Deciphera Pharmaceuticals
NASDAQ:DCPHDeciphera Pharmaceuticals, Inc., a biopharmaceutical company, develops drugs to enhance the lives of cancer patients by addressing key mechanisms of drug resistance that limit the rate and durability of response to existing cancer therapies in the United States and internationally. Its lead drug candidate is QINLOCK used for the treatment of gastrointestinal stromal tumors (GIST), as well as in INTRIGUE Phase 3 study to treat second-line GIST. The company is also developing vimseltinib, an orally administered, potent, and highly selective switch-control kinase inhibitor of colony stimulating factor 1 receptor (CSF1R) for the treatment of tenosynovial giant cell tumor (TGCT); and DCC-3116 to treat RAS/RAF mutant cancers that is in the preclinical-stage. Deciphera Pharmaceuticals, Inc. was founded in 2003 and is headquartered in Waltham, Massachusetts.
Global Blood Therapeutics
NASDAQ:GBTGlobal Blood Therapeutics, Inc., a biopharmaceutical company, engages in the discovery, development, and delivery of treatments for underserved patient communities with sickle cell disease (SCD). The company offers Oxbryta tablets, an oral, once-daily therapy for SCD. It also evaluates the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase II a clinical trial of adolescent and pediatric patients with SCD. In addition, the company is developing its lead product candidate inclacumab, novel human monoclonal antibody, which is in Phase III a clinical trial to reduce the incidence of painful vaso-occlusive crises, and resulting hospital admissions; and GBT601, a hemoglobin polymerization inhibitor that is in Phase I development. It has a license and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD and beta thalassemia; and Sanofi S.A. to two early-stage research programs in SCD. Global Blood Therapeutics, Inc. was incorporated in 2011 and is headquartered in South San Francisco, California.
Legend Biotech
NASDAQ:LEGNLegend Biotech Corporation, a clinical-stage biopharmaceutical company, through its subsidiaries, engages in the discovery, development, manufacturing, and commercialization of novel cell therapies for oncology and other indications in the United States, China, and internationally. Its lead product candidate, LCAR- B38M, is a chimeric antigen receptor for the treatment of multiple myeloma (MM). The company also has a portfolio of earlier-stage autologous CAR-T product candidates targeting various cancers, including Non-Hodgkins Lymphoma, acute lymphoblastic leukemia, gastric cancer, esophageal cancer, pancreatic cancer, colorectal cancer, hepatocellular carcinoma, small cell lung cancer, and non-small cell lung cancer. It has collaboration and license agreement with Janssen Biotech, Inc. for the development and commercialization of ciltacabtagene autoleucel. The company was founded in 2014 and is based in Somerset, New Jersey. Legend Biotech Corporation is a subsidiary of Genscript Biotech Corporation.
PTC Therapeutics
NASDAQ:PTCTPTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.
Sage Therapeutics
NASDAQ:SAGESage Therapeutics, Inc., a biopharmaceutical company, develops and commercializes brain health medicines. Its product candidates include ZULRESSO, a CIV injection for the treatment of postpartum depression (PPD) in adults; and ZURZUVAE, a neuroactive steroid, a positive allosteric modulator of GABAA receptors, targeting both synaptic and extrasynaptic GABAA receptors, for the treatment of postpartum depression. Its product pipeline also comprises SAGE-324, a compound that is in Phase II clinical trial to treat essential tremors, as well as has completed Phase I clinical trial for epilepsy and Parkinson's diseases; and SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor, which is in Phase II clinical trial for the treatment of depression, Huntington's disease, Parkinson's diseases, Alzheimer's disease, attention deficit hyperactivity disorder, schizophrenia, and neuropathic pain. The company has a strategic collaboration with Shionogi & Co., Ltd. for the development and commercialization of zuranolone in Japan, Taiwan, and South Korea; and a collaboration and license agreement with Biogen MA Inc. to jointly develop and commercialize SAGE-217 and SAGE-324 products. The company was formerly known as Sterogen Biopharma, Inc. and changed its name to Sage Therapeutics, Inc. in September 2011. Sage Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.