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IRD vs ZVRA

Opus Genetics, Inc. Common Stock and Zevra Therapeutics, Inc. Common Stock

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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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Zevra Therapeutics, Inc. Common Stock

Zevra Therapeutics, Inc., a commercial-stage company, focuses on addressing unmet needs for the treatment of rare diseases in the United States. The company develops its products through Ligand Activated Technology platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase 1/2 clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 3 clinical trial for the treatment of vascular Ehlers Danlos syndrome. In addition, it offers AZSTARYS, a once-daily treatment for attention deficit and hyperactivity disorder in patients aged six years and older; OLPRUVA to treat urea cycle disorders; and MIPLYFFA for the treatment of niemann pick disease type C, an ultra-rare neurodegenerative lysosomal storage disorder. The company has collaboration and license agreement with Commave Therapeutics SA to develop, manufacture and commercialize the company's product candidates containing SDX and d-methylphenidate; and license agreement with Acer and Relief Therapeutics, Inc. to develop and commercialize rights for OLPRUVA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.

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