202 / HER2 / Nanobody Radiotherapeutic / Oncology
HER2-positive advanced solid tumorsPhase 1ActiveUSGlobal
177Lu-RAD204RAD 204 / PD-L1 / Nanobody Radiotherapeutic / Oncology
PD-L1 positive advanced solid tumorsPhase 1ActiveUSGlobal
68Ga-TrivehexinRAD 301 / αvβ6-integrin / Peptide Radiopharmaceutical / Diagnostics
Loco-regional pancreatic cancer (Imaging)Phase 2ActiveGlobal
177Lu-D0301RAD 302 / αvβ6-integrin / Peptide Radiotherapeutic / Oncology
Solid tumors (PDAC, NSCLC, HNSCC)PreclinicalActiveGlobal
BetaBartRV-01 / B7-H3 / Radiotherapeutic / Oncology
B7H3-expressing solid tumorsPhase 1/2aActiveUS
161Tb-RAD 402RAD 402 / KLK3 (PSA) / Monoclonal Antibody Radiotherapeutic / Oncology
Advanced prostate cancerPhase 1ActiveAustralia

SLDB

Solid Bio

solidbio.com

7assets

SGT-003SGT-003 / Gene Therapy / Neuromuscular
Duchenne muscular dystrophy (INSPIRE)Phase 1/2ActiveGlobalDuchenne muscular dystrophy (IMPACT)Phase 3Activeex-U.S.
SGT-212SGT-212 / Gene Therapy / Neuromuscular
Friedreich's ataxia (FA)Phase 1/2ActiveGlobal
SGT-501SGT-501 / Gene Therapy / Cardiac
RYR2-Mediated CPVTPhase 1/2ActiveGlobalCASQ2-Mediated CPVTPreclinicalActiveGlobal
SGT-601SGT-601 / TNNT2 / Gene Therapy / Cardiac
TNNT2-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
SGT-401SGT-401 / BAG3 / Gene Therapy / Cardiac
BAG3-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
SGT-701SGT-701 / RBM20 / Gene Therapy / Cardiac
RBM20-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
Undisclosed Cardiac TargetsGene Therapy / Cardiac
Genetic cardiomyopathies and channelopathiesDiscoveryActiveGlobal

SYRE

Spyre

spyre.com

7assets

SPY001SPY001 / α4β7 / Monoclonal antibody / Gastroenterology
Inflammatory Bowel Disease (IBD)Ulcerative Colitis
SPY002SPY002 / TL1A / Monoclonal antibody / Gastroenterology / Rheumatology
Inflammatory Bowel Disease (IBD)Rheumatoid Arthritis (RA)
SPY003SPY003 / IL-23 / Monoclonal antibody / Gastroenterology
Inflammatory Bowel Disease (IBD)
SPY120SPY120 / α4β7 + TL1A / Combination / Gastroenterology
Inflammatory Bowel Disease (IBD)
SPY130SPY130 / α4β7 + IL-23 / Combination / Gastroenterology
Inflammatory Bowel Disease (IBD)
SPY230SPY230 / TL1A + IL-23 / Combination / Gastroenterology
Inflammatory Bowel Disease (IBD)Ulcerative Colitis
SPY072SPY072 / TL1A / Monoclonal antibody / Rheumatology
Rheumatoid Arthritis (RA)Psoriatic Arthritis (PsA)Axial Spondyloarthritis (axSpA)

TNXP

Tonix Pharma

tonixpharma.com

7assets

URGN

Urogen

urogen.com

7assets

JelmytoMitomycin for Pyelocalyceal Solution
UGN-102Mitomycin for Intravesical Solution
UGN-103
UGN-104
UGN-201
UGN-301
UGN-501ICVB-1042

VYGR

Voyager Therapeutics

voyagertherapeutics.com

7assets

VY7523VY-TAU01 / Tau / Monoclonal Antibody / Neurology
Alzheimer's DiseasePhase 1OngoingUnited States
VY1706Tau / AAV Gene Therapy / Neurology
Alzheimer's DiseaseIND-EnablingOngoingUnited States
APOE Gene TherapyAPOE / AAV Gene Therapy / Neurology
Alzheimer's DiseasePreclinicalOngoing
NBIB-'223NBIB-223 / Frataxin (FXN) / AAV Gene Therapy / Neurology
Friedreich's Ataxia (FA)IND-EnablingOngoing
GBA1 Gene TherapyGBA1 / AAV Gene Therapy / Neurology
Parkinson's Disease and GBA1-mediated diseasesIND-EnablingOngoing
Novartis Partnered ProgramsUndisclosed / AAV Gene Therapy / Neurology
Huntington's Disease, Spinal Muscular Atrophy (SMA), UndisclosedPreclinicalOngoing
Alexion Partnered ProgramUndisclosed / AAV Gene Therapy / Neurology
Undisclosed Neurological DiseasePreclinicalOngoing

ACAD

Acadia Pharmaceuticals

acadia.com

6assets

RemlifanserinACP-204 / 5-HT2A receptor / inverse agonist / Neurological Disorders
Alzheimer’s Disease PsychosisPhase 2RecruitingLewy Body Dementia with PsychosisPhase 2
ACP-211ACP-211 / selectively deuterated R-norketamine / Neurological Disorders
Major Depressive DisorderPhase 2
ACP-711ACP-711 / GABAA α3 / modulator / Neurological Disorders
Essential TremorPhase 1
ACP-271ACP-271 / GPR88 / agonist / Neurological Disorders; Rare Diseases
Tardive DyskinesiaIND EnablingHuntington's DiseaseIND Enabling
ACP-2591ACP-2591 / cGP analog / Rare Diseases
Rett Syndrome; Fragile X SyndromePhase 1
STOKE ASOantisense oligonucleotide / Rare Diseases
SYNGAP1Discovery

ACIU

AC Immune

acimmune.com

6assets

ACI-24.060ACI-24.060 / Abeta / active immunotherapy / neurodegenerative disease
Alzheimer's diseasePhase 2ongoing ABATE trialDown syndrome-related Alzheimer's diseasePhase 1b/2ongoing ABATE trial
ACI-7104.056ACI-7104.056 / alpha-synuclein / active immunotherapy / neurodegenerative disease
Parkinson's diseasePhase 2ongoing VacSYn trial
ACI-35.030JNJ-2056; JNJ-64042056 / phosphorylated Tau / active immunotherapy / neurodegenerative disease
preclinical Alzheimer's diseasePhase 2bongoing ReTain trial
ACI-19764ACI-19764 / NLRP3 inflammasome / small molecule inhibitor / neurodegenerative and inflammatory diseases
healthy volunteersPhase 1ongoing
Morphomer-Tau aggregation inhibitorsTau / small molecule aggregation inhibitor / neurodegenerative disease
Alzheimer's disease and other neurodegenerative diseasesIND-enablingplanned/initiating
Morphomer alpha-synuclein aggregation inhibitoralpha-synuclein / small molecule aggregation inhibitor / neurodegenerative disease
Parkinson's diseasepreclinicallead declaration/readiness for IND-enabling

ALEC

Alector

alector.com

6assets

AL101PGRN / antibody / Neurodegenerative diseases
Alzheimer’s diseasePhase 2
AL137-ABCAmyloid beta / ABC-enabled antibody / Neurodegenerative diseases
Alzheimer’s diseasePreclinical
AL050-ABCGCase / ABC-enabled enzyme replacement therapy / Neurodegenerative diseases
Parkinson’s diseasePreclinicalGaucher diseasePreclinicalLewy body dementiaPreclinical
AL064-ABCTau / ABC-enabled siRNA / Neurodegenerative diseases
Alzheimer’s diseaseResearchFrontotemporal dementiaResearch
ADP062-ABCAlpha-synuclein / ABC-enabled siRNA / Neurodegenerative diseases
Parkinson’s diseaseResearch
ADP065-ABCNLRP3 / ABC-enabled siRNA / Neurodegenerative diseases
Alzheimer’s diseaseResearchParkinson’s diseaseResearch

APLS

Apellis

apellis.com

6assets

APL-3007 + SYFOVREAPL-3007 / SYFOVRE / C3 / siRNA + pegcetacoplan injection / Ophthalmology
Geographic atrophyPhase 2
EMPAVELIpegcetacoplan / EMPAVELI / C3 / C3 inhibitor / Rare disease
FSGSPivotal Phase 2/3DGFPivotal Phase 3
RNA therapiesRNA therapy / Neurology
UndisclosedPreclinical
APL-9099: Gene-edited FcRn therapyAPL-9099 / FcRn / gene-edited therapy / Multiple therapeutic areas
UndisclosedPreclinical
Gene-edited complement therapiescomplement / gene-edited therapy / Multiple therapeutic areas
UndisclosedPreclinical
Oral complement inhibitorcomplement / oral complement inhibitor / Multiple therapeutic areas
UndisclosedPreclinical

AXSM

Axsome

axsome.com

6assets

AXS-05AXS-05 / AUVELITY / NMDA receptor; sigma-1 receptor; CYP2D6 / oral small molecule / CNS
Smoking cessationPhase 2/3 plannedon track to initiate in 2Q 2026US
SolriamfetolSUNOSI / dopamine and norepinephrine reuptake; TAAR1; 5-HT1A / oral small molecule / CNS
Pediatric ADHDPhase 3 plannedtwo trials on track to initiate in 2Q 2026USMDD with excessive daytime sleepiness symptomsPhase 3CLARITY initiated February 2026USBinge eating disorderPhase 3ENGAGE ongoingUS
AXS-12AXS-12 / norepinephrine reuptake inhibitor; cortical dopamine modulator / oral small molecule / CNS
Cataplexy in narcolepsyNDA submittedFDA filing acceptance decision pendingUS
AXS-14AXS-14 / norepinephrine reuptake inhibitor / oral small molecule / CNS
FibromyalgiaPhase 3FORWARD ongoingUS
AXS-17AXS-17 / GABAA receptor alpha2,3 positive allosteric modulator / oral small molecule / CNS
EpilepsyPhase 2-enablingtrial-enabling activities underwayUS
AXS-20AXS-20 / PDE10A / oral small molecule / CNS
SchizophreniaPhase 3-enablingtrial-enabling activities anticipated in 2026USTourette syndromeplanned evaluationUS

BEAM

Beamtx

beamtx.com

6assets

Ristoglogene autogetemcelBEAM-101 / Risto-cel / HBG1/2 promoter; fetal hemoglobin activation / Ex vivo autologous CD34+ HSPC base-edited cell therapy / Hematology
Sickle cell diseasePhase I/IIOngoing BEACON Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
ESCAPEBEAM-103 / CD117; HbF edit + CD117 edit-antibody pair / Anti-CD117 monoclonal antibody plus edited cell therapy; in vivo and/or ex vivo / Hematology
Sickle cell diseasePhase I-EnablingBeta-thalassemiaPhase I-Enabling
In vivo HSC editingHbF activation / In vivo LNP base editing / Hematology
Sickle cell diseaseResearchBeta-thalassemiaResearch
BEAM-302BEAM-302 / SERPINA1 E342K/PiZ mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
Alpha-1 antitrypsin deficiencyPhase I/IIOngoing Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
BEAM-301BEAM-301 / G6PC R83C mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
Glycogen storage disease IaPhase I/IIOngoing Phase 1/2 trial
BEAM-304BEAM-304 / PAH mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
PhenylketonuriaPhase I-EnablingPlanned Phase 1/2 trialU.S.

BMRN

Biomarin

biomarin.com

6assets

VosoritideBMN 111 / VOXZOGO / CNP analog / FGFR3 pathway / Peptide / Skeletal Conditions
HypochondroplasiaPhase 3Active clinical developmentIdiopathic short staturePhase 2Active clinical developmentNoonan syndromePhase 2Active clinical development
BMN 333BMN 333 / Long-acting C-type natriuretic peptide (CNP) / Peptide / Skeletal Conditions
AchondroplasiaPhase 1Clinical development; Phase 2/3 plannedHypochondroplasiaPreclinicalNot started / planned
PegvaliasePALYNZIQ / Phenylalanine ammonia lyase enzyme substitution / Enzyme biologic / Enzyme Therapies
Phenylketonuria, adolescents aged 12-17Phase 3Active, not recruitingUnited StatesEuropePhenylketonuria, pregnancy and breastfeeding exposure registryPhase 4RecruitingPhenylketonuria, hypersensitivity rapid drug desensitizationPhase 4Recruiting
BMN 401BMN 401; formerly INZ-701 / ENPP1 enzyme replacement / Enzyme biologic / Innovation
ENPP1 DeficiencyPhase 3Active, not recruiting / recruiting studiesABCC6 DeficiencyPhase 1Recruiting / active long-term study
BMN 351BMN 351 / Exon 51 skipping / Antisense oligonucleotide / Innovation
Duchenne muscular dystrophy amenable to exon 51 skippingPhase 1/2Recruiting
BMN 349BMN 349 / Small molecule / Innovation
Alpha-1 antitrypsin deficiency-associated liver diseasePhase 1Recruiting

BTAI

Bioxcel Therapeutics

bioxceltherapeutics.com

6assets

BXCL501BXCL501 / IGALMI / alpha-2 receptor agonist / orally dissolving film formulation of dexmedetomidine / Neuroscience
Acute treatment of agitation associated with bipolar I/II disorder or schizophrenia in adults under healthcare provider supervisionMarketedApprovedUSAcute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home settingPhase 3Company-sponsored SERENITY programAcute treatment of agitation associated with Alzheimer’s dementiaPhase 3Company-sponsored TRANQUILITY program; paused subject to funding
BXCL502BXCL502 / latrepirdine / Neuroscience
Chronic agitation in dementiaPreclinicalDevelopment paused due to Strategic Reprioritization announced August 14, 2023
BXCL503BXCL503 / Neuroscience
Apathy in dementiaPreclinicalDevelopment paused due to Strategic Reprioritization announced August 14, 2023
BXCL504BXCL504 / Neuroscience
Aggression in dementiaPreclinicalDevelopment paused due to Strategic Reprioritization announced August 14, 2023
BXCL701BXCL701 / DPP8/9 and FAP inhibitor; innate immune activator / oral small molecule / Oncology
Small cell neuroendocrine prostate cancer (SCNC)Phase 2Company-sponsored trial; expected FDA meetingUSUKSmall cell lung cancer (SCLC)PreclinicalCompany-sponsored; initiate Phase 1b/2 expectedMetastatic pancreatic ductal adenocarcinomaPhase 1Investigator-sponsored trial; Phase 2 readout expected
BXCL702BXCL702 / novel DPP inhibitor / Oncology
Solid tumorsPreclinicalBXCL701 follow-on; candidate nomination expected

COGT

Cogent Bio

cogentbio.com

6assets

BezuclastinibCGT9486 / KIT activation loop mutations / KIT D816V / small-molecule tyrosine kinase inhibitor / Oncology; hematology
NonAdvanced systemic mastocytosis (NonAdvSM)NDA acceptedPDUFA target action date December 30, 2026; based on SUMMIT pivotal trialUSAdvanced systemic mastocytosis (AdvSM)registration-directed trialAPEX trial; NDA submission planned in 1H 2026GlobalUSGastrointestinal stromal tumors (GIST), imatinib-resistant / second-line, in combination with sunitinibNDA submitted; Phase 3PEAK Phase 3 positive; NDA submitted under RTORGlobalUS
CGT4859CGT4859 / FGFR2/3 / small-molecule inhibitor / Oncology
FGFR2/3-altered cancersPhase 1/2Clinical data planned in 2026
CGT4255CGT4255 / ErbB2 / HER2 / small-molecule inhibitor / Oncology
HER2/ErbB2-altered cancersPhase 1Dose escalation expected to complete in 2026
CGT6297CGT6297 / PI3Kα mutant / small-molecule inhibitor / Oncology
PI3Kα-mutant cancersPhase 1Dose escalation expected to complete in 2026
Selective pan-KRAS inhibitorKRAS / small-molecule inhibitor / Oncology
KRAS-mutant cancerspreclinicalIND application expected in 2026
JAK2 V617F inhibitorJAK2 V617F / small-molecule inhibitor / Hematology
JAK2 V617F-driven diseasepreclinicalIND application expected in 2026

CRNX

Crinetics

crinetics.com

6assets

ETON

Eton Pharma

etonpharma.com

6assets

AmglidiaAmglidia / glyburide oral suspension / Pediatric endocrinology / rare disease
Neonatal diabetes mellitusLate-stage development; NDA submission anticipated in 2026In developmentUnited States
ET-700ET-700 / extended-release zinc acetate formulation / Rare metabolic disease
Wilson diseasePilot clinical study / proof-of-conceptFirst patient dosed; topline results expected H2 2026; potential pivotal study in early 2027United States
ET-800ET-800 / Rare disease
UndisclosedLate-stage developmentIn development
ZENEO hydrocortisone autoinjectorZENEO / hydrocortisone autoinjector / Endocrinology
Adrenal insufficiency / adrenal crisis managementLate-stage developmentIn developmentUnited States
KHINDIVI revised formulationKHINDIVI / hydrocortisone oral solution / Pediatric endocrinology
Adrenocortical insufficiency label expansion to younger pediatric patientsBioequivalence study; supplemental NDA plannedStudy initiated; supplement expected in Q3 2026; potential mid-2027 approvalUnited States
INCRELEX label expansionINCRELEX / IGF-1 receptor pathway / mecasermin injection / Pediatric endocrinology
Severe primary IGF-1 deficiency definition harmonization / label expansionPlanned clinical studyFDA feedback requested; study could initiate in H1 2026 if favorableUnited States

FATE

Fate Therapeutics

fatetherapeutics.com

6assets

FT819FT819 / CD19 / iPSC-derived off-the-shelf CAR T-cell therapy / Autoimmunity
Systemic Lupus ErythematosusPhase 1RMATUSSystemic Sclerosis (SSc)Phase 1USANCA associated Vasculitis (AAV)Phase 1US
FT839FT839 / CD19/CD38/CD20; CD19/CD38/BCMA/GPRC5D / Next-generation CAR T-cell therapy / Autoimmunity; Oncology
Pan-indication without lympho-conditioning chemotherapyPhase 1USPan-indication (hematologic) without lympho-conditioning chemotherapyPhase 1US
FT522FT522 / CD19/CD20 / Cellular immunotherapy / Autoimmunity
Pan-indication without lympho-conditioning chemotherapyPhase 1US
FT825FT825 / HER2/EGFR / iPSC-derived off-the-shelf CAR T-cell therapy / Oncology
Solid tumorsPhase 1EnrollingUS
UndisclosedUndisclosed / Cellular immunotherapy / Oncology
Solid tumorsPhase 1US
FT836FT836 / MICA/B/EGFR/HER2 / Next-generation CAR T-cell therapy / Oncology
Pan-indication (solid/hematologic) without lympho-conditioning chemotherapyPhase 1US

HROW

Harrow

harrow.com

6assets

MELT-300Sublingual Sedation
IHEEZOchloroprocaine hydrochloride
VEVYEcyclosporine ophthalmic solution
TRIESENCEtriamcinolone acetonide
BYOOVIZranibizumab-nuna
OPUVIZaflibercept-yszy

HUMA

Humacyte

humacyte.com

6assets

Symvess (ATEV)Symvess / Acellular Tissue Engineered Vessel / Vascular Disease
Vascular trauma repairApprovedFDA Approved (Dec 2024)United StatesArteriovenous access for hemodialysisPhase 3V007 met primary endpoints; V012 ongoing; sBLA planned H2 2026United StatesPeripheral artery disease (PAD)Phase 2Completed Phase 2 trials and expanded access; positive limb salvage dataUnited StatesEurope
CTEVCoronary Tissue Engineered Vessel / Cardiovascular Disease
Coronary artery bypass grafting (CABG)PreclinicalPositive NHP data; IND submitted; first-in-human planned H2 2026United States
Biovascular Pancreas (BVP)Biovascular Pancreas™ / Bioengineered Organ / Diabetes
Type 1 diabetesPreclinicalPositive preclinical data; NHP studies planned; first-in-human planned 2027
Urinary ConduitAdvanced Tissue Construct / Urology
Urinary diversion after cystectomyPreclinical
Tracheal ReplacementAdvanced Tissue Construct / Otolaryngology
Tracheal replacementPreclinical
Esophageal ReplacementAdvanced Tissue Construct / Gastroenterology
Esophageal replacementPreclinical

IMNM

Immunome

immunome.com

6assets

INO

Inovio

inovio.com

6assets

INO-3107INO-3107 / HPV-6 and HPV-11 / DNA immunotherapy / HPV-related rare disease
Recurrent Respiratory Papillomatosis (RRP) in adultsBLA accepted under accelerated approval, PDUFA Oct 30 2026United States
VGX-3100VGX-3100 / HPV-16/18 / DNA immunotherapy / HPV-related diseases
Cervical dysplasia (HSIL)3Ongoing (partnered)ChinaAnal dysplasia
INO-3112INO-3112 / HPV-16/18 / DNA immunotherapy / Oncology
HPV16/18-positive oropharyngeal squamous cell carcinoma (OPSCC)3Phase 3 planningUnited StatesEurope
INO-5401INO-5401 / Tumor-associated antigens / DNA immunotherapy / Oncology
Glioblastoma (GBM)1Ongoing Phase 1; Phase 2 planning (as INO-5412 w/ Akeso)United StatesPrevention in BRCA1/2 mutation carriers1Ongoing Phase 1United States
INO-4201INO-4201 / Ebola virus / DNA medicine / Infectious diseases
Ebola vaccine booster (to ERVEBO)2Phase 2 planning; Phase 1b completed
DMAb SARS-CoV-2SARS-CoV-2 / DNA-encoded monoclonal antibody / Infectious diseases
SARS-CoV-21Ongoing Phase 1; data expected/reportedUnited States

IVVD

Invivyd

invivyd.com

6assets

PemivibartPEMGARDA / SARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
Pre-exposure prophylaxis (PrEP) of COVID-19
VYD2311VYD2311 / SARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
COVID-19 prevention
AdintrevimabSARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
COVID-19 prevention and treatment
Measles mAbMeasles (rubeola) virus / Monoclonal Antibody / Infectious Diseases
Measles treatment and post-exposure prophylaxis
RSV mAbRespiratory Syncytial Virus (RSV) / Monoclonal Antibody / Infectious Diseases
RSV infection
Influenza mAbInfluenza virus / Monoclonal Antibody / Infectious Diseases
Influenza infection

MOLN

Molecularpartners

molecularpartners.com

6assets

MP0712MP0712 / DLL3 / Radio-DARPin Therapy / Oncology
Small Cell Lung Cancer (SCLC)Neuroendocrine Tumors
MP0726MP0726 / MSLN / Radio-DARPin Therapy / Oncology
Ovarian Cancer
MP0533MP0533 / CD33 x CD123 x CD70 x CD3 / Multispecific T Cell Engager / Oncology
Relapsed/Refractory AMLAML/MDS
MP0317MP0317 / FAP x CD40 / Tumor-localized CD40 Agonist / Oncology
Advanced Solid TumorsCholangiocarcinoma
MP0632MP0632 / CD2 x CD3 x MSLN x EpCAM / Switch-DARPin T Cell Engager / Oncology
MSLN/EpCAM Expressing Solid TumorsOvarian Cancer
MP0621MP0621 / cKIT x CD16a x CD47 / Switch-DARPin / Hematology
Conditioning for Hematopoietic Stem Cell Transplantation

MPLT

Maplightrx

maplightrx.com

6assets

MTVA

Metaviatx

metaviatx.com

6assets

DA-1726DA-1726 / GLP1R / GCGR (Glucagon-like peptide-1 receptor and glucagon receptor) / Oxyntomodulin (OXM) analogue / Cardiometabolic Diseases
ObesityPhase 1ActiveGlobalMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1ActiveGlobal
VanoglipelDA-1241 / GPR119 (G-protein-coupled receptor 119) / Small Molecule / Cardiometabolic Diseases
Metabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2aActiveGlobalType 2 Diabetes (T2D)Phase 1CompletedGlobal
ANA001ANA001 / Unknown / Oral Niclosamide Formulation / Infectious Diseases
Moderate COVID-19Phase 2LegacyUS
NB-01NB-01 / Unknown / Unknown / Neurology
Painful Diabetic NeuropathyUnknownLegacyGlobal
NB-02NB-02 / Unknown / Unknown / Neurology
Cognitive Impairment / Neurodegenerative DiseasesUnknownLegacyGlobal
GemcabeneUnknown / Small Molecule / Cardiovascular / Infectious Diseases
DyslipidemiaUnknownLegacyGlobalAcute COVID-19UnknownLegacyGlobal

