OPGx-BEST1 - (BIRD-1)

September 9, 2026: Opus Genetics (NASDAQ: IRD) reported positive interim 3- and 6-month Phase 1/2 BIRD-1 data for OPGx-BEST1 in BEST1-related retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB). In low-dose Cohort 1 (1.5 x 10⁹ vg/eye; 5 participants), no serious adverse events, dose-limiting toxicities, intraocular inflammation, or notable vital sign/lab findings were observed; treatment-related AEs were all mild or moderate.

All 5 participants showed clinically meaningful visual-function improvement. BCVA improved in 3/5, low-luminance visual acuity in 2/5, contrast sensitivity in 2/5, and microperimetry retinal sensitivity in 3/4 evaluable participants. Structural improvement was seen in 4/5: reduced vitelliform material in 2/3 BVMD participants and reduced intraretinal fluid in 2/2 ARB participants.

The company said FDA aligned in August 2026 on a potential pivotal endpoint of ≥3 dB microperimetry improvement in ≥5 prespecified loci plus a patient-reported outcome in a randomized controlled trial; BCVA, LLVA, and contrast sensitivity may also be acceptable endpoints. Opus expects Phase 3/commercial manufacturing readiness in early 2027, Phase 3 dosing to begin in 2027, 6-month BVMD Cohort 1 data in Q2 2027, and higher-dose Cohort 2 topline 3-month data in Q2 2027 after dosing completion in Q4 2026.