Opus Genetics Doses First Pediatric Patient in Phase 1/2 Trial for OPGx-LCA5 Gene Therapy

Opus Genetics has reached a significant milestone in its ongoing Phase 1/2 clinical trial for OPGx-LCA5, an investigational gene therapy for Leber congenital amaurosis (LCA). The company announced that the first pediatric patient has been dosed, with initial data from this cohort expected by Q3 2025.

Promising Early Results in Adult Patients

The trial has already demonstrated early clinical proof of concept, with meaningful visual improvement observed in adult patients as early as one month post-treatment.

Key findings from the trial include:

       • Six-month data, released in October 2024, confirmed significant visual improvements.

       • Twelve-month data on adult LCA5 patients will be presented at a major medical conference in Q2 2025.

       • An FDA Type D meeting is scheduled for March 2025 to discuss the pivotal trial design and registrational endpoints.

"We are proud to expand the OPGx-LCA5 clinical trial to pediatric patients. Early intervention is crucial to preserving or restoring vision before disease progression,” said George Magrath, MD, CEO of Opus Genetics

Dr. Magrath also emphasized that the durability of positive responses observed at six and twelve months will be a key focus in upcoming FDA discussions regarding a potential Phase 3 trial.

Phase 1/2 Trial Design and Key Efficacy Endpoints

The open-label Phase 1/2 trial evaluates the safety and preliminary efficacy of subretinal gene therapy with OPGx-LCA5 in patients with biallelic LCA5 gene mutations.

Efficacy endpoints include:

       • Multi-Luminance Orientation and Mobility Test (MLoMT) to measure functional vision.

       • Full-Field Stimulus Testing (FST) to assess retinal sensitivity to light.

       • Microperimetry to evaluate point-wise sensitivity to light.

Advancing Toward a Potential Treatment for LCA5

Opus Genetics continues to work closely with the University of Pennsylvania, regulatory agencies, the medical community, and patient advocates to advance this promising therapy.

With encouraging early results and the expansion of the trial to pediatric patients, OPGx-LCA5 has the potential to become a transformative treatment for individuals with LCA5-related vision loss.