- Opus Genetics completed enrollment in its registrational phase 3 trial of OPGx-LCA5 for LCA5-associated inherited retinal disease
- The FDA-designed study uses a self-controlled run-in period and is intended to support a future Biologics License Application
- Dosing is expected to begin in the fourth quarter of 2026, with topline data anticipated by the end of 2027
Opus Genetics has completed patient enrollment in its registrational phase 3 clinical trial evaluating OPGx-LCA5, an investigational gene therapy for patients with LCA5-associated inherited retinal disease
The company announced that the final patient has been enrolled in the study, which is intended to support a future Biologics License Application with the FDA. If approved, OPGx-LCA5 could become the first therapy available for patients with LCA5-associated inherited retinal disease
The enrollment milestone follows the FDA’s acceptance of the program into its Rare Disease Evidence Principles (RDEP) program in May 2026. According to the company, the phase 3 study was designed in collaboration with the agency to address the challenges of conducting registrational trials in ultra-rare diseases
“Achieving full enrollment in our registrational phase 3 trial is a significant milestone for the OPGx-LCA5 program and reflects the dedication of participating patients and families, our clinical investigators, and our team,” said George Magrath, MD, chief executive officer of Opus Genetics. “With enrollment now complete, we remain on track to initiate dosing in the fourth quarter of 2026, report topline data by the end of 2027, and continue advancing OPGx-LCA5 to bring the first potential treatment to patients with LCA5-associated inherited retinal disease.”
The registrational study 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. In line with discussions held under the FDA’s RDEP program, the trial incorporates an evidence-generation strategy tailored to an ultra-rare disease population, including a 6-month run-in period during which participants serve as their own controls before receiving treatment
Patients are currently completing the run-in phase, with dosing expected to begin in the fourth quarter of 2026. Opus Genetics anticipates reporting topline efficacy and safety results by the end of 2027
OPGx-LCA5 has received multiple FDA regulatory designations, including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy status. The therapy’s inclusion in the RDEP program is intended to facilitate the development of evidence supporting regulatory review in rare diseases where conventional trial designs may not be feasible
