Opus Genetics sets September timeline for OPGx-BEST1 trial data

Opus Genetics, Inc. (Nasdaq: IRD) said it expects to announce three-month topline data from Cohort 1 of its Phase 1/2 OPGx-BEST1 clinical trial during the second week of September 2026, contingent on all participants completing their scheduled assessments.

The company also plans to present the data at the EURETINA Congress in Vienna, Austria, scheduled for October 1-4, 2026.

The trial, called BIRD-1, is an adaptive, open-label study evaluating the safety and efficacy of OPGx-BEST1, a gene therapy candidate for Best Vitelliform Macular Dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). Enrollment in Cohort 1 was completed in May 2026 with five participants — three with BVMD and two with ARB.

The primary endpoint for Cohort 1 is safety and tolerability. The trial also measures structural parameters, including subretinal fluid via Optical Coherence Tomography scans. A reduction trending toward 20% in subretinal fluid may be considered clinically meaningful and could support advancing into Cohort 2. If a 100% fluid reduction is observed in the majority of Cohort 1 patients, the trial may be expanded into a potential pivotal study.

Following the Month 3 data readout, an Independent Data Monitoring Committee will review Cohort 1 results to determine next steps. Opus Genetics said it plans to present the data to the U.S. Food and Drug Administration to align on further clinical development.

The trial evaluates two doses of OPGx-BEST1: 1.5E9 vg/eye in Cohort 1 and 4.5E9 vg/eye in Cohort 2. The company said potential participants for Cohort 2 have already been identified.

Opus Genetics said it will enter an investor relations quiet period beginning July 15, 2026, lasting until the public announcement of the data.

According to the company, estimated global prevalence of BEST1-associated inherited retinal diseases is approximately 21,800 patients, with roughly 8,400 in the U.S. No approved treatments currently exist for these conditions.

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