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Companies

Amphastar Pharmaceuticals, Inc.

amphastar.com
8assets
  • Insulin AspartAMP-004 / interchangeable biosimilar insulin / Diabetes
  • Recombinant Human InsulinAMP-005 / interchangeable biosimilar insulin / Diabetes
  • AMP-028AMP-028 / biosimilar
  • Intranasal epinephrineAMP-019 / adrenergic receptors / intranasal small molecule / Allergy/Immunology
    Anaphylaxis/allergic reactionsdevelopment
  • AMP-105AMP-105 / peptide / Oncology
    Poorly treated cancersdevelopmentUnited StatesCanada
  • AMP-107AMP-107 / VEGFR / topical eye-drop peptide / Ophthalmology
    Wet age-related macular degenerationdevelopmentUnited StatesCanada
  • AMP-109AMP-109 / peptide-docetaxel conjugate / Oncology
    CancerdevelopmentUnited StatesCanada
  • AMP-110AMP-110 / ACTH receptor pathway / synthetic human corticotropin analog peptide
    Multiple indicationsdevelopmentUnited StatesCanada

Crinetics Pharmaceuticals, Inc.

crinetics.com
8assets
  • PaltusotineCRN00808 / PALSONIFY / SST2 / oral nonpeptide SST2 agonist / Endocrinology; oncology
    Acromegaly (US)ApprovedapprovedUSAcromegaly (EU)Registrationalunder reviewEUCarcinoid syndrome associated with NETsPhase 3clinicalGlobal
  • AtumelnantCRN04894 / MC2R/ACTH receptor / oral nonpeptide ACTH receptor antagonist / Endocrinology
    Congenital adrenal hyperplasia (adult)Phase 3clinicalCongenital adrenal hyperplasia (pediatric)Phase 2/3clinicalACTH-dependent Cushing's syndromePhase 2/3planned
  • CRN09682CRN09682 / SST2 / nonpeptide drug conjugate; MMAE payload / Oncology
    SST2-expressing NETs and other SST2-expressing solid tumorsPhase 1/2clinical
  • PTH AntagonistPTH receptor / oral nonpeptide antagonist / Endocrinology
    Hyperparathyroidism and other diseases of hypercalcemiaPreclinicalIND-enabling
  • TSH AntagonistTSH receptor / oral nonpeptide antagonist / Endocrinology
    Graves' disease; thyroid eye diseasePreclinicalIND-enabling
  • SST3 AgonistSST3 / oral nonpeptide agonist / Nephrology
    Polycystic kidney diseasePreclinicalIND-enabling
  • Oral GLP-1 NonpeptideGLP-1 receptor / oral nonpeptide agonist / Metabolic disease
    Diabetes; obesityPreclinicaldisclosed
  • Oral GIP NonpeptideGIP receptor / oral nonpeptide agonist / Metabolic disease
    Diabetes; obesityPreclinicaldisclosed

CureVac N.V.

curevac.com
8assets
  • CVGBMCVGBM / tumor-associated antigens / unmodified mRNA cancer precision immunotherapy / Oncology
    newly diagnosed surgically resected MGMT-unmethylated glioblastoma or astrocytoma with molecular signature of glioblastomaPhase 1ongoing
  • sqNSCLC shared-antigen cancer precision immunotherapy candidateshared tumor antigens / unmodified mRNA cancer precision immunotherapy / Oncology
    squamous non-small cell lung cancerIND/CTA filedIND and CTA submissions filed Q1 2025
  • CV0601CV0601 / SARS-CoV-2 Omicron BA.4-5 spike antigen / mRNA vaccine / Infectious disease
    COVID-19Phase 2study completed August 2024; GSK assumed full control
  • CV0701CV0701 / SARS-CoV-2 Omicron BA.4-5 and original SARS-CoV-2 antigens / bivalent mRNA vaccine / Infectious disease
    COVID-19Phase 2study completed August 2024; GSK assumed full control
  • seasonal influenza mRNA vaccine candidateinfluenza antigens / multivalent mRNA vaccine / Infectious disease
    seasonal influenzaPhase 2GSK assumed full control
  • avian influenza H5N1 mRNA vaccine candidateH5N1 influenza antigens / mRNA vaccine / Infectious disease
    avian influenza H5N1Phase 2GSK assumed full control
  • COVID-19/influenza combination mRNA vaccine candidateSARS-CoV-2 and influenza antigens / combination mRNA vaccine / Infectious disease
    COVID-19 and influenzaPhase 1/2GSK initiated study November 2024; GSK assumed full control
  • UPEC FimH ferritin nanoparticle mRNA vaccine candidateFimH / mRNA vaccine / Infectious disease
    urinary tract infections caused by uropathogenic E. coliPreclinicallead candidate identified

Dyne Therapeutics, Inc.

dyne-tx.com
8assets

Evaxion A/S

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

Exelixis, Inc.

