Opus Genetics Aligns with FDA to Advance LCA5 Clinical Trial

Opus Genetics announced that it has reached alignment with the FDA in a Type B Rare Disease Evidence Principles, or RDEP, meeting on the design of its registrational Phase 3 clinical trial evaluating OPGx-LCA5 for LCA5-associated IRD, an early-onset severe inherited retinal dystrophy. Opus Genetics has received the meeting minutes from the Type B meeting confirming several key elements of the trial. The Phase 3 study is expected to enroll eight participants who are able to complete microperimetry testing with both eyes treated. The study is also expected to include a six-month run-in period, allowing each participant to serve as their own natural history control prior to receiving treatment. Seven of the eight planned participants have already been enrolled and are currently completing the run-in period, and the company expects to initiate dosing in the fourth quarter of 2026. The primary efficacy endpoint is a mean improvement of at least 7 decibels in retinal sensitivity across the central 16 test loci, a clinically meaningful measure of visual function. The Phase 3 study is designed with greater than 90% statistical power to detect a treatment effect of at least seven decibels. The Phase 1/2 trial supports this outcome measure as those participants able to complete microperimetry demonstrated an average improvement of approximately 10.5 dB. Importantly, the FDA indicated that Opus Genetics may submit a Biologics License Application based on compelling efficacy at the six-month primary endpoint, with 12-month durability data submitted during the BLA review process.