Opus Genetics announces completion of Type B RMAT meeting with FDA

Opus Genetics (IRD) announced completion of a Type B Regenerative Medicine Advanced Therapy meeting with the U.S. Food and Drug Administration regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis caused by mutations in the LCA5 gene. The meeting provided constructive feedback from the FDA on key elements of Opus’s registration strategy, including Chemistry, Manufacturing and Controls (CMC), and the pivotal trial design. The FDA acknowledged the significant unmet medical need for individuals with LCA5-related blindness and reaffirmed its commitment to regulatory flexibility for rare genetic diseases. To date in the Phase 1/2 portion of the trial, six late-stage participants have been treated with OPGx-LCA5, and all six have experienced clinically meaningful improvements in vision, providing evidence of biological activity with the potential for functional restoration of vision in individuals with advanced disease. Opus will incorporate the FDA’s feedback into its updated clinical development and CMC plans for the Phase 3 portion of the study to include enrolling as few as 8 participants in a single arm, 12-month study utilizing an adaptive design, which provides flexibility on endpoints and number of participants, reflective of LCA5 as a rare condition with an urgent medical need. The Company expects the Phase 3 portion of the trial will include a run-in period prior to dosing to evaluate the natural history of each participant to serve as their own control in the study. Opus is actively identifying patients for this segment and has enrolled the first participant. Efficacy and safety will be assessed using measures such as visual acuity, full-field stimulus testing, microperimetry, and Multi-Luminance Orientation and Mobility Test. Following availability of validated clinical drug supply manufactured with the intended commercial processes, dosing with OPGx-LCA5 is anticipated in the second half of 2026, with topline clinical data expected approximately one year later.