Key Takeaways
- In September, Opus Genetics expects 3-month topline data from cohort 1 of the BIRD-1 clinical trial of OPGx-BEST1 for BEST-1 associated inherited retinal diseases.
- BIRD-1 is an adaptive, open-label, dose-escalation phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adults with Best vitelliform macular dystrophy or autosomal-recessive bestrophinopathy.
Opus Genetics announced that it expects 3-month topline data from cohort 1 of the phase 1/2 BIRD-1 clinical trial of OPGx-BEST1 for BEST-1 associated inherited retinal diseases in mid-September 2026. OPGx-BEST1 uses a proprietary AAV-based gene therapy platform to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium cells.
Assuming the participants (n = 5) complete their scheduled assessments, company plans to present the data at the Euretina Congress in Vienna, Austria.
BIRD-1 is an adaptive, open-label, dose-escalation phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adults with Best vitelliform macular dystrophy or autosomal-recessive bestrophinopathy. The trial is evaluating two doses of OPGx-BEST1: 1.5E9 vg/eye (cohort 1) and 4.5E9 vg/eye (cohort 2).
The primary endpoint for cohort 1 is the safety and tolerability of OPGx-BEST1, with secondary analyses of several structural parameters such as subretinal fluid as measured by OCT. A reduction trending towards 20% may be considered clinically meaningful and will to be used to provide the rationale to advance the trial into cohort 2 to optimize dose selection. If OPGx-BEST1 demonstrates a 100% reduction in fluid in most patients in cohort 1, the trial may be expanded into a potential pivotal trial, according to the company.
In addition to OCT structural assessments, other functional endpoints evaluated include microperimetry, BCVA, low luminance visual acuity, and contrast sensitivity.
