Opus Genetics has reached alignment with the US Food and Drug Administration (FDA) on the Phase 3 clinical trial design for OPGx-LCA5, the company’s emerging LCA5 gene therapy. If OPGx-LCA5 is safe and meets its primary outcome measure in the trial at six months, Opus will likely seek FDA approval by submitting a Biologics License Application or BLA. Treatment durability data at 12 months will also be considered by the FDA during its review.
Leber congenital amaurosis 5 (LCA5) is one of the rarest and most severe forms of LCA affecting approximately 5,000 globally.
The Phase 3 trial investigators plan to treat both eyes in eight patients. The trial includes a pre-treatment run-in period in which the natural history (i.e., the natural disease course) for each planned participant will be evaluated. Seven of the eight patients are currently enrolled in the run-in study. Investigators plan to begin dosing patients in the fourth quarter of 2026.
Investigators will use microperimetry, which measures retinal sensitivity at different loci (points) in the retina, as the primary outcome measure in the Phase 3 clinical trial. A mean improvement of at least seven decibels (dB) in retinal sensitivity across the central 16 test loci is considered clinically meaningful.
Opus Genetics previously reported meaningful vision improvements in three adult and three pediatric patients (aged 16-17) in its Phase 1/2 LCA5 gene therapy clinical trial at the University of Pennsylvania. Some patients saw objects for the first time after treatment. Others had meaningful improvements in visual acuity.
OPGx-LCA5 is administered by an injection underneath the retina. The gene therapy uses an engineered adeno-associated virus (AAV) to deliver health copies of the LCA5 gene into retinal cells. Researchers believe that retinal gene therapies can work for many years, perhaps the lifetime of the patient.
Opus Genetics is a Hope in Focus partner.