Clinical Read2026-09-18

OPGx-BEST1 - (BIRD-1)

Opus Genetics Inc.IRD
Indication
Bestrophin-1 (BEST1)-related IRD.
Stage
phase1
Event
Topline Data
Details

August 6, 2026: Opus Genetics (NASDAQ: IRD) gave a pipeline update centered on inherited retinal disease gene therapies. For OPGx-BEST1, enrollment is complete in Cohort 1 of the Phase 1/2 BIRD-1 trial, with 3-month topline data expected in the second week of September 2026. Baseline demographics were previously presented at ARVO 2026.

For OPGx-LCA5, the company said it aligned with FDA on a registrational Phase 3 trial in eight participants, using a six-month run-in period so each participant serves as their own control before bilateral treatment. Enrollment is complete, dosing is planned for Q4 2026 using clinical supply made with intended commercial processes, and topline data are expected by the end of 2027.

Other programs: OPGx-RDH12 is expected to enter the clinic in Q4 2026 and is partially funded by the RDH12 Alliance; OPGx-MERTK is expected to start clinical testing at Cleveland Clinic Abu Dhabi in Q1 2027; OPGx-RHO presented preclinical safety and efficacy data in two large animal models of autosomal-dominant retinitis pigmentosa at ARVO 2026 and the Foundation Fighting Blindness summit, with global clinical testing planned for 2H 2027.

Opus reported $88.8 million in cash as of June 30, 2026 and said its runway extends into 2029.