Agios Pharmaceuticals
NASDAQ:AGIOAgios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
Blueprint Medicines
NASDAQ:BPMCBlueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of indolent SM, and other mast cell disorders. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered thyroid carcinoma, and medullary thyroid carcinoma; BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, 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.
Ultragenyx Pharmaceutical
NASDAQ:RAREUltragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.
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.