NRIX

Nurixtx

nurixtx.com

6assets

NTLA

Intelliatx

intelliatx.com

6assets

OCGN

Ocugen

ocugen.com

6assets

OGN

Organon

organon.com

6assets

ebopiprantPreterm labor
OG-6219Endometriosis
OG-7191PCOS
Cirqle Biomedical candidateNon-hormonal contraceptive
Xaciatoclindamycin phosphate vaginal gel
Jada systemPostpartum hemorrhage device

OSTX

Ostherapies

ostherapies.com

6assets

OST-HER2OST31-164 / HER2 / Bioengineered Listeria monocytogenes (Immunotherapy) / Oncology
Osteosarcoma (Pulmonary Metastatic)Phase 2bActiveGlobalBreast CancerPhase 1ActiveGlobalCanine OsteosarcomaApprovedMarketedUS
OST-AXALAXAL / ADXS-HPV / HPV / Listeria-based Immunotherapy / Oncology
HPV-associated cancersPhase 3ActiveGlobal
OST-503ADXS-503 / KRAS G12 position-related antigens / Listeria-based Immunotherapy / Oncology
Non-Small Cell Lung Cancer (NSCLC)Phase 2ActiveGlobalGlioblastoma (GBM)Phase 2ActiveGlobalPancreatic CancerPreclinicalActiveGlobal
OST-504OST-PSA / ADXS-504 / ADXS31142 / PSA / Listeria-based Immunotherapy / Oncology
Prostate Cancer (Castration-Resistant)Phase 1bActiveGlobal
OST-tADCVarious / Tunable Antibody-Drug Conjugate (ADC) / Oncology
Ovarian CancerPreclinicalActiveGlobalBreast CancerPreclinicalActiveGlobal
OST-HOT constructsShared hotspot mutations and cancer-testes antigens / Listeria-based Immunotherapy / Oncology
Solid TumorsPreclinicalActiveGlobal

PRQR

Proqr

proqr.com

6assets

AX-0810AX-0810 / NTCP (SLC10A1) / RNA Editing Oligonucleotide (Axiomer EON) / Liver
Biliary AtresiaPrimary Sclerosing Cholangitis
AX-0811AX-0811 / NTCP / RNA Editing Oligonucleotide (Axiomer EON) / Liver
Cholestatic Diseases
AX-0422AX-0422 / IDUA / RNA Editing Oligonucleotide (Axiomer EON) / Metabolic / Rare Disease
Hurler Syndrome
AX-2402AX-2402 / MECP2 (R270X) / RNA Editing Oligonucleotide (Axiomer EON) / CNS / Rare Disease
Rett Syndrome
AX-2911AX-2911 / PNPLA3 / RNA Editing Oligonucleotide (Axiomer EON) / Liver / Metabolic
MASH
AX-1412AX-1412 / B4GALT1 / RNA Editing Oligonucleotide (Axiomer EON) / Cardiovascular
Cardiovascular Disease

RARE

Ultragenyx

ultragenyx.com

6assets

rivunatpagene miziparvovecUX701 / ATP7B / AAV9 gene therapy / Inborn Errors of Metabolism
Wilson disease
rebisufligene etisparvovecUX111 / AAV gene therapy / Neurogenetic
Sanfilippo syndrome type A (MPS IIIA)
avalotcagene ontaparvovecDTX301 / OTC / AAV8 gene therapy / Inborn Errors of Metabolism
Ornithine transcarbamylase (OTC) deficiency
DTX401DTX401 / AAV gene therapy / Inborn Errors of Metabolism
Glycogen storage disease type Ia (GSDIa)
GTX-102GTX-102 / UBE3A / Antisense oligonucleotide (ASO) / Neurogenetic
Angelman Syndrome
setrusumabUX143 / Biologic / Bone-Endocrine
Osteogenesis imperfecta

RVMD

Revmed

revmed.com

6assets

DaraxonrasibRMC-6236 / RAS(ON) multi-selective / Small Molecule / Oncology
Pancreatic Ductal Adenocarcinoma (PDAC)Non-Small Cell Lung Cancer (NSCLC)Colorectal Cancer (CRC)
ZoldonrasibRMC-9805 / RAS(ON) G12D / Small Molecule / Oncology
Non-Small Cell Lung Cancer (NSCLC)Solid Tumors
ElironrasibRMC-6291 / RAS(ON) G12C / Small Molecule / Oncology
KRAS-G12C mutant cancersNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
RMC-5127RMC-5127 / RAS(ON) G12V / Small Molecule / Oncology
RAS-G12V mutant cancers
RMC-0708RMC-0708 / RAS(ON) Q61H / Small Molecule / Oncology
RAS-Q61H mutant cancers
RMC-8839RMC-8839 / RAS(ON) G13C / Small Molecule / Oncology
RAS-G13C mutant cancers

RXRX

Recursion

recursion.com

6assets

REC-4881Familial adenomatous polyposis
REC-1245Biomarker-enriched solid tumors and lymphoma
REC-3565MALT1 Inhibitor / B-cell malignancies
Advanced solid tumors program
Solid tumors incl. HR+ breast cancer program
Hypophosphatasia program

SDGR

Schrodinger

schrodinger.com

6assets

SGR-1505SGR-1505 / MALT1 / Small Molecule / Oncology
Relapsed/Refractory B-cell MalignanciesPhase 1Active
SGR-3515SGR-3515 / PKMYT1 / Wee1 / Small Molecule / Oncology
Advanced Solid TumorsPhase 1Active
SGR-4174SGR-4174 / SOS1 / Small Molecule / Oncology
KRAS-driven CancersPreclinicalActive
SGR-2921SGR-2921 / CDC7 / Small Molecule / Oncology
Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome (MDS)Phase 1Discontinued
ACCG-2671ACCG-2671 / Calcitonin Receptor (DACRA) / Small Molecule / Metabolic
ObesityPhase 1Active
AJ1-11095AJ1-11095 / JAK2 / Small Molecule / Hematology
MyelofibrosisPreclinicalActiveUS

SLN

Silence Therapeutics

silence-therapeutics.com

6assets

DivesiranSLN124 / TMPRSS6 / siRNA / Hematologic Conditions
Polycythemia VeraOther Blood Disorders
ZerlasiranSLN360 / LPA / siRNA / Cardiovascular Disease
Elevated Lp(a)Atherosclerotic Cardiovascular Disease
SLN312SLN312 / ANGPTL3 / siRNA / Cardiometabolic
Dyslipidemia
SLN548SLN548 / CFB / siRNA / Complement
SLN365SLN365 / GPR146 / siRNA / Cardiometabolic
Homozygous familial hypercholesterolemia (HoFH)
SLN098SLN098 / INHBE / siRNA / Metabolic
Obesity

TCMD

Tactilemedical

tactilemedical.com

6assets

TPST

Tempesttx

tempesttx.com

6assets

AmezalpatTPST-1120 / PPARα antagonist / Small Molecule / Oncology
Hepatocellular Carcinoma (HCC)CholangiocarcinomaRenal Cell Carcinoma (RCC)
TPST-1495TPST-1495 / Dual EP2/EP4 Antagonist / Small Molecule / Oncology / Gastrointestinal
Familial Adenomatous Polyposis (FAP)Solid Tumors
TREX-1 InhibitorTREX-1 / Small Molecule / Oncology
Solid Tumors
TPST-2003TPST-2003 / CD19/BCMA / Autologous CAR-T Cell Therapy / Oncology
Relapsed/Refractory Multiple Myeloma (rrMM)
TPST-3003TPST-3003 / CD19/BCMA / Allogeneic Cell Therapy / Oncology
Relapsed/Refractory Multiple Myeloma (rrMM)
TPST-4003TPST-4003 / CD19/BCMA / In Vivo Cell Therapy / Immunology / Autoimmune
Systemic Lupus Erythematosus (SLE)Immunology Disorders

TRDA

Entradatx

entradatx.com

6assets

UTHR

Unither

unither.com

6assets

TreprostinilTyvaso / Prostacyclin receptor / Small Molecule / Respiratory
Idiopathic Pulmonary Fibrosis (IPF)
RalinepagProstacyclin receptor / Small Molecule / Cardiopulmonary
Pulmonary Arterial Hypertension (PAH)
UKidneyUKidney / Xenotransplantation / Organ Transplantation
End Stage Renal Disease
UThymoKidneyUThymoKidney / Xenotransplantation / Organ Transplantation
End Stage Renal Disease
miroliverELAPmiroliverELAP / Regenerative Medicine / Organ Transplantation
Acute Liver Failure
Centralized Lung Evaluation System (CLES)Ex-vivo Perfusion System / Organ Transplantation
Lung Transplant

VALN

Valneva

valneva.com

6assets

Chikungunya vaccineVLA1553 / IXCHIQ
Lyme Disease vaccineVLA15
Shigella vaccineS4V2
Zika vaccineVLA1601
Human metapneumovirus (hMPV) vaccineVLA1554
Epstein-Barr Virus (EBV) vaccineVLA2112

VIR

Vir

vir.bio

6assets

ElebsiranVIR-2218 / HBV / siRNA / Infectious Diseases
Chronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
TobevibartVIR-3434 / HBV / Monoclonal Antibody / Infectious Diseases
Chronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
VIR-1388VIR-1388 / HIV / T Cell Vaccine / Infectious Diseases
HIV PreventionPhase 1ActiveUnited StatesSouth Africa
VIR-5500VIR-5500 / PSMA / Masked T-Cell Engager / Oncology
Solid TumorsPhase 1ActiveGlobal
VIR-5525VIR-5525 / EGFR / Masked T-Cell Engager / Oncology
Solid TumorsPhase 1ActiveGlobal
VIR-5818VIR-5818 / HER2 / Masked T-Cell Engager / Oncology
Solid TumorsPhase 1ActiveGlobal

VKTX

Viking Therapeutics

vikingtherapeutics.com

6assets

VK2735 (Subcutaneous)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic Disorders
Obesity
VK2735 (Oral)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic Disorders
Obesity
VK2809VK2809 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Metabolic Disorders
NASHFibrosisNAFLD
VK0214VK0214 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Rare Diseases
X-linked adrenoleukodystrophy (X-ALD)
Amylin Agonist (DACRAs)Dual amylin and calcitonin receptor agonist (DACRA) / Agonist / Metabolic Disorders
ObesityMetabolic disorders
VK5211VK5211 / Selective androgen receptor modulator (SARM) / Small molecule / Musculoskeletal Disorders
Hip fracture recoveryMuscle and bone disorders

VTGN

Vistagen

vistagen.com

6assets

fasedienolPH94B / peripheral nasal chemosensory receptors / Intranasal pherine / Psychiatry
Social Anxiety Disorder (SAD)
itruvonePH10 / peripheral nasal chemosensory receptors / Intranasal pherine / Psychiatry
Major Depressive Disorder (MDD)
refisolonePH80 / peripheral nasal chemosensory receptors / Intranasal pherine / Women's Health
Menopausal Vasomotor SymptomsPremenstrual Dysphoric Disorder (PMDD)
PH15PH15 / peripheral nasal chemosensory receptors / Intranasal pherine / Neurology/Psychiatry
Psychomotor and cognitive impairment due to mental fatigue
PH284PH284 / peripheral nasal chemosensory receptors / Intranasal pherine / Oncology Supportive Care
Cancer Cachexia
AV-101AV-101 / NMDA receptor glycine site / Small molecule / Neurology/Psychiatry
Neurological disorders

XLO

Xiliotx

xiliotx.com

6assets

VilastobartXTX101 / CTLA-4 / Monoclonal Antibody / Oncology
Advanced solid tumors / MSS mCRCPhase 2ActiveGlobal
Efarindodekin alfaXTX301 / IL-12 / Cytokine / Oncology
Advanced solid tumorsPhase 2ActiveGlobal
XTX501XTX501 / PD-1 / IL-2 / Bispecific Antibody / Cytokine / Oncology
Non-small cell lung cancer (NSCLC) and other solid tumorsPreclinicalActiveGlobal
PSMA+STEAP1 Masked T Cell EngagerPSMA / STEAP1 / T Cell Engager / Oncology
Prostate cancerPreclinicalActiveGlobal
CLDN18.2 Masked T Cell EngagerCLDN18.2 / T Cell Engager / Oncology
Gastric, pancreatic, esophageal, and lung cancersPreclinicalActiveGlobal
Undisclosed Masked T Cell EngagerUndisclosed / T Cell Engager / Oncology
CancerPreclinicalActiveGlobal

ABEO

Abeona Therapeutics

abeonatherapeutics.com

5assets

ABO-503ABO-503 / RS1 / AAV gene therapy / Ophthalmology
X-linked retinoschisis (XLRS)Preclinical
ABO-504ABO-504 / ABCA4 / dual AAV gene therapy / Ophthalmology
Stargardt diseasePreclinical
ABO-505ABO-505 / OPA1 / AAV gene therapy / Ophthalmology
Autosomal dominant optic atrophy (ADOA)Preclinical
UX111UX111 / SGSH / AAV9 gene therapy / Rare disease; CNS
Sanfilippo syndrome type A (MPS IIIA)Phase 1/2
TSHA-102TSHA-102 / MECP2 / AAV9 gene therapy / Rare disease; CNS
Rett syndromePhase 1/2

ALLO

Allogene

allogene.com

5assets

Cemacabtagene ansegedleucelALLO-501 / CD19 / Allogeneic CAR T cell therapy / Hematologic malignancies
Large B-cell lymphoma with MRD after first-line therapyPivotal Phase 2EnrollingNorth AmericaSouth KoreaAustraliaRelapsed/refractory large B-cell lymphomaPhase 1Clinical data reported
ALLO-316ALLO-316 / CD70 / Allogeneic CAR T cell therapy / Oncology
Advanced or metastatic clear cell renal cell carcinomaPhase 1TRAVERSE Phase 1b enrollment completed; partnering soughtCD70+ hematologic malignanciesIND-enablingSolid tumorsIND-enabling
ALLO-213ALLO-213 / DLL3 / Allogeneic CAR T cell therapy / Solid tumors
Small cell lung cancerIND-enabling
ALLO-182ALLO-182 / Claudin 18.2 / Allogeneic CAR T cell therapy / Solid tumors
Gastric and pancreatic solid tumorsIND-enabling
ALLO-329ALLO-329 / CD19/CD70 / Allogeneic CAR T cell therapy / Autoimmune disease
Systemic lupus erythematosusPhase 1RESOLUTION enrollingLupus nephritisPhase 1RESOLUTION enrollingSclerodermaPhase 1RESOLUTION enrolling

ANNX

Annexon Bio

annexonbio.com

5assets

TanruprubartANX005 / C1q/classical complement pathway / IV mAb / Autoimmune
Guillain-Barré Syndrome (GBS)MAA filed Jan 2026; FDA discussions ongoing
VonaprumentANX007 / C1q/classical complement pathway / IVT Fab / Ophthalmology
Geographic Atrophy (GA)Phase 3ARCHER II enrollment completed; Phase 3 data 2H26
ANX1502ANX1502 / Classical complement pathway / Oral small molecule / Autoimmune
Cold Agglutinin Disease (CAD)Proof-of-concept study; update upon CAD study completion in 2026
ANX005ANX005 / C1q/classical complement pathway / IV mAb / Neurodegenerative
Huntington’s Disease (HD)Phase 2b/3Poised for late-stage Phase 2b/3 developmentAmyotrophic Lateral Sclerosis (ALS)Phase 2b/3Poised for late-stage Phase 2b/3 development
ANX009ANX009 / C1q/classical complement pathway / Subcutaneous Fab / Autoimmune
Lupus Nephritis (LN)Evaluating options for future development

ASND

Ascendis Pharma

ascendispharma.com

5assets

TransCon hGHlonapegsomatropin / SKYTROFA / growth hormone receptor / long-acting TransCon prodrug of somatropin / Endocrinology rare diseases
Turner syndrome (children aged 1-10)Phase 2New InsiGHTS TrialUnited StatesTurner syndrome, ISS, SHOX deficiency, SGA (children aged 2-17)Phase 3HighLiGHts TrialMultinationalPediatric GHDPhase 3Partner programChinaJapan
TransCon CNPnavapegritide / YUVIWEL / C-type natriuretic peptide pathway / long-acting TransCon prodrug of CNP / Endocrinology rare diseases
Achondroplasia (infants aged 0-<2)Pivotal Phase 2reACHin TrialMultinationalAchondroplasia (children aged 2-11)Pivotal Phase 2ApproaCH TrialMultinationalAchondroplasia (adolescents aged 12-18)Pivotal Phase 2bteACH TrialMultinational
TransCon CNP + TransCon hGHnavapegritide + lonapegsomatropin / CNP pathway + growth hormone receptor / combination of long-acting TransCon prodrugs / Endocrinology rare diseases
Achondroplasia (children aged 2-11)Phase 2COACH TrialMultinational
TransCon PTHpalopegteriparatide / YORVIPATH / parathyroid hormone receptor / long-acting TransCon prodrug of PTH / Endocrinology rare diseases
Adult hypoparathyroidism (dosing expansion)Phase 3PaTHway60 TrialUnited StatesAdult hypoparathyroidismPhase 3Partner programChina
TransCon IL-2 β/γ (Systemic)IL-2 beta/gamma receptor / TransCon prodrug cytokine / Oncology
Various tumor typesPhase 2IL-Believe TrialMultinational

AUTL

Autolus

autolus.com

5assets

obe-celobecabtagene autoleucel / AUCATZYL / CD19 / autologous CAR T cell therapy / Oncology; Autoimmune disease
B-NHLPhase 1enrollingPediatric B-ALL & B-NHLPhase 1enrollingPCNSLPhase 1enrolling
AUTO1/22CD19; CD22 / autologous CAR T cell therapy / Oncology
Pediatric ALLPhase 1enrolling
AUTO4TRBC1 / autologous CAR T cell therapy / Oncology
PTCLPhase 1enrolling
AUTO6NGGD2 / autologous CAR T cell therapy / Oncology
Neuroblastoma; other tumor typesPhase 1enrolling
AUTO8BCMA; CD19 / autologous CAR T cell therapy / Oncology
Multiple myelomaPhase 1enrolling

AVBP

Arrivent

arrivent.com

5assets

FirmonertinibEGFR TKI / oral small molecule / Oncology
1L NSCLC EGFR exon 20 insertion mutationsPhase 3Monotherapyglobal1L+ NSCLC EGFR PACC mutationsPhase 3MonotherapyglobalAdjuvant EGFR uncommon mutationsPhase 3Monotherapy
ARR-217ARR-217 / CDH17 ADC / antibody-drug conjugate / Oncology
GI tumorsPreclinical
ARR-002ARR-002 / antibody-drug conjugate / Oncology
Solid tumorsDiscovery
ARR-421ARR-421 / antibody-drug conjugate / Oncology
Solid tumorsDiscovery
ARR-173ARR-173 / antibody-drug conjugate / Oncology
Solid tumorsDiscovery

BBIO

Bridge Bio

bridgebio.com

5assets

AcoramidisAttruby / TTR (Transthyretin) / Small Molecule / ATTR amyloidosis
Early-Stage Variant Transthyretin Amyloidosis (ATTRv)Phase 3Investigational; ACT-EARLY prevention trial
BBP-418BBP-418 / FKRP / Small Molecule / Muscular dystrophy
Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)Phase 3FORTIFY met interim endpoints; additional studies planned
EncaleretCaSR / Small Molecule / Endocrinology
Autosomal Dominant Hypocalcemia Type 1 (ADH1)Phase 3NDA planned H1 2026; pediatric registrational trial initiatedUSEUChronic HypoparathyroidismPhase 3 plannedNIH investigator-initiated study
InfigratinibFGFR3 / Small Molecule / Skeletal dysplasias
AchondroplasiaPhase 3PROPEL 3; topline results expected early 2026USEUHypochondroplasiaPhase 2ACCEL 2/3 Phase 2 portion dosed April 2025; data expected H2 2026
BBP-812BBP-812 / ASPA / AAV Gene Therapy / Neurology; genetic disease
Canavan DiseasePhase 1/2 pivotalInvestigationalUSEU

BCRX

Biocryst

biocryst.com

5assets

ORLADEYOberotralstat / ORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Hereditary angioedema
Hereditary angioedema prophylaxis, adults and pediatric patients 12 years and olderApproved/CommercialapprovedHereditary angioedema prophylaxis, pediatric patients 2 to <12 yearsApproved/Commercialapproved
NavenibartSTAR-0215 / plasma kallikrein / monoclonal antibody / Hereditary angioedema
Hereditary angioedema prophylaxisPhase 3investigational
BCX17725BCX17725 / KLK5 / protein therapeutic / Rare disease
Netherton syndromePhase 1investigational
Avoralstatplasma kallikrein / ocular plasma kallikrein inhibitor / Ophthalmology
Diabetic macular edemainvestigational
STAR-0310STAR-0310 / OX40 / monoclonal antibody antagonist / Dermatology
Atopic dermatitisinvestigational

BCYC

Bicycle Therapeutics

bicycletherapeutics.com

5assets

nuzefatide pevedotinBT5528 / EphA2 / Bicycle Drug Conjugate / Oncology
metastatic pancreatic ductal adenocarcinomaPhase IIongoingEphA2-expressing tumorsPhase I/IIongoing
zelenectide pevedotinBT8009 / Nectin-4 / Bicycle Drug Conjugate / Oncology
Nectin-4 expressing tumorsPhase I/IIongoing; deprioritizedmetastatic urothelial cancerPhase IIongoing; deprioritized
BT7480BT7480 / Nectin-4 / CD137 / Bicycle tumor-targeted immune cell agonist / Oncology
solid tumorsPhase I/IIseeking partner; no longer internally developed
BT1702BT1702 / MT1-MMP / Bicycle Radioligand / Oncology
MT1-MMP overexpressing tumorsIND-enabling / Human Imagingfirst company-sponsored BRC trial planned in 2027
ION286/AZD4063ION286/AZD4063 / TfR1 / TfR1-based precision-guided nucleic acid delivery / Cardiomyopathy
cardiomyopathyPhase Iongoing

CAPR

Capricor

capricor.com

5assets

Deramiocelallogeneic CDCs / Neuromuscular disease
Duchenne muscular dystrophy (DMD)Phase 3in progress
Deramiocelallogeneic CDCs / Neuromuscular disease
Becker muscular dystrophy (BMD)Preclinicalin progress
StealthX™Exosome-based vaccine / Infectious diseases
SARS-CoV-2Phase 1in progress
StealthX™Therapeutic exosomes
EvaluatingPreclinicalin progress
CDC-ExosomesExosome platform / Neuromuscular disease
Duchenne muscular dystrophyPreclinicalin progress

CNTA

Centessa

centessa.com

5assets

COCP

Cocrystal Pharma

cocrystalpharma.com

5assets

CDI-988CDI-988 / pan-viral protease inhibitor / oral small molecule antiviral / Infectious disease / antiviral
Norovirus gastroenteritisPhase 1Phase 1 in progress; Phase 1b challenge study initiatedCoronavirus infectionPhase 1Phase 1 in progress; Phase 1 study completed
Pan-viral protease inhibitorpan-viral protease inhibitor / small molecule antiviral / Infectious disease / antiviral
RhinovirusPreclinicalPreclinical in progress; lead discovery ongoing
CC-42344 oral PB2 inhibitorCC-42344 / PB2 inhibitor / oral small molecule antiviral / Infectious disease / antiviral
Influenza APhase 2Phase 2 in progress; Phase 2a study completed, additional Phase 2a needed
CC-42344 inhaled PB2 inhibitorCC-42344 / PB2 inhibitor / inhaled small molecule antiviral / Infectious disease / antiviral
Influenza APreclinicalGLP tox study complete
Oral replication inhibitorreplication inhibitor / oral small molecule antiviral / Infectious disease / antiviral
Influenza A & BPreclinicalPreclinical in progress; lead discovery ongoing, NIH SBIR funded

