Pipeline Watchlist
Company Pipelines
Companies
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- ERAS-0015ERAS-0015 / RAS / Small molecule molecular glue / OncologyRAS-mutant solid tumorsPhase 1AURORAS-1; in clinicWorldwide
- ERAS-4001ERAS-4001 / KRAS / Small molecule / OncologyKRAS-mutant solid tumorsPhase 1BOREALIS-1; in clinicWorldwide
- ERAS-12ERAS-12 / EGFR D2/D3 / Large molecule; bispecific antibody / OncologyEGFR and RAS/MAPK altered tumorsWorldwide
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- DMR-001DMR-001 / mutant calreticulin (mutCALR) / Fc-null, half-life extended anti-mutCALR monoclonal antibody; subcutaneous biologic / Hematology/oncologymutCALR-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/oncologymutCALR-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/oncologymutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2027USex-US
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- quaratusugene ozeplasmid (REQORSA)REQORSA / TUSC2 / non-viral gene therapy / OncologyEGFR-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 / DiabetesType 1 DiabetesPreclinicalType 2 DiabetesPreclinical
- NPRL2 gene therapyNPRL2 / non-viral gene therapy / OncologyKRAS/STK11 mutant anti-PD1-resistant NSCLCPreclinical
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- HLP0035-HT2A (and other serotonin receptors) / Deuterated NSA (oral) / Mental HealthAdjunctive treatment of major depressive disorder (MDD)Phase 3Dosing underway (PARADIGM program); FDA Breakthrough Therapy DesignationUnited StatesEuropeAustralia
- HLP004Serotonin receptors / Deuterated NSA (intramuscular) / Mental HealthGeneralized anxiety disorder (GAD)Phase 2Positive topline results reportedUnited States
- HLP005NSA (phenethylamines and tryptamines) / Mental HealthCNS disordersPreclinical
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- IFx-2.0plasmid DNA (innate immune agonist) / OncologyAdvanced 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 / OncologyNPM1-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) / OncologyAcute myeloid leukemia (AML)PreclinicalLead selection
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- Vidofludimus calciumIMU-838 / DHODH inhibitor and Nurr1 activator / Oral small molecule / Multiple sclerosisRelapsing 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 diseasesCeliac diseasePhase 1bCompleted (positive proof-of-concept)Global
- IMU-381IMU-381 / Nurr1 / Oral small molecule / Autoimmune diseasesNeurologic, gastrointestinal and other autoimmune diseasesPreclinicalPreclinical
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- tarcocimab tedromerZenkuda / VEGF / Antibody Biopolymer Conjugate (ABC) / OphthalmologyWet Age-Related Macular Degeneration (wet AMD)Diabetic RetinopathyRetinal Vein Occlusion (RVO)
- KSI-501KSI-501 / IL-6 / VEGF / Bispecific Antibody Biopolymer Conjugate (ABC) / OphthalmologyWet Age-Related Macular Degeneration (wet AMD)Diabetic Macular Edema (DME)
- KSI-101KSI-101 / IL-6 / VEGF / Bispecific Protein / OphthalmologyMacular Edema Secondary to Inflammation (MESI)
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- mivocabtagene autoleucelKYV-101 / CD19 / Autologous CAR T-cell therapy / Autoimmune DiseasesStiff 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 DiseasesAutoimmune DiseasesPhase 1IND ActiveUS
- KYV-201KYV-201 / CD19 / Allogeneic CAR T-cell therapy / Autoimmune DiseasesAutoimmune DiseasesPreclinicalActiveUS
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- SotagliflozinINPEFA / ZYNQUISTA / SGLT1 / SGLT2 / Small Molecule / CardiometabolicHeart FailureApprovedactiveUnited StatesHypertrophic Cardiomyopathy (HCM)Phase 3activeGlobalType 1 Diabetes (T1D)RegistrationactiveUnited States
- PilavapadinLX9211 / AAK1 / Small Molecule / Pain / NeurologyDiabetic Peripheral Neuropathic Pain (DPNP)Phase 2bactiveUnited StatesPostherpetic Neuralgia (PHN)Phase 2inactiveUnited States
- LX9851LX9851 / ACSL5 / Small Molecule / MetabolismObesity / Weight ManagementPhase 1activeGlobal
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- ZidesamtinibNVL-520
- NeladalkibNVL-655
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- LicaminlimabOCS-02
- PrivosegtorOCS-05
