Opus Genetics Announces FDA Alignment on Phase 3 Registrational Trial Design for OPGx-LCA5 in LCA5-Associated Inherited Retinal Disease

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Successful Type B RDEP Meeting confirms FDA alignment on pivotal Phase 3 study design FDA indicates Opus Genetics may submit a BLA based on 6-month efficacy data, with 12-month durability data provided during review Company expects to initiate Phase 3 dosing in 4Q 2026 RESEARCH TRIANGLE PARK, N.C., July 06, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc . (Nasdaq: IRD) (the "Company" or "Opus Genetics"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) in a Type B Rare Disease Evidence Principles (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 (dB) 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 (BLA) based on compelling efficacy at the six-month primary endpoint, with 12-month durability data submitted during the BLA review process. "We believe this alignment with the FDA provides a clear roadmap toward a potential BLA submission and, most importantly, brings us one step closer to delivering a treatment for patients living with LCA5-associated blindness," said George Magrath, M.D., Chief Executive Officer, Opus Genetics. "We have had a very positive experience working with the FDA through the RDEP program to develop a registrational pathway for this ultra-rare inherited retinal disease. With enrollment nearly complete, we anticipate initiating dosing in the Phase 3 study during the fourth quarter of 2026. In addition, OPGx-LCA5 may qualify to receive a Priority Review Voucher, representing a potentially significant strategic asset."

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