COYA

Coya Therapeutics

coyatherapeutics.com

5assets

COYA 302COYA 302 / Treg-enhancing / T effector & macrophage-depleting biologics / Low-dose IL-2 + CTLA4-Ig biologic combination / Neurodegenerative diseases
Amyotrophic lateral sclerosis (ALS)Phase 2Enrolling ALSTARS Phase 2 trial; completed POC IIT open-label studyU.S.CanadaFrontotemporal dementia (FTD)Phase 1Planned Phase 2 FTD IND filing disclosed as 2H 2025 milestoneWorldwideAlzheimer's disease (AD)IND-enablingClinical data release from LD IL-2 IIT disclosed as Q1 2025 milestoneWorldwide
COYA 301COYA 301 / Treg enhancement / Proprietary low-dose IL-2 / Neurodegenerative diseases
Alzheimer’s disease (AD)IND-enablingRandomized double-blind placebo-controlled IIT data presented at CTAD 2024; additional biomarker/blood panel data disclosed as 1H 2025 milestone
COYA 303COYA 303 / Treg enhancement / GLP-1 receptor / COYA 301 + GLP-1 agonist combination / Inflammatory diseases
UndisclosedPreclinicalAdditional IP filings, pre-clinical in-vitro data, pre-clinical translational study, and partnership discussions disclosed as 1H 2025 milestones
COYA 201COYA 201 / Allogeneic Treg-derived exosomes
UndisclosedIND-enablingPre-clinical characterization disclosed as 2025 milestone
COYA 206COYA 206 / Antigen-directed allogeneic Treg-derived exosomes
UndisclosedPreclinicalPre-clinical characterization disclosed as 2025 milestone

CYTK

Cytokinetics

cytokinetics.com

5assets

MYQORZOaficamten / MYQORZO / cardiac myosin / cardiac myosin inhibitor / Cardiovascular
oHCMCommercialapproved/marketed
AficamtenMYQORZO / cardiac myosin / cardiac myosin inhibitor / Cardiovascular
oHCM (Monotherapy vs. SOC)Phase 3activePediatric oHCMPhase 2/3activenHCMPhase 3active
Omecamtiv Mecarbilcardiac myosin / cardiac myosin activator / Cardiovascular
HFrEFPhase 3active
UlacamtenCK-586 / cardiac myosin / cardiac myosin inhibitor / Cardiovascular
HFpEFPhase 2active
CK-089CK-089 / fast skeletal muscle troponin / fast skeletal muscle troponin activator / Neuromuscular
Additional skeletal muscle activators / neuromuscular diseasePhase 1active

ENTX

Entera Bio

enterabio.com

5assets

EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Osteoporosis
Postmenopausal women with low BMD and high-risk osteoporosisPhase 3Phase 3 protocol submitted to FDA; registrational program plannedU.S.
EB612EB612 / LA-PTH 1-34 / oral long-acting PTH analog peptide tablet / Hypoparathyroidism
HypoparathyroidismPreclinicalDevelopment ongoing; pre-IND initiatives underway
OXMOPK-88006 / GLP-1 & glucagon agonist / oral oxyntomodulin peptide tablet / Obesity / metabolic diseases
Obesity and metabolic diseasesPreclinicalIND filing expected late H1 2026U.S.
GLP-2Long-acting GLP-2 / oral GLP-2 peptide tablet / Short bowel syndrome
Short bowel syndromePreclinicalProof-of-concept single-dose pharmacokinetic study completed in rodents
EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Stress fractures
Stress fracturesInvestigator Sponsored Trial

GYRE

Gyretx

gyretx.com

5assets

HydronidoneF351 / TGFβRI/p38γ (anti-fibrotic) / Small molecule / Fibrosis
CHB-associated liver fibrosisNDA (priority review)NDA submission for conditional approval expected 1H 2026; Breakthrough Therapy designation (NMPA, Mar 2021)ChinaMASH-associated liver fibrosisIND-enablingU.S. IND application anticipated in 2026United States
PirfenidoneETUARY / Anti-fibrotic / Small molecule / Fibrosis/Respiratory
Idiopathic pulmonary fibrosis (IPF)Approved/MarketedApproved in China (2011); commercializedChinaPneumoconiosis (PD)Phase 352-week Phase 3 trial enrollment completed (272 patients, 18 sites)ChinaRadiation-induced lung injury (RILI) with or without immune-related pneumonitisClinicalIn developmentChina
F573F573 / Caspase inhibitor / Small molecule / Hepatology
Acute/acute-on-chronic liver failure (ALF/ACLF)ClinicalIn developmentChina
F528F528 / Small molecule / Respiratory
Chronic obstructive pulmonary disease (COPD)PreclinicalIn developmentChina
F230F230 / Small molecule / Cardiopulmonary
Pulmonary arterial hypertension (PAH)PreclinicalIn developmentChina

JAGX

Jaguar

jaguar.health

5assets

CrofelemerMytesi / CFTR and CaCC channels / Botanical Drug / Gastrointestinal
HIV-Related DiarrheaApprovedMarketedUnited StatesCancer Therapy-related Diarrhea (CTD) / Chemotherapy-induced overactive bowelPhase 3ActiveUnited StatesIrritable Bowel SyndromeIn DevelopmentActiveUnited States
Crofelemer Powder for Oral SolutionCFTR and CaCC channels / Botanical Drug / Gastrointestinal / Rare Disease
Microvillus Inclusion Disease (MVID)Late StageActiveUnited StatesEuropeShort Bowel Syndrome with Intestinal Failure (SBS-IF)Late StageActiveUnited StatesEurope
NP-300NP-300 / Unknown / Botanical Drug / Gastrointestinal / Infectious Disease
Cholera Diarrhea / Infectious DiarrheaIn DevelopmentActiveGlobal
GelclairGelclair / Unknown / Bioadherent Oral Gel / Supportive Care / Oncology
Oral MucositisApprovedMarketedUnited States
Crofelemer (Animal Health)Canalevia-CA1 / CFTR and CaCC channels / Botanical Drug / Animal Health
Chemotherapy-induced diarrhea in dogsApprovedMarketedUnited States

KRRO

Korro Bio

korrobio.com

5assets

KRRO-121KRRO-121 / Glutamine Synthetase (GS) / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / Liver
Urea Cycle Disorders (UCD) / Hepatic Encephalopathy (HE)PreclinicalActiveGlobal
AATD GalNAc ProgramSERPINA1 mRNA / GalNAc-conjugated oligonucleotide (RNA Editing) / Rare Diseases / Respiratory / Liver
Alpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
AMPKγ1 ProgramAMPKγ1 / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / Liver
Liver Function / LongevityPreclinicalActiveGlobal
ALS ProgramTDP-43 mRNA / Oligonucleotide (RNA Editing) / Neurology
Amyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobal
Cardiometabolic ProgramUndisclosed / Oligonucleotide (RNA Editing) / Metabolic / Cardiovascular
Cardiometabolic DiseasePreclinicalActiveGlobal

KYKOF

Kyowakirin

kyowakirin.com

5assets

ziftomenibKOMZIFTI / Menin / Small Molecule / Oncology
Acute Myeloid Leukemia (AML) (Combination)Phase 3ActiveGlobalRelapsed or refractory (R/R) NPM1-mutant AMLPre-RegistrationNDA Submitted / Priority ReviewUS
rocatinlimabKHK4083 / AMG 451 / OX40 / Monoclonal Antibody / Immunology/Allergy
Moderate to Severe Atopic DermatitisPhase 3ActiveGlobalUSEUJPPrurigo NodularisPhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
atidarsagene autotemcelOTL-200 / Lenmeldy / Libmeldy / ARSA / Hematopoietic Stem Cell (HSC) Gene Therapy / Rare Diseases
Early-onset Metachromatic Leukodystrophy (MLD)Pre-RegistrationPreparation underway for clinical trial / Regulatory ReviewJP
tivozanibKHK4951 / VEGF Receptor Tyrosine Kinase / Small Molecule / Ophthalmic
Ophthalmic DiseasesPhase 2/3ActiveGlobal
mogamulizumabKW-0761 / POTELIGEO / CCR4 / Monoclonal Antibody / Oncology
Mycosis fungoides and Sézary syndromePre-RegistrationFiledCanada

LEXX

Lexariabioscience

lexariabioscience.com

5assets

LGND

Ligand

investor.ligand.com

5assets

sparsentansparsentan / Filspari / ETA/AT1 receptors / Small molecule / Nephrology
IgA Nephropathy
ensifentrineensifentrine / Ohtuvayre / PDE3/PDE4 / Small molecule / Respiratory
COPD
pneumococcal 15-valent conjugate vaccineV114 / Vaxneuvance / Pneumococcal serotypes / Vaccine / Infectious Disease
Pneumococcal disease
carfilzomibcarfilzomib / Kyprolis / Proteasome / Small molecule / Oncology
Multiple Myeloma
berdazimerberdazimer / Zelsuvmi / Nitric oxide / Small molecule / Dermatology
Molluscum contagiosum

LSTA

Lisata

lisata.com

5assets

CertepetideLSTA1 / alpha-v integrins / Cyclic Peptide / Oncology
Advanced Solid TumorsPancreatic Ductal Adenocarcinoma (PDAC)Cholangiocarcinoma (CCA)
LSTA12LSTA12 / HONEDRA / CD34+ / Cell Therapy / Cardiovascular
Critical Limb Ischemia (CLI)
LSTA14LSTA14 / OLOGO / CD34+ / Cell Therapy / Cardiovascular
No-Option Refractory Disabling Angina (NORDA)
XOWNAXOWNA / CD34+ / Cell Therapy / Cardiovascular
Coronary Microvascular Dysfunction (CMD)
LSTA201LSTA201 / CD34+ / Cell Therapy / Nephrology
Diabetic Kidney Disease (DKD)

LTRN

Lantern Pharma

lanternpharma.com

5assets

LP-300LP-300 / PTPN11 / Small molecule / Oncology
Non-small cell lung cancer (NSCLC) in never-smokers
LP-184LP-184 / PTGR1-activated alkylating agent / Small molecule / Oncology
Solid tumorsCNS cancersGlioblastoma multiforme (GBM)
LP-284LP-284 / PKC-beta / Small molecule / Oncology
Hematologic malignanciesAggressive B-cell lymphomas
STAR-001STAR-001 / Unknown / Small molecule / Oncology
CNS cancersBrain metastases
ADC ProgramUnknown / Antibody-drug conjugate (ADC) / Oncology
Cancer

MANE

Veradermics

veradermics.com

5assets

VDPHL01VDPHL01 / Small molecule / Dermatology
Androgenetic Alopecia (Pattern Hair Loss)
VDAAVDAA / Topical small molecule / Dermatology
Alopecia Areata
VDADVDAD / Dermatology
Atopic Dermatitis
VDMCVDMC / Dermatology
Molluscum Contagiosum
VDMN-21VDMN-21 / Immune stimulatory / Microarray / Dermatology
Warts

MBX

Mbx Bio

mbxbio.com

5assets

MGNX

Macrogenics

macrogenics.com

5assets

MGC026B7-H3 ADC
MGC028ADAM9 ADC
MGC030TOP1i-based ADC
lorigerlimabPD-1 x CTLA-4 bispecific DART
MGD024CD123 x CD3 DART

MGTX

Meiragtx

meiragtx.com

5assets

MIRM

Mirum Pharma

mirumpharma.com

5assets

MaralixibatLIVMARLI / IBAT / Small molecule / Rare Liver Diseases
Alagille syndrome (ALGS)Progressive familial intrahepatic cholestasis (PFIC)Biliary atresia
VolixibatSHP626 / IBAT / Small molecule / Rare Liver Diseases
Primary sclerosing cholangitis (PSC)Primary biliary cholangitis (PBC)
MRM-3379MRM-3379 / PDE4D / Small molecule / Neurocognitive Disorders
Fragile X syndrome
Cholic acidCHOLBAM / Bile acid replacement / Small molecule / Rare Liver Diseases
Bile acid synthesis disordersPeroxisomal disorders (Zellweger spectrum disorders)
ChenodiolCTEXLI / Bile acid replacement / Small molecule / Rare Genetic Disorders
Cerebrotendinous xanthomatosis (CTX)

MNPR

Monopartx

monopartx.com

5assets

ALXN1840ALXN1840 / Copper / Small Molecule / Rare Diseases
Wilson Disease
MNPR-101-ZrMNPR-101-Zr / uPAR / Radiopharmaceutical / Oncology
Advanced Solid Cancers (Imaging)
MNPR-101-LuMNPR-101-Lu / uPAR / Radiopharmaceutical / Oncology
Advanced Solid Cancers
MNPR-101-AcMNPR-101-Ac / uPAR / Radiopharmaceutical / Oncology
Advanced Solid Cancers
MNPR-101MNPR-101 / uPAR / Monoclonal Antibody / Oncology
Solid Tumors

MRUS

Merus

merus.nl

5assets

PetosemtamabMCLA-158 / EGFR x LGR5 / Bispecific Antibody / Oncology
1L PD-L1+ r/m head and neck squamous cell carcinoma (with pembrolizumab)Phase 3OngoingGlobal2/3L r/m head and neck squamous cell carcinomaPhase 3OngoingGlobal1L metastatic colorectal cancer (with standard chemotherapy)Phase 2OngoingGlobal
ZenocutuzumabMCLA-128 / BIZENGRI / HER2 x HER3 / Bispecific Antibody / Oncology
Pancreatic adenocarcinoma (NRG1 fusion-positive)ApprovedApprovedUnited StatesNon-small cell lung cancer (NRG1 fusion-positive)ApprovedApprovedUnited StatesOther non-NRG1+ cancerPhase 2OngoingGlobal
MCLA-129MCLA-129 / EGFR x c-MET / Bispecific Antibody / Oncology
Solid tumorsPhase 1/Phase 2OngoingGlobal2L+ EGFRm non-small cell lung cancer (with chemotherapy)Phase 2OngoingGlobal
BesufetamigONO-4685 / PD-1 x CD3 / Bispecific Antibody / Oncology, Immunology
Relapsed/Refractory T Cell LymphomaPhase 1OngoingGlobalPsoriasisPhase 1OngoingGlobal
INCA33890INCA33890 / PD-1 x TGFBr2 / Bispecific Antibody / Oncology
Select advanced solid tumorsPhase 1OngoingGlobal

NKTR

Nektar

nektar.com

5assets

NMRA

Neumoratx

neumoratx.com

5assets

navacaprantNMRA-140 / Kappa opioid receptor (KOR) antagonist / Small molecule / Neuropsychiatry
Major Depressive Disorder
NMRA-511NMRA-511 / Vasopressin 1a receptor (V1aR) antagonist / Small molecule / Neuropsychiatry / Neurodegenerative
Alzheimer's disease agitation
NMRA-215NMRA-215 / NLRP3 inhibitor / Small molecule / Endocrinology / Metabolic
Obesity
NMRA-861NMRA-861 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
SchizophreniaNeuropsychiatric disorders
NMRA-898NMRA-898 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
SchizophreniaNeuropsychiatric disorders

NVAX

Novavax

novavax.com

5assets

PCRX

Pacira

pacira.com

5assets

triamcinolone acetonideZILRETTA / Small Molecule Formulation / Musculoskeletal Pain
Shoulder Osteoarthritis (OA)Phase 3Active
iovera°iovera° / Device / Neurology/Pain
SpasticityClinicalActive
bupivacaine liposomeEXPAREL / Small Molecule Formulation / Pain Management
Acute Post-Surgical Pain (Pediatric 0 to < 6)ClinicalActive
enekinragene inzadenovecPCRX-201 / IL-1Ra / Gene Therapy / Musculoskeletal Pain
Osteoarthritis of the Knee (OAK)Phase 2Active
PCRX-2002PCRX-2002 / Unknown / Pain Management
Post-Surgical PainClinicalActive

PCSA

Processa Pharmaceuticals

processapharmaceuticals.com

5assets

PCS6422NGC-Capecitabine
PCS499
PCS12852
PCS3117NGC-Gemcitabine
PCS11TATT-11T

PCVX

Vaxcyte

vaxcyte.com

5assets

VAX-31VAX-31 / Streptococcus pneumoniae (31 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
Invasive Pneumococcal Disease (IPD) and Pneumonia in AdultsPhase 3OngoingUnited StatesInvasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
VAX-24VAX-24 / Streptococcus pneumoniae (24 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
Invasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
VAX-A1VAX-A1 / Group A Streptococcus / Conjugate Vaccine / Infectious Diseases
Group A Strep InfectionsPreclinicalOngoing
VAX-GIVAX-GI / Shigella / Vaccine / Infectious Diseases
Shigellosis / Shigella InfectionsPreclinicalOngoing
VAX-XLVAX-XL / Streptococcus pneumoniae / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
Invasive Pneumococcal Disease (IPD)PreclinicalOngoing

PEPG

Pepgen

pepgen.com

5assets

PGEN

Precigen

precigen.com

5assets

PMN

Promisneuro Sciences

promisneurosciences.com

5assets

PMN310PMN310 / Amyloid-Beta / Monoclonal Antibody / Neurology
Alzheimer's Disease
PMN267PMN267 / TDP-43 / Monoclonal Antibody / Neurology
Amyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Limbic-predominant age-related TDP-43 encephalopathy (LATE)
PMN442PMN442 / Alpha-Synuclein / Monoclonal Antibody / Neurology
Parkinson's Disease (PD)Multiple System Atrophy (MSA)Dementia with Lewy Bodies (DLB)
PMN311PMN311 / Amyloid-Beta / Vaccine / Neurology
Alzheimer's Disease
PMN440PMN440 / Alpha-Synuclein / Vaccine / Neurology
Parkinson's Disease

PRTC

Puretech Health

puretechhealth.com

5assets

deupirfenidoneLYT-100 / Small Molecule / Respiratory / Fibrotic Diseases
Idiopathic Pulmonary Fibrosis (IPF)Phase 3ActiveGlobal
LYT-200LYT-200 / Biologic / Oncology
Relapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
GlyphAlloSPT-300 / Allopregnanolone / Small Molecule / Neuropsychiatry
Anxiety and Mood DisordersPhase 2ActiveGlobal
GlyphAgoSPT-320 / Small Molecule / Neuropsychiatry
Neurological / Neuropsychiatric IndicationsPhase 1ActiveGlobal
xanomeline and trospium chlorideKarXT / Cobenfy / Muscarinic Receptors / Small Molecule / Neuropsychiatry
SchizophreniaApprovedActiveUS

PTGX

Protagonist Inc

protagonist-inc.com

5assets

Rusfertide
IcotrokinraJNJ-77242113 / PN-21235
PN-881
Oral metabolic/obesity peptide
Oral hepcidin mimetic/ferroportin blocker

QURE

Uniqure

uniqure.com

5assets

AMT-130AMT-130 / Huntingtin protein (mHTT) / Gene Therapy (AAV5) / Central Nervous System
Huntington's DiseasePhase 1/2ActiveGlobal
AMT-191AMT-191 / Alpha-galactosidase A (GLA) / Gene Therapy / Rare Diseases
Fabry DiseasePhase 1/2aActiveGlobal
AMT-260AMT-260 / Undisclosed / Gene Therapy / Central Nervous System
Refractory Mesial Temporal Lobe EpilepsyPhase 1/2aActiveGlobal
AMT-162AMT-162 / SOD1 / Gene Therapy / Central Nervous System
Amyotrophic Lateral Sclerosis (ALS)Preclinical/ClinicalActiveGlobal
etranacogene dezaparvovecAMT-061 / HEMGENIX / Factor IX / Gene Therapy (AAV5) / Hematology
Hemophilia BApprovedMarketedUSGlobal

ROIV

Roivant

roivant.com

5assets

BrepocitinibJAK1 / TYK2 / Small Molecule / Autoimmune & Inflammatory
DermatomyositisPhase 3ActiveGlobalNon-infectious UveitisPhase 3ActiveGlobalCutaneous SarcoidosisPhase 2ActiveGlobal
IMVT-1402IMVT-1402 / FcRn / Monoclonal Antibody / Autoimmune
Rheumatoid Arthritis (difficult-to-treat)Phase 2ActiveGlobalGraves' DiseasePhase 2/3ActiveGlobal
BatoclimabFcRn / Monoclonal Antibody / Autoimmune
Thyroid Eye Disease (TED)Phase 3ActiveGlobalMyasthenia Gravis (MG)Phase 3ActiveGlobalChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2bActiveGlobal
MosliciguatRVT-304 / sGC / Small Molecule / Pulmonary
Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 2ActiveGlobal
NamilumabGM-CSF / Monoclonal Antibody / Autoimmune & Inflammatory
SarcoidosisPhase 2ActiveGlobal

SHPH

Shuttle Pharma

shuttlepharma.com

5assets

RopidoxuridineIPdR / Radiation Sensitizer / Small Molecule / Oncology
GlioblastomaPhase 2ActiveUnited StatesSarcomasPhase 1/Phase 2ActiveUnited States
SP-2-225SP-2-225 / HDAC6 / Small Molecule / Oncology
Breast Cancer / Solid TumorsPreclinicalActiveUnited States
SP-1-303SP-1-303 / Class I HDAC / Small Molecule / Oncology
ER+ Breast CancerPreclinicalActiveUnited States
SP-1-161SP-1-161 / HDAC / Small Molecule / Oncology
Solid TumorsDiscoveryActiveUnited States
PSMA-B ligandPSMA-B / PSMA / Radiopharmaceutical / Oncology
Metastatic Prostate CancerPreclinicalActiveUnited States

SPTX

Seaporttx

seaporttx.com

5assets

GlyphAlloSPT-300 / Allopregnanolone prodrug
GlyphAgoSPT-320 / Agomelatine prodrug
Glyph2BLSDSPT-348 / Non-hallucinogenic neuroplastogen prodrug
GlyphCeleCelecoxib prodrug
GlyphCBD

SRRK

Scholarrock

scholarrock.com

5assets

VRDN

Viridian Therapeutics

viridiantherapeutics.com

5assets

VTRS

Viatris

viatris.com

5assets

MR-146MR-146 / AAV Gene Therapy / Ophthalmology
Neurotrophic keratopathy (NK)
Low Dose Estrogen Weekly PatchEstrogen / Progestin receptors / Transdermal Patch / Women's Health
Contraception
PitolisantHistamine H3 receptor / Small molecule / Neurology
Obstructive Sleep Apnea Syndrome
SelatogrelP2Y12 receptor / Small molecule / Cardiovascular
Acute Myocardial Infarction
CenerimodS1P1 receptor / Small molecule / Immunology
Systemic lupus erythematosus (SLE)

WVE

Wavelife Sciences

wavelifesciences.com

5assets

WVE-006WVE-006 / AAT transcript (Pi*ZZ mutation) / RNA editing oligonucleotide (AIMer) / Genetic/Respiratory/Liver Diseases
Alpha-1 antitrypsin deficiency (AATD)
WVE-007WVE-007 / INHBE mRNA / siRNA / Metabolic Diseases
Obesity
WVE-008WVE-008 / PNPLA3 I148M / RNA editing oligonucleotide / Liver Diseases
PNPLA3-related liver disease
WVE-N531WVE-N531 / DMD Exon 53 / Exon skipping oligonucleotide / Neuromuscular Diseases
Duchenne muscular dystrophy (DMD)
WVE-003WVE-003 / Mutant huntingtin (mHTT) mRNA / Allele-selective silencing oligonucleotide / Neurological Diseases
Huntington's disease (HD)

ABCL

AbCellera

abcellera.com

4assets

ABCL635ABCL635 / NK3R / antibody medicine; GPCR / Endocrinology / Women's Health
Vasomotor Symptoms (VMS) associated with menopausePhase 1/2clinical trial ongoing
ABCL575ABCL575 / OX40L / monoclonal antibody / Immunology & Inflammation
Atopic Dermatitis (AD)Phase 1clinical trial initiated Q3 2025
ABCL688ABCL688 / Undisclosed GPCR / Ion Channel / antibody medicine / Autoimmunity
Undisclosed autoimmunity indicationIND-Enablingentered IND-enabling studies Q2 2025
ABCL386ABCL386 / Undisclosed / antibody medicine / Oncology
Undisclosed oncology indicationIND-Enabling

ABSI

Absci

absci.com

4assets

ABS-101ABS-101 / TL1A / antibody / Inflammatory bowel disease
Inflammatory bowel diseasePhase 1
ABS-201ABS-201 / PRLR / anti-PRLR antibody / Dermatology; gynecology
Androgenetic alopeciaPhase 1/2aEndometriosisPhase 1
ABS-301ABS-301 / Undisclosed immuno-oncology target / antibody / Immuno-oncology
Immuno-oncologyLead ID
ABS-501ABS-501 / HER2 / anti-HER2 antibody / Oncology
OncologyCandidate

