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Arena Pharmaceuticals, Inc., a biopharmaceutical company, focuses on providing novel medicines with pharmacology and pharmacokinetics to patients worldwide. The company's investigational clinical programs include Etrasimod (APD334) that is in Phase III clinical trial for ulcerative colitis, Phase IIb/III clinical trial for Crohn's disease, Phase II clinical trial for alopecia areata, Phase III clinical trial for atopic dermatitis, and Phase IIb clinical trial for eosinophilic esophagitis; Olorinab (APD371), which is in Phase IIb clinical trial for the treatment of abdominal pain associated with irritable bowel syndrome; APD418 that is in Phase II clinical trial for acute heart failure; and Temanogrel for coronary microvascular obstruction that is in Phase II clinical trial. It also develops ralinepag (APD811), which is in Phase III clinical trial for pulmonary arterial hypertension. The company has collaboration agreements with United Therapeutics Corporation; Everest Medicines Limited; Eisai Co., Ltd. and Eisai Inc.; Boehringer Ingelheim International GmbH; Beacon Discovery, Inc.; and Aristea Therapeutics, Inc. Arena Pharmaceuticals, Inc. was incorporated in 1997 and is based in Park City, Utah. As of March 11, 2022, Arena Pharmaceuticals, Inc. operates as a subsidiary of Pfizer Inc.

Biohaven Ltd., together with its subsidiaries, focuses on discovering, developing, and commercializing therapies for immunology, neuroscience, and oncology worldwide. The company's pipeline products include Troriluzole, which is in Phase 3 clinical trial for the treatment of neurological and neuropsychiatric illnesses; BHV-5500 that blocks glutamate signaling mediated by post-synaptic NMDA receptors; Taldefgrobep Alfa, which is in Phase 3 clinical trial for the treatment of spinal muscular atrophy and obesity; BHV-7000, a candidate in Phase 2/3 clinical trials for the treatment of focal and generalized epilepsy, bipolar disorder, and major depressive disorder; BHV-2100 that is in Phase 1 clinical trials for the treatment of migraines and neuropathic pain; and BHV-8000, a product candidate in Phase 1 clinical trials for the treatment of early Alzheimer's and Parkinson's disease, sclerosis, and amyloid-related imaging abnormalities. It also offers BHV-1300, a product candidate in Phase 1 clinical trials to treat rheumatoid arthritis; BHV-1310 for the treatment of generalized myasthenia gravis and acute exacerbations or flares; BHV-1400 to treat IgA Nephropathy; and BHV-1600 for the treatment of dilated cardiomyopathy. In addition, the company develops BHV-1100, a product candidate in Phase 1a/1b clinical trials for multiple myeloma patients; BHV-1510, a preclinical product that targets carcinomas; and BHV-1500 for Hodgkin's lymphoma. It has license, development, and commercialization agreements with Yale University, AstraZeneca, University of Connecticut, Artizan Biosciences Inc., Reliant Glycosciences LLC, Katholieke Universiteit Leuven, BMS, and Highlightll Pharmaceutical Co. Ltd. The company was formerly known as Biohaven Research Ltd and changed its name to Biohaven Ltd. in September 2022. Biohaven Ltd. was incorporated in 2022 and is headquartered in New Haven, Connecticut.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

MyoKardia, 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, 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.