Opus Genetics announced positive interim data from the low-dose cohort of its Phase 1/2 clinical trial (BIRD-1) for OPGx-BEST1, a gene therapy for inherited retinal diseases. The treatment demonstrated a favorable safety profile and all five participants showed clinically meaningful improvement in visual function. The company also announced a successful meeting with the FDA, aligning on a potential pivotal endpoint for a Phase 3 trial.
Key Details
- Clinical Data: In the 5-participant low-dose cohort, OPGx-BEST1 was well-tolerated with no serious adverse events. 60% of participants (3/5) showed improvement in best-corrected visual acuity (BCVA), and 75% of evaluable participants (3/4) showed improvement in retinal sensitivity by microperimetry.
- Regulatory Progress: Following an August 2026 meeting, Opus Genetics aligned with the U.S. FDA on a potential pivotal endpoint for a Phase 3 trial, based on microperimetry improvement in conjunction with a patient-reported outcome.
- Next Steps: The company has advanced to a higher-dose Cohort 2, with dosing expected to be complete in Q4 2026 and topline data anticipated in Q2 2027. Planning for a Phase 3 trial is underway, with dosing expected to begin in 2027.