Opus Genetics Secures FDA Alignment on Phase 3 OPGx-LCA5 Trial with 7 dB Endpoint
IRD•Opus Genetics obtained FDA alignment on its Phase 3 trial for OPGx-LCA5, confirming eight participants, a six-month run-in, and a 7 dB improvement in retinal sensitivity as the primary endpoint. FDA permits BLA submission on six-month efficacy with 12-month durability follow-up, with dosing to start in 4Q 2026.
1. FDA Alignment on Trial Design
Opus Genetics reached FDA alignment in a Type B RDEP meeting on its registrational Phase 3 design for OPGx-LCA5 targeting LCA5-associated inherited retinal disease. The meeting confirmed key protocol elements to support a pivotal registrational filing.
2. Study Structure and Endpoints
The Phase 3 study will enroll eight participants able to complete microperimetry in both eyes, including a six-month run-in period as each subject’s own control. The primary efficacy endpoint is a mean improvement of at least 7 dB in retinal sensitivity, with over 90% statistical power to detect this treatment effect.
3. BLA Submission Pathway
FDA indicated that a Biologics License Application may be submitted based on compelling six-month efficacy data, with 12-month durability data provided during the review. This pathway could accelerate access to OPGx-LCA5 if results meet the threshold.
4. Timeline and Next Steps
Seven of eight participants have completed enrollment and are in the run-in phase. Opus Genetics expects to initiate Phase 3 dosing in the fourth quarter of 2026, and OPGx-LCA5 may qualify for a Priority Review Voucher, enhancing its regulatory and commercial strategy.
