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AMLX vs IRD

Amylyx Pharmaceuticals, Inc. Common Stock and Opus Genetics, Inc. Common Stock

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Amylyx Pharmaceuticals, Inc. Common Stock

Amylyx Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, engages in the discovery and development of treatment options for neurodegenerative diseases and endocrine conditions in the United States. Its lead products candidate includes avexitide, an investigational, first-in-class glucagon-like peptide-1, or GLP-1 receptor antagonist, which is in phase 3 clinical trial to treat post-bariatric hypoglycemia and congenital hyperinsulinism. The company also develops AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol which is phase 2 clinical trial for the treatment of wolfram syndrome, as well as in phase 2b/3 clinical trial to treat progressive supranuclear palsy; AMX0114, which is in phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis; and long-acting GLP-1 receptor antagonist to treat post-bariatric hypoglycemia and other rare diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.

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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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