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Axsome Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel therapies for central nervous system (CNS) disorders in the United States. The company's commercial product portfolio includes Auvelity (dextromethorphan-bupropion), a N-methyl-D-aspartate receptor antagonist with multimodal activity indicated for the treatment of major depressive disorder; and Sunosi (solriamfetol), a medication indicated to the treatment of excessive daytime sleepiness in patients with narcolepsy or obstructive sleep apnea. It is also developing AXS-05, which is in Phase III clinical trial to treat Alzheimer's disease agitation, as well as that has completed phase II clinical trial for the treatment of smoking cessation; AXS-07, an investigational medicine that has completed Phase III trials for the acute treatment of migraine; AXS-12, an investigational medicine, which is in Phase III trial to treat narcolepsy; AXS-14, a selective and potent norepinephrine reuptake inhibitor that has completed Phase III trial for the treatment of fibromyalgia and other conditions; and solriamfetol, a dual-acting dopamine and norepinephrine reuptake inhibitor, which has completed a Phase 2 trial for treating attention-deficit/hyperactivity disorder, and is in phase 2 major depressive, binge eating, and shift work disorder. Axsome Therapeutics, Inc. has a research collaboration agreement with Duke University for evaluating AXS-05 in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.

Deciphera 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.

FibroGen, Inc., a biopharmaceutical company, discovers, develops, and commercializes therapeutics to treat serious unmet medical needs. Its lead product candidates are Pamrevlumab, a human monoclonal antibody targeting connective tissue growth factor that is in Phase III clinical development for the treatment of locally advanced pancreatic cancer; and Roxadustat, an oral small molecule inhibitor of hypoxia-inducible factor prolyl hydroxylase activity, which has completed Phase III clinical development for the treatment of anemia in chronic kidney disease in China, Europe, Japan, and other countries, as well as in Phase III clinical development for anemia related with myelodysplastic syndromes. The company has collaboration agreements with Astellas Pharma Inc. and AstraZeneca AB. FibroGen, Inc. was incorporated in 1993 and is headquartered in San Francisco, California.

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.

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.