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Axsome Therapeutics, Inc and Ultragenyx Pharmaceutical Inc.

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Axsome Therapeutics, Inc

Axsome Therapeutics, Inc., a biopharmaceutical company, develops and delivers novel therapies for the management of 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, sigma-1 receptor agonist, aminoketone, and CYP2D6 inhibitor indicated for the treatment of major depressive disorder in adults; Sunosi (solriamfetol), a dopamine and norepinephrine reuptake inhibitor, trace amine-associated receptor 1 agonist, and 5-HT1A agonist indicated for the treatment of excessive daytime sleepiness in patients with narcolepsy or obstructive sleep apnea; and Symbravo (MoSEIC meloxicam rizatriptan), or AXS-07, a rapidly absorbed, multi-mechanistic, selective COX-2 inhibitor, and 5-HT1B/1D agonist indicated for the acute treatment of migraine with or without aura. It also develops AXS-05 (dextromethorphan-bupropion), an investigational N-methyl-D-aspartate receptor antagonist, which has completed 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-12 (reboxetine), a highly selective and potent norepinephrine reuptake inhibitor and cortical dopamine modulator, which has completed Phase III trial to treat narcolepsy; AXS-14 (esreboxetine), a selective and potent norepinephrine reuptake inhibitor that has completed Phase III trial for the treatment of fibromyalgia and other conditions; and Solriamfetol, a dopamine and norepinephrine reuptake inhibitor, which is in Phase III trial for treating attention deficit hyperactivity, major depressive, binge eating, and excessive sleepiness associated with shift work disorder. It has a research collaboration agreement with Duke University for evaluating AXS-05 in a Phase 2 clinical trial in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.

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Ultragenyx Pharmaceutical Inc.

Ultragenyx 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, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; 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; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial 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.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.

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