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HRMY vs RGNX

Harmony Biosciences Holdings, Inc. Common Stock and REGENXBIO Inc.

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Harmony Biosciences Holdings, Inc. Common Stock

Harmony Biosciences Holdings, Inc., a commercial-stage pharmaceutical company, focuses on developing and commercializing therapies for patients with rare and other neurological diseases in the United States. The company offers WAKIX (pitolisant), a molecule with a novel mechanism of action for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It also develops Pitolisant that is in Phase 3 clinical trial for the treatment of Prader-Willi Syndrome (PWS); in Phase 2 clinical trial to treat Myotonic Dystrophy (DM1); and is in Phase 1 clinical trials for the treatment of Pitolisant Gastro-Resistant (GR) and High-Dose (HD). In addition, the company's products under development include BP1.15205, an orexin 2 receptor agonist for the treatment of narcolepsy and other potential indications; and HBS-102, a melanin-concentrating hormone receptor type 1 (MCHR1) antagonist. Further, it develops ZYN-002 that is in a Phase 3 registrational trial for the treatment of Fragile X Syndrome (FXS), rare genetic disorder that causes inherited intellectual disability and autism spectrum disorder; and 22q Deletion Syndrome, a disorder caused by a small missing piece on the long arm of the 22nd chromosome. Additionally, the company is developing EPX-100 (clemizole hydrochloride), a serotonin (5HT-2) receptor agonist to treat dravet syndrome and lenox-gastaut syndrome; and EPX-200 (liquid formulation of lorcaserin), a selective 5HT-2C agonist to treat developmental and epileptic encephalopathies (DEE). The company was formerly known as Harmony Biosciences II, Inc. and changed its name to Harmony Biosciences Holdings, Inc. in February 2020. Harmony Biosciences Holdings, Inc. was incorporated in 2017 and is headquartered in Plymouth Meeting, Pennsylvania.

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REGENXBIO Inc.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

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