Akouos
NASDAQ:AKUSAkouos, Inc., a precision genetic medicine company, developing gene therapies to restore, improve, and preserve physiologic hearing for individuals. The company's precision genetic medicine platform incorporates a proprietary adeno-associated viral vector library and a novel delivery approach. Its lead product candidate is AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. The company is also developing AK-CLRN1 for the auditory manifestations of Usher syndrome 3A, or USH3A; and AK-antiVEGF for vestibular schwannoma. In addition, its precision genetic medicine platform addresses hearing loss related to genes needed for supporting cell function. The company was incorporated in 2016 and is based in Boston, Massachusetts.
Clovis Oncology
NASDAQ:CLVSClovis Oncology, Inc, a biopharmaceutical company, focuses on acquiring, developing, and commercializing anti-cancer agents in the United States, Europe, and internationally. Its commercial product includes Rubraca (rucaparib) tablet, a small molecule poly ADP-ribose polymerase inhibitor, used as monotherapy for the treatment of patients with deleterious BRCA mutation associated advanced ovarian cancer, who have been treated with two or more chemotherapies, and selected for therapy by an FDA-approved companion diagnostic for Rubraca. As of 4/6/18, Rubraca® (rucaparib) is also approved by the FDA for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. FDA granted regular approval for Rubraca in this second, broader and earlier-line indication on a priority review timeline based on positive data from the phase 3 ARIEL3 clinical trial. Biomarker testing is not required for patients to be prescribed Rubraca in this maintenance treatment indication.
Chimerix
NASDAQ:CMRXChimerix, Inc., a biopharmaceutical company, develops medicines to improve and extend the lives of patients facing deadly diseases. Its pipeline products include ONC201 a program that is in Phase 3 clinical trial for treating patients with H3 K27M-mutant diffuse glioma, as well as in Phase 2 clinical trial for the treatment of rare neuroendocrine tumors; and ONC206, an imipridone, Dopamine Receptor D2 (DRD2) antagonist, and caseinolytic protease P (ClpP) agonist, which is in Phase 1 clinical trial for adult and pediatric patients with primary central nervous system tumors. The company also develops ONC212, an imipridone agonist of the orphan G protein-coupled receptors (GPCR) tumor suppressor GPR132, as well as ClpP for oncology indications; and CMX521, a nucleoside analog antiviral drug candidate for the treatment of SARS-CoV-2. It has license agreement with SymBio Pharmaceuticals to develop and commercialize TEMBEXA for human diseases other than orthopoxviruses, including smallpox. Chimerix, Inc. was incorporated in 2000 and is headquartered in Durham, North Carolina.
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.
Inventiva
NASDAQ:IVAInventiva S.A., a clinical-stage biopharmaceutical company, focuses on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH) and other diseases. Its lead product candidate is Lanifibranor, which is in Phase III clinical trial to treat NASH. The company also develops Odiparcil for the treatment of patients with mucopolysaccharidoses type VI. In addition, it has a pipeline of earlier stage programs in oncology and other diseases. The company was founded in 2011 and is headquartered in Daix, France.