Core Insights - Opus Genetics has reached an agreement with the FDA on a Special Protocol Assessment (SPA) for a Phase 3 clinical trial of oral APX3330 targeting moderate to severe nonproliferative diabetic retinopathy (NPDR) [1][2][3] - The primary endpoint of the trial is a reduction in 3-step or greater worsening on the binocular diabetic retinopathy severity scale (DRSS) compared to placebo [2] - APX3330 has shown potential in previous trials to slow or prevent the progression of diabetic retinopathy and has a favorable safety profile [2][4] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases (IRDs) and other ophthalmologic disorders [5] - The company’s pipeline includes AAV-based gene therapies for various retinal diseases and small-molecule drugs like APX3330 [5] - APX3330 is a first-in-class oral inhibitor of the transcription factor regulator Ref-1, targeting pathways involved in angiogenesis, oxidative stress, and inflammation [4] Market Context - Diabetic retinopathy is a leading cause of blindness among working-age adults, affecting approximately 10 million patients in the U.S. [3] - The successful development and approval of APX3330 could provide a transformative treatment option for NPDR patients [3]
Opus Genetics Receives FDA Agreement Under Special Protocol Assessment for Phase 3 Trial of APX3330 in Diabetic Retinopathy