Biotech Distilled
Substack ↗ Subscribe
Subscribe Substack ↗ About

Home › Modalities › Small Molecule

Small Molecule (105 companies)

Ticker Company Therapeutic areas Programs Lead stage Programs
ACIU AC Immune Neurology Rare 6 Phase 3 10
ACHV Achieve Life Sciences — 2 NDA Submitted 2
ACRS Aclaris Therapeutics DermatologyGI/Hepatology+1 Rare 4 Phase 2 8
ACRV Acrivon Therapeutics Oncology 6 Phase 2 6
ACTU Actuate Therapeutics Oncology 4 Phase 2 4
AGMB Agomab Therapeutics ImmunologyRespiratory Rare 2 Phase 2 4
AKBA Akebia Therapeutics HematologyOphthalmology+2 Rare 7 Approved 10
ALDX Aldeyra Therapeutics DermatologyOncology+1 Rare 7 NDA Submitted 7
ATHE Alterity Therapeutics Neurology Rare 2 Phase 2 2
AMLX Amylyx Pharmaceuticals MetabolicNeurology Rare 1 Phase 3 5
ANNX Annexon HematologyNeurology+2 Rare 1 Phase 3 6
RCUS Arcus Biosciences DermatologyImmunology+1 2 Phase 3 6
ARDX Ardelyx GI/HepatologyRenal 4 Approved 4
ARVN Arvinas NeurologyOncology Rare 12 Approved 12
ATAI Atai Beckley Neurology 5 Phase 2 5
BLTE Belite Bio MetabolicOphthalmology Rare 6 NDA Submitted 6
BIOA BioAge Labs CardiovascularOphthalmology 2 Phase 2 2
BMEA Biomea Fusion MetabolicOncology 4 Phase 2 4
BBOT BridgeBio Oncology Therapeutics Oncology 8 Phase 1 8
CCCC C4 Therapeutics Oncology 2 Phase 2 2
CRDL Cardiol Therapeutics Cardiovascular Rare 4 Phase 3 4
CRVO CervoMed Neurology Rare 4 Phase 3 4
CING Cingulate Neurology 1 NDA Submitted 1
CLNN Clene Neurology Rare 5 Phase 3 5
CLYM Climb Bio HematologyImmunology+1 Rare 1 Phase 2 7
COGT Cogent Biosciences HematologyOncology Rare 7 NDA Submitted 7
CGTX Cognition Therapeutics Neurology 2 Phase 2 2
CMPS COMPASS Pathways Neurology 3 NDA Submitted 3
CRVS Corvus Pharmaceuticals DermatologyOncology 3 Phase 3 4
CRNX Crinetics Pharmaceuticals MetabolicOncology Rare 6 Approved 6
CGEM Cullinan Therapeutics HematologyImmunology+2 Rare 1 NDA Submitted 10
HELP Cybin Neurology 3 Phase 3 3
DFTX Definium Therapeutics Neurology 4 Phase 3 4
EIKN Eikon Therapeutics Oncology 13 Phase 2 13
ELOX Eloxx Pharmaceuticals DermatologyRenal Rare 4 Phase 2 4
ENTA Enanta Pharmaceuticals DermatologyImmunology+1 6 Phase 2 6
ERAS Erasca Oncology 2 Phase 1 3
ETON Eton Pharmaceuticals GI/HepatologyInfectious Disease+2 Rare 11 Approved 14
EVMN Evommune DermatologyImmunology+1 3 Phase 2 6
EXEL Exelixis CardiovascularOncology 17 Approved 22
EYPT EyePoint Ophthalmology 3 Phase 3 3
FHTX Foghorn Therapeutics Oncology 2 Phase 1 5
FBIO Fortress Biotech DermatologyHematology+2 Rare 8 Approved 12
GANX Gain Therapeutics MetabolicNeurology Rare 4 Phase 1 5
GHRS GH Research NeurologyWomen's Health 4 Phase 2 4
GOSS Gossamer Bio Cardiovascular Rare 4 Phase 3 4
GRCE Grace Therapeutics Neurology 1 NDA Submitted 1
IMRX Immuneering Oncology 5 Phase 3 5
IMUX Immunic GI/HepatologyNeurology 4 Phase 3 4
IBRX ImmunityBio Infectious DiseaseOncology 1 Approved 16
IMNM Immunome Oncology Rare 1 NDA Submitted 9
IXHL Incannex Healthcare ImmunologyNeurology+1 3 Phase 2 3
INSM Insmed CardiovascularDermatology+2 Rare 6 Approved 9
