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HRMY vs IRD

Harmony Biosciences Holdings, Inc. Common Stock and Opus Genetics, Inc. Common Stock

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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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Opus Genetics, Inc. Common Stock

Opus Genetics, Inc., a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of inherited retinal diseases (IRDs). It develops OPGx-LCA5, an early-onset retinal degeneration that is in Phase 1/2 clinical trial for the treatment of LCA5-associated IRD; and OPGx-BEST1 to treat BEST1-associated retinal disease. The company also develops various pre-clinical IRD programs, including OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations; OPGx-RDH12 that is designed to restore protein expression and halt functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase gene; OPGx-MERTK, an AAV vector for the treatment of MERTK IRD; OPGx-NMNAT1, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 gene; and OPGx-CNGB1, an AAV gene therapy for retinitis pigmentosa due to mutations in the CNGB1 gene. In addition, it develops Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as for the treatment of presbyopia and dim light or night vision disturbances; and APX3330, a small-molecule inhibitor of Ref-1 for the treatment of diabetic retinopathy. The company was formerly known as Ocuphire Pharma, Inc. Opus Genetics, Inc. was founded in 2018 and is headquartered in Durham, North Carolina.

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