Opus Genetics is advancing OPGx-BEST1 into higher-dose Cohort 2 at 4.5 x 10⁹ vg/eye after low-dose Cohort 1 showed visual-function gains in all five participants. The move sets up further safety and efficacy testing, with dosing expected to finish in 2026, topline data expected in 2027, and results intended to inform a potential pivotal trial.

Key Highlights

  • No serious adverse events, dose-limiting toxicities or intraocular inflammation were observed.
  • The FDA aligned with Opus Genetics on a potential microperimetry-based endpoint for a randomized, controlled key trial.
  • Opus Genetics reported visual-function improvement across all five low-dose Cohort 1 participants.

Opus Genetics, Inc. (NASDAQ: IRD) is moving OPGx-BEST1 into higher-dose Cohort 2 at 4.5 x 10⁹ vg/eye after low-dose Cohort 1 produced clinically meaningful visual-function improvement in all five participants. The company said the early results also showed structural changes and a favorable safety profile.

Low-Dose Results Support Higher-Dose Testing

BCVA improved in 60% of participants, while LLVA and contrast sensitivity each improved in 40%. Among evaluable participants, 75% showed clinically meaningful retinal-sensitivity improvement on microperimetry.

FDA Alignment Shapes Key-Trial Planning

BCVA, LLVA and contrast sensitivity may also be acceptable endpoints. The company said dosing in Cohort 2 is expected to finish in 2026, with topline data expected in 2027. The company also expects to complete Phase 3 and commercial Manufacturing requirements in early 2027.