Cytokinetics
NASDAQ:CYTKCytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases. The company develops small molecule drug candidates primarily engineered to impact muscle function and contractility. Its drug candidates include omecamtiv mecarbil, a novel cardiac myosin activator that is in Phase III clinical trial in patients with heart failure. The company also develops CK-136, a novel cardiac troponin activator that is in Phase I clinical trial; CK-586, a small molecule cardiac myosin inhibitor, that is in Phase I clinical trial; and aficamten, a novel cardiac myosin inhibitor, which is in Phase III clinical trial for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. Cytokinetics, Incorporated has a strategic alliance with Ji Xing Pharmaceuticals Limited. The company was incorporated in 1997 and is headquartered in South San Francisco, 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.
Praxis Precision Medicines
NASDAQ:PRAXPraxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. It is developing ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase III clinical trial for the treatment of essential tremor; PRAX-562 for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEE); and PRAX-628 to treat focal epilepsy. The company also develops PRAX-222 for the treatment of pediatric patients with early-onset SCN2A-DEE; PRAX-020 to treat KCNT1 related epilepsies; PRAX-080 for the treatment of PCDH19; and PRAX-090 and PRAX-100 for SYNGAP1 and SCN2A-LoF. It has a license agreement with RogCon Inc.; a research collaboration and license agreement with Ionis Pharmaceuticals, Inc.; a strategic collaboration and license agreement with UCB Biopharma SRL; and collaboration with The Florey Institute to develop three novel ASOs. The company was incorporated in 2015 and is based in Boston, Massachusetts.
Travere Therapeutics
NASDAQ:RTRXTravere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.
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.