Annexon
NASDAQ:ANNXAnnexon, Inc., a clinical-stage biopharmaceutical company, discovers and develops medicines for treating inflammatory-related diseases. Its lead candidate is ANX005, an investigational full-length monoclonal antibody, which is in Phase 3 clinical trial for the treatment of patients with guillain-barré syndrome; completed Phase II clinical trial for treating Huntington's disease; and in Phase II clinical trial for the treatment of amyotrophic lateral sclerosis. The company is also developing ANX007, an antigen-binding fragment (Fab) that is in Phase 3 program for the treatment of patients with geographic atrophy; and ANX1502, a novel oral small molecule inhibitor, which is in Phase 1 clinical trials for autoimmune indications. In addition, it develops ANX009, a C1q-blocking Fab that is in Phase I clinical trial for treating patients with lupus nephritis. The company was incorporated in 2011 and is headquartered in Brisbane, California.
Arcturus Therapeutics
NASDAQ:ARCTArcturus Therapeutics Holdings Inc., a late-stage clinical messenger RNA medicines and vaccine company, focuses on the development of infectious disease vaccines and other products within liver and respiratory rare diseases. Its technology platforms include LUNAR lipid-mediated delivery and STARR mRNA. The company is developing ARCT-810 (LUNAR-OTC), a mRNA-based therapeutic candidate, which is in Phase 2 clinical trial for treating ornithine transcarbamylase deficiency; and ARCT-154 (LUNAR-COV19), a mRNA vaccine candidate that is in Phase 3 arm of a Phase 1/2/3 study in Vietnam for the treatment of COVID-19, as well as ARCT-032 (LUNAR-CF), a mRNA therapeutic candidate for cystic fibrosis. Its product pipeline includes, ARCT-2301 for bivalent: ancestral/omicron which is in Phase 3; ARCT-2303 for monovalent that is in Phase 3; ARCT-2138 for quadrivalent which is in Phase 1; and LUNAR-FLU which is in pre-clinical trial. Arcturus Therapeutics Holdings Inc. was founded in 2013 and is headquartered in San Diego, California.
ChemoCentryx
NASDAQ:CCXIChemoCentryx, Inc., a biopharmaceutical company, focuses on the development and commercialization of new medications for inflammatory disorders, autoimmune diseases, and cancer in the United States. It offers TAVNEOS (avacopan), an orally administered selective C5aR inhibitor for the treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis. The company also develops TAVNEOS for the treatment of patients with severe hidradenitis suppurativa, as well as patients with complement 3 glomerulopathy, and lupus nephritis. In addition, it develops CCX559, an orally administered inhibitor for programmed death protein 1/programmed death-ligand 1 for the treatment of various cancers; and CCX507, an orally administered inhibitor of the chemokine receptor known as CCR9, which has completed Phase I clinical trial for the treatment of inflammatory bowel disease. Further, the company has early-stage drug candidates that targets Th17 driven diseases and CCR6. ChemoCentryx, Inc. was incorporated in 1996 and is headquartered in San Carlos, California.
Epizyme
NASDAQ:EPZMEpizyme, Inc., a commercial-stage biopharmaceutical company, discovers, develops, and commercializes novel epigenetic medicines for patients with cancer and other diseases in the United States. The company offers Tazemetostat for the treatment of metastatic or locally advanced epithelioid sarcoma for adults and pediatric patients. It also develops R-CHOP in front-line patients with high risk diffuse large B-cell lymphoma; and PARP inhibitor in patients with PARPi-resistant solid tumors, such as castration-resistant prostate cancer, small cell lung cancer, and others. In addition, the company develops Tazemetostat in patients with INI1-negative tumors in adults and pediatrics; PRMT5 inhibitor for patients with solid tumors; and PRMT1 inhibitor. It has collaboration agreements with Roche Molecular Systems, Inc.; Lymphoma Academic Research Organization; Eisai Co. Ltd.; HUTCHMED (China) Limited; and Roche Sequencing Solutions, Inc. The company was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
Travere Therapeutics
NASDAQ:TVTXTravere Therapeutics, Inc., a biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases. Its products include FILSPARI (sparsentan), a once-daily, oral medication designed to target two critical pathways in the disease progression of IgA Nephropathy (endothelin 1 and angiotensin-II); and Thiola and Thiola EC (tiopronin tablets) for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. The company's clinical-stage programs consist of Sparsentan, a novel investigational product candidate, which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis in the U.S. and Europe; and Pegtibatinase (TVT-058), a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria. It has a cooperative research and development agreement with National Institutes of Health's National Center for Advancing Translational Sciences and Alagille Syndrome Alliance for the identification of potential small molecule therapeutics for Alagille syndrome. The company was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in November 2020. Travere Therapeutics, Inc. was incorporated in 2008 and is headquartered in San Diego, California.