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

Immunocore Holdings PLC

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

Innate Pharma S.A.

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

Opus Genetics, Inc.

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

Kymera Therapeutics, Inc.

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

PTC Therapeutics, Inc.

ptcbio.com
8assets

Roche Holding AG

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

Royalty Pharma PLC

royaltypharma.com
8assets

Sionna Therapeutics, Inc.

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

Sarepta Therapeutics, Inc.

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

Zenas BioPharma, Inc.

zenasbio.com
8assets

Zymeworks Inc.

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

Ascentage Pharma

ir.ascentage.com
7assets
  • OlverembatinibHQP1351 / BCR-ABL / small molecule TKI / Oncology
    CML-CP/AP with T315I; CML-CP resistant/intolerant to 1st/2nd gen TKIsMarketedApproved in ChinaChinaCMLPhase 3 registrationalOngoing; POLARIS-2GlobalFirst-line Ph+ ALLPhase 3 registrationalOngoing; POLARIS-1Global
  • LisaftoclaxAPG-2575 / Bcl-2 / small molecule inhibitor / Oncology
    CLL/SLL after systemic therapy including BTK inhibitorsMarketedApproved in China 2025ChinaCLL/SLL after BTK inhibitorsPhase 3 registrationalOngoing; GLORA; +BTK inhibitorGlobalFirst-line CLL/SLLPhase 3 registrationalOngoing; GLORA-2; +acalabrutinibGlobal
  • APG-2449APG-2449 / FAK/ALK/ROS1 / small molecule inhibitor / Oncology
    ALK-positive advanced or locally advanced NSCLC, treatment-naivePhase 3 registrationalOngoing; CDE-clearedChinaNSCLC resistant/intolerant to 2nd generation ALK TKIsPhase 3 registrationalOngoing; CDE-clearedChinaPlatinum-resistant ovarian cancerPhase 1b/2Ongoing; +liposomal doxorubicin
  • AlrizomadlinAPG-115 / MDM2-p53 / small molecule inhibitor / Oncology
    Unresectable/metastatic melanoma and other advanced solid tumorsPhase 1b/2Ongoing; monotherapy or +pembrolizumabUnited StatesAustraliaR/R T-PLL or NHLPhase 2aOngoing; monotherapy or +lisaftoclaxUnited StatesAustraliaSalivary gland cancerPhase 2Collaborative research study
  • PelcitoclaxAPG-1252 / Bcl-2/Bcl-xL / small molecule inhibitor / Oncology
    EGFR-mutant NSCLCPhase 1bOngoing; +osimertinibChinaR/R NHLPhase 1b/2OngoingChinaRecurrent ovarian and endometrial cancersPhase 1Ongoing; +cobimetinib
  • APG-5918APG-5918 / EED/PRC2 / small molecule inhibitor / Oncology; Hematology
    Advanced solid tumors and hematologic malignanciesPhase 1OngoingChinaUnited StatesAnemia-related indicationsPhase 1OngoingChina
  • APG-3288APG-3288 / BTK / PROTAC degrader / Oncology
    R/R B-cell malignanciesPhase 1IND cleared; planned/initiating in 2026United StatesChina

Alto Neuroscience, Inc.