ACRS

Aclaristx

aclaristx.com

4assets

BosakitugATI-045 / TSLP / monoclonal antibody / Immuno-inflammatory diseases
Atopic dermatitisPhase 2Enrollment complete; top-line results expected 4Q 2026
ATI-052ATI-052 / TSLP; IL-4Rα / humanized bispecific antibody / Immuno-inflammatory diseases
Atopic dermatitisPhase 1b POCDosing ongoing; top-line results expected 2H 2026AsthmaPhase 1b POCDosing ongoing; Phase 2b program planned 4Q 2026
ATI-2138ATI-2138 / ITK; JAK3 / oral small molecule inhibitor / Immuno-inflammatory diseases
Atopic dermatitisPhase 2aCompleteLichen planusPhase 2bPlanned initiation 2H 2026
ATI-9494ATI-9494 / ITK / small molecule inhibitor / Immuno-inflammatory diseases
Undisclosed immuno-inflammatory indicationsPreclinical/IND-enablingIND expected 2H 2026

AGIO

Agios

agios.com

4assets

MitapivatPYRUKYND®; AQVESME™ / PK / PK activator / Rare diseases; hematology
Adult PK deficiencyApprovedApprovedU.S.EUGreat BritainAdult thalassemiaApprovedApprovedU.S.KSAUAEAdult sickle cell diseaseRegulatory submissionPlanned sNDA submissionU.S.
TebapivatAG-946 / PK / PK activator / Rare diseases; hematology
Lower-risk myelodysplastic syndromesPhase 2bEnrollment completedSickle cell diseasePhase 2Enrollment completed
AG-181AG-181 / PAH / PAH stabilizer / Rare diseases; metabolic disorders
PhenylketonuriaPhase 1/1bPhase 1 completed; Phase 1b planned
AG-236AG-236 / TMPRSS6 / siRNA / Rare diseases; hematology
Polycythemia veraPhase 1Healthy volunteer trial ongoing

AKBA

Akebia

ir.akebia.com

4assets

PraliciguatsGC / soluble guanylate cyclase stimulator / Kidney disease
Focal segmental glomerulosclerosisPhase 2first patient enrolled Dec 2025
Ebribafusp alfaAKB-097 / C3d/factor H / anti-C3d-factor H fusion protein; complement inhibitor / Rare kidney diseases
IgA nephropathyPhase 2 plannedbasket trial planned 2H 2026Lupus nephritisPhase 2 plannedbasket trial planned 2H 2026C3 glomerulopathyPhase 2 plannedbasket trial planned 2H 2026
AKB-9090AKB-9090 / HIF-PH / HIF-PH inhibitor / Kidney disease; acute care
Cardiac surgery-associated acute kidney injuryPhase 1first participants dosed Apr 2026Acute respiratory distress syndromePreclinical
AKB-10108AKB-10108 / HIF-PH / HIF-PH inhibitor / Other disease areas
Retinopathy of prematurityPreclinical

ALMS

Alumis

alumis.com

4assets

EnvudeucitinibESK-001 / TYK2 / oral small molecule TYK2 inhibitor / Immunology
Plaque psoriasisPhase 3ONWARD3 LTE ongoing; NDA planned 2H 2026GlobalJapanSystemic lupus erythematosusPhase 2LUMUS ongoing; topline data expected 2026Global
A-005A-005 / TYK2 / CNS-penetrant allosteric TYK2 inhibitor / Neuroinflammation
Multiple sclerosisPhase 1 completed; Phase 2 plannedPhase 2 initiation planned H1 2026
LonigutamabIGF-1R / subcutaneous humanized IgG1 monoclonal antibody / Endocrinology/Immunology
Thyroid eye diseaseDevelopment program evaluation ongoing
IRF5/Additional TargetsIRF5, additional targets
Undisclosed

AMLX

Amylyx

amylyx.com

4assets

AvexitideGLP-1 receptor / GLP-1 receptor antagonist / Endocrine/metabolic diseases
Post-bariatric hypoglycemia (PBH)Phase 3LUCIDITY completed enrollment; topline data expected Q3 2026USCongenital hyperinsulinism (HI)Clinical development next steps under discussionUS
AMX0035AMX0035 / ER stress and mitochondrial dysfunction / Oral fixed-dose combination of sodium phenylbutyrate and taurursodiol / Neurodegenerative/rare diseases
Wolfram syndromePhase 2HELIOS positive Week 48 data; pivotal Phase 3 planned pending FDA alignmentUSEU
AMX0114AMX0114 / Calpain-2 (CAPN2) / Antisense oligonucleotide (ASO) / Neurodegenerative diseases
Amyotrophic lateral sclerosis (ALS)Phase 1LUMINA Cohort 2 fully enrolled; biomarker data expected H1 2026US
AMX0318AMX0318 / GLP-1 receptor / Long-acting GLP-1 receptor antagonist / Endocrine/metabolic diseases
PBH and other rare diseasesIND-enablingIND-enabling studies underway; IND targeted for 2027

APGE

Apogee Therapeutics

apogeetherapeutics.com

4assets

ZumilokibartAPG777 / IL-13 / SQ half-life extended monoclonal antibody / Inflammation & immunology
Atopic dermatitisPhase 2in-progress; positive Part A 52-week data; Part B enrollment completedAsthmaPhase 1complete; positive Phase 1b dataEosinophilic esophagitisPhase 1in-progress
APG279APG279 / IL-13 + OX40L / coformulation of zumilokibart + APG990 extended half-life monoclonal antibodies / Inflammation & immunology
Atopic dermatitisPhase 1in-progress; Phase 1b PoC data expected 2H 2026
APG273APG273 / IL-13 + TSLP / combination of zumilokibart + APG333 antibodies / Inflammation & immunology
Asthma / COPDPreclinicalin-progress; additional trial plans to be announced in 2026
APG808APG808 / IL-4Rα / half-life extended monoclonal antibody / Inflammation & immunology
AsthmaPhase 1bpositive interim results reported May 2025

ATAI

Ataibeckley

ataibeckley.com

4assets

BPL-003BPL-003 / serotonergic neuromodulator / intranasal mebufotenin benzoate / Mental health
Treatment-Resistant DepressionPhase 2
VLS-01VLS-01 / 5-HT1/2/6/7 sub-receptors / buccal film DMT / Mental health
Treatment-Resistant DepressionPhase 2
EMP-01EMP-01 / oral R-MDMA / Mental health
Social Anxiety DisorderPhase 2
Novel 5-HT2A Receptor Agonists5-HT2A receptor / non-hallucinogenic neuroplastogens / Mental health
Opioid Use DisorderIn developmentTreatment-Resistant DepressionIn development

BOLT

Bolt Bio

boltbio.com

4assets

BDC-4182BDC-4182 / Claudin 18.2 / next-generation Boltbody ISAC / Oncology
Gastric cancerPhase 1Ongoing dose-escalationGastroesophageal cancerPhase 1Ongoing dose-escalation
BDC-3042BDC-3042 / Dectin-2 / agonist antibody / Oncology
Non-small cell lung cancerPhase 1Dose-escalation complete; seeking partnerMelanomaPhase 1Dose-escalation complete; seeking partnerOther solid tumorsPhase 1Dose-escalation complete; seeking partner
CEA ISACCEA / Boltbody ISAC / Oncology
Colorectal cancerPreclinicalPre-IND; seeking partnerNon-small cell lung cancerPreclinicalPre-IND; seeking partnerPancreatic cancerPreclinicalPre-IND; seeking partner
PD-L1 ISACPD-L1 / ISAC / Oncology
Solid tumors resistant to checkpoint inhibitorsPreclinicalPre-IND; seeking partner

CGEM

Cullinan Therapeutics

cullinantherapeutics.com

4assets

CLN-978CLN-978 / CD19xCD3 / bispecific T cell engager / Immunology
OUTRACE: Rheumatoid arthritisPhase 1Initial data in RA in Q2 2026 and repeat dosing data in Q3 2026EuropeOUTRACE: Systemic lupus erythematosusPhase 1bInitial data in SLE in Q2 2026GlobalOUTRACE: Sjögren’s diseasePhase 1Initial data in Sjögren’s disease in Q4 2026Global
VelinotamigBCMAxCD3 / bispecific T cell engager / Immunology
Autoimmune diseasesIND-EnablingGenrix Bio initiated a Phase 1 study in China in Q4 2025; initial data expected in Q4 2026; clinical trial status not yet recruitingChina
ZipalertinibEGFR ex20ins / tyrosine kinase inhibitor (TKI) / Oncology
REZILIENT1: Non-small cell lung cancerPhase 2U.S. FDA accepted NDA for relapsed EGFR ex20ins NSCLC; PDUFA target action date February 27, 2027United StatesREZILIENT2: Non-small cell lung cancerPhase 2Parallel cohort study ongoingREZILIENT3: Non-small cell lung cancerPhase 3Phase 3 1L study fully enrolled; Taiho expects topline results by end of 2026Global
CLN-049CLN-049 / FLT3xCD3 / bispecific T cell engager / Oncology
Acute myeloid leukemia and myelodysplastic syndromePhase 1Dose escalation update in H2 2026; dose expansion cohorts to initiate in Q2 2026 and complete enrollment in Q4 2026Measurable residual disease in acute myeloid leukemiaPhase 1Phase 1 study ongoing

CGEN

Cgen

cgen.com

4assets

COM701COM701 / PVRIG / Fc-reduced anti-PVRIG antibody / Oncology / immuno-oncology
Relapsed platinum-sensitive ovarian cancer; maintenance therapyAdaptive platform trial; sub-trial 1 randomized vs. placeboongoing
COM701 + COM902 + pembrolizumabPVRIG, TIGIT, PD-1 / Antibody combination / Oncology / immuno-oncology
Platinum-resistant ovarian cancerProof of conceptongoing
RilvegostomigPD-1 / TIGIT / Fc-reduced anti-PD-1/TIGIT bispecific antibody / Oncology / immuno-oncology
Non-small-cell lung cancer (NSCLC)Phase 3ongoingGastrointestinal cancerPhase 3ongoingEndometrial cancerPhase 3ongoing
GS-0321GS-0321 / IL-18 binding protein / Anti-IL18BP antibody / Oncology / immuno-oncology
Solid tumorsPhase 1ongoing

CLNN

Clene

clene.com

4assets

CNM-Au8®CNM-Au8 / mitochondrial function / NAD pathway; neuroprotection and remyelination / oral gold nanocrystal suspension / Neurology
Amyotrophic Lateral Sclerosis (ALS)Global Phase 3 plannedPlanned; EAPs ongoing; regulatory agency engagement ongoingUSEUGlobalMultiple Sclerosis (MS)Phase 3 plannedPlanned; EAP ongoing; Repair-MS target engagement and safety confirmedUSGlobalParkinson’s Disease (PD)In development; REPAIR-PD trial listed
CNM-ZnAgCNM-ZnAg / zinc-silver ionic solution / Infectious disease
Antiviral and antimicrobial usesDemonstrated antiviral and antimicrobial properties
CNM-AgZn17CNM-AgZn17 / topical gel polymer suspension of silver and zinc ions / Infectious disease / wound healing
Infectious diseasesIn developmentWound healingIn development
CNM-PtAu7CNM-PtAu7 / gold-platinum nanotherapeutic / Oncology
CancerIn vitroInitial research

CMPX

Compass Therapeutics

compasstherapeutics.com

4assets

CTX-009 (tovecimig)CTX-009 / DLL4 x VEGF-A / Bispecific antibody / Oncology
Biliary Tract Cancer (2L)Phase 2/317.1% ORR; met primary endpoint; PFS/OS data expected late Q1 2026Colorectal Cancer (monotherapy 3L/4L)Phase 2Completed; monotherapy activityBasket Study — DLL4+ tumorsAdditional indications planned H1 2026: CRC, gastric, ovarian, renal, HCC
CTX-471CTX-471 / CD137 (4-1BB) / Agonistic antibody / Oncology
Basket Study — NCAM (CD56)Phase 2Trial initiation expected Q1 2026Basket Study — Post-checkpointPhase 1Completed
CTX-8371CTX-8371 / PD-1 x PD-L1 / Bispecific antibody / Oncology
Solid TumorsPhase 1Expansion cohorts initiated; Phase 1 data expected H1 2026Non-small cell lung cancer (NSCLC)Phase 1 expansionEnrollingTriple-negative breast cancer (TNBC)Phase 1 expansionEnrolling
CTX-10726CTX-10726 / PD-1 x VEGF-A / Bispecific antibody / Oncology
Solid TumorsIND-enabling preclinicalIND filing expected 4Q2025; Phase 1 initiation expected Q1 2026

CORT

Corcept

corcept.com

4assets

Korlymmifepristone
Lifyorlirelacorilant
dazucorilant
miricorilant

CTNM

Contineum Tx

contineum-tx.com

4assets

PIPE-791PIPE-791 / LPA1R / Oral small molecule LPA1R antagonist / Inflammation and immunology; neuroscience
Idiopathic pulmonary fibrosis (IPF)Phase 2PROPEL-IPF patient dosing initiatedProgressive multiple sclerosis (PrMS)Phase 1bFurther clinical development deferred until funding obtainedChronic painPhase 1bExploratory trial ongoing; topline data expected Q2 2026United States
CTX-343CTX-343 / LPA1R / Peripherally restricted oral small molecule LPA1R antagonist / Inflammation and immunology
Peripheral indicationsPreclinicalClinical development initiation deferred until funding obtained
PIPE-307PIPE-307; JNJ-89495120 / M1R / Oral small molecule M1 receptor antagonist / Neuroscience
Relapsing-remitting multiple sclerosis (RRMS)Phase 2VISTA topline data reported; further development at Janssen discretionMajor depressive disorder (MDD)Phase 2Janssen-sponsored Moonlight-1 trial
CalpainCalpain / Calpain inhibitor
UndisclosedPreclinicalActive preclinical program

DRUG

Brightminds Bio

brightmindsbio.com

4assets

DSGN

Designtx

designtx.com

4assets

DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurology
Friedreich ataxiaPhase 1/2RESTORE-FA multiple-dose trial opened/ongoing; evaluates IV and subcutaneous administrationex-U.S.
DT-168DT-168 / TCF4 CTG repeat expansion / GeneTAC small molecule eye drop / Ophthalmology / genetic disease
Fuchs endothelial corneal dystrophyPhase 2Biomarker trial opened/ongoing in FECD patients scheduled for corneal transplant surgery
DT-818DT-818 / DMPK CTG repeat expansion / GeneTAC small molecule / Neuromuscular / genetic disease
Myotonic dystrophy type 1Phase 1 plannedPatient dosing in Phase 1 MAD trial anticipated in H1 2026Australia
Huntington's disease GeneTAC programHTT CAG repeat expansion / mutant huntingtin expression / GeneTAC small molecule / Neurology / genetic disease
Huntington's diseasePreclinicalProgram disclosed; candidate not named

DWTX

Virios

dwtx.com

4assets

Halneuron® NaV1.7 InjectionHAL / Halneuron® / NaV1.7 / subcutaneous small-molecule/peptide analgesic injection / Pain / neuropathy
Chemotherapy-induced neuropathic pain (CINP)Phase 2bOngoing; FDA Fast Track designationU.S.General cancer painPhase 2aCompleteAcute surgical painPreclinical
SP16 Intravenous AdministrationSP16 IV / LRP1 agonist / IV peptide therapeutic / Neuropathy / cancer-related pain
Chemotherapy-induced peripheral neuropathy (CIPN)Phase 1bNCI funded / forthcoming
IMC-1IMC-1 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illness
FibromyalgiaPhase 2aPreviously demonstrated efficacy; disclosed as a novel therapeutic under antiviral combinations
IMC-2IMC-2 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illness
Long COVIDOpen-label investigator-initiated study completed/reported; disclosed as a novel therapeutic under antiviral combinations

EWTX

Edgewisetx

edgewisetx.com

4assets

SevasemtenMyosin ATPase / Oral fast skeletal myosin inhibitor / Muscular dystrophy
Becker muscular dystrophyPivotal / Phase 3Duchenne muscular dystrophyPhase 2
EDG-7500EDG-7500 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
Hypertrophic cardiomyopathyPhase 2
EDG-15400EDG-15400 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
HFpEFPhase 1
EDG-003EDG-003 / Undisclosed target / Cardiometabolic
CardiometabolicPreclinical

FDMT

4dmolecular Therapeutics

4dmoleculartherapeutics.com

4assets

4D-1504D-150 / VEGF family members / Intravitreal AAV genetic medicine / Retina
Wet AMDPivotal / Phase 34FRONT-1 and 4FRONT-2 Phase 3; PRISM Phase 1/2 ongoingDiabetic macular edemaPivotal readySPECTRA clinical trial Part 1
4D-7104D-710 / CFTR / Aerosol-delivered AAV genetic medicine / Pulmonology
Cystic fibrosis lung disease; not modulator-amenable / ineligible or discontinued CFTR modulator therapyPhase 2AEROW Phase 1/2 study; pipeline lists Phase 2
4D-1754D-175 / Complement factor H / complement pathway / Intravitreal AAV genetic medicine / Retina
Geographic atrophySeeking partnerships to advance
4D-7254D-725 / Aerosol-delivered AAV genetic medicine / Pulmonology
Alpha-1 antitrypsin lung diseasePreclinicalSeeking partnerships to advance

GLPG

Glpg

glpg.com

4assets

GLUE

Monterosatx

monterosatx.com

4assets

MRT-6160MRT-6160 / VAV1 / Oral molecular glue degrader / Immunology & Inflammation
Immune-mediated diseasesClinical
MRT-8102MRT-8102 / NEK7 / Oral molecular glue degrader / Immunology & Inflammation
IL-1β/NLRP3-driven inflammatory diseasesPhase 1
MRT-2359MRT-2359 / GSPT1 / Oral molecular glue degrader / Oncology
Castration-resistant prostate cancerPhase 1/2Ongoing
Cyclin E1 degrader programCCNE1 / CDK2 / Molecular glue degrader / Oncology
CCNE1-amplified tumorsPreclinicalLead optimizationER+ breast cancerPreclinicalLead optimization

HCWB

Hcwbiologics

hcwbiologics.com

4assets

HCW9302HCW9302 / IL-2Rα / IL-2 fusion protein / Autoimmune
Alopecia areataPhase 1OngoingUS
HCW11-006HCW11-006 / Multiple cytokines (IL-15/IL-15Rα, IL-7, IL-21, IL-2) / Multi-functional fusion immunotherapeutic / Oncology
CancerPhase 1Planned by licenseeChina
HCW11-018bHCW11-018b / Tissue Factor/CD3 / Tetra-valent bispecific T-cell engager (Big BiTE) / Oncology
Solid tumorsPreclinicalIND-enablingGlobal
HCW11-040HCW11-040 / PD-1 (pembrolizumab-based) + TGF-β / Tetra-valent immune checkpoint inhibitor fusion / Oncology
Solid tumorsPreclinicalIND-enablingGlobal

IBRX

Immunity Bio

immunitybio.com

4assets

nogapendekin alfa inbakiceptN-803 / ANKTIVA® / IL-15 receptor / Cytokine fusion protein / Interleukin-15 superagonist / Oncology, Infectious Diseases
BCG Unresponsive NMIBC Carcinoma In-Situ & Papillary (QUILT-3.032)ApprovedMarketedUSBCG Naïve NMIBC (QUILT-2.005)Phase 2/Phase 3ActiveUSNon-Small Cell Lung Cancer (ResQ201A)Phase 3ActiveGlobal
PD-L1 t-haNKPD-L1 / Cell Therapy / Engineered NK cells / Oncology
Advanced Solid TumorsPhase 2ActiveUS
AldoxAlbumin-bound doxorubicin complex / Oncology
Advanced Solid TumorsPhase 2ActiveUS
Recombinant BCGVaccine / Immunotherapy / Oncology
Bladder CancerExpanded AccessActiveUS

IMTX

Immatics

immatics.com

4assets

anzu-cel (IMA203)IMA203 / anzutresgene autoleucel / PRAME / Autologous TCR-T cell therapy / Solid Tumors
2L+ cutaneous melanomaPhase 3ongoingUSEU5Uveal melanomaPhase 1bongoingUSEU5PRAME+ solid tumorsPhase 1ongoingUSEU5
IMA203CD8 (GEN2)IMA203CD8 / PRAME / Autologous TCR-T cell therapy (next-gen with CD8) / Solid Tumors
PRAME+ advanced solid tumorsPhase 1dose escalation ongoingUSEU5Ovarian and endometrial cancersPhase 1ongoingUSEU5
IMA402IMA402 / PRAME / Half-life extended TCR bispecific (TCER) / Solid Tumors
Recurrent/refractory solid tumors (melanoma, gynecologic, sqNSCLC)Phase 1/2Phase 1b expansion ongoingUSEU
IMA401IMA401 / MAGEA4/8 / TCR bispecific (TCER) / Solid Tumors
Recurrent/refractory solid tumors (HNSCC, sqNSCLC, melanoma)Phase 1dose escalation completedUSEU

INAB

In8 Bio

in8bio.com

4assets

INB-100INB-100 / DeltEx Allo allogeneic γδ T cell therapy / Oncology
Acute leukemia post-HSCTPhase 1Expansion cohort enrollingUS
INB-200INB-200 / DeltEx DRI autologous genetically modified γδ T cell therapy / Oncology
Newly diagnosed glioblastomaPhase 1Treatment completed; long-term follow-upUS
INB-400INB-400 / DeltEx DRI autologous and allogeneic γδ T cell therapy / Oncology
Newly diagnosed glioblastomaPhase 2Enrollment suspended; treatment completed; data collection ongoingUS
INB-619INB-619 / CD19 / γδ T cell engager (TCE) / Oncology and autoimmune diseases
CD19+ B-cell malignanciesPreclinicalIND-enabling studiesB-cell mediated autoimmune diseasesPreclinicalIND-enabling studies

INKT

Mink Therapeutics

investor.minktherapeutics.com

4assets

agenT-797agenT-797 / Allogeneic native iNKT cell therapy / Oncology and Immunology
Critical pulmonary immune failure (severe hypoxemic respiratory failure/pneumonia/ARDS)Phase 2randomized adaptive placebo-controlled ongoingUSSolid tumor cancers (checkpoint-refractory, incl. gastric/esophageal/GEJ adenocarcinoma)Phase 2investigator-sponsored ongoing (NCT06251973)USGraft-versus-host disease (GvHD prevention)expanding development
MiNK-215MiNK-215 / FAP / IL-15 armored FAP-CAR-iNKT cell therapy / Oncology
Solid tumors (NSCLC, CRC liver metastases)PreclinicalIND-enabling studies underway
MiNK-413MiNK-413 / BCMA / IL-15 armored BCMA-CAR-iNKT cell therapy / Oncology
Multiple myelomaPreclinical
PRAME TCR-iNKTPRAME / TCR-engineered iNKT cell therapy / Oncology
Pediatric cancers/solid tumorsPreclinicalIND-enabling

IRON

Discmedicine

discmedicine.com

4assets

bitopertinGlyT1 / Small Molecule / Hematology
Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)Phase 3OngoingGlobalDiamond-Blackfan AnemiaPhase 2OngoingUS
DISC-0974DISC-0974 / Hemojuvelin (HJV) / Monoclonal Antibody / Hematology
Anemia of Myelofibrosis (MF)Phase 2OngoingGlobalAnemia of Chronic Kidney Disease (CKD)Phase 1/Phase 2OngoingGlobalAnemia of Inflammatory Bowel Disease (IBD)Phase 2PlannedGlobal
DISC-3405DISC-3405 / TMPRSS6 (Matriptase-2) / Monoclonal Antibody / Hematology
Polycythemia Vera (PV)Phase 2OngoingGlobalSickle Cell Disease (SCD)Phase 1bOngoingGlobal
DISC-0998DISC-0998 / Hemojuvelin (HJV) / Monoclonal Antibody / Hematology
Inflammatory-disease-associated anemiaPreclinicalOngoingGlobal

