Core Insights - Opus Genetics, Inc. announced one-year results from its Phase 1/2 study of OPGx-LCA5, showing sustained improvements in vision for patients with severe inherited retinal degeneration due to LCA5 gene mutations [1][2] - The treatment was well tolerated, with no serious adverse events reported, indicating a favorable safety profile [7] Efficacy and Functional Endpoints - Significant improvements in cone-mediated vision were observed, with all treated subjects identifying more objects compared to baseline at 12 months [5] - Visual acuity improved by an average of 0.35 logMAR, equivalent to a 3.5 line improvement across three participants [5] - Full-field stimulus testing showed a 0.86 log improvement in retinal sensitivity at 12 months compared to control eyes [5][6] - Pupillary light reflex responses increased in treated eyes, indicating enhanced cone-mediated vision [12] Study Design - The clinical trial is an open-label, Phase 1/2 study evaluating the safety and efficacy of OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene [8] - The trial has been enrolling both adult and pediatric patients, with dosing for pediatric patients starting in February 2025 [8] Company Pipeline and Future Plans - Opus Genetics is developing gene therapies for multiple inherited retinal diseases, with plans to enter Phase 1/2 trials for the BEST-1 program later this year [2] - Discussions with the U.S. FDA regarding the registration trial design for OPGx-LCA5 are ongoing, with the goal to initiate the study in 2026 [2]
Opus Genetics Announces Presentation of OPGX-LCA5 Gene Therapy Data at ARVO; 12 Month Phase 1/2 Results Support Potential to Restore to Meaningful Vision