small-molecule-drugs
(21)
Teon Therapeutics, Inc.
Teon Therapeutics is a clinical-stage, privately held biopharmaceutical company in Redwood City, California, developing a focused portfolio of orally administered small-molecule drugs that block immunosuppressive, cancer-promoting signaling pathways in the tumor microenvironment. Its GPCR-directed candidates - TT-702, a first-in-class adenosine A2B receptor antagonist, and TT-816, a first-in-class cannabinoid CB2 receptor antagonist - aim to restore anti-tumor immunity for the roughly 88% of solid-tumor patients who do not benefit from existing immunotherapies. The founding team members were primary inventors of Lexiscan, the only FDA-approved selective adenosine therapeutic.
Dominique Verhelle
Dominique Verhelle is a French-born scientist and biotech entrepreneur who co-founded and leads NextRNA Therapeutics, a Boston company building small-molecule drugs that target long non-coding RNA to treat cancers and neurological disorders. With a PhD in life sciences and an MBA in entrepreneurship, she has spent more than two decades across big pharma, venture capital, and startups - including Celgene, Pfizer, Third Rock Ventures, and Takeda - and helped launch Fulcrum Therapeutics and Cedilla Therapeutics before starting NextRNA. She is also an outspoken advocate for women founders in biotech.
Lina Yao
Lina Yao is a pharmaceutical scientist and the founder of Teon Therapeutics, a Redwood City, California biotech developing first-in-class, orally administered small-molecule cancer drugs that target G-protein coupled receptors to reverse tumor-driven immune suppression. A molecular pharmacologist by training with an MD and PhD and more than two decades in drug discovery, she built her career at CV Therapeutics and Gilead Sciences before launching Teon in 2018. She led the company through an oversubscribed $30 million Series A in 2021 and into the clinic with two novel programs: TT-702, an adenosine A2B receptor antagonist, and TT-816, a cannabinoid CB2 receptor antagonist being studied in combination with Merck's KEYTRUDA.
Vivace Therapeutics, Inc.
Vivace Therapeutics is a San Mateo, California oncology company turning novel Hippo-YAP pathway biology into first-in-class cancer drugs. Its lead small molecule, VT3989, is a first-in-class YAP/TEAD inhibitor that blocks TEAD auto-palmitoylation and has shown durable responses in treatment-resistant mesothelioma and NF2-mutant tumors. Built on a capital-efficient US-China 'virtual biotech' model, the company has raised roughly $105 million across four rounds and is preparing a registrational Phase 3 trial.
Nurix Therapeutics
Nurix Therapeutics is a clinical-stage biopharmaceutical company in San Francisco pioneering targeted protein degradation - medicines that destroy disease-causing proteins rather than merely blocking them. Built on its DELigase discovery engine that harnesses the cell's ubiquitin-proteasome system and DNA-encoded library screening, Nurix is advancing a pipeline of degraders and degrader antibody conjugates for cancer and autoimmune disease, led by the BTK degrader bexobrutideg (NX-5948). The company is partnered with Roche, Sanofi, Gilead, and Pfizer (via Seagen) and trades on Nasdaq under NRIX.
Michael Shih
Michael Shih is the Chief Executive Officer of NeoPhore Ltd, a London-based small molecule neoantigen immuno-oncology company developing first-in-class drugs that target the DNA mismatch repair (MMR) pathway to make cancers more visible to the immune system. An attorney by training with degrees in biology, he spent nearly two decades structuring biotech deals before sitting in the chief executive's chair, with senior business development and corporate development roles at Kite Pharma, Biogen, Sanofi, Epizyme, Forest Laboratories, Kastle Therapeutics and Eisai. He took the helm at NeoPhore in December 2024, right after the company closed an oversubscribed Series B round backed by Bristol Myers Squibb.
858 Therapeutics
858 Therapeutics is a clinical-stage biotechnology company in San Diego building small-molecule cancer drugs aimed at novel targets in DNA damage response, RNA modulation, and innate immunity. Its lead candidate, the oral PARG inhibitor ETX-19477, is in a first-in-human Phase 1/2 trial for advanced solid tumors and has won FDA Fast Track designation for BRCA-mutated, platinum-resistant ovarian cancer. Founded in 2019 by a serial team of San Diego drug developers, the company has raised $110 million across Series A and B financings.