KLRA

Kailera

kailera.com

4assets

KPTI

Karyopharm

karyopharmmedicalaffairs.com

4assets

SelinexorKPT-330 / XPOVIO / XPO1 (Exportin 1) / Small Molecule / Oncology
Multiple MyelomaMarketedActiveUSGlobalEndometrial CancerPhase 3ActiveUSGlobalMyelofibrosisPhase 3ActiveUSGlobal
EltanexorKPT-8602 / XPO1 (Exportin 1) / Small Molecule / Oncology
Myelodysplastic NeoplasmsPhase 2PausedUSColorectal CancerPhase 2PausedUSProstate CancerPhase 2PausedUS
KPT-9274KPT-9274 / PAK4 / NAMPT / Small Molecule / Oncology
Solid Tumors & LymphomaPhase 1PausedUS
VerdinexorKPT-335 / XPO1 (Exportin 1) / Small Molecule / Veterinary Oncology / Infectious Diseases
Canine LymphomaMarketedActiveUS

KTRA

Tuhura Bio

tuhurabio.com

4assets

IFx-2.0IFx-Hu2.0 / Innate Immune System / Personalized Cancer Vaccine / Innate Immune Agonist / Oncology
Advanced or Metastatic Merkel Cell Carcinoma (1st line, adjunctive to pembrolizumab)Phase 3OngoingUnited StatesMerkel Cell Carcinoma of Unknown Primary (MCCUP)Phase 1/Phase 2OngoingUnited States
TBS-2025KVA12123 / VISTA / Monoclonal Antibody / Oncology
Advanced Solid Tumor CancerPhase 1OngoingUnited StatesmutNPM1 Acute Myeloid Leukemia (AML)Phase 1/Phase 2PlannedUnited States
Bi-functional ADCsMyeloid-derived suppressor cells (MDSCs) / Antibody-Drug Conjugate (ADC) / Oncology
Solid TumorsPreclinicalOngoingUnited States
REM-001Unknown / Photodynamic Therapy / Oncology
Cutaneous Metastatic Breast Cancer (CMBC)Phase 3OngoingUnited States

KTTA

Pasithea

pasithea.com

4assets

PAS-004PAS-004 / MEK 1/2 / Macrocyclic Small Molecule / Oncology / Rare Diseases
Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)MAPK pathway-driven advanced solid tumors
PAS-003PAS-003 / α5β1 Integrin / Monoclonal Antibody / Neurology
Amyotrophic Lateral Sclerosis (ALS)
PAS-001PAS-001 / C4A / Small Molecule / Psychiatry
Schizophrenia
PAS-002PAS-002 / Neurology
Multiple Sclerosis (MS)

KURA

Kuraoncology

kuraoncology.com

4assets

ZiftomenibKO-539 / KOMZIFTI / Menin-KMT2A/MLL interaction / Small molecule / Hematologic Malignancies
R/R NPM1-mutated AMLNewly Diagnosed AMLFLT3-mutated AML
DarlifarnibKO-2806 / Farnesyl transferase / Small molecule / Oncology
Clear cell renal cell carcinoma (ccRCC)Solid tumors
TipifarnibFarnesyl transferase / Small molecule / Oncology
HRAS-mutant Head and Neck Squamous Cell Carcinoma (HNSCC)PIK3CA-dependent HNSCC
KO-7246KO-7246 / Menin / Small molecule / Cardiometabolic
DiabetesCardiometabolic disease

MDGL

Madrigal Pharma

madrigalpharma.com

4assets

MNOV

Medicinova

medicinova.com

4assets

MN-166ibudilast
MN-001tipelukast
MN-221bedoradrine
MN-029denibulin

MSLE

Satellos

satellos.com

4assets

SAT-3247SAT-3247 / AAK1 / Small molecule / Degenerative muscle diseases
Duchenne Muscular Dystrophy (DMD)Facioscapulohumeral Muscular Dystrophy (FSHD)
SAT-3153SAT-3153 / Notch polarity kinase / Small molecule / Degenerative muscle diseases
Duchenne Muscular Dystrophy (DMD)
BertilimumabiCo-008 / Eotaxin-1 (CCL11) / Monoclonal antibody / Immunology
Bullous Pemphigoid
iCo-019iCo-019 / Fungal cell membrane / Small molecule (Oral Amphotericin B) / Infectious Diseases
Fungal infections

NBP

Novabridge

novabridge.com

4assets

GivastomigCLDN18.2 x 4-1BB / Bispecific Antibody / Oncology
Gastric CancerGastrointestinal Malignancies
RagistomigPD-L1 x 4-1BB / Bispecific Antibody / Oncology
Solid Tumors
UliledlimabCD73 / Monoclonal Antibody / Oncology
Cancer
VIS-101VEGF-A x ANG-2 / Bispecific Antibody / Ophthalmology
Wet Age-Related Macular Degeneration (AMD)Diabetic Macular Edema (DME)

NTHI

Neonc

neonc.com

4assets

NEO100NEO-100 / Unknown / Small Molecule / Oncology, Neurology
Malignant GliomasGrade 4 AstrocytomaParkinson's Disease
NEO212NEO-212 / DNA / Small Molecule Conjugate / Oncology
Malignant Gliomas
NEO214NEO-214 / DR5, PDE4 / Small Molecule Conjugate / Oncology
Glioblastoma (GBM)Multiple Myeloma
NEO400NEO-400 / Unknown / Small Molecule Conjugate / Dermatology, Oncology
UV-Induced Skin DamageTopical Oncology

NUVB

Nuvation Bio

nuvationbio.com

4assets

TaletrectinibIBTROZI / ROS1 / Small Molecule / Oncology
Advanced ROS1+ NSCLC
SafusidenibmIDH1 / Small Molecule / Oncology
Diffuse IDH1-mutant gliomaAstrocytomaOligodendroglioma
NUV-1511NUV-1511 / Undisclosed / Drug-Drug Conjugate (DDC) / Oncology
Advanced solid tumors
NUV-868NUV-868 / BET (BD2-selective) / Small Molecule / Oncology
Advanced solid tumors

OMER

Omeros

omeros.com

4assets

NarsoplimabOMS721 / Yartemlea / MASP-2 / Monoclonal antibody / Hematology / Complement-Mediated Diseases
Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)
ZaltenibartOMS906 / MASP-3 / Monoclonal antibody / Hematology / Nephrology
Paroxysmal nocturnal hemoglobinuria (PNH)C3 glomerulopathy
OMS1029OMS1029 / MASP-2 / Biologic / Complement-Mediated Diseases
OMS527OMS527 / PDE7 / Small molecule / Addiction
Cocaine use disorder

PHAR

Pharming

pharming.com

4assets

PRAX

Praxis Medicines

praxismedicines.com

4assets

UlixacaltamidePRAX-944 / T-type calcium channel / Small molecule / Neurology
Essential Tremor
RelutriginePRAX-562 / Persistent sodium current (NaV) / Small molecule / Neurology
Developmental and Epileptic Encephalopathies (DEEs)SCN2A-DEESCN8A-DEE
VormatriginePRAX-628 / Sodium channels (NaV) / Small molecule / Neurology
Focal Onset EpilepsyEpilepsy
ElsunersenSCN2A mRNA / Antisense oligonucleotide (ASO) / Neurology
SCN2A Developmental and Epileptic Encephalopathy

PVLA

Palvellatx

palvellatx.com

4assets

RGNX

Regenx Bio

regenxbio.com

4assets

ABBV-RGX-314surabgene lomparvovec / sura-vec
RGX-121clemidsogene lanparvovec / NAVSUNLI
RGX-111
RGX-202

RLAY

Relaytx

relaytx.com

4assets

ZovegalisibRLY-2608 / Mutant PI3Kα / Small molecule / Oncology & Genetic Disease
HR+/HER2- metastatic breast cancer (PI3Kα-mutated)Phase 3ActiveGlobalPIK3CA-driven vascular malformations/anomaliesPhase 1ActiveGlobal
LirafugratinibRLY-4008 / FGFR2 / Small molecule / Oncology
FGFR2-altered solid tumorsClinicalOut-licensedGlobal
RLY-8161RLY-8161 / NRAS / Small molecule / Oncology
NRAS-driven solid tumorsPreclinicalActiveGlobal
Fabry disease programUndisclosed / Non-inhibitory chaperone / Genetic Disease
Fabry diseasePreclinicalActiveGlobal

RYTM

Rhythmtx

rhythmtx.com

4assets

IMCIVREEsetmelanotide
bivamelagonLB54640
RM-718
Preclinical small moleculesCongenital hyperinsulinism (CHI)

SCYX

Scynexis

scynexis.com

4assets

SCY-770PXL-770 / AMP-activated protein kinase (AMPK) / Small Molecule / Rare Diseases / Nephrology
Autosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 2ActiveGlobal
SCY-247SCY-247 / 1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
Treatment and Prevention of Invasive Fungal InfectionsPhase 1ActiveUnited States
Fungerp analogs1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
Treatment of resistant fungiPreclinicalActiveGlobal
IbrexafungerpSCY-078 / BREXAFEMME / 1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
Vulvovaginal candidiasis (VVC) and Recurrent VVCApprovedActiveUnited StatesInvasive Candidiasis (IC)Phase 3ActiveGlobal

SEPN

Septerna

septerna.com

4assets

SEP-479SEP-479 / PTH1R / Small Molecule / Endocrinology
HypoparathyroidismPhase 1Active
SEP-631SEP-631 / MRGPRX2 / Small Molecule / Immunology and Inflammation
Chronic spontaneous urticaria (CSU)Phase 1ActiveOther mast cell diseasesPhase 1Active
TSHR ProgramTSHR / Small Molecule / Endocrinology
Graves' diseasePreclinicalActiveThyroid eye diseasePreclinicalActive
Novo Nordisk Partnered ProgramsGLP-1R, GIPR, GCGR, Undisclosed / Small Molecule / Metabolic Diseases
Metabolic diseasesDiscoveryActive

SILO

Silo Pharma

silopharma.com

4assets

SPC-15SPC-15 / 5-HT4 receptor agonist / Small Molecule / Psychiatry
PTSD and stress-induced anxiety disordersPreclinicalActiveGlobal
SP-26SP-26 / NMDA receptor antagonist / Implant / Neurology
Fibromyalgia and chronic painPreclinicalActiveGlobal
SPC-14SPC-14 / Unknown / Small Molecule / Neurology
Alzheimer's diseasePreclinicalActiveGlobal
SPU-16SPU-16 / Unknown / Peptide / Neurology
Multiple sclerosisPreclinicalActiveGlobal

SLGL

Sol Gel

sol-gel.com

4assets

patidegibSGT-610 / Hedgehog signaling pathway (SMO) / Small molecule / Dermatology
Gorlin syndrome (prevention of new BCC lesions)High-frequency basal cell carcinoma (BCC)
erlotinibSGT-210 / EGFR / Small molecule / Dermatology
Undisclosed indications
tretinoin and benzoyl peroxide cream, 0.1%/3%TWYNEO / Retinoid receptors / Bacterial proteins / Small molecule / Dermatology
Acne vulgaris
benzoyl peroxide cream, 5%EPSOLAY / Bacterial proteins / Small molecule / Dermatology
Inflammatory lesions of rosacea

STRO

Sutro Bio

sutrobio.com

4assets

STRO-004Tissue Factor ADC
STRO-006Integrin beta-6 ADC
STRO-227PTK7-targeting dual-payload ADC
ASP2998TROP2-targeted iADC

TCRX

Tscan

tscan.com

4assets

TSC-100TSC-100 / HA-1 / TCR-T Cell Therapy / Hematologic Malignancies
Acute Myeloid Leukemia (AML) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesAcute Lymphoblastic Leukemia (ALL) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesMyelodysplastic Syndromes (MDS) (Post-HCT Relapse Prevention)Phase 1ActiveUnited States
TSC-101TSC-101 / HA-2 / TCR-T Cell Therapy / Hematologic Malignancies
Acute Myeloid Leukemia (AML) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesAcute Lymphoblastic Leukemia (ALL) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesMyelodysplastic Syndromes (MDS) (Post-HCT Relapse Prevention)Phase 1ActiveUnited States
ImmunoBank TCR-T CandidatesT-Plex / Multiple Targets (e.g., PRAME, HPV16, MAGE-A1) / Multiplexed TCR-T Cell Therapy / Oncology
Solid TumorsPhase 1ActiveUnited States
Novel TargetsUnidentified Autoantigens / Targeted Therapeutics / Autoimmune Disorders
Crohn's DiseaseDiscoveryActiveUnited States

TNGX

Tangotx

tangotx.com

4assets

PRMT5vopimetostat
PRMT5TNG456
HBS1LTNG961
CoRESTTNG260

VXRT

Vaxart

vaxart.com

4assets

VXA-CoV2-3.3VXA-CoV2-3.3 / SARS-CoV-2 / Oral recombinant adenovirus vaccine / Infectious Diseases
COVID-19
Norovirus Oral Vaccine CandidateNorovirus (GI.1 and GII.4) / Oral recombinant adenovirus vaccine / Infectious Diseases
Norovirus InfectionAcute Gastroenteritis
Seasonal Influenza Oral Vaccine CandidateVXA-A1 / Influenza Virus / Oral recombinant adenovirus vaccine / Infectious Diseases
Seasonal Influenza
HPV Therapeutic Vaccine CandidateHuman Papillomavirus (HPV) / Oral recombinant therapeutic vaccine / Oncology
HPV-associated Cancers / Immuno-oncology

XENE

Xenon Pharma

xenon-pharma.com

4assets

AzetukalnerXEN1101 / Kv7.2/Kv7.3 potassium channel opener / Small Molecule / Neurology and Psychiatry
Focal Onset Seizures (FOS)Phase 3OngoingGlobalPrimary Generalized Tonic-Clonic Seizures (PGTCS)Phase 3OngoingGlobalMajor Depressive Disorder (MDD)Phase 3OngoingGlobal
XEN1120XEN1120 / Kv7 potassium channel opener / Small Molecule / Pain
PainPhase 1OngoingUnited States
XEN1701XEN1701 / NaV1.7 sodium channel modulator / Small Molecule / Pain
PainPhase 1OngoingUnited States
NBI-921355NBI-921355 / NaV1.2 / NaV1.6 sodium channel inhibitor / Small Molecule / Neurology
EpilepsyPhase 1OngoingGlobal

ABOS

Acumen Pharmaceuticals

acumenpharm.com

3assets

SabirnetugACU193 / soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; immunotherapy / Neurology
Early Alzheimer's diseasePhase 2ongoing ALTITUDE-AD; IV formulationglobal
Sabirnetug subcutaneous formulationACU193 / soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; subcutaneous formulation with ENHANZE / Neurology
Early Alzheimer's diseasePhase 1subcutaneous formulation study
Enhanced Brain Delivery (EBD™)TfR-Brain transporter + AβO mAb / bispecific/brain-delivered antibody program / Neurology
Early Alzheimer's diseasePreclinicalPre-clinical Alzheimer's diseasePreclinical

ALKS

Alkermes

alkermes.com

3assets

Sodium oxybate extended-release oral suspensiononce-nightly extended-release oral suspension / Sleep disorders
Idiopathic hypersomniaPhase 3Evaluating in REVITALYZ
Olanzapine and samidorphanLYBALVI / oral small molecule combination / Psychiatry
Schizophrenia and bipolar I disorder (pediatric)Phase 3Evaluating in ENLIGHTEN-Youth
AlixorextonALKS 2680 / orexin 2 receptor / oral small molecule agonist / Sleep disorders
Narcolepsy type 1Phase 3Brilliance Studies programNarcolepsy type 2Phase 3Brilliance Studies programIdiopathic hypersomniaPhase 2Vibrance-3

APRE

Aprea

aprea.com

3assets

APR-1051APR-1051 / WEE1 kinase / Oral small-molecule inhibitor / Oncology
Advanced solid tumorsPhase 1Monotherapy; dose escalation ongoing
ATRN-119 (Mosipasertib)ATRN-119 / Mosipasertib / ATR kinase / Oral macrocyclic small-molecule inhibitor / Oncology
Advanced solid tumors (ovarian, CRC, lung)Phase 1Monotherapy; RP2D determinedHematological cancers (AML, MDS)Phase 1Monotherapy; RP2D determined
APR-1602APR-1602 / DYRK1 / Oral small-molecule inhibitor / Oncology
Solid tumors and hematological cancersPreclinicalReady to enter IND-enabling studies

AQST

Aquestive

aquestive.com

3assets

AnaphylmAQST-109 / Anaphylm / epinephrine / dibutepinephrine sublingual film / Allergy
Type I allergic reactions, including anaphylaxisNDA/FiledCRL received; NDA resubmission planned Q3 2026USType I allergic reactions, including anaphylaxisRegulatory application plannedApplications planned in 2026CanadaEU
AQST-108AQST-108 / epinephrine / topical gel / Dermatology
Alopecia areataPhase 1second Phase 1 dosing completed; readout expected Q2 2026USMultiple potential dermatologic/localized indicationsPreclinicalevaluating
LibervantLibervant / diazepam / diazepam buccal film / CNS
Acute repetitive seizures / seizure clustersTentative approvalUS market access subject to orphan exclusivity expiration; relaunch targeted 2027 or sooner if full approvalUS

ARQT

Arcutis

arcutis.com

3assets

Topical Roflumilast CreamARQ-151 / ZORYVE / PDE4 / small molecule topical cream / Dermatology
Plaque psoriasis 0.3% (2-5 yr)sNDA submittedPDUFA June 29, 2026USAtopic dermatitis 0.05% (3-24 mo)Phase 2planned sNDA submission Q2 2026US
Topical Roflumilast FoamARQ-154 / ZORYVE / PDE4 / small molecule topical foam / Dermatology
Vitiligo 0.3%Phase 2acurrently enrolling; advancement decision Q4 2026Hidradenitis suppurativa 0.3%Phase 2acurrently enrolling; advancement decision Q1 2027
ARQ-234ARQ-234 / CD200R / fusion protein biologic / Dermatology
Atopic dermatitisPhase 1a/1benrolling

ARTV

Artiva Bio

artivabio.com

3assets

AlloNKAB-101 / AlloNK® / CD20 combinations / Allogeneic off-the-shelf non-genetically modified cryopreserved NK cell therapy / Autoimmune disease; oncology
Refractory rheumatoid arthritisPhase 2aongoing; lead indication; Fast TrackUSSjögren’s diseasePhase 2aongoingUSIdiopathic inflammatory myopathiesPhase 2aongoingUS
CAR-NKAB-205 / CD5 / Allogeneic anti-CD5 CAR-NK cell therapy expressing soluble IL-15 / Oncology
Hematologic malignanciesPreclinicalcollaborator-funded
CAR-NKAB-201 / HER2 / Allogeneic anti-HER2 CAR-NK cell therapy expressing soluble IL-15 / Oncology
Solid tumorsPreclinicalcollaborator-funded; US orphan drug designationUS

ATYR

Atyr Pharma

atyrpharma.com

3assets

EfzofitimodNRP2 modulator / tRNA synthetase-derived biologic immunomodulator / Interstitial lung disease
Pulmonary sarcoidosisPhase 3SSc-ILDPhase 2Other ILD (CTD-ILD; CHP)Phase 1
ATYR0101ATYR0101 / LTBP1 modulator / DARS tRNA synthetase-derived candidate / Fibrosis
FibrosisPreclinical
ATYR0750ATYR0750 / FGFR4 modulator / AARS tRNA synthetase-derived candidate / Liver disorders
Liver disordersPreclinical

AZTR

Azitrainc

azitrainc.com

3assets

ATR-12ATR-12 / LEKTI replacement / SPINK5 / Topical live biotherapeutic; engineered S. epidermidis expressing rhLEKTI-D6 / Dermatology
Netherton syndromePhase 1bclinicalUS
ATR-04ATR-04 / IL-36g reduction; S. aureus inhibition / Topical live biotherapeutic; auxotrophic engineered S. epidermidis strain SE484 / Dermatology / oncology supportive care
EGFR inhibitor-associated rashPhase 1/2clinicalUS
ATR-01ATR-01 / Filaggrin / FLG / Topical protein candidate; FLG5-6 with cell-penetrating peptide / Dermatology
Ichthyosis vulgarisPreclinicalpreclinical

BIOA

Bioage Labs

bioagelabs.com

3assets

BGE-102BGE-102 / NLRP3 / Oral CNS-penetrant small-molecule NLRP3 inhibitor / Cardiometabolic / ophthalmology
CV riskPhase 1Diabetic macular edemaPhase 1
APJ OralApelin receptor APJ / Oral APJ agonist / Metabolic disease
ObesityLead optimization
APJ ParenteralApelin receptor APJ / Parenteral APJ agonist / Metabolic disease
ObesityLead optimization

BRNS

Barinthus Bio

barinthusbio.com

3assets

VTP-1000VTP-1000 / Gluten antigens; immune tolerance via SNAP-TI / Antigen-specific immunotherapy / Autoimmunity
Celiac diseasePhase 1Active; AVALON trial ongoingWorldwide rights
VTP-300VTP-300 / HBsAg/full-length surface, modified polymerase and core antigens / ChAdOx/MVA viral-vector immunotherapeutic / Infectious disease
Chronic hepatitis B virus infectionPhase 2 / Phase 2bDeprioritized for direct operations; seeking partner; HBV003 fully enrolledWorldwide rights
VTP-800/850VTP-800/850 / Prostate cancer antigens including 5T4, PSA, PAP and STEAP1 / ChAdOx/MVA viral-vector immunotherapeutic / Oncology
Prostate cancer; biochemical recurrence after definitive local therapyPhase 1Deprioritized for direct operations; seeking partner; PCA001 complete per IR updatesWorldwide rights

CGON

Cgoncology

cgoncology.com

3assets

Cretostimogene grenadenorepvec monotherapyintravesically delivered oncolytic immunotherapy / Oncology
High-risk BCG-unresponsive non-muscle invasive bladder cancer (BOND-003 Cohort C; CIS with or without high-grade Ta/T1)Phase 3Enrollment complete; data presented at AUA 2025High-risk BCG-unresponsive non-muscle invasive bladder cancer (BOND-003 Cohort P; high-grade Ta/T1)Phase 2Enrollment complete; topline results at SUO 2025Intermediate-risk non-muscle invasive bladder cancer (PIVOT-006)Phase 3Enrollment complete; topline data expected 1H 2026
Cretostimogene grenadenorepvec + gemcitabineoncolytic immunotherapy + chemotherapy combination / Oncology
High-risk BCG-exposed non-muscle invasive bladder cancer (CORE-008 Cohort CX)Trial in progress; initiated 2Q 2025; data expected 1H 2026
Cretostimogene grenadenorepvec + pembrolizumabPD-1 combination / oncolytic immunotherapy + checkpoint inhibitor combination / Oncology
High-risk BCG-unresponsive non-muscle invasive bladder cancer (CORE-001)Phase 2Final results manuscript; 24-month data presented at ASCO 2024

CHRS

Coherus

coherus.com

3assets

LOQTORZI (toripalimab-tpzi)LOQTORZI / PD-1 / Anti-PD-1 monoclonal antibody / Oncology
1L Nasopharyngeal Carcinoma (combo with chemotherapy)ApprovedApprovedUnited States2L+ Nasopharyngeal Carcinoma (monotherapy)ApprovedApprovedUnited States
TagmokitugCHS-114 / CCR8 / Anti-CCR8 monoclonal antibody / Oncology
4L+ Colorectal Cancer (combo with toripalimab)Phase 22L HNSCC (combo with toripalimab)Phase 12L Gastric Cancer/GEJ/EAC (combo with toripalimab)Phase 1
CasdozokitugIL-27 / IL-27 antagonist monoclonal antibody / Oncology
1L Hepatocellular Carcinoma (combo with toripalimab + bevacizumab)Phase 2

CING

Cingulate

cingulate.com

3assets

CTx-1301CTx-1301 / Small molecule; once-daily multi-core PTR extended-release tablet of dexmethylphenidate / CNS / neuropsychiatry
Attention deficit/hyperactivity disorder (ADHD)NDA under FDA review; PDUFA May 31, 2026ActiveUnited States
CTx-1302CTx-1302 / Small molecule; multi-core PTR formulation of dextroamphetamine / CNS / neuropsychiatry
Attention deficit/hyperactivity disorder (ADHD)Phase 3 plannedActiveUnited States
CTx-2103CTx-2103 / Small molecule; once-daily multi-release PTR formulation of buspirone hydrochloride / CNS / neuropsychiatry
Anxiety disorders / symptoms of anxietyPre-IND / formulation-stageActiveUnited States

