Topline six-month efficacy data expected by end of 2027

Phase 3 study follows FDA alignment on registrational trial design through Rare Disease Evidence Principles (RDEP) program

Opus Genetics may submit a BLA based on 6-month efficacy data, with 12-month durability data provided during review

 

RESEARCH TRIANGLE PARK, N.C., Aug. 03, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (NASDAQ:IRD) ("Opus Genetics" or the "Company"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that the last patient has been enrolled in its registrational Phase 3 clinical trial evaluating OPGx-LCA5, the Company’s investigational gene therapy for LCA5-associated inherited retinal disease.

Completion of enrollment marks a significant milestone in the development of OPGx-LCA5 and follows the program’s acceptance into the U.S. Food and Drug Administration’s (FDA) Rare Disease Evidence Principles (RDEP) program in May 2026. The Phase 3 study was designed in collaboration with the FDA to support a potential Biologics License Application (BLA) for what could become the first approved therapy for patients living with LCA5-associated inherited retinal disease.

The registrational Phase 3 trial is evaluating the safety and efficacy of a one-time subretinal administration of OPGx-LCA5 in patients with genetically confirmed LCA5-associated inherited retinal disease. Consistent with the Company’s RDEP discussions with the FDA, the study incorporates innovative evidence-generation approaches appropriate for an ultra-rare disease, including a six-month run-in period in which patients serve as their own control. Participants are currently completing the run-in period, and the Company expects to initiate dosing of OPGx-LCA5 in the fourth quarter of 2026, with topline data expected by the end of 2027.

OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and has been accepted into the FDA’s Rare Disease Evidence Principles (RDEP) program.

The Company continues to expect that OPGx-LCA5 may qualify for a Rare Pediatric Disease Priority Review Voucher upon approval, representing a potentially significant strategic asset.