Key Highlights

  • IRD shares are up 9.31% to $4.74 in pre-market trading.
  • The company reported positive low-dose Cohort 1 data from its Phase 1/2 trial of OPGx-BEST1 in patients with BEST1-related retinal diseases.
  • All five participants demonstrated clinically meaningful improvement in visual function, with structural improvements observed in four participants.
  • The FDA aligned on a potential pivotal endpoint of at least 3 decibels of microperimetry improvement alongside patient-reported outcomes, following a successful Type C meeting.
  • Cohort 2 has been over-enrolled with eight participants, with topline three-month data expected in the second quarter of 2027.

Shares Rise on Positive Gene Therapy Data

Opus Genetics (NASDAQ:IRD) shares are trading higher in pre-market trading after the company reported positive low-dose Cohort 1 data from its ongoing Phase 1/2 BIRD-1 trial evaluating OPGx-BEST1 in patients with BEST1-related retinal diseases, including Best vitelliform macular dystrophy and autosomal recessive bestrophinopathy. Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases.

All five participants in Cohort 1 demonstrated clinically meaningful improvement in visual function, with structural improvements observed in four participants, and the therapy showed a favorable safety and tolerability profile with no serious adverse events or dose-limiting toxicities.

FDA Alignment Clears a Path Toward Pivotal Development

The company also announced a successful FDA Type C meeting, in which the agency aligned on a potential pivotal endpoint based on at least 3 decibels of microperimetry improvement alongside patient-reported outcomes for a randomized, controlled trial. That regulatory clarity supports the company's plan to begin planning for a Phase 3 trial immediately, with participant dosing expected to begin in 2027.

Higher-Dose Cohort Already Over-Enrolled

Based on the safety profile and positive proof-of-concept findings from Cohort 1, the company has advanced to a higher-dose Cohort 2, which was originally designed to enroll five participants but has been over-enrolled with eight, most of whom have Best vitelliform macular dystrophy. Dosing is expected to be completed in the fourth quarter of 2026, with topline three-month data expected in the second quarter of 2027.

New Epidemiology Data Points to a Larger Market

New epidemiology research cited by the company estimates approximately 23,600 symptomatic BEST1 patients in the U.S., including roughly 10,600 who remain undiagnosed, and approximately 45,400 symptomatic patients globally, suggesting a substantially larger addressable patient population than previously estimated. Continued progress in Cohort 2 dosing and the path toward Phase 3 initiation will likely be the key catalysts for the stock going forward.