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- D-PLEX100D-PLEX100 / Bacterial Ribosome / Small Molecule Formulation / Infectious Diseases / Surgical Site CarePrevention of surgical site infections (SSIs) following abdominal colorectal surgeryPhase 3OngoingUnited StatesEuropeIsrael
- PP03APP03A / GLP-1 Receptor / Peptide Formulation / Metabolic DiseaseObesity and Type 2 DiabetesPreclinicalOngoing
- PP04APP04A / OncoPLEX / STING / Small Molecule Formulation / OncologySolid TumorsPreclinicalOngoing
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- QRX003QRX003 / Serine protease inhibitor / Small molecule / DermatologyNetherton SyndromePhase 3OngoingUnited StatesEuropeMiddle EastPeeling Skin SyndromePhase 2OngoingNew Zealand
- Topical rapamycinQRX009 / mTOR / Small molecule / DermatologyMicrocystic Lymphatic MalformationsPreclinicalOngoingGlobalVenous MalformationsPreclinicalOngoingGlobalAngiofibromaPreclinicalOngoingGlobal
- QRX008QRX008 / VLA-4 / VCAM-1 inhibitor / Small molecule / DermatologySclerodermaPreclinicalOngoingGlobal
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- RAP-219RAP-219 / TARPγ8 AMPAR / Small molecule / Neurology/PsychiatryFocal onset seizuresPrimary generalized tonic-clonic seizuresBipolar mania
- α6β4 nAChR Agonistα6β4 nAChR / Small molecule / NeurologyChronic painMigraine
- α9α10 nAChR Modulatorα9α10 nAChR / Small molecule / Hearing/VestibularHearing disordersVestibular disorders
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- PC111PC111 / sFasL (soluble Fas Ligand) / Monoclonal Antibody / Immunology / DermatologyPemphigusPreclinicalActiveGlobalStevens-Johnson syndrome / toxic epidermal necrolysis (SJS/TEN)PreclinicalActiveGlobal
- Intradermal IL-17A/F NanoAbIL-17A / IL-17F / VHH Antibody Fragment (NanoAb) / Immunology / DermatologyMild-to-moderate plaque psoriasisPreclinicalActiveGlobal
- Systemic Bi-Specific IL-17 NanoAbIL-17A / IL-17F and secondary proprietary target / Bi-specific VHH Antibody Fragment (NanoAb) / Immunology / DermatologyModerate-to-severe plaque psoriasisPreclinicalActiveGlobalPsoriatic arthritisPreclinicalActiveGlobal
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- DenifanstatASC40 / FASN / Small molecule / Metabolic disease, Dermatology, OncologyMASH (F2/F3)Phase 3ActiveModerate to severe acnePhase 3ActiveRecurrent glioblastoma (GBM)Phase 3Active
- TVB-3567FASN / Small molecule / Dermatology, OncologyAcnePhase 1ActiveSolid tumorsPreclinicalActive
- FASNi TopicalFASN / Small molecule / DermatologyAcnePreclinicalActive
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- lotilaner ophthalmic solution 0.25%TP-03 / XDEMVY / Small molecule / Eye CareDemodex blepharitisMeibomian Gland Disease
- lotilaner ophthalmic gelTP-04 / Small molecule / Dermatology / Eye CareRosaceaOcular rosacea
- lotilaner oral tabletTP-05 / Small molecule / Infectious Disease PreventionLyme disease preventionCommunity reduction of malaria
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- TX45TX-000045 / RXFP1 / Fc-relaxin fusion protein / CardiopulmonaryPH-HFpEF (Group 2 Pulmonary Hypertension)PH-HFrEF (Group 2 Pulmonary Hypertension)PH-ILD (Group 3 Pulmonary Hypertension)
- TX2100TX002100 / GPCR / Biotherapeutic / GPCR Antagonist / Hematology / Rare DiseasesHereditary Hemorrhagic Telangiectasia (HHT)
- Fibrosis ProgramGPCR / Bispecific GPCR Antagonist / FibrosisFibrosis
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- ublituximabTG-1101 / BRIUMVI / CD20 / Monoclonal Antibody / Autoimmune DiseasesRelapsing Multiple Sclerosis (RMS)
- ublituximab subcutaneousCD20 / Monoclonal Antibody / Autoimmune DiseasesRelapsing Multiple Sclerosis (RMS)
- azercabtagene zapreleucelazer-cel / CD19 / Allogeneic CAR-T Cell Therapy / Autoimmune DiseasesProgressive Multiple Sclerosis (PMS)Autoimmune Diseases
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- Intranasal ForalumabTZLS-401 / CD3 epsilon / Monoclonal Antibody / Neurology/ImmunologyNon-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 / OncologyNSCLC with pan KRAS-positive mutations
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- TN-201TN-201 / MYBPC3 / AAV-based gene therapy / CardiovascularMYBPC3-associated Hypertrophic Cardiomyopathy (HCM)
- TN-401TN-401 / PKP2 / AAV9-based gene therapy / CardiovascularPKP2-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