CLRB

Cellectar

cellectar.com

3assets

Iopofosine I 131tumor-cell phospholipid ether/lipid raft targeting; iodine-131 radiotherapeutic payload / phospholipid drug conjugate radiotherapeutic / Oncology
Relapsed/refractory Waldenström macroglobulinemiaPhase 2 pivotal completedBreakthrough Therapy Designation; EMA CMA submission planned Q3 2026United StatesEuropeRelapsed/refractory multiple myelomaPhase 2bongoing / studiedCentral nervous system lymphomaPhase 2bongoing / studied
CLR 121125CLR 125 / tumor-cell phospholipid ether/lipid raft targeting; iodine-125 Auger emitter payload / phospholipid ether radioconjugate / Oncology
Relapsed or refractory triple-negative breast cancerPhase 1bdose-finding study ongoingUnited States
CLR 121225CLR 225 / tumor-cell phospholipid ether/lipid raft targeting; actinium-225 alpha emitter payload / phospholipid ether radioconjugate / Oncology
Pancreatic cancerPreclinical / planned Phase 1clinical evaluation planned

CLYM

Climb Bio

climbbio.com

3assets

Budoprutug IVCD19 / monoclonal antibody / Immune-mediated diseases
Primary Membranous NephropathyPhase 2Immune ThrombocytopeniaPhase 1Systemic Lupus ErythematosusPhase 1
Budoprutug SCCD19 / monoclonal antibody / Immune-mediated diseases
Autoimmune DiseasePhase 1
CLYM116CLYM116 / APRIL / monoclonal antibody / Immune-mediated diseases / renal disease
IgA NephropathyPhase 1

CNTX

Context Therapeutics

contexttherapeutics.com

3assets

CTIM-76CTIM-76 / Claudin 6 (CLDN6) x CD3 / Bispecific antibody / T-cell engager / Oncology
Ovarian cancerPhase 1 dose escalationOngoingUnited StatesEndometrial cancerPhase 1 dose escalationOngoingUnited StatesTesticular cancerPhase 1 dose escalationOngoingUnited States
CT-95CT-95 / Mesothelin (MSLN) x CD3 / Bispecific antibody / T-cell engager / Oncology
Pancreatic cancerPhase 1 dose escalationOngoingUnited StatesNon-small cell lung cancerPhase 1 dose escalationOngoingUnited StatesOvarian cancerPhase 1 dose escalationOngoingUnited States
CT-202CT-202 / Nectin-4 x CD3 / Bispecific antibody / T-cell engager / Oncology
Bladder cancerPreclinicalRegulatory filings completed to support first-in-human trialUnited StatesNon-small cell lung cancerPreclinicalRegulatory filings completed to support first-in-human trialUnited StatesColorectal cancerPreclinicalRegulatory filings completed to support first-in-human trialUnited States

CRVS

Corvus Pharma

corvuspharma.com

3assets

SoquelitinibCPI-818 / ITK / Oral small molecule ITK inhibitor / Immune diseases; oncology
Relapsed/refractory peripheral T-cell lymphoma (PTCL)Registration Phase 3EnrollingUnited StatesModerate-to-severe atopic dermatitisPhase 2Initiated/enrollingUnited StatesModerate-to-severe atopic dermatitisPhase 1b/2Enrolling; partner-ledChina
CiforadenantCPI-444 / Adenosine A2A receptor / Oral small molecule checkpoint inhibitor / Oncology
Cancer indicationsClinical-stageGreater China
MupadolimabCPI-006 / CD73 / Anti-CD73 monoclonal antibody / Oncology
Non-small cell lung cancer (NSCLC)Phase 1/1bPartner-led evaluationChina

CTMX

Cytomx

cytomx.com

3assets

Varseta-MCX-2051 / EpCAM / Probody ADC; Topo-1 ADC / Oncology
3L+ metastatic colorectal cancer (mCRC)Phase 1 expansion2L/3L mCRC, in combination with bevacizumabTrials anticipated to begin in 2H 20262L mCRC, in combination with bevacizumab + chemotherapyTrials anticipated to begin in 2H 2026
CX-801CX-801 / IFN-α2b / Interferon alpha-2b Probody cytokine / Oncology
Advanced melanomaPhase 1
CX-908CX-908 / P-Cadherin x CD3 / Probody T-cell engager / TCB / Oncology
Solid tumorsPreclinical

CYBN

Cybin

helus.com

3assets

HLP003CYB003 / 5-HT2A receptor / Deuterated novel serotonergic agonist; oral NCE / Mental health / neuropsychiatry
Adjunctive treatment of Major Depressive Disorder (MDD)Phase 3Dosing underway in Phase 3 PARADIGM program; FDA Breakthrough Therapy Designation
HLP004CYB004 / Serotonin receptors / Deuterated novel serotonergic agonist; intramuscular dDMT program / Mental health / neuropsychiatry
Generalized Anxiety Disorder (GAD)Phase 2Phase 2 study enrollment complete; topline safety and efficacy data expected Q1 2026United States
HLP005CYB005 / Phenethylamines and tryptamines / CNS disorders
CNS DisordersPreclinicalPreclinical

DAWN

Dayone Bio

dayonebio.com

3assets

TovorafenibDAY101 / OJEMDA / RAF / MAPK pathway / Oral small-molecule type II RAF inhibitor / Oncology
BRAF-altered relapsed pLGGApprovedOJEMDA received FDA accelerated approval; FIREFLY-1 pivotal Phase 2 follow-up ongoing/disclosedU.S.Frontline RAF-altered pLGGPhase 3 / RegistrationalFIREFLY-2 pivotal Phase 3
Emiltatug ledadotinEmi-Le / B7-H4 / Antibody-drug conjugate (ADC) / Oncology
Adenoid cystic carcinoma (ACC), adult and pediatric solid tumorsPhase 1B7-H4-targeted ADC; Phase 1 data anticipated mid-2026 per company investor materials
DAY301MTX-13 / CB-002 / PTK7 / Antibody-drug conjugate (ADC) / Oncology
Adult and pediatric solid tumorsPhase 1Phase 1a data anticipated 2H 2026 per company investor materials

EDSA

Edesa Biotech

edesabiotech.com

3assets

EB06 Anti-CXCL10 Monoclonal antibodyEB06 / CXCL10 / Monoclonal antibody / Dermatological diseases
VitiligoPhase 2Health Canada CTA approved; IND in processCanadaUnited States
EB01 (daniluromer)EB01 / sPLA2 / Topical sPLA2 inhibitor / Dermatological diseases
Allergic contact dermatitisPhase 2b completedTopline results available
ParidiprubartEB05 / TLR4 / Monoclonal antibody / Respiratory diseases
Acute respiratory distress syndromePhase 3Phase 3 results reported; selected for U.S. government-funded Phase 2 platform studyUnited StatesCanada

ERAS

Erasca

erasca.com

3assets

ERAS-0015ERAS-0015 / RAS / Small molecule molecular glue / Oncology
RAS-mutant solid tumorsPhase 1AURORAS-1; in clinicWorldwide
ERAS-4001ERAS-4001 / KRAS / Small molecule / Oncology
KRAS-mutant solid tumorsPhase 1BOREALIS-1; in clinicWorldwide
ERAS-12ERAS-12 / EGFR D2/D3 / Large molecule; bispecific antibody / Oncology
EGFR and RAS/MAPK altered tumorsWorldwide

GENB

Generatebio Medicines

generatebiomedicines.com

3assets

GLTO

Damoratx

damoratx.com

3assets

DMR-001DMR-001 / mutant calreticulin (mutCALR) / Fc-null, half-life extended anti-mutCALR monoclonal antibody; subcutaneous biologic / Hematology/oncology
mutCALR-driven essential thrombocythemiaPreclinical / IND-enablingIND or CTA submission planned mid-2026; clinical proof-of-concept datasets expected beginning mid-2027USex-USmutCALR-driven myelofibrosisPreclinical / IND-enablingIND or CTA submission planned mid-2026; clinical proof-of-concept datasets expected beginning mid-2027USex-US
DMR-002DMR-002 / mutant calreticulin (mutCALR) / mutCALR-targeted mechanism; undisclosed / Hematology/oncology
mutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2H 2026USex-US
DMR-003DMR-003 / mutant calreticulin (mutCALR) x CD3 / bispecific antibody; T-cell engager / Hematology/oncology
mutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2027USex-US

GNPX

Genprex

genprex.com

3assets

quaratusugene ozeplasmid (REQORSA)REQORSA / TUSC2 / non-viral gene therapy / Oncology
EGFR-mutant NSCLC (osimertinib-resistant)Phase 1/2Phase 2a expansion enrollingUnited Statesextensive-stage SCLC (maintenance with atezolizumab)Phase 1/2expansion enrollingUnited States
GPX-002GPX-002 / PDX1 and MafA / AAV gene therapy / Diabetes
Type 1 DiabetesPreclinicalType 2 DiabetesPreclinical
NPRL2 gene therapyNPRL2 / non-viral gene therapy / Oncology
KRAS/STK11 mutant anti-PD1-resistant NSCLCPreclinical

HELP

Helus

helus.com

3assets

HLP0035-HT2A (and other serotonin receptors) / Deuterated NSA (oral) / Mental Health
Adjunctive treatment of major depressive disorder (MDD)Phase 3Dosing underway (PARADIGM program); FDA Breakthrough Therapy DesignationUnited StatesEuropeAustralia
HLP004Serotonin receptors / Deuterated NSA (intramuscular) / Mental Health
Generalized anxiety disorder (GAD)Phase 2Positive topline results reportedUnited States
HLP005NSA (phenethylamines and tryptamines) / Mental Health
CNS disordersPreclinical

HURA

Tuhura Bio

tuhurabio.com

3assets

IFx-2.0plasmid DNA (innate immune agonist) / Oncology
Advanced or metastatic Merkel cell carcinoma (first-line, adjunctive to pembrolizumab)Phase 3OngoingUnited StatesMetastatic Merkel cell carcinoma of unknown primary (MCCUP, first-line)Phase 1b/2aOngoingUnited States
TBS-2025VISTA / monoclonal antibody / Oncology
NPM1-mutated relapsed/refractory AML (with menin inhibitor)Phase 1b/2PlannedUnited States
DOR bi-specific ADCDelta opioid receptor (DOR) on MDSCs/TAMs / antibody-drug conjugate (ADC) / Oncology
Acute myeloid leukemia (AML)PreclinicalLead selection

IMUX

Imux

imux.com

3assets

Vidofludimus calciumIMU-838 / DHODH inhibitor and Nurr1 activator / Oral small molecule / Multiple sclerosis
Relapsing multiple sclerosisPhase 3Ongoing (ENSURE-1/2 fully enrolled; top-line data expected end 2026)GlobalProgressive multiple sclerosisPhase 2Completed (CALLIPER positive; PPMS Phase 3 initiation expected 2026)North AmericaEurope
IMU-856IMU-856 / SIRT6 modulator / Oral small molecule / Gastrointestinal diseases
Celiac diseasePhase 1bCompleted (positive proof-of-concept)Global
IMU-381IMU-381 / Nurr1 / Oral small molecule / Autoimmune diseases
Neurologic, gastrointestinal and other autoimmune diseasesPreclinicalPreclinical

IVA

Inventiva Pharma

inventivapharma.com

3assets

KNSA

Kiniksa

kiniksa.com

3assets

KOD

Kodiak

kodiak.com

3assets

tarcocimab tedromerZenkuda / VEGF / Antibody Biopolymer Conjugate (ABC) / Ophthalmology
Wet Age-Related Macular Degeneration (wet AMD)Diabetic RetinopathyRetinal Vein Occlusion (RVO)
KSI-501KSI-501 / IL-6 / VEGF / Bispecific Antibody Biopolymer Conjugate (ABC) / Ophthalmology
Wet Age-Related Macular Degeneration (wet AMD)Diabetic Macular Edema (DME)
KSI-101KSI-101 / IL-6 / VEGF / Bispecific Protein / Ophthalmology
Macular Edema Secondary to Inflammation (MESI)

KYTX

Kyvernatx

kyvernatx.com

3assets

mivocabtagene autoleucelKYV-101 / CD19 / Autologous CAR T-cell therapy / Autoimmune Diseases
Stiff Person Syndrome (SPS)Phase 2Completed (Preparing BLA)USGeneralized Myasthenia Gravis (gMG)Phase 3OngoingUSLupus Nephritis (LN)Phase 1/2OngoingUSGermany
KYV-102KYV-102 / CD19 / Autologous CAR T-cell therapy / Autoimmune Diseases
Autoimmune DiseasesPhase 1IND ActiveUS
KYV-201KYV-201 / CD19 / Allogeneic CAR T-cell therapy / Autoimmune Diseases
Autoimmune DiseasesPreclinicalActiveUS

LXRX

Lex Pharma

lexpharma.com

3assets

SotagliflozinINPEFA / ZYNQUISTA / SGLT1 / SGLT2 / Small Molecule / Cardiometabolic
Heart FailureApprovedactiveUnited StatesHypertrophic Cardiomyopathy (HCM)Phase 3activeGlobalType 1 Diabetes (T1D)RegistrationactiveUnited States
PilavapadinLX9211 / AAK1 / Small Molecule / Pain / Neurology
Diabetic Peripheral Neuropathic Pain (DPNP)Phase 2bactiveUnited StatesPostherpetic Neuralgia (PHN)Phase 2inactiveUnited States
LX9851LX9851 / ACSL5 / Small Molecule / Metabolism
Obesity / Weight ManagementPhase 1activeGlobal

MDWD

Mediwound

mediwound.com

3assets

NUVL

Nuvalent

nuvalent.com

3assets

OCS

Oculis

oculis.com

3assets

OCUL

Ocutx

ocutx.com

3assets

PHAT

Phathom Pharma

phathompharma.com

3assets

PHIO

Phio Pharma

phiopharma.com

3assets

PH-762PH-762 / PD-1 / INTASYL (sdRNA) / Oncology
Cutaneous Squamous Cell CarcinomaMelanomaMerkel Cell Carcinoma
PH-894PH-894 / BRD4 / INTASYL (sdRNA) / Oncology / Infectious Disease
Advanced MelanomaMerkel Cell CarcinomaHepatocellular Carcinoma
PH-804PH-804 / TIGIT / INTASYL (sdRNA) / Oncology
Cancer

PYPD

Polypid

polypid.com

3assets

D-PLEX100D-PLEX100 / Bacterial Ribosome / Small Molecule Formulation / Infectious Diseases / Surgical Site Care
Prevention of surgical site infections (SSIs) following abdominal colorectal surgeryPhase 3OngoingUnited StatesEuropeIsrael
PP03APP03A / GLP-1 Receptor / Peptide Formulation / Metabolic Disease
Obesity and Type 2 DiabetesPreclinicalOngoing
PP04APP04A / OncoPLEX / STING / Small Molecule Formulation / Oncology
Solid TumorsPreclinicalOngoing

QNRX

Quoin Pharma

quoinpharma.com

3assets

QRX003QRX003 / Serine protease inhibitor / Small molecule / Dermatology
Netherton SyndromePhase 3OngoingUnited StatesEuropeMiddle EastPeeling Skin SyndromePhase 2OngoingNew Zealand
Topical rapamycinQRX009 / mTOR / Small molecule / Dermatology
Microcystic Lymphatic MalformationsPreclinicalOngoingGlobalVenous MalformationsPreclinicalOngoingGlobalAngiofibromaPreclinicalOngoingGlobal
QRX008QRX008 / VLA-4 / VCAM-1 inhibitor / Small molecule / Dermatology
SclerodermaPreclinicalOngoingGlobal

RAPP

Rapportrx

rapportrx.com

3assets

RAP-219RAP-219 / TARPγ8 AMPAR / Small molecule / Neurology/Psychiatry
Focal onset seizuresPrimary generalized tonic-clonic seizuresBipolar mania
α6β4 nAChR Agonistα6β4 nAChR / Small molecule / Neurology
Chronic painMigraine
α9α10 nAChR Modulatorα9α10 nAChR / Small molecule / Hearing/Vestibular
Hearing disordersVestibular disorders

SCNI

Scinai

scinai.com

3assets

PC111PC111 / sFasL (soluble Fas Ligand) / Monoclonal Antibody / Immunology / Dermatology
PemphigusPreclinicalActiveGlobalStevens-Johnson syndrome / toxic epidermal necrolysis (SJS/TEN)PreclinicalActiveGlobal
Intradermal IL-17A/F NanoAbIL-17A / IL-17F / VHH Antibody Fragment (NanoAb) / Immunology / Dermatology
Mild-to-moderate plaque psoriasisPreclinicalActiveGlobal
Systemic Bi-Specific IL-17 NanoAbIL-17A / IL-17F and secondary proprietary target / Bi-specific VHH Antibody Fragment (NanoAb) / Immunology / Dermatology
Moderate-to-severe plaque psoriasisPreclinicalActiveGlobalPsoriatic arthritisPreclinicalActiveGlobal

SGMT

Sagimet

sagimet.com

3assets

DenifanstatASC40 / FASN / Small molecule / Metabolic disease, Dermatology, Oncology
MASH (F2/F3)Phase 3ActiveModerate to severe acnePhase 3ActiveRecurrent glioblastoma (GBM)Phase 3Active
TVB-3567FASN / Small molecule / Dermatology, Oncology
AcnePhase 1ActiveSolid tumorsPreclinicalActive
FASNi TopicalFASN / Small molecule / Dermatology
AcnePreclinicalActive

SUPN

Supernus

supernus.com

3assets

TARS

Tarsusrx

tarsusrx.com

3assets

lotilaner ophthalmic solution 0.25%TP-03 / XDEMVY / Small molecule / Eye Care
Demodex blepharitisMeibomian Gland Disease
lotilaner ophthalmic gelTP-04 / Small molecule / Dermatology / Eye Care
RosaceaOcular rosacea
lotilaner oral tabletTP-05 / Small molecule / Infectious Disease Prevention
Lyme disease preventionCommunity reduction of malaria

TECX

Tectonictx

tectonictx.com

3assets

TX45TX-000045 / RXFP1 / Fc-relaxin fusion protein / Cardiopulmonary
PH-HFpEF (Group 2 Pulmonary Hypertension)PH-HFrEF (Group 2 Pulmonary Hypertension)PH-ILD (Group 3 Pulmonary Hypertension)
TX2100TX002100 / GPCR / Biotherapeutic / GPCR Antagonist / Hematology / Rare Diseases
Hereditary Hemorrhagic Telangiectasia (HHT)
Fibrosis ProgramGPCR / Bispecific GPCR Antagonist / Fibrosis
Fibrosis

TGTX

Tg Therapeutics

tgtherapeutics.com

3assets

ublituximabTG-1101 / BRIUMVI / CD20 / Monoclonal Antibody / Autoimmune Diseases
Relapsing Multiple Sclerosis (RMS)
ublituximab subcutaneousCD20 / Monoclonal Antibody / Autoimmune Diseases
Relapsing Multiple Sclerosis (RMS)
azercabtagene zapreleucelazer-cel / CD19 / Allogeneic CAR-T Cell Therapy / Autoimmune Diseases
Progressive Multiple Sclerosis (PMS)Autoimmune Diseases

TLSA

Tizianalife Sciences

tizianalifesciences.com

3assets

Intranasal ForalumabTZLS-401 / CD3 epsilon / Monoclonal Antibody / Neurology/Immunology
Non-Active Secondary Progressive Multiple Sclerosis (SPMS)Multiple System Atrophy (MSA)Mild Alzheimer's Disease
Anti IL-6R mAbTZLS-501 / IL-6R / Monoclonal Antibody / Immunology
MilciclibTZLS-201 / CDKs / Small Molecule / Oncology
NSCLC with pan KRAS-positive mutations

TNYA

Tenaya Therapeutics

tenayatherapeutics.com

3assets

TN-201TN-201 / MYBPC3 / AAV-based gene therapy / Cardiovascular
MYBPC3-associated Hypertrophic Cardiomyopathy (HCM)
TN-401TN-401 / PKP2 / AAV9-based gene therapy / Cardiovascular
PKP2-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
TN-301TN-301 / HDAC6 / Small Molecule / Cardiometabolic / Neuromuscular
Heart Failure with preserved Ejection Fraction (HFpEF)Duchenne Muscular Dystrophy (DMD)

TVTX

Travere

travere.com

3assets

sparsentanFILSPARI
pegtibatinaseTVT-058
tioproninThiola / Thiola EC

TYRA

Tyra

tyra.bio

3assets

dabogratinibTYRA-300 / FGFR3 / Small molecule / Oncology / Skeletal Dysplasia
Metastatic Urothelial Cancer (mUC)Non-Muscle Invasive Bladder Cancer (IR NMIBC)Achondroplasia
TYRA-200TYRA-200 / FGFR1/2/3 / Small molecule / Oncology
Intrahepatic CholangiocarcinomaAdvanced Solid Tumors
TYRA-430TYRA-430 / FGFR4/3 / Small molecule / Oncology
Hepatocellular Carcinoma (HCC)Advanced Solid Tumors

VERA

Veratx

veratx.com

3assets

AtaciceptVT-001 / BAFF and APRIL / Recombinant Fusion Protein / Immunology / Nephrology
IgA Nephropathy (IgAN)BLA SubmittedPriority Review (PDUFA July 2026)USGlobalLupus NephritisPhase 2OngoingGlobalFocal Segmental Glomerulosclerosis (FSGS)Phase 2OngoingGlobal
MAU868MAU868 / BK Virus / Monoclonal Antibody / Infectious Disease / Transplantation
BK Viremia in Kidney Transplant RecipientsPhase 2Evaluating for future developmentGlobal
VT-109VT-109 / BAFF and APRIL / Fusion Protein / Immunology
B Cell Mediated DiseasesPreclinicalActiveGlobal

VSTM

Verastem

verastem.com

3assets

Avutometinib and DefactinibVS-6766 / VS-6063 / AVMAPKI FAKZYNJA CO-PACK / RAF/MEK and FAK / Small Molecule / Oncology
KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC)ApprovedActiveUnited StatesRecurrent low-grade serous ovarian cancer (LGSOC)Phase 3ActiveGlobalMetastatic Pancreatic Cancer (PDAC)Phase 2ActiveGlobal
AvutometinibVS-6766 / RAF/MEK / Small Molecule / Oncology
BRAF, KRAS and NRAS-Mutant Solid TumorsPhase 1ActiveGlobal
VS-7375GFH375 / KRAS G12D / Small Molecule / Oncology
KRAS G12D-Mutated Solid TumorsPhase 1/Phase 2ActiveUnited StatesChina

ZURA

Zura Bio

zurabio.com

3assets

tibulizumabZB-106 / IL-17A and BAFF / Bispecific dual-antagonist antibody / Autoimmune and Inflammatory Diseases
Hidradenitis suppurativaSystemic sclerosis
crebankitugZB-168 / IL-7Rα / Monoclonal antibody / Autoimmune and Inflammatory Diseases
Autoimmune and inflammatory conditions
torudokimabZB-880 / IL-33 / Monoclonal antibody / Autoimmune and Inflammatory Diseases
Autoimmune and inflammatory conditions

ABUS

Arbutus Biopharma

arbutusbio.com

2assets

ImdusiranAB-729 / HBV viral antigens including HBsAg / RNAi therapeutic; subcutaneous GalNAc-conjugated / Infectious disease; chronic HBV
Chronic hepatitis B virus infectionPhase 2aActive clinical studies; IM-PROVE I and IM-PROVE II combinations
AB-101AB-101 / PD-L1 / Oral PD-L1 inhibitor / Infectious disease; chronic HBV immunotherapy
Chronic hepatitis B virus infectionPhase 1a/1bAB-101-001 in healthy subjects and cHBV patients

ACET

Adicet Bio

adicetbio.com

2assets

Prulacabtagene leucelprula-cel; formerly ADI-001 / CD20 / Allogeneic gamma delta CAR T cell therapy / Autoimmune diseases
Lupus nephritisPhase 1activeSystemic lupus erythematosusPhase 1activeSystemic sclerosisPhase 1active
ADI-212ADI-212 / PSMA / Next-generation gene-edited and armored allogeneic CAR gamma delta T cell therapy / Oncology
Metastatic castration-resistant prostate cancerPreclinicalactive

