Alkermes
NASDAQ:ALKSAlkermes plc, a biopharmaceutical company, researches, develops, and commercializes pharmaceutical products to address unmet medical needs of patients in therapeutic areas in the United States, Ireland, and internationally. It has a portfolio of proprietary commercial products for the treatment of alcohol dependence, opioid dependence, schizophrenia and bipolar I disorder and a pipeline of clinical and preclinical product candidates in development for neurological disorders. Its marketed products include ARISTADA, an intramuscular injectable suspension for the treatment of schizophrenia; ARISTADA INITIO for the treatment of schizophrenia in adults; VIVITROL for the treatment of alcohol and prevention of opioid dependence; and LYBALVI, an oral atypical antipsychotic drug candidate for the treatment of adults with schizophrenia and bipolar I disorder. It has collaboration agreements primarily with Janssen Pharmaceutica N.V., Janssen Pharmaceutica Inc, and Janssen Pharmaceutica International. The company also offers proprietary technology platforms to third parties to enable them to develop, commercialize, and manufacture products. Alkermes plc was founded in 1987 and is headquartered in Dublin, Ireland.
BridgeBio Pharma
NASDAQ:BBIOBridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.
Mirati Therapeutics
NASDAQ:MRTXMirati Therapeutics, Inc., a commercial-stage oncology company, develops novel therapeutics to address the genetic and immunological promoters of cancer in the United States. The company provides KRAZATI, an oral targeted treatment option for adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung (NSCLC), as well as in clinical development as a monotherapy and in combination with other agents. It also develops Sitravatinib, an investigational spectrum-selective kinase inhibitor in Phase 3 clinical development that inhibits receptor tyrosine kinases (RTKs), including TAM family receptors, split family receptors, and RET, which overcomes resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment that enhances antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation. In addition, the company pipeline comprises the MRTX1719, an investigational synthetic lethal PRMT5 inhibitor designed to target the PRMT5/methylthioadensoine (MTA) complex and is in clinical development; MRTX0902, a selective SOS1 inhibitor that improves anti-tumor efficacy in combination with targeted mitogen-activated protein kinase (MAPK)-pathway inhibitors, and is in clinical development; and MRTX1133, an investigational lead KRAS G12D compound. It has a collaboration and license agreement with BeiGene, Ltd. to develop, manufacture, and commercialize sitravatinib; and Zai Lab Ltd. to research, develop, manufacture, and commercialize adagrasib in various indications. Mirati Therapeutics, Inc. was founded in 1995 and is headquartered in San Diego, California.
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.
United Therapeutics
NASDAQ:UTHRUnited Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. The company offers Tyvaso DPI, an inhaled dry powder via pre-filled and single-use cartridges; Tyvaso, an inhaled solution via ultrasonic nebulizer; Remodulin (treprostinil) injection to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Orenitram, a tablet dosage form of treprostinil, to delay disease progression and improve exercise capacity in PAH patients; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. It also markets and sells Unituxin (dinutuximab) injection, a monoclonal antibody for treating high-risk neuroblastoma; and Remunity Pump, which contains a pump and separate controller for Remodulin. In addition, the company engages in developing RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Nebulized Tyvaso, for the treatment of idiopathic pulmonary fibrosis, as well as xenografts, which are development-stage organ products. It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.