- TN-301TN-301 / HDAC6 / Small Molecule / Cardiometabolic / NeuromuscularHeart Failure with preserved Ejection Fraction (HFpEF)Duchenne Muscular Dystrophy (DMD)
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- sparsentanFILSPARI
- pegtibatinaseTVT-058
- tioproninThiola / Thiola EC
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- dabogratinibTYRA-300 / FGFR3 / Small molecule / Oncology / Skeletal DysplasiaMetastatic Urothelial Cancer (mUC)Non-Muscle Invasive Bladder Cancer (IR NMIBC)Achondroplasia
- TYRA-200TYRA-200 / FGFR1/2/3 / Small molecule / OncologyIntrahepatic CholangiocarcinomaAdvanced Solid Tumors
- TYRA-430TYRA-430 / FGFR4/3 / Small molecule / OncologyHepatocellular Carcinoma (HCC)Advanced Solid Tumors
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- AtaciceptVT-001 / BAFF and APRIL / Recombinant Fusion Protein / Immunology / NephrologyIgA Nephropathy (IgAN)BLA SubmittedPriority Review (PDUFA July 2026)USGlobalLupus NephritisPhase 2OngoingGlobalFocal Segmental Glomerulosclerosis (FSGS)Phase 2OngoingGlobal
- MAU868MAU868 / BK Virus / Monoclonal Antibody / Infectious Disease / TransplantationBK Viremia in Kidney Transplant RecipientsPhase 2Evaluating for future developmentGlobal
- VT-109VT-109 / BAFF and APRIL / Fusion Protein / ImmunologyB Cell Mediated DiseasesPreclinicalActiveGlobal
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- Avutometinib and DefactinibVS-6766 / VS-6063 / AVMAPKI FAKZYNJA CO-PACK / RAF/MEK and FAK / Small Molecule / OncologyKRAS-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 / OncologyBRAF, KRAS and NRAS-Mutant Solid TumorsPhase 1ActiveGlobal
- VS-7375GFH375 / KRAS G12D / Small Molecule / OncologyKRAS G12D-Mutated Solid TumorsPhase 1/Phase 2ActiveUnited StatesChina
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- tibulizumabZB-106 / IL-17A and BAFF / Bispecific dual-antagonist antibody / Autoimmune and Inflammatory DiseasesHidradenitis suppurativaSystemic sclerosis
- crebankitugZB-168 / IL-7Rα / Monoclonal antibody / Autoimmune and Inflammatory DiseasesAutoimmune and inflammatory conditions
- torudokimabZB-880 / IL-33 / Monoclonal antibody / Autoimmune and Inflammatory DiseasesAutoimmune and inflammatory conditions
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- ImdusiranAB-729; IDR / HBV viral antigens/HBV RNA transcripts / GalNAc-conjugated RNAi therapeutic; subcutaneous / Infectious disease; hepatitis BChronic hepatitis B (cHBV); IM-PROVE I: imdusiran + peg-IFNα-2a + NAPhase 2aclinicalChronic hepatitis B (cHBV); IM-PROVE II: imdusiran + nivolumab + NA + VTP-300Phase 2aclinical
- AB-101AB-101 / PD-L1 / Oral PD-L1 inhibitor / Infectious disease; hepatitis BChronic hepatitis B (cHBV); AB-101-001 in healthy subjects and cHBV patientsPhase 1a/1bclinical; Part 3 dosing in cHBV patients
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- Prulacabtagene leucelprula-cel; formerly ADI-001 / CD20 / Allogeneic gamma delta CAR T cell therapy / Autoimmune diseasesLupus nephritisPhase 1activeSystemic lupus erythematosusPhase 1activeSystemic sclerosisPhase 1active
- ADI-212ADI-212 / PSMA / Next-generation gene-edited and armored allogeneic CAR gamma delta T cell therapy / OncologyMetastatic castration-resistant prostate cancerPreclinicalactive
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- AKY-1189AKY-1189 / Nectin-4 / miniprotein radioconjugate; 225Ac targeted alpha radiopharmaceutical / OncologyNectin-4 expressing solid tumorsPhase 1bongoingUSLocally advanced or metastatic urothelial cancer, breast cancer, NSCLC, colorectal cancer, cervical cancer, head and neck cancerPhase 1bongoingUS
- AKY-2519AKY-2519 / B7-H3 (CD276) / miniprotein radioconjugate; 225Ac targeted alpha radiopharmaceutical; 64Cu imaging pair / OncologyMetastatic castration-resistant prostate cancerPhase 1bongoingUSB7-H3 expressing solid tumors, including lung and colorectal cancerPhase 1bplannedUS
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- AKTX-101AKTX-101 / TROP2 / Antibody-drug conjugate with PH1 RNA splicing modulator payload / OncologyTROP2-expressing solid tumorsPreclinical / IND-enablingPhase 1 planned late 2026 or early 2027
- AKTX-102AKTX-102 / CEACAM5 / Antibody-drug conjugate with PH1 RNA splicing modulator payload / OncologyCEACAM5-expressing solid tumorsDiscoveryAdvancing