AKTS

Aktisoncology

aktisoncology.com

2assets

AKY-1189AKY-1189 / Nectin-4 / miniprotein radioconjugate; Actinium-225 radiopharmaceutical / Oncology
Nectin-4 expressing solid tumors; locally advanced or metastatic UC, breast cancer, NSCLC, colorectal cancer, cervical cancerPhase 1bongoing
AKY-2519AKY-2519 / B7-H3 (CD276) / miniprotein radioconjugate; Actinium-225 radiopharmaceutical / Oncology
B7-H3 expressing solid tumors; prostate, lung and other solid tumorsIND-enablingcurrent

AKTX

Akaritx

akaritx.com

2assets

AKTX-101AKTX-101 / Trop-2 / antibody-drug conjugate; PH1 RNA-splicing payload / Oncology
Urothelial cancerIND-enablingPhase 1 filing expected 4Q 2026Other solid tumorsIND-enablingplanned expansion
AKTX-102AKTX-102 / CEACAM5 / antibody-drug conjugate; PH1 RNA-splicing payload / Oncology
Colon, gastric and lung cancersPreclinicaldeveloping best-in-class antibody/PH1 ADC

ALXO

Alxoncology

alxoncology.com

2assets

evorpaceptCD47 / CD47-blocking immuno-oncology biologic / Oncology
ENHERTU-experienced HER2-positive breast cancer; with trastuzumab + chemotherapyPhase 2FPI mid-year '25RRMM; with SARCLISA + dexamethasonePhase 1Currently enrolling2L/3L advanced HER2-overexpressing gastric/GEJ cancer; with trastuzumab, CYRAMZA + paclitaxelPhase 2Completed; established POC; seeking partner ex-US
ALX2004ALX2004 / EGFR / ADC / Oncology
EGFR-expressing solid tumorsPhase 1FPI mid-year '25

ALZN

Alzamend

alzamend.com

2assets

AL001AL001 / LISPRO / ionic cocrystal of lithium, proline and salicylate / Neurology/Psychiatry
Alzheimer's diseasePhase 2clinical developmentUSBipolar disorderPhase 2clinical developmentUSMajor depressive disorderPhase 2anticipated initiation in 2026US
ALZN002ALZN002 / E22W / beta-amyloid / autologous mutant-peptide sensitized dendritic-cell therapeutic vaccine / Neurology
Mild to moderate dementia of the Alzheimer's typePhase 1/2apaused in 2024; expected to resume by mid-2026US

ANAB

Anaptys Bio

anaptysbio.com

2assets

dostarlimabJemperli / PD-1 / antibody / Oncology
rectal cancerstudiedcolon cancerstudiedhead and neck squamous cell carcinomastudied
imsidolimabIL-36R / antibody / Inflammation/Dermatology
generalized pustular psoriasisBLA acceptedtarget action date December 12, 2026US

ANTX

An2 Therapeutics

an2therapeutics.com

2assets

EpetraboroleLeucyl-tRNA synthetase / oral boron-based small molecule antibiotic / Hematology; Infectious disease
Phlebotomy-dependent polycythemia veraPhase 2proof-of-concept studyMycobacterium abscessus complex lung diseasePhase 2IND cleared; investigator-initiated studyU.S.MelioidosisPhase 2 planneddevelopment
AN2-502998AN2-502998 / CPSF3 / oral boron-based benzoxaborole small molecule / Infectious disease
Chagas diseasePhase 1first-in-human trial underway

ARDX

Ardelyx

ardelyx.com

2assets

IBSRELA®tenapanor / IBSRELA® / NHE3 / small molecule / Gastrointestinal
Chronic Idiopathic Constipation (CIC)Phase III
RDX10531RDX10531 / NHE3 / small molecule / Multiple Therapeutic Areas
UndisclosedPreclinical

AVIR

Atea Pharma

ateapharma.com

2assets

Bemnifosbuvir / Ruzasvir fixed-dose combinationBEM/RZR / HCV NS5B polymerase / NS5A / Oral small-molecule antiviral combination / Infectious disease
Chronic hepatitis C virus infectionPhase 3C-BEYOND enrollment completed; C-FORWARD ongoingUSCanadaOutside North America
AT-587AT-587 / HEV replication / Nucleotide prodrug / Infectious disease
Hepatitis E virus infectionIND/CTA-enablingPhase 1 initiation targeted mid-2026USEU

BHC

Bausch Health

bauschhealth.com

2assets

LarsucosterolEpigenetic modulator / Hepatology
Alcohol-associated hepatitis (AH)Phase 3Registrational Phase 3 program initiated in early 2026; FDA Breakthrough Therapy DesignationUS
AmiselimodS1P / S1P modulator / Gastroenterology
Mild- to moderate-ulcerative colitisPhase 2Internal review of opportunity ongoing

BIVI

Biovie Pharma

bioviepharma.com

2assets

bezisterimNE3107 / ERK / NF-κB / TNF-α inflammatory signaling; insulin resistance / Oral small molecule / Neurology / neurodegenerative disease
Parkinson’s diseasePhase 2Currently in human clinical studies; SUNRISE-PD Phase 2 listed by companyLong COVID neurological symptomsPhase 2ADDRESS-LC Phase 2 listed by companyAlzheimer’s diseasePhase 3Studied; company pipeline states currently being studied
BIV201BIV201 / Vasopressin receptor activity via terlipressin / Continuous infusion formulation of terlipressin / Hepatology
Refractory ascites / ascites due to liver cirrhosisPhase 2b / Phase 3 preparationInvestigational; Orphan Drug and Fast Track designations; Phase 2b data reported and Phase 3 discussed in company updateUnited StatesHepatorenal syndromeOrphan Drug designation disclosedUnited States

BLRX

Biolinerx

biolinerx.com

2assets

GLIX1GLIX1 / DNA damage response; restores TET2 activity / oral small molecule / Oncology
GlioblastomaPhase 1first patients expected Q1 2026Other cancers / solid tumorsPreclinicaladditional indications to be explored in preclinical models
MotixafortideAPHEXDA / CXCR4 / CXCR4 inhibitor / Oncology; hematology / stem cell mobilization; rare disease
First-line metastatic pancreatic cancerPhase 2randomized Phase 2 with Columbia University; GloriaBio plans large randomized Phase 2 in AsiaAsiaStem cell mobilization for autologous transplantation in multiple myelomaApproved / Bridging Studyapproved in US; China bridging study planned/led by Gloria BiosciencesUSChinaAsiaSickle cell disease - CD34+ HSC mobilization for gene therapyPhase 2 / Phase 1investigator-initiated study; Gamida Cell leading Phase 1 program under Ayrmid agreement

BLTE

Belite Bio

belitebio.com

2assets

TinlarebantLBS-008 / RBP4 / Oral small-molecule RBP4 antagonist / Ophthalmology / retinal degenerative diseases
Stargardt disease type 1 (STGD1)Phase 3; Phase 2/3Ongoing clinical evaluation; rolling NDA submission initiated in U.S.U.S.EuropeJapanGeographic atrophy (GA) in advanced dry AMDPhase 3Ongoing clinical evaluation
LBS-009LBS-009 / RBP4 / Oral small-molecule RBP4 antagonist / Metabolic disease / liver disease
Non-alcoholic fatty liver disease (NAFLD)PreclinicalPreclinical developmentNon-alcoholic steatohepatitis (NASH)PreclinicalPreclinical developmentType 2 diabetes (T2D)PreclinicalPreclinical development

BMEA

Biomeafusion

biomeafusion.com

2assets

IcovamenibBMF-219 / menin / oral covalent small molecule menin inhibitor / Metabolic disease
Type 1 diabetes mellitusPhase IICOVALENT-112; study ongoing / 52-week follow-up completed with data expected Q2 2026Insulin-deficient type 2 diabetesPhase IICOVALENT-211; enrolling / first patient dosedType 2 diabetes uncontrolled with GLP-1 RA-based therapyPhase IICOVALENT-212; enrolling / first patient dosed
BMF-650BMF-650 / GLP-1 receptor / oral small-molecule GLP-1 receptor agonist / Metabolic disease
ObesityPhase IGLP-131; clinical study enrolling / first patient dosed

CADL

Candeltx

candeltx.com

2assets

CAN-2409CAN-2409 / Adenovirus platform viral immunotherapy / Oncology
Borderline resectable pancreatic adenocarcinomaPhase IIFast Track Designation (FDA); enrollment paused subject to additional fundingUSNSCLC + PD-1/PD-(L)1Phase IIFast Track Designation (FDA)USLocalized intermediate/high-risk prostate cancerBLA ReadinessFast Track Designation (FDA); Special Protocol Assessment (FDA)US
CAN-3110CAN-3110 / HSV platform viral immunotherapy / Oncology
Recurrent high-grade glioma / recurrent GBMPhase IFast Track Designation (FDA)US

CLDX

Celldex

celldex.com

2assets

BarzolvolimabCDX-0159 / KIT / KIT antagonist humanized monoclonal antibody / Allergic, inflammatory and autoimmune diseases
Chronic spontaneous urticaria (CSU)Phase 3ongoingCold urticaria (ColdU)Phase 3ongoingSymptomatic dermographism (SD)Phase 3ongoing
CDX-622CDX-622 / TSLP and SCF / Bispecific antibody / Inflammatory and fibrotic disorders
Healthy volunteer studyPhase 1ongoingAsthma proof-of-mechanism studyPhase 1planned/ongoing

CMPS

Compasspathways

compasspathways.com

2assets

COMP360 psilocybin treatmentCOMP360 / synthetic psilocybin formulation / Mental health / psychiatry
Treatment-resistant depression (TRD)Phase 3Post-traumatic stress disorder (PTSD)Phase 2Anorexia nervosaPhase 2
Prodrug programmeProdrug

CRBP

Corbus Pharma

corbuspharma.com

2assets

CRB-701CRB-701 / Nectin-4 / Antibody-drug conjugate (ADC) / Oncology
Nectin-4 expressing solid tumorsPhase 1In progress; dose optimization in HNSCC, cervical & mUC; FDA Fast Track Designation granted for HNSCC and cervical cancerUSEuropeCervical cancerPhase 3PendingChina
CRB-913CRB-913 / CB1 receptor / Highly peripherally restricted CB1 inverse agonist / Obesity / metabolic disease
Obesity and related conditionsPhase 1In progress; 12-week dose-range study in obesity planned mid-2026

CRBU

Caribou Bio

cariboubio.com

2assets

Vispacabtagene regedleucelCB-010 / vispa-cel / CD19 / Allogeneic anti-CD19 CAR-T cell therapy; PD-1 knockout / Hematologic malignancies
Relapsed or refractory B cell non-Hodgkin lymphomaPhase 1Ongoing
CB-011CB-011 / BCMA / Allogeneic anti-BCMA CAR-T cell therapy; B2M knockout and B2M–HLA-E insertion / Hematologic malignancies
Relapsed or refractory multiple myelomaPhase 1Ongoing

DBVT

Dbv Technologies

dbv-technologies.com

2assets

VIASKIN® Peanut PatchDBV712 / Peanut allergen extract / Epicutaneous immunotherapy patch; drug-device combination / Food allergy
Peanut allergy; children 4–7 years oldPhase 3 completed; BLA submission anticipated 1H 2026Primary endpoint met in VITESSE Phase 3USPeanut allergy; toddlers 1–3 years oldPhase 3 / BLA submission anticipated 2H 2026Accelerated Approval pathway; COMFORT Toddlers safety trial ongoingUSPeanut allergy; children 1–7 years oldMAA planningEMA guidance received for circular patchEU
VIASKIN® Milk PatchDBV135 / Milk allergen / Epicutaneous immunotherapy patch; drug-device combination / Food allergy / immunology
Cow’s milk allergy; ages 2–17 yearsPhase 2MILES study referencedMilk-induced eosinophilic esophagitis; ages 4–17 yearsPhase 2SMILEE study referenced

DFTX

Definiumtx

definiumtx.com

2assets

DT120 ODTDT120 / serotonin receptors / Orally disintegrating tablet formulation of lysergide (LSD) D-tartrate / Psychiatry / brain health
Generalized anxiety disorderPhase 3Two Phase 3 registrational trials ongoing/evaluating efficacy and safetyU.S.EuropeMajor depressive disorderPhase 3Emerge Phase 3 ongoing; Ascend Phase 3 expected to initiate mid-2026U.S.
DT402DT402 / serotonergic system / R-enantiomer of MDMA (R(-)-MDMA) / Neurodevelopmental disorders
Autism spectrum disorderPhase 2aPhase 2a study initiated after completion of Phase 1 single-ascending dose study

DMAC

Diamedica

diamedica.com

2assets

DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / Neurological
Acute Ischemic StrokePhase 2/3ReMEDy2 Study; in progress
DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / Pregnancy Complications
PreeclampsiaPhase 2Part 1A Dose-Selection / Part 1B Expansion Cohort; in progressPreeclampsiaPhase 2Parts 2 & 3 Expected Management Study; in progressFetal Growth RestrictionPhase 2Part 3 Fetal Growth Restriction; in progress

DNTH

Dianthustx

dianthustx.com

2assets

ClaseprubartDNTH103 / active C1s / long-acting monoclonal antibody / autoimmune / neuromuscular disorders
Generalized Myasthenia Gravis (gMG)Phase IIPhase 2 data reported; Phase 3 trial planned mid-2026Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase IIICAPTIVATE; interim responder analysis completed and GO decision announced Q1 2026Multifocal Motor Neuropathy (MMN)Phase IITop-line Phase 2 data expected 2H 2026
DNTH212DNTH212 / LBL-047 / BDCA2 and BAFF/APRIL / extended half-life bifunctional fusion protein / autoimmune disorders
Systemic lupus erythematosus (SLE)Phase ITwo-part Phase 1 study in China ongoing; healthy-volunteer results expected 2H 2026China

DTIL

Precision Biosciences

precisionbiosciences.com

2assets

PBGENE-HBVPBGENE-HBV / HBV cccDNA and integrated HBV DNA / in vivo ARCUS gene editing therapy / Infectious disease
Chronic hepatitis BPhase 1/2aELIMINATE-B trial ongoingGlobalUnited States
PBGENE-DMDPBGENE-DMD / Dystrophin gene exons 45-55 region / in vivo ARCUS gene editing therapy / gene excision / Neuromuscular disease
Duchenne muscular dystrophyPhase 1/2FUNCTION-DMD study; first clinical trial site activated and enrollment begun per company IR overviewUnited States

EBS

Emergentbiosolutions

emergentbiosolutions.com

2assets

CDC Category A AgentCDC Category A agent / Medical countermeasures
Undisclosed Disease Area
CDC Category B AgentCDC Category B agent / Medical countermeasures
Undisclosed Disease Area

ELVN

Enliven Therapeutics

enliventherapeutics.com

2assets

ELVN-001BCR-ABL Program
ELVN-002HER2 Program

EPRX

Eupraxia Pharma

eupraxiapharma.com

2assets

EP-104GIEP-104GI / long-acting controlled-release fluticasone propionate formulation; intra-esophageal injection / Gastrointestinal / inflammatory disease
Eosinophilic esophagitis (EoE)Phase 1b/2Ongoing
EP-104IAREP-104IAR / long-acting controlled-release fluticasone propionate formulation; intra-articular injection / Pain / osteoarthritis
Pain due to knee osteoarthritisPhase 2Completed

EVMN

Evommune

evommune.com

2assets

EVO756EVO756 / MRGPRX2 / Oral small-molecule antagonist / Chronic inflammatory diseases
Chronic Spontaneous UrticariaPhase 2Atopic DermatitisPhase 2MigrainePhase 1
EVO301EVO301 / IL-18 / Long-acting injectable SAFA-IL-18BP fusion protein / Chronic inflammatory diseases
Atopic DermatitisPhase 2Ulcerative ColitisPhase 1

EYPT

Eyepoint

eyepoint.bio

2assets

DURAVYUEYP-1901 / DURAVYU / VEGF receptors; JAK1; PDGFR / vorolanib intravitreal insert; tyrosine kinase inhibitor in Durasert E bioerodible sustained-release insert / Retinal diseases
Wet age-related macular degenerationPhase 3Trial underway; LUGANO and LUCIA fully enrolled; LUGANO topline data expected mid-2026, LUCIA to closely followGlobalDiabetic macular edemaPhase 3COMO and CAPRI pivotal trials underway; enrollment completion expected 3Q 2026Global
Undisclosed candidatesDurasert E programs / Retinal diseases
Retinal diseasesPre-clinicalIn progress

FLGT

Fulgentgenetics

ir.fulgentgenetics.com

2assets

FID-007FID-007 / Cytotoxic / Nanoencapsulated paclitaxel / Oncology
Head and Neck (H&N) cancer, 2nd linePhase 2Ongoing; interim findings expected June 2026Ampullary or ICI resistant cancerGo/no-go based on H&N study
FID-022FID-022 / Cytotoxic / Oncology
Colon cancerPhase 1Ongoing; dosing for 3rd dose level ongoingPancreatic cancerPhase 1Ongoing; dosing for 3rd dose level ongoingOvarian cancerPhase 1Ongoing; dosing for 3rd dose level ongoing

GUTS

Fractyl

ir.fractyl.com

2assets

RevitaDuodenal mucosa / Endoscopic hydrothermal ablation / Obesity
Weight maintenance post-GLP-1 in obesityPivotalREMAIN-1 randomization completed; 6-mo topline expected early Q4 2026US
RejuvaRJVA-001 / Pancreatic beta cells / AAV9 gene therapy / Type 2 Diabetes
Type 2 diabetesPhase 1/2CTA submitted; FIH dosing & preliminary data expected H2 2026 pending approvalEUAustralia

IMVT

Immunovant

immunovant.com

2assets

BatoclimabIMVT-1401
IMVT-1402

KALV

Kalvista

kalvista.com

2assets

sebetralstatEKTERLY
Oral Factor XIIa inhibitor

KYNB

Kyntra Bio

kyntrabio.com

2assets

FG-3246FOR46 / CD46 / Antibody-Drug Conjugate (ADC) / Oncology
Metastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 2OngoingUnited States
roxadustatFG-4592 / HIF-PH / Small Molecule / Rare Disease
Anemia associated with lower-risk myelodysplastic syndromes (LR-MDS)Phase 3PlannedUnited States

LQDA

Liquidia

liquidia.com

2assets

YUTREPIAtreprostinil inhalation powder
L606liposomal treprostinil inhalation suspension

LYEL

Lyell

lyell.com

2assets

rondecabtagene autoleucelLYL314 / Ronde-cel / CD19 / CD20 / CAR T-cell therapy / Oncology
Relapsed/Refractory Large B-cell Lymphoma (LBCL) - 2LPhase 3ActiveUSRelapsed/Refractory Large B-cell Lymphoma (LBCL) - 3L+PivotalActiveUS
LYL273LYL273 / GCC (Guanylyl Cyclase-C) / CD19 / CAR T-cell therapy / Oncology
Refractory Metastatic Colorectal Cancer (mCRC)Phase 1ActiveUS

MAZE

Mazetx

mazetx.com

2assets

MRKR

Marker Therapeutics

markertherapeutics.com

2assets

MT-601neldaleucel / Survivin, PRAME, NY-ESO-1, MAGE-A4, SSX2, WT-1 / T Cell Therapy (MAR-T cell) / Oncology
LymphomaPancreatic Cancer
MT-401-OTSzelenoleucel / Multi-Tumor Associated Antigens / Allogeneic T Cell Therapy / Oncology
Acute Myeloid Leukemia (AML)Myelodysplastic Syndrome (MDS)

NAMS

Newamsterdam Pharma

newamsterdampharma.com

2assets

ObicetrapibAMG-899 / CETP / Small Molecule / Cardiometabolic
Primary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEuropeAtherosclerotic Cardiovascular Disease (ASCVD)Phase 3OngoingGlobal
Obicetrapib / Ezetimibe FDCCETP / NPC1L1 / Small Molecule / Cardiometabolic
Primary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEurope

NCNA

Nucana

nucana.com

2assets

NUC-3373NUC-3373 / Thymidylate Synthase (TS) / Nucleoside analog (ProTide) / Oncology
Advanced solid tumorsColorectal cancerLung cancer
NUC-7738NUC-7738 / RNA polyadenylation / Nucleoside analog (ProTide) / Oncology
Advanced solid tumorsMelanoma

NGNE

Neurogene

neurogene.com

2assets

NRXP

Nrx Pharma

nrxpharma.com

2assets

NRX-101NMDA and 5-HT2A receptors / Small molecule / Central Nervous System
Bipolar Depression with Acute Suicidal Ideation and Behavior (ASIB)Phase 3ActiveUnited StatesBipolar Depression with Subacute Suicidal Ideation and Behavior (SSIB)Phase 2/Phase 3ActiveUnited StatesDepression and Suicidality (in association with Transcranial Magnetic Stimulation)Phase 3ActiveUnited States
NRX-100KETAFREE / NMDA receptor / Small molecule / Central Nervous System
Acute Suicidal Depression / Suicidal Ideation in DepressionRegistrationActiveUnited States

NXTC

Nextcure

nextcure.com

2assets

SIM0505SIM0505 / CDH6 / Antibody-drug conjugate (ADC) / Oncology
Solid tumorsOvarian cancerLung cancer
LNCB74LNCB74 / B7-H4 / Antibody-drug conjugate (ADC) / Oncology
Solid tumorsBreast cancerOvarian cancer

OKUR

Onkure Therapeutics

onkuretherapeutics.com

2assets

OLMA

Olema

olema.com

2assets

PalazestrantOP-1250
OP-3136

ORIC

Oric Pharma

oricpharma.com

2assets

RinzimetostatORIC-944 / PRC2 (EED subunit) / Small molecule / Oncology
Metastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 1bActiveGlobal
EnozertinibORIC-114 / EGFR / HER2 (exon 20 and PACC/atypical mutations) / Small molecule / Oncology
Non-Small Cell Lung Cancer (NSCLC)Phase 1bActiveUnited StatesAustraliaCanadaHong KongMalaysiaPolandSouth KoreaSpainTaiwanUnited Kingdom

ORKA

Orukatx

orukatx.com

2assets

ORKA-001ORKA-001 / IL-23p19 / Monoclonal Antibody / Dermatology / Immunology
Plaque PsoriasisPhase 2ActiveUnited States
ORKA-002ORKA-002 / IL-17A/F / Monoclonal Antibody / Dermatology / Immunology
Plaque PsoriasisPhase 1ActiveUnited StatesHidradenitis SuppurativaPhase 1ActiveUnited States

PBYI

Pumabiotechnology

pumabiotechnology.com

2assets

NeratinibNERLYNX
Alisertib

PSTV

Plus Therapeutics

plustherapeutics.com

2assets

rhenium Re186 obisbemeda186RNL / REYOBIQ / Radiotherapeutic / Oncology
Recurrent GlioblastomaLeptomeningeal MetastasesPediatric Brain Cancer
Rhenium-188 NanoLiposome Biodegradable Alginate Microsphere188RNL-BAM / Radiotherapeutic / Oncology
Hepatocellular CarcinomaMetastatic Colorectal Cancer

PYXS

Pyxisoncology

pyxisoncology.com

2assets

Micvotabart pelidotinMICVO, PYX-201 / EDB+FN / Antibody-Drug Conjugate (ADC) / Oncology
Solid TumorsRecurrent and Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)
PYX-106PYX-106 / Siglec-15 / Monoclonal Antibody (mAb) / Oncology
Solid Tumors

QNTM

Quantum Biopharma

quantumbiopharma.com

2assets

Lucid-MSLucid-21-302 / Demyelination / Small Molecule (NCE) / Neurology
Multiple Sclerosis
FSD202FSD-202 / Mast cell activation / Small Molecule / Immunology / Pain
Idiopathic Mast Cell Activation Syndrome (MCAS)

QTTB

Q32 Bio

q32bio.com

2assets

BempikibartADX-914
ADX-096

REPL

Replimune

replimune.com

2assets

vusolimogene oderparepvecRP1 / HSV-1 oncolytic virus encoding GALV-GP R-protein and GM-CSF / Oncolytic Virus / Oncology
Advanced MelanomaPhase 3Complete Response LetterUSNon-melanoma Skin Cancers (CSCC, MCC, BCC, Angiosarcoma)Phase 2ActiveUS
RP2RP2 / Oncolytic virus / Oncolytic Virus / Oncology
Metastatic Uveal MelanomaPhase 2ActiveUSHepatocellular CarcinomaPhase 2ActiveUS

