Acasti Pharma
NASDAQ:ACSTAcasti Pharma Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was incorporated in 2002 and is headquartered in Laval, Canada.
Bicycle Therapeutics
NASDAQ:BCYCBicycle Therapeutics plc, a clinical-stage biopharmaceutical company, develops a class of medicines for diseases that are underserved by existing therapeutics in the United States and the United Kingdom. Its product pipeline comprising BT8009, a bicycle toxin conjugate (BTC) which is in phase I/II and phase II/III clinical trial for the treatment of high nectin-4 expressing tumors; BT5528, a BTC which is in phase I/II clinical trial for the treatment of Ephrin type A receptor 2 expressing tumor; BT7480, a Bicycle TICA molecule which is in phase I/II targeting Nectin-4 and agonizing CD137; and BT7455, a Bicycle TICA molecule targeting Ephrin type A receptor 2 and CD137 and is in preclinical trial. The company also developing BT1718, a bicycle toxin conjugate (BTC), which is in Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease; and BT7401, a multivalent bicycle CD137 agonist which is in phase IIa to treat advanced solid tumors. In addition, it develops Novel anti-infective, which is in preclinical trial targeting anti-infectives disease; CNS targets, which is in preclinical trial targeting CNS disease; and Novel neuromuscular targets, which is in preclinical trial targeting neuromuscular disease. Further, the company collaborates with biopharmaceutical companies and organizations to develop programs in therapeutic areas. The company has collaboration and license agreement with Bayer Consumer Care AG; Novartis Pharma AG; Cancer Research UK; Cancer Research Technology Ltd; and Oxurion NV. Bicycle Therapeutics plc was incorporated in 2009 and is headquartered in Cambridge, the United Kingdom.
Concert Pharmaceuticals
NASDAQ:CNCEConcert Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company, which discovers and develops novel small molecule drugs through the use of deuterium technology. Its product candidates target a range of diseases including autoimmune disorders, inflammation, and central nervous system disorders. The company was founded by Richard H. Aldrich, Roger D. Tung and Christoph H. Westphal on April 12, 2006 and is headquartered in Lexington, MA.
IDEAYA Biosciences
NASDAQ:IDYAIDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.
Mirum Pharmaceuticals
NASDAQ:MIRMMirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.