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- evorpaceptALX148 / CD47 / CD47-targeted investigational therapy / OncologyENHERTU-experienced HER2-positive breast cancer; with trastuzumab + chemotherapy (ASPEN-Breast)Phase 2FPI mid-year 2025Relapsed or refractory multiple myeloma; with SARCLISA + dexamethasonePhase 1FPI Q3 2024, currently enrolling2L/3L advanced HER2-overexpressing gastric/GEJ cancer; with trastuzumab, CYRAMZA + paclitaxel (ASPEN-06)Phase 2Completed, established POC, seeking partner ex-US
- ALX2004ALX2004 / EGFR / ADC / OncologyEGFR-expressing solid tumorsPhase 1FPI mid-year 2025
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- AL001AL001 / LISPRO / ionic cocrystal of lithium, proline and salicylate / Neurology/PsychiatryAlzheimer'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 / NeurologyMild to moderate dementia of the Alzheimer's typePhase 1/2apaused in 2024; expected to resume by mid-2026US
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- dostarlimabJemperli / PD-1 / antibody / OncologyEndometrial cancerApprovedapproveddMMR pan tumorsApprovedapprovedRectal cancerbeing studied
- imsidolimabIL-36R / antibody / Dermatology/InflammationGeneralized pustular psoriasisBLA submitted/acceptedFDA target action date December 12, 2026US
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- EpetraboroleLeucyl-tRNA synthetase / oral boron-based small molecule antibiotic / Hematology; Infectious diseasePhlebotomy-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 diseaseChagas diseasePhase 1first-in-human trial underway
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- bemnifosbuvir/ruzasvir fixed-dose combinationBEM/RZR / HCV NS5B polymerase; HCV NS5A / oral small-molecule antiviral regimen / viral hepatitishepatitis C virus (HCV) infectionPhase 3activeglobal
- AT-587AT-587 / hepatitis E virus (HEV) / oral nucleotide analog / viral hepatitischronic hepatitis E virus (HEV) infection in immunocompromised patientspreclinicalPhase 1 anticipated mid-2026
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- LarsucosterolDUR-928 / Epigenetic modulator / HepatologyAlcohol-associated hepatitisPhase 3Registrational Phase 3 initiated in early 2026US
- AmiselimodMT-1303 / S1P receptor / Oral S1P modulator / GastroenterologyMild-to-moderate ulcerative colitisPhase 2Internal review of opportunity ongoing
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- BezisterimNE3107 / ERK; NF-κB; TNF-α / oral small molecule anti-inflammatory insulin sensitizer / neurology; neurodegenerative diseaseAlzheimer’s diseasePhase 2/3 conductedclinicalParkinson’s diseasePhase 2SUNRISE-PD last patient evaluation completed; topline targeted Q3 2026USLong COVID neurological symptomsPhase 2ADDRESS-LC enrollingUS
- BIV201BIV201 / continuous infusion formulation of terlipressin / liver diseaseascites due to liver cirrhosisPhase 3 planningclinical developmentUSrefractory ascitesPhase 2b completedclinical developmentUS
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- GLIX1GLIX1 / DNA damage response; restores TET2 activity / oral small molecule / OncologyGlioblastomaPhase 1Other cancersPreclinical
- MotixafortideBL-8040 / APHEXDA / CXCR4 / peptide CXCR4 inhibitor / Oncology; rare diseases; stem cell mobilizationPancreatic cancerPhase 2Stem cell mobilization in multiple myelomaApprovedApproved in US; bridging study in AsiaUSAsiaSickle cell disease stem cell mobilizationPhase 1/2
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- TinlarebantLBS-008 / RBP4 / Oral small-molecule RBP4 antagonist / Ophthalmology / retinal degenerative diseasesStargardt 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 diseaseNon-alcoholic fatty liver disease (NAFLD)PreclinicalPreclinical developmentNon-alcoholic steatohepatitis (NASH)PreclinicalPreclinical developmentType 2 diabetes (T2D)PreclinicalPreclinical development
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- IcovamenibMenin / Oral small molecule menin inhibitor / Diabetes; metabolic diseaseType 1 diabetes mellitusPhase IIOngoingInsulin-deficient type 2 diabetesPhase IIEnrollingType 2 diabetes uncontrolled with a GLP-1 RA-based therapyPhase IIEnrolling
- BMF-650BMF-650 / GLP-1 receptor / Oral small molecule GLP-1 receptor agonist / Obesity; metabolic diseaseHealthy participants / obesity and metabolic disordersPhase IOngoing