RLMD

Relmada

relmada.com

2assets

RNXT

Renovorx

renovorx.com

2assets

RZLT

Rezolute Bio

rezolutebio.com

2assets

ErsodetugRZ358 / Insulin receptor / Monoclonal antibody / Endocrinology/Rare Diseases
Congenital hyperinsulinismTumor hyperinsulinism
RZ402RZ402 / Plasma kallikrein / Small molecule / Ophthalmology
Diabetic macular edema (DME)Diabetic retinopathy (DR)

SANA

Sana

sana.com

2assets

SC451SC451 / Pancreatic islet cell replacement / HIP-modified stem-cell-derived pancreatic islet cell therapy / Endocrinology / Autoimmune
Type 1 diabetes
SG293SG293 / B-cells / In vivo CAR-T therapy / Oncology / Autoimmune
B-cell cancersB-cell mediated autoimmune diseases

SGP

Spyglass Pharma

spyglasspharma.com

2assets

BIM-IOL SystemBimatoprost Drug Pad-IOL System
BIM-DRSBimatoprost Drug Ring System

SLS

Sellaslife Sciences

sellaslifesciences.com

2assets

Galinpepimut-SGPS
SLS009tambiciclib

SNDX

Syndax

syndax.com

2assets

RevumenibSNDX-5613 / Revuforj / Menin / Small Molecule / Oncology
R/R acute leukemia with KMT2A translocation (AUGMENT-101-2A)ApprovedApprovedUSR/R NPM1m AML (AUGMENT-101-2C)ApprovedApprovedUSFrontline unfit NPM1m and KMT2Ar acute leukemias (EVOLVE-2)Phase 3ActiveUS
AxatilimabSNDX-6352 / Niktimvo / CSF-1R / Monoclonal Antibody / Oncology / Immunology
Refractory Chronic GVHD (AGAVE-201)ApprovedApprovedUSFrontline Chronic GVHD (corticosteroids combination)Phase 3ActiveUSFrontline Chronic GVHD (ruxolitinib combination)Phase 2ActiveUS

SPRB

Spruce Biosciences

sprucebio.com

2assets

Tralesinidase AlfaTA-ERT / Alpha-N-acetylglucosaminidase (NAGLU) / Enzyme Replacement Therapy / Neurology / Rare Disease
Mucopolysaccharidosis Type IIIB (MPS IIIB) / Sanfilippo Syndrome Type BPhase 3ActiveGlobal
TildacerfontCorticotropin-releasing factor type-1 (CRF1) receptor / Small Molecule / Neurology / Psychiatry
Major Depressive Disorder (MDD)Phase 2ActiveEurope

STOK

Stoke Therapeutics

stoketherapeutics.com

2assets

zorevunersenSTK-001
STK-002

STTK

Shattuck Labs

shattucklabs.com

2assets

SL-325SL-325 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated Diseases
Inflammatory Bowel Disease (IBD)Crohn's DiseaseUlcerative Colitis
SL-425SL-425 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated Diseases
Inflammatory Bowel Disease (IBD)

TARA

Protaratx

protaratx.com

2assets

TARA-002TARA-002 / Broad immunopotentiator / Cell therapy / Oncology / Rare Diseases
Non-Muscle Invasive Bladder Cancer (NMIBC)Phase 2ActiveUnited StatesLymphatic Malformations (LMs)Phase 2ActiveUnited States
IV Choline ChloridePhospholipid substrate replacement / Small molecule / Intravenous replacement therapy / Rare Diseases
Patients dependent on long-term parenteral support (PS) / Choline deficiencyPhase 3ActiveUnited States

TBPH

Theravance

theravance.com

2assets

Yupelrirevefenacin
Trelegy (economic interest)

TENX

Tenaxthera

tenaxthera.com

2assets

TNX-103oral levosimendan
TNX-201imatinib

UNCY

Unicycive

unicycive.com

2assets

Oxylanthanum CarbonateOLC / Renazorb / Phosphate / Small molecule / Nephrology
Hyperphosphatemia in chronic kidney disease (CKD) on dialysisNDAResubmittedUnited States
UNI-494UNI-494 / KATP channel / Nitric oxide (nicorandil prodrug) / Small molecule / Nephrology
Acute Kidney Injury (AKI)Phase 1CompletedUnited KingdomUnited StatesPrevention of Delayed Graft Function (DGF) in kidney transplant patientsPhase 1ActiveUnited States

VERU

Veru Pharma

verupharma.com

2assets

EnobosarmVERU-024 / GTx-024 / Ostarine
SabizabulinVERU-111

VRCA

Verrica

verrica.com

2assets

cantharidinVP-102 / YCANTH / Unknown / Small Molecule / Dermatology
Molluscum ContagiosumApprovedActiveCommon WartsPhase 3ActiveExternal Genital WartsPhase 2Active
VP-315VP-315 / Oncolytic peptide / Peptide / Dermatologic Oncology
Basal Cell CarcinomaPhase 2ActiveSquamous Cell CarcinomaPhase 1/2Active

ZVSA

Zyversa

zyversa.com

2assets

VAR 200Cholesterol Efflux Pathway (ABCA1 and ABCG1) / Cholesterol Efflux Mediator / Renal Diseases
Focal Segmental Glomerulosclerosis (FSGS)Phase 2activeUnited StatesDiabetic Kidney Disease (DKD)Phase 2activeUnited StatesAlport SyndromePreclinicalactiveUnited States
IC 100Inflammasome ASC / Monoclonal Antibody / Inflammatory Diseases
Cardiometabolic Conditions (Obesity-associated)PreclinicalactiveUnited StatesParkinson's DiseasePreclinicalactiveUnited StatesOrphan Renal DiseasesPreclinicalactiveUnited States

ABVX

Abivax

abivax.com

1assets

ObefazimodABX464 / miR-124 enhancer / oral small molecule / Inflammatory diseases
Moderately to severely active ulcerative colitisPhase 3ABTECT induction data readout reported; maintenance data readout planned Q2 2026Crohn’s diseasePhase 2bENHANCE-CD initiated; 12-week induction data readout planned Q4 2026Moderately to severely active ulcerative colitis, combination therapyPreclinicalevaluation with other agents ongoing

ACHV

Achieve Life Sciences

achievelifesciences.com

1assets

Cytisiniclinenicotinic acetylcholine receptor / plant-based alkaloid; small molecule / Nicotine dependence
Smoking cessation in adultsNDA reviewFDA accepted NDA; PDUFA June 20, 2026; CRL expected due to third-party manufacturing OAI; resubmission planned Q4 2026USE-cigarette/vaping cessationPhase 3 plannedPhase 2 completed; successful end-of-Phase 2 meeting; Breakthrough Therapy designation; National Priority Voucher awardedUS

ACTU

Actuate Therapeutics

actuatetherapeutics.com

1assets

elraglusibGSK-3β / small-molecule kinase inhibitor / Oncology
1L metastatic pancreatic cancer with GnPPhase 3 in planningupdated Phase 2 data presented at ASCO GI; FDA/EMA meetings planned 1H 20261L metastatic pancreatic cancer with FOLFIRINOXPhase 2study closed to enrollment; data readout mid-2026Advanced pancreatic cancer with mFOLFIRINOX + retifanlimabPhase 2enrollment open

ADCT

Adc Therapeutics

adctherapeutics.com

1assets

Loncastuximab tesirine-lpylZYNLONTA / CD19 / antibody-drug conjugate / Oncology
3L+ relapsed/refractory DLBCL (LOTIS-2)Phase 3/Confirmatoryrelapsed/refractory DLBCL (LOTIS-5, with rituximab)Phase 3/Confirmatoryrelapsed/refractory B-NHL (LOTIS-7, with glofitamab)Phase 1b

AEON

Aeon Biopharma

aeonbiopharma.com

1assets

ABP-450 (prabotulinumtoxinA)ABP-450 / SNAP-25 / acetylcholine release / 900 kDa botulinum toxin type A complex / Neurology / neuromuscular disorders
Episodic migrainePhase 2Enrollment completed; P2 data anticipatedChronic migrainePhase 2P2 data anticipatedCervical dystoniaPhase 2P2 data reported; P3 initiation anticipated

AIM

Aimimmuno

aimimmuno.com

1assets

Ampligenrintatolimod; poly(I):poly(C12U) / Ampligen / TLR3 / dsRNA immunomodulator/TLR3 agonist / Oncology; immune disorders; viral diseases
Late-stage/metastatic pancreatic cancerPhase 2; Phase 3 plannedongoing/planningNetherlandsLocally advanced pancreatic adenocarcinomaPhase 2suspendedUSEuropeAdvanced recurrent ovarian cancerPhase 2completedUS

ALT

Altimmune

altimmune.com

1assets

pemvidutideALT-801 / glucagon/GLP-1 receptors / peptide dual receptor agonist / serious liver diseases
metabolic dysfunction-associated steatohepatitis (MASH)Phase 3 plannedBreakthrough Therapy Designation; Phase 3 initiation planned in 2026Globalalcohol use disorder (AUD)Phase 2RECLAIM ongoing; topline data expected Q3 2026alcohol-associated liver disease (ALD)Phase 2RESTORE ongoing/enrolling

ATOS

Atossa Therapeutics

atossatherapeutics.com

1assets

(Z)-endoxifenEstrogen receptor; PKCβ1 / Oral small molecule SERM/D / Oncology; rare disease
Metastatic breast cancerearly-stage trialsFDA pathway discussionsUSAdjuvant breast cancerFDA pathway discussionsUSNeoadjuvant breast cancerPhase 2ongoing EVANGELINE; I-SPY2US

AURA

Aura Biosciences

aurabiosciences.com

1assets

belzupacap sarotalocanAU-011 / bel-sar / tumor-associated GAGs/HSPGs / virus-like drug conjugate (VDC) / oncology
early choroidal melanoma (small choroidal melanoma and/or indeterminate lesions)Phase 3ongoingGlobalmetastases to the choroidPhase 2ongoingcancers of the ocular surfacePhase 1initiating

AVTX

Avalotx

avalotx.com

1assets

abdakibartAVTX-009 / IL-1β / humanized IgG4 monoclonal antibody / immune-mediated inflammatory diseases
hidradenitis suppurativaPhase 2LOTUS trial; topline data expected Q2 2026global

BCAX

Bicara

ir.bicara.com

1assets

ficerafusp alfaBCA101 / EGFR/TGF-β / bifunctional antibody; EGFR-directed mAb with TGF-β ligand trap / Oncology; solid tumors
1L HPV-negative recurrent/metastatic HNSCC; with pembrolizumabPhase 2/3ongoing; FORTIFI-HN01 pivotal studyGlobal3L+ MSS RAS wild-type metastatic colorectal cancer; monotherapy and with pembrolizumabPhase 1/1benrolling expansion cohort

BFRI

Biofrontera Us

biofrontera-us.com

1assets

BF-200 ALABF-200 ALA / Ameluz® / Topical aminolevulinic acid gel with photodynamic therapy / Dermatology
Actinic keratosis: pharmacokinetics studyCompletedActinic keratosis: safety study for use of 3 tubesCompletedSuperficial basal cell carcinomaLast patient in

BNTC

Benitec

benitec.com

1assets

BB-301BB-301 / PABPN1 / ddRNAi gene therapy; intramuscular; modified AAV9 silence-and-replace construct / Neuromuscular disease
Oculopharyngeal Muscular Dystrophy (OPMD)Phase 1b/2aClinical-stageGlobal rights

CABA

Cabaletta Bio

cabalettabio.com

1assets

rese-celCABA-201 / CD19 / Autologous fully human 4-1BB CD19-CAR T cell therapy / Autoimmune disease
Myositis / idiopathic inflammatory myopathyRegistrational; Phase 1/2Recruiting / activeUSDermatomyositisRegistrational; Phase 1/2ActiveUSAntisynthetase syndromeRegistrational; Phase 1/2ActiveUS

CELC

Celcuity

celcuity.com

1assets

gedatolisibPI3K/AKT/mTOR (PAM) pathway; pan-class I PI3K and mTORC1/2 / Small molecule inhibitor / Oncology
HR+/HER2- advanced or metastatic breast cancer, 2L, with palbociclib (Ibrance) + fulvestrantPhase 3VIKTORIA-1; FDA Breakthrough Therapy DesignationHR+/HER2- advanced or metastatic breast cancer, 2L, with fulvestrantPhase 3VIKTORIA-1HR+/HER2- advanced or metastatic breast cancer, 1L, with palbociclib (Ibrance) or ribociclib (Kisqali) + fulvestrantPhase 3VIKTORIA-2; enrolling

CNTB

Connectbiopharm

connectbiopharma.com

1assets

RademikibartCBP-201 / IL-4Rα / Fully human monoclonal antibody / Inflammatory diseases
Acute exacerbations in asthmaPhase 2Recruitment ongoing; topline data expected mid-2026GlobalAcute exacerbations in COPDPhase 2Recruitment ongoing; topline data expected mid-2026GlobalModerate-to-severe atopic dermatitisPhase 3 / NDA submittedPhase 3 data reported by Simcere; China NDA submitted July 2025Greater China

CRDF

Cardiffoncology

cardiffoncology.com

1assets

onvansertibPLK1 / oral small-molecule inhibitor / Oncology
1L RAS-mutated metastatic colorectal cancerPhase 2CRDF-004 completed enrollment; planned registrational developmentMetastatic pancreatic ductal adenocarcinomaPhase 2investigator-initiated / ongoing or plannedRelapsed small cell lung cancerPhase 2investigator-initiated / ongoing

EDIT

Editasmedicine

editasmedicine.com

1assets

EDIT-401EDIT-401 / LDLR / in vivo CRISPR gene editing medicine / Cardiovascular diseases
Hyperlipidemia / LDL-C reductionPreclinicalIND/CTA submission planned by mid-2026; human proof-of-concept targeted by end of 2026Atherosclerotic cardiovascular disease (ASCVD)PreclinicalIn developmentHeterozygous familial hypercholesterolemia (HeFH)PreclinicalFirst-in-human trial planned in 2026

FBRX

Fortebiorx

fortebiorx.com

1assets

FB102FB102 / CD122 / monoclonal antibody / Autoimmune and autoimmune-related diseases
Celiac diseasePhase 2Topline results expected in 2026; U.S. arm included after FDA IND approvalUnited StatesVitiligoPhase 1bTopline results expected in 1H 2026Alopecia areataPhase 1bData readout expected in 2026

FENC

Fennec Pharma

investors.fennecpharma.com

1assets

PEDMARKPEDMARK / PEDMARQSI / cisplatin-induced ototoxicity / small molecule; sodium thiosulfate injection / oncology supportive care
Reduce risk of ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumorsApprovedMarketed; ongoing evidence generationUSEUUKCisplatin-induced ototoxicity in pediatric and AYA patients with non-metastatic solid tumorsPhase 2/3Positive topline results; pursuing registrationJapanOtoprotection in adults receiving cisplatin chemotherapy, including head and neck and testicular cancersPhase I/IIInvestigator-sponsored study initiatedUS

FULC

Fulcrumtx

fulcrumtx.com

1assets

pociredirFTX-6058 / EED / PRC2; HbF induction / oral small molecule / Hematology
Sickle cell diseasePhase 1bactive; potential registration-enabling trial planned for 2H 2026United States

GERN

Geron

geron.com

1assets

imetelstatRYTELO / telomerase / telomerase inhibitor / Hematologic malignancies
JAK inhibitor relapsed/refractory myelofibrosisPhase 3open for screening and enrollmentfrontline myelofibrosis, intermediate-1/intermediate-2/high-risk; combination with ruxolitinibPhase 1open for screening and enrollmentrelapsed/refractory/intolerant acute myeloid leukemia or high-risk MDS after hypomethylating agentsPhase 2investigator-led; open for screening and enrollment

GLSI

Greenwichlife Sciences

greenwichlifesciences.com

1assets

GLSI-100GP2 + GM-CSF / HER2/neu / peptide immunotherapy vaccine / Oncology
Prevention of recurrence in HER2/neu-positive breast cancer after surgery and trastuzumab-based therapyPhase 3Ongoing; Fast Track designated; FLAMINGO-01United StatesEurope

GNLX

Genelux

genelux.com

1assets

Olvi-Vecolvimulogene nanivacirepvec / Oncolytic vaccinia virus immunotherapy / Oncology
Platinum-resistant/refractory ovarian cancer (PRROC)Phase 3Enrolling; topline data expected 2H 2026 (OnPrime/GOG-3076; NCT05281471)USNon-small cell lung cancer (NSCLC), advanced/metastatic recurrentPhase 2Dose-escalation ongoing (VIRO-25; NCT06463665); systemic IV Olvi-Vec + platinum-doublet + ICI vs docetaxelUSSmall cell lung cancer (SCLC), platinum-relapsed/refractoryPhase 1b/2Dose selection ongoing (OLVI-VEC-SCLC-202; NCT07136285); Olvi-Vec + platinum + etoposideChinaUS

IMMX

Immix Bio

immixbio.com

1assets

NXC-201NXC-201 / BCMA / sterically-optimized autologous CAR-T / Hematology
Relapsed/refractory AL AmyloidosisPhase 2BLA-enabling; enrollment completedUSIsraelSelect immune-mediated diseasesExpanding

KLRS

Kalaristx

kalaristx.com

1assets

TH103TH103 / VEGF / HSPG / Recombinant fusion protein / Retinal Diseases
Neovascular age-related macular degeneration (nAMD)Phase 1b/2OngoingDiabetic eye diseasePlannedPlannedRetinal vein occlusionPlannedPlanned

LBRX

Lb Pharma

lbpharma.us

1assets

LB-102

LGVN

Longeveron

longeveron.com

1assets

Lomecel-Blaromestrocel

LIXT

Lixte

lixte.com

1assets

LB-100LB-100 / Protein Phosphatase 2A (PP2A) / Small Molecule / Oncology
Ovarian Clear Cell CarcinomaPhase 2ActiveUnited StatesMetastatic Colon CancerPhase 1bActiveGlobalAdvanced Soft Tissue SarcomaPhase 1/2PausedEurope

LRMR

Larimartx

larimartx.com

1assets

nomlabofuspCTI-1601

MLTX

Moonlaketx

moonlaketx.com

1assets

SonelokimabM1095 / IL-17A and IL-17F (tri-specific Nanobody) / Nanobody (VHH) / Immunology (Dermatology and Rheumatology)
Hidradenitis Suppurativa (HS)Phase 3OngoingGlobalAdolescent Hidradenitis Suppurativa (Teen HS)Phase 3OngoingGlobalPsoriatic Arthritis (PsA)Phase 3OngoingGlobal

MLYS

Mineralystx

mineralystx.com

1assets

lorundrostatMLS-101 / Aldosterone synthase (CYP11B2) / Small molecule / Cardio-Renal-Metabolic Syndrome
Uncontrolled and treatment-resistant hypertensionPhase 3CompletedGlobalObstructive Sleep Apnea (OSA) and HypertensionPhase 2OngoingGlobalChronic Kidney Disease (CKD)Phase 2OngoingGlobal

NBTX

Nanobiotix

nanobiotix.com

1assets

NBTXR3JNJ-1900 / Hensify / Tumor cell (Radioenhancer) / Functionalized hafnium oxide (HfO2) nanoparticles / Oncology
Locally Advanced Head and Neck Squamous Cell Carcinoma (LA-HNSCC) - Platinum-ineligiblePhase 3OngoingGlobalRecurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)Phase 1/2OngoingGlobalSoft Tissue SarcomaApprovedMarketedEurope

NKTX

Nkartatx

nkartatx.com

1assets

NKX019NKX019 / CD19 / Allogeneic CAR-NK Cell Therapy / Autoimmune Diseases
Lupus NephritisSystemic SclerosisInflammatory Myopathy

NVCR

Novocure

novocure.com

1assets

Tumor Treating FieldsTTFields / Optune Gio, Optune Lua, Optune Pax, Optune Mya / Mitotic spindle / Electric Field Therapy / Oncology
Glioblastoma (GBM)ApprovedMarketedUnited StatesEuropeJapanGlioblastoma (GBM) - Earlier use (TRIDENT)Phase 3ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)ApprovedMarketedUnited StatesJapanEurope

PALI

Palisade Bio

palisadebio.com

1assets

PALI-2108PALI-2108 / PDE4 / Small Molecule Prodrug / Gastroenterology
Ulcerative Colitis (UC)Phase 2ActiveGlobalCrohn's Disease (CD)Phase 2ActiveGlobal

PHVS

Pharvaris

pharvaris.com

1assets

DeucrictibantPHA121

PMVP

Pmv Pharma

pmvpharma.com

1assets

RezatapoptPC14586

PROK

Prokidney

prokidney.com

1assets

RilparencelREACT

SKYE

Skyebioscience

skyebioscience.com

1assets

NimacimabCB1 / Monoclonal antibody / Metabolic Diseases
ObesityPhase 2activeUnited States

SLNO

Soleno

soleno.life

1assets

Diazoxide Choline Extended-Release (DCCR)VYKAT XR

SMMT

Smmttx

smmttx.com

1assets

IvonescimabSMT112 / AK112

SPRY

Ars Pharma

ars-pharma.com

1assets

Epinephrine nasal sprayARS-1 / neffy / Alpha and beta-adrenergic receptors / Small Molecule / Immunology & Allergy
Type I Allergic Reactions (including Anaphylaxis)ApprovedActiveUnited StatesEuropean UnionCanadaUrticaria (Acute Flares of Chronic Spontaneous Urticaria)Phase 2ActiveUnited States

SVRA

Savara Pharma

savarapharma.com

1assets

MolgramostimMolbreevi / GM-CSF receptor / Recombinant protein (rhGM-CSF) / Respiratory / Rare Lung Diseases
Autoimmune Pulmonary Alveolar Proteinosis (aPAP)SubmissionRegulatory SubmissionUSUKEU

TERN

Terns Pharma

ternspharma.com

1assets

TERN-701TERN-701 / BCR::ABL1 / Small Molecule / Oncology
Chronic Myeloid Leukemia (CML)Phase 1/2ActiveGlobal

TRML

Tourmaline Bio

tourmalinebio.com

1assets

pacibekitugTOUR006 / IL-6 / Monoclonal Antibody / Cardiovascular and Immune Diseases
Atherosclerotic Cardiovascular Disease (ASCVD)Phase 2ActiveGlobalThyroid Eye Disease (TED)Phase 2ActiveGlobalAbdominal Aortic Aneurysm (AAA)Phase 2PlannedGlobal

TSHA

Tayshagtx

tayshagtx.com

1assets

TSHA-102

TSVT

2seventy Bio

2seventybio.com

1assets

idecabtagene vicleucelAbecma / BCMA / CAR-T Cell Therapy / Oncology
Relapsed or refractory multiple myelomaMarketedApprovedUSEU

UPB

Upstream Bio

upstreambio.com

1assets

verekitugUPB-101 / TSLP receptor (TSLPR) / Monoclonal antibody / Respiratory/Inflammatory Diseases
Severe AsthmaChronic rhinosinusitis with nasal polyps (CRSwNP)Chronic obstructive pulmonary disease (COPD)

VOR

Vor Bio

vorbio.com

1assets

TelitaciceptRC18 / Taiai / BAFF/APRIL (BLyS/APRIL) / Recombinant Fusion Protein / Autoimmune Diseases
Generalized Myasthenia Gravis (gMG)Phase 3Active / Approved in ChinaGlobalChinaSystemic Lupus Erythematosus (SLE)Phase 3Active / Approved in ChinaGlobalChinaRheumatoid Arthritis (RA)Phase 3Active / Approved in ChinaGlobalChina

VTVT

Vtv Therapeutics

vtvtherapeutics.com

1assets

CadisegliatinTTP399 / Liver-selective glucokinase / Small molecule / Endocrinology
Type 1 Diabetes (adjunctive to insulin)Phase 3OngoingUnited StatesType 2 DiabetesPhase 2PlannedUnited Arab Emirates

ZNTL

Zentalis

zentalis.com

1assets

AzenosertibZN-c3 / WEE1 / Small Molecule / Oncology
Cyclin E1-positive platinum-resistant ovarian cancer (PROC)Advanced solid tumors

ADPT

Adaptive Biotech

No pipeline

sec.gov

0assets

No disclosed assets in this row.