Ardelyx
NASDAQ:ARDXArdelyx, Inc., a biopharmaceutical company, discovers, develops, and commercializes medicines to treat gastrointestinal and cardiorenal therapeutic areas in the United States and internationally. The company's lead product candidate is tenapanor for the treatment of patients with irritable bowel syndrome with constipation. It also develops XPHOZAH, which is in Phase III clinical trial to reduce serum phosphorus in adults with chronic kidney disease (CKD)on dialysis, or hyperphosphatemia; RDX013, a potassium secretagogue, for the treatment of elevated serum potassium, or hyperkalemia, a problem among patients with kidney and/or heart failure; and RDX020, for adult patients with metabolic acidosis, a serious electrolyte disorder. The company has agreements with Kyowa Kirin, Fosun Pharmaceutical Industrial Development Co. Ltd., and Knight Therapeutics, Inc. for the development and commercialization of tenapanor in their respective territories. The company was formerly known as Nteryx, Inc. and changed its name to Ardelyx, Inc. in June 2008. Ardelyx, Inc. was incorporated in 2007 and is headquartered in Waltham, Massachusetts.
Dynavax Technologies
NASDAQ:DVAXDynavax Technologies Corporation, a commercial stage biopharmaceutical company, focuses on developing and commercializing vaccines in the United States. It markets HEPLISAV-B, a hepatitis B vaccine for prevention of infection caused by all known subtypes of hepatitis B virus in age 18 years and older in the United States and Europe. The company also manufactures and sells CpG 1018, the adjuvant used in HEPLISAV-B. Dynavax Technologies Corporation has a collaboration agreement with Valneva Scotland Limited; Zhejiang Clover Biopharmaceuticals, Inc.; Clover Biopharmaceuticals (Hong Kong) Co., Limited; Biological E. Limited; PT Bio Farma; Medigen Vaccine Biologics; and Serum Institute of India Pvt. Ltd. The company was formerly known as Double Helix Corporation and changed its name to Dynavax Technologies Corporation in September 1996. The company was incorporated in 1996 and is headquartered in Emeryville, California.
Homology Medicines
NASDAQ:FIXXHomology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.
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.
Ocular Therapeutix
NASDAQ:OCULOcular Therapeutix, Inc., a biopharmaceutical company, focuses on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its bioresorbable hydrogel-based formulation technology in the United States. The company markets DEXTENZA, a dexamethasone ophthalmic insert to treat post-surgical ocular inflammation and pain following ophthalmic surgery, as well as allergic conjunctivitis. It is developing AXPAXLI, an axitinib intravitreal implant that is in phase 3 trials for the treatment of wet age-related macular degeneration and other retinal diseases; PAXTRAVA, a travoprost intracameral implant, which is in phase 2 clinical trials for the treatment of open-angle glaucoma or ocular hypertension; OTX-CSI, a cyclosporine intracanalicular insert that has completed phase 2 clinical trials for the treatment of dry eye disease; and OTX-DED, a dexamethasone intracanalicular insert, which is in phase 2 clinical trials for the short-term treatment of the signs and symptoms of dry eye disease. In addition, the company offers modulator for intermediate and late dry age-related macular degeneration; and gene delivery for inherited retinal degenerations and protein biofactory indications. The company has a strategic collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) for the development and commercialization of products using the company's sustained-release hydrogel in combination with Regeneron's large molecule VEGF-targeting compounds for the treatment of retinal diseases; and AffaMed Therapeutics Limited for the development and commercialization of DEXTENZA and OTX-TIC. Ocular Therapeutix, Inc. was incorporated in 2006 and is headquartered in Bedford, Massachusetts.