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.
HUTCHMED
NASDAQ:HCMHUTCHMED (China) Limited, together with its subsidiaries, discovers, develops, and commercializes targeted therapeutics and immunotherapies for cancer and immunological diseases in Hong Kong and internationally. The company develops Savolitinib for the treatment of non-small cell lung cancer (NSCLC), papillary renal cell carcinoma (RCC), and gastric cancer (GC); and Fruquintinib, an inhibitor for colorectal cancer (CRC), breast cancer, gastric cancer, microsatellite stable-CRC endometrial cancer (EMC), NSCLC, RCC, gastrointestinal, cervical, and solid tumors. It also develops Surufatinib, which is used for the treatment of pancreatic neuroendocrine tumor (NET), non-pancreatic NET, neuroendocrine carcinoma, SCLC, biliary tract cancer, and solid tumors; and Sovleplenib that treats hematological cancers and certain chronic immune diseases. In addition, it develops Tazemetostat for the treatment of certain epithelioid sarcoma and follicular lymphoma patients; HMPL-306, an inhibitor for hematological malignancies, gliomas, chondrosarcomas, cholangiocarcinomas, and solid tumors; HMPL-760, a Bruton's tyrosine kinase inhibitor; HMPL-453 for intrahepatic cholangiocarcinoma and solid tumors; HMPL-295 and HMPL-415 for solid tumors; HMPL-653 for metastatic solid tumors and tenosynovial giant cell tumors; and HMPL-A83 for the treatment of malignant neoplasms. It has collaboration agreements with AstraZeneca AB (publ), Lilly (Shanghai) Management Company Limited, Takeda, BeiGene Ltd., Inmagene Biopharmaceuticals Co. Ltd., Innovent Biologics Co., Inc., Genor Biopharma Co. Ltd., Shanghai Junshi Biosciences Co. Ltd., and Epizyme, Inc. The company was formerly known as Hutchison China MediTech Limited and changed its name to HUTCHMED (China) Limited in May 2021. HUTCHMED (China) Limited was incorporated in 2000 and is headquartered in Hong Kong, Hong Kong.
MyoKardia
NASDAQ:MYOKMyoKardia, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is Mavacamten, an orally administered allosteric modulator of cardiac myosin, which is in Phase III clinical trial for the treatment of hypertrophic cardiomyopathy (HCM). The company also develops Danicamtiv, an orally administered small molecule, which completed Phase I and Phase IIa clinical trials on patients with dilated cardiomyopathy. Its preclinical programs include MYK-224, a small molecule program for the treatment of HCM targeting hypercontractility and impaired relaxation; ACT-1 for the treatment of diastolic dysfunction; and LUS-1 for the treatment of diastolic dysfunction. Myokardia, Inc. was founded in 2012 and is headquartered in Brisbane, California.
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.
uniQure
NASDAQ:QUREuniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.