BlossomHill Therapeutics, Inc.
BlossomHill Therapeutics is a San Diego clinical-stage biopharmaceutical company designing small-molecule precision medicines for cancer and autoimmune disease. Founded in 2020 by drug designer J. Jean Cui and biotech veteran Y. Peter Li - the team behind Turning Point Therapeutics (acquired by Bristol Myers Squibb in 2022) - the company is advancing a wholly-owned pipeline led by BH-30643, a first-in-class macrocyclic OMNI-EGFR inhibitor for EGFR-mutant non-small cell lung cancer, and BH-30236, a CLK inhibitor targeting aberrant RNA splicing in AML and high-risk MDS.
Darrin Miles
Darrin Miles is the President and CEO of Nested Therapeutics, a Cambridge, Massachusetts precision oncology company that launched in 2022 with $125 million in financing to hunt for cancer-driving mutations hiding in plain sight. A commercialization leader with more than two decades in healthcare, Miles spent 14 years at Genentech shepherding landmark cancer brands and later served as Chief Commercial Officer at Agios Pharmaceuticals, where he helped bring the first mutant-IDH therapies to acute myeloid leukemia patients. At Nested, he is translating computational biophysics, genomics, and chemical biology into drugs aimed at targets the rest of the field calls undruggable.
Neomorph, Inc.
Neomorph is a San Diego clinical-stage biotech building molecular glue degraders - small molecules that recruit the cell's own disposal machinery to destroy disease-driving proteins long written off as 'undruggable.' Founded in 2020 out of Deerfield Management with scientific founders who decoded how thalidomide-class drugs work, the company has assembled what it calls the world's largest proprietary molecular glue target space across a broad portfolio of E3 ubiquitin ligases. It has signed multibillion-dollar discovery pacts with AbbVie, Novo Nordisk, and Biogen, dosed its first patient with lead candidate NEO-811 in kidney cancer, and raised roughly $209M across Series A and B.
NeoPhore Ltd
NeoPhore is a UK small-molecule immuno-oncology company developing first-in-class inhibitors of the DNA mismatch repair (MMR) pathway. By temporarily switching off MMR, its drugs are designed to make tumours generate fresh neoantigens, become visible to the immune system, and respond to checkpoint immunotherapy - extending the benefit of drugs like Keytruda to patients whose cancers are otherwise immune-cold. A 2017 spin-out of Cambridge's PhoreMost, NeoPhore has raised roughly $47M and counts Bristol Myers Squibb and Memorial Sloan Kettering among its backers and collaborators.
Newleos Therapeutics
Newleos Therapeutics is a Boston-based, clinical-stage biotechnology company building a new generation of treatments for neuropsychiatric disorders. Launched in February 2025 with an oversubscribed $93.5 million Series A, the company in-licensed four clinical-ready oral small molecules from Roche - targeting GABAA-y1, V1a, TAAR1, and GABAA-a5 - aimed at generalized anxiety, social anxiety, substance use disorders, and cognitive impairment. Its name nods to 'eos,' the Greek word for dawn: a new dawn for mental health treatment.
Quanta Therapeutics
Quanta Therapeutics is a clinical-stage biopharmaceutical company in South San Francisco building allosteric small-molecule inhibitors for RAS-driven cancers. Using a proprietary Second Harmonic Generation (SHG) optical platform that detects subtle changes in protein conformation, Quanta designs oral drugs aimed at KRAS mutations long considered undruggable - including G12D and G12V - that together drive a large share of pancreatic, colorectal, lung and endometrial cancers. Its lead candidate QTX3034 is in Phase 1 trials, with QTX3544 and a pan-KRAS program close behind.
Jeffrey Stafford
Jeffrey Stafford is the CEO and co-founder of 858 Therapeutics, a San Diego clinical-stage biotech developing small-molecule cancer drugs against novel targets in DNA-damage repair, innate immunity and RNA epigenetics. A chemist by training, he has spent three decades turning molecules into medicines: his discovery teams contributed to three FDA-approved drugs, and he previously led Jecure Therapeutics (acquired by Genentech in 2018) and co-founded Quanticel Pharmaceuticals (acquired by Celgene in 2015). 858's lead asset, the PARG inhibitor ETX-19477, won FDA Fast Track status and posted a 57% response rate in BRCA-mutated ovarian cancer at ASCO 2026.