KAPA Kairos Pharma Oncology 3 Phase 2 9
KPRX Kiora Pharmaceuticals Ophthalmology Rare 4 Phase 2 4
KURA Kura Oncology MetabolicOncology 12 Approved 12
KYMR Kymera Therapeutics DermatologyRespiratory Rare 4 Phase 2 4
LENZ LENZ Therapeutics Ophthalmology 1 Approved 1
MAIA MAIA Biotechnology Oncology 7 Phase 3 7
MPLT MapLight Therapeutics Neurology 6 Phase 2 6
MIST Milestone Pharmaceuticals Cardiovascular 2 Approved 2
GLUE Monte Rosa Therapeutics CardiovascularDermatology+2 Rare 6 Phase 2 6
NGEN NervGen Pharma Neurology 1 Phase 1 3
NRSN NeuroSense Therapeutics Neurology Rare 3 Phase 2 3
NAMS NewAmsterdam Pharma CardiovascularMetabolic+1 6 NDA Submitted 6
NRXP NRX Pharmaceuticals Neurology 5 NDA Submitted 5
NRIX Nurix Therapeutics ImmunologyNeurology+1 16 Phase 3 16
NVCT Nuvectis Pharma HematologyOncology+1 Rare 8 Phase 3 8
OCS Oculis Holding NeurologyOphthalmology Rare 3 Phase 3 4
OMER Omeros HematologyNeurology+2 Rare 2 Approved 6
IRD Opus Genetics Ophthalmology Rare 4 Approved 14
OVID Ovid Therapeutics Neurology Rare 4 Phase 2 4
PALI Palisade Bio Immunology 2 Phase 1 3
KTTA Pasithea Therapeutics NeurologyOncology Rare 4 Phase 1 4
PMVP PMV Pharmaceuticals Oncology 7 Phase 2 7
PYPD PolyPid Infectious DiseaseOncology 2 NDA Submitted 2
PCSA Processa Pharmaceuticals GI/HepatologyImmunology+2 Rare 7 Phase 2 7
TARA Protara Therapeutics DermatologyMetabolic+1 Rare 1 Phase 2 3
QNRX Quoin Pharmaceuticals DermatologyImmunology Rare 4 Phase 2 4
RLAY Relay Therapeutics DermatologyOncology Rare 4 NDA Submitted 4
RLMD Relmada Therapeutics MetabolicNeurology+1 Rare 4 Phase 2 4
RVPH Reviva Pharmaceuticals CardiovascularDermatology+2 Rare 10 Phase 3 10
RYTM Rhythm Pharmaceuticals Metabolic Rare 1 Approved 13
SGMT Sagimet Biosciences DermatologyMetabolic 3 NDA Submitted 3
SCYX Scynexis Infectious DiseaseRenal Rare 5 Approved 5
SLS SELLAS Life Sciences Group Oncology 4 Phase 3 9
SION Sionna Therapeutics Respiratory Rare 5 Phase 2 5
GPCR Structure Therapeutics MetabolicRespiratory Rare 5 Phase 3 5
TNGX Tango Therapeutics Oncology 4 Phase 1 4
TENX Tenax Therapeutics Cardiovascular Rare 2 Phase 3 2
TVTX Travere Therapeutics MetabolicRenal Rare 6 Approved 7
TTRX Turn Therapeutics DermatologyInfectious Disease Rare 3 Phase 2 4
TYRA Tyra Biosciences MetabolicOncology Rare 5 Phase 2 5
RARE Ultragenyx Pharmaceutical CardiovascularGI/Hepatology+2 Rare 1 Approved 12
UNCY Unicycive Therapeutics Renal 2 NDA Submitted 2
VNDA Vanda Pharmaceuticals CardiovascularDermatology+6 Rare 25 Approved 29
VSTM Verastem Oncology 5 Approved 5
VRCA Verrica Pharmaceuticals DermatologyOncology 2 Approved 3
VKTX Viking Therapeutics MetabolicNeurology Rare 5 Phase 3 8
VYGR Voyager Therapeutics MetabolicNeurology Rare 4 Phase 1 9
VTVT vTv Therapeutics MetabolicRespiratory Rare 8 Phase 3 8
XENE Xenon Pharmaceuticals Neurology Rare 8 Phase 3 8
XERS Xeris Biopharma MetabolicNeurology Rare 3 Approved 4
ZBIO Zenas BioPharma DermatologyImmunology+2 Rare 4 NDA Submitted 11
ZNTL Zentalis Pharmaceuticals Oncology 1 Phase 3 1