altoneuroscience.com
7assets
  • ALTO-207CTC-501 / D3/D2 & 5-HT3 / fixed-dose combination of pramipexole and ondansetron / Neuropsychiatry
    Treatment-resistant depressionPhase 2bactive
  • ALTO-300MT1/2 & 5HT2C / oral small molecule / Neuropsychiatry
    Major depressive disorderPhase 2bactiveUnited States
  • ALTO-100BDNF/neural plasticity / oral small molecule / Neuropsychiatry
    Bipolar depressionPhase 2bactive
  • ALTO-101PDE4 / transdermal PDE4 inhibitor / Neuropsychiatry
    Cognitive impairment associated with schizophreniaPhase 2active
  • ALTO-203Histamine H3 receptor / oral small molecule inverse agonist / Neuropsychiatry
    Major depressive disorder with anhedoniaPhase 2aactive
  • ALTO-202NMDA NR2B/GluN2B / oral small molecule negative allosteric modulator / Neuropsychiatry
    Major depressive disorderactive
  • ALTO-208CTC 413 / D3/D2 & NK-1 / fixed-dose combination of pramipexole and aprepitant / Neurology
    Parkinson's diseaseactive

Arvinas, Inc.

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

Genmab A/S

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

Structure Therapeutics Inc.

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

Harmony Biosciences Holdings, Inc.

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

Innoviva, Inc.

inva.com
7assets

Iovance Biotherapeutics, Inc.

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

Janux Therapeutics, Inc.

januxrx.com
7assets

Legend Biotech Corporation

legendbiotech.com
7assets

Lexeo Therapeutics, Inc.

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

MannKind Corporation

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

OPKO Health, Inc.

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

Radiopharm Theranostics Limited

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

Solid Biosciences Inc.

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

Spyre Therapeutics, Inc.

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)

Tonix Pharmaceuticals Holding Corp.

tonixpharma.com
7assets

UroGen Pharma Ltd.

urogen.com
7assets

Voyager Therapeutics, Inc.

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

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

AC Immune

acimmune.com
6assets
  • ACI-35.030JNJ-2056; JNJ-64042056 / phosphorylated Tau (pTau) / active immunotherapy / neurodegenerative diseases
    preclinical Alzheimer's diseasePhase 2bongoing
  • ACI-24.060amyloid beta (Abeta) / active immunotherapy / neurodegenerative diseases
    Alzheimer's diseasePhase 2ongoingAlzheimer's disease in Down syndromePhase 2ongoing
  • ACI-7104.056alpha-synuclein (a-syn) / active immunotherapy / neurodegenerative diseases
    Parkinson's diseasePhase 2ongoing
  • ACI-19764NLRP3 inflammasome / small molecule inhibitor / neuroinflammation
    neuro-inflammationPhase 1ongoing
  • Morphomer TauTau / small molecule / neurodegenerative diseases
    Alzheimer's diseasePreclinicalIND-enabling studies planned
  • Morphomer a-synalpha-synuclein (a-syn) / small molecule / neurodegenerative diseases
    Parkinson's diseasePreclinicalreadiness to initiate IND-enabling studies

Alector, Inc.

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

Allogene Therapeutics, Inc.

allogene.com
6assets
  • cemacabtagene ansegedleucelcema-cel; ALLO-501A / CD19 / Allogeneic CAR T cell therapy / Hematologic malignancies
    Large B-cell lymphoma, MRD-positive first-line consolidationPivotal Phase 2EnrollingUSCanadaSouth KoreaAustralia
  • ALLO-316ALLO-316 / CD70 / Allogeneic CAR T cell therapy / Solid tumors; hematologic malignancies
    Advanced/metastatic clear cell renal cell carcinomaPhase 1bExpansion cohort enrollment completed; strategic opportunities being exploredCD70+ hematologic malignanciesIND-enablingCD70+ solid tumorsIND-enabling
  • ALLO-329ALLO-329 / CD19/CD70 / Allogeneic dual CAR T cell therapy; CRISPR gene-edited; Dagger technology / Autoimmune disease
    Systemic lupus erythematosus including lupus nephritisPhase 1OngoingIdiopathic inflammatory myopathiesPhase 1OngoingSystemic sclerosisPhase 1Ongoing
  • 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
  • BCMA/CD70 dual CAR T constructBCMA/CD70 / Allogeneic dual CAR T cell therapy; Dagger technology / Hematologic malignancies
    Multiple myelomaPreclinical

Apellis Pharmaceuticals, Inc.