Synnovation Therapeutics
Synnovation Therapeutics is a Wilmington, Delaware precision medicine company building small-molecule cancer drugs aimed at validated oncology targets where greater potency and selectivity could beat the current standard of care. Founded in 2024 by a team of former Incyte medicinal chemistry leaders, it launched with a $102 million Series A led by Third Rock Ventures. Its lead programs are SNV1521, a CNS-penetrant, highly selective PARP1 inhibitor, and SNV4818, a mutant-selective PI3K-alpha inhibitor whose subsidiary Pikavation Therapeutics was acquired by Novartis in March 2026 for $2 billion upfront.
Maureen Hillenmeyer
Maureen Hillenmeyer is the founder and CEO of Hexagon Bio, a Menlo Park drug-discovery company that mines the genomes of fungi for hidden small molecules and engineers yeast to brew them into potential medicines. A former competitive swimmer turned computational biologist, she holds a PhD in biomedical informatics from Stanford and built the company out of a Stanford research program she led, raising more than $270 million to turn DNA sequence data into cancer and infectious-disease therapeutics.
Nocion Therapeutics, Inc.
Nocion Therapeutics is a clinical-stage biopharmaceutical company in the Boston area developing a new class of small-molecule 'charged' sodium channel blockers - nocions - that selectively, locally, and durably silence overactive sensory neurons (nociceptors) without numbing everything else. Its lead candidate, taplucainium, is an inhaled powder aimed at refractory and unexplained chronic cough, with broader potential across itch, pain, and neurogenic inflammation. The science was born in the Harvard Medical School and Boston Children's Hospital labs of Bruce Bean and Clifford Woolf.
Soongyu Choi
Soongyu Choi is the co-CEO and co-founder of Avelos Therapeutics, a Seoul-based clinical-stage precision oncology company chasing first-in-class small molecules in synthetic lethality, DNA damage response, and cell cycle control. A Harvard-trained chemist who spent a decade at PTC Therapeutics and led R&D at Yuhan and Hana Pharm, he co-founded Avelos in 2021 and was elevated from CTO to co-CEO in January 2024. His flagship program, AD1208, is a first-in-class MASTL kinase inhibitor he frames as a precise strike on cancers that have stopped responding to standard therapy.
Frontier Medicines
Frontier Medicines is a precision-medicine biotech using chemoproteomics, covalent chemistry, and machine learning to drug proteins long considered 'undruggable.' Its lead candidate, FMC-376, is a first-in-class dual ON/OFF inhibitor of KRAS G12C now in the Phase 1/2 PROSPER trial. Backed by $315M+ in venture funding and a major AbbVie partnership, the company is one of the most-watched names in next-generation targeted cancer therapy.
Terry Rosen
Terry Rosen is the CEO and co-founder of Arcus Biosciences, a clinical-stage biopharmaceutical company focused on developing combination cancer immunotherapies. A medicinal chemist by training with a Ph.D. from UC Berkeley, Rosen has spent over 30 years at the intersection of chemistry and oncology - from Abbott Laboratories and Pfizer to Amgen, and then co-founding Flexus Biosciences which sold to Bristol-Myers Squibb for $1.25 billion in 2015. He immediately used that platform to launch Arcus, which has since secured a landmark 10-year partnership with Gilead Sciences and advanced multiple molecules into pivotal clinical studies, including casdatifan, a best-in-class HIF-2α inhibitor showing strong results in kidney cancer.
Jung Choi
Jung Choi is the President and CEO of Alterome Therapeutics, a clinical-stage precision oncology company pioneering mutation-specific cancer drugs. With over 20 years in biopharmaceutical leadership - spanning Gilead Sciences, InterMune, Chimerix, and Global Blood Therapeutics where he led a $5.4B Pfizer acquisition - Choi brought deal-making, strategy, and operational depth to Alterome when he was appointed CEO in April 2025. Under his leadership, Alterome has advanced two first-in-class inhibitors into Phase 1/1b trials targeting KRAS and AKT1 E17K oncogenic drivers, backed by $231M in total funding including a $132M Series B.