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Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on ophthalmology. The company is dedicated to discovering and commercializing innovative therapies to address significant unmet medical needs in patients suffering from refractive and retinal eye disorders. Its portfolio includes Phentolamine Ophthalmic Solution, currently used to reverse pharmacologically induced mydriasis (pupil dilation). This solution is also advancing through Phase III clinical trials for the treatment of presbyopia and impaired vision in dim light or at night. A leading candidate in its retinal pipeline is APX3330, a small-molecule inhibitor targeting the reduction oxidation effector factor-1 protein. APX3330 has successfully completed Phase II clinical trials for diabetic retinopathy. Furthermore, Opus Genetics is developing APX2009 and APX2014, two preclinical product candidates also aimed at various retinal indications. Originally founded in 2018 as Ocuphire Pharma, Inc., the company maintains its headquarters in Farmington Hills, Michigan.
Opus Genetics reached alignment with the FDA on the Phase 3 design for OPGx-LCA5, allowing for a BLA submission based on 6-month efficacy data with 12-month durability data to follow during review.
The registrational trial plans to enroll eight participants using a six-month run-in period as a natural history control, with seven patients already enrolled and dosing anticipated to begin in the fourth quarter of 2026.
The trial is designed with greater than 90% statistical power to detect a mean improvement of at least 7 decibels in retinal sensitivity, supported by Phase 1/2 data showing an approximate 10.5 dB improvement.