Opus Genetics Completes Enrollment in OPGx-LCA5 Phase 3 Trial
Opus Genetics, Inc. announced the completion of enrollment in its registrational Phase 3 clinical trial for OPGx-LCA5, an investigational gene therapy for LCA5-associated inherited retinal disease. This milestone follows FDA alignment on the trial design through the Rare Disease Evidence Principles program. The company expects to initiate dosing in Q4 2026 and report topline six-month efficacy data by the end of 2027, with potential for BLA submission based on this data.
Key Highlights
- Opus Genetics completed enrollment in its registrational Phase 3 clinical trial for OPGx-LCA5.
- Topline six-month efficacy data for OPGx-LCA5 is expected by the end of 2027.
- Dosing of OPGx-LCA5 is anticipated to initiate in the fourth quarter of 2026.
- The Phase 3 study design was aligned with the FDA through the 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.
- OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and RMAT designations from the FDA.
- The company expects OPGx-LCA5 may qualify for a Rare Pediatric Disease Priority Review Voucher upon approval.
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