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HRMY vs PTCT

Harmony Biosciences Holdings, Inc. Common Stock and PTC Therapeutics, 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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PTC Therapeutics, Inc.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living 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 inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. 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. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

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