apellis.com
6assets

Autolus Therapeutics PLC

autolus.com
6assets
  • obe-celAUCATZYL / CD19 / autologous CAR T-cell therapy / Hematology/oncology; autoimmune
    r/r adult ALLApprovedFDA/MHRA/EMA approvedUSUKEUPediatric B-ALLPhase 2CATULUSSystemic Lupus ErythematosusPhase 1CARLYSLE
  • AUTO1/22CD19 & CD22 / dual-targeting autologous CAR T-cell therapy / Hematology/oncology
    Pediatric B-ALLPhase 1CARPALL
  • AUTO8BCMA & CD19 / autologous CAR T-cell therapy / Hematology/oncology
    Multiple MyelomaPhase 1MCARTYLight Chain AmyloidosisPhase 1ALARIC
  • AUTO4/5TRBC1/2 / programmed T-cell therapy / Hematology/oncology
    TRBC1/2+ Peripheral TCLPreclinicalLibrA T1
  • AUTO6NGGD2 / autologous CAR T-cell therapy / Solid tumors
    NeuroblastomaPhase 1MAGNETO
  • AUTO9programmed T-cell therapy / Hematology/oncology
    Acute Myeloid LeukemiaPreclinicalTBD

Axsome Therapeutics, Inc.

axsome.com
6assets
  • AXS-05AXS-05 / AUVELITY / NMDA receptor; sigma-1 receptor; CYP2D6 / oral small molecule; dextromethorphan-bupropion / CNS
    Smoking cessationPhase 2/3 plannedOn track to initiate in Q2 2026US
  • SolriamfetolSUNOSI / DNRI; TAAR1; 5-HT1A / oral small molecule / CNS
    ADHD, childrenPhase 3 plannedOn track to initiate in Q2 2026USADHD, adolescentsPhase 3 plannedOn track to initiate in Q2 2026USMDD with EDS symptomsPhase 3CLARITY initiatedUS
  • AXS-12AXS-12 / Norepinephrine reuptake inhibitor; cortical dopamine modulator / oral small molecule; reboxetine / CNS
    Cataplexy in narcolepsyNDA submittedFDA filing acceptance decision pendingUS
  • AXS-14AXS-14 / Norepinephrine reuptake inhibitor / oral small molecule; esreboxetine / CNS
    FibromyalgiaPhase 3FORWARD ongoingUS
  • AXS-17AXS-17 / GABA-A receptor α2,3 subtype-selective PAM / oral small molecule / CNS
    EpilepsyPhase 2 trial-enablingUnderwayUS
  • AXS-20AXS-20 / PDE10A / oral small molecule; balipodect / CNS
    SchizophreniaPre-Phase 3Phase 3 trial-enabling activities anticipated in 2026USTourette syndromePlanned evaluationUS

Beam Therapeutics Inc.

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.

BioXcel Therapeutics, Inc.

bioxceltherapeutics.com
6assets
  • BXCL501BXCL501 / IGALMI / alpha-2 receptor agonist / orally dissolving/sublingual film formulation of dexmedetomidine / Neuroscience
    Acute agitation associated with bipolar disorders or schizophrenia, at homePhase 3Company-sponsored SERENITY programAcute agitation associated with Alzheimer's dementiaPhase 3Company-sponsored TRANQUILITY program; paused subject to fundingOpioid use disorderPhase 2Investigator-sponsored
  • BXCL701BXCL701 / DPP8/9 and FAP / oral innate immune activator / Immuno-oncology
    Small cell neuroendocrine prostate cancerPhase 2Company-sponsored; FDA meeting expectedUSUKSmall cell lung cancerPreclinicalCompany-sponsored; initiate Phase 1b/2 expectedMetastatic pancreatic ductal adenocarcinomaPhase 2Investigator-sponsored; Phase 2 readout expected
  • BXCL502BXCL502 / latrepirdine / Neuroscience
    Chronic agitation in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL503BXCL503 / Neuroscience
    Apathy in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL504BXCL504 / Neuroscience
    Aggression in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL702BXCL702 / DPP inhibitor / novel DPP inhibitor / Immuno-oncology
    Solid tumorsPreclinicalBXCL701 follow-on; candidate nomination expected

Cocrystal Pharma, Inc.

cocrystalpharma.com
6assets

Eton Pharmaceuticals, Inc.

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 Therapeutics, Inc.

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

Harrow, Inc.

harrow.com
6assets

Humacyte, Inc.

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