Opus Genetics reports positive Cohort 1 data for OPGx-BEST1; FDA aligns on pivotal endpoint

Opus Genetics, Inc.

All 5 Cohort 1 participants showed clinically meaningful visual function improvement; BCVA improved in 3/5, microperimetry in 3/4 evaluable.
No serious adverse events or dose-limiting toxicities; structural improvements in 4/5, including vitelliform reduction in 2/3 BVMD.
FDA aligned on ≥3 dB microperimetry improvement in ≥5 loci with patient-reported outcome as potential pivotal endpoint.
Cohort 2 over-enrolled to 8 participants; dosing complete Q4 2026, topline data Q2 2027; cash runway into 2029.
New epidemiology estimates ~23,600 symptomatic BEST1 patients in US and ~45,400 globally.

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