Opus Genetics has reached alignment with the US 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 8 participants who are able to complete microperimetry testing with both eyes treated. The study is also expected to include a 6-month run-in period, allowing each participant to serve as their own natural history control prior to receiving treatment. Seven of the 8 planned participants have already been enrolled and are currently completing the run-in period, and the company expects to initiate dosing in the 4^th 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 percent statistical power to detect a treatment effect of at least 7 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.

George Magrath, MD, Chief Executive Officer (CEO), Opus Genetics, said, “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. 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 4^th quarter of 2026. In addition, OPGx-LCA5 may qualify to receive a Priority Review Voucher, representing a potentially significant strategic asset.”

The FDA indicated that Opus Genetics may submit a Biologics License Application (BLA) based on compelling efficacy at the 6-month primary endpoint, with 12-month durability data submitted during the BLA review process.

Jean Bennett, MD, PhD, Co-Founder and Board Member, Opus Genetics, added, “LCA5-associated inherited retinal disease is among the most severe forms of childhood blindness, with patients experiencing profound vision loss at an early age and no approved treatment options. We are encouraged by the FDA's continued engagement and look forward to advancing this important program as rapidly as possible for patients and families.”