Opus Genetics, Inc. (NASDAQ:IRD) stock is surging on Wednesday after the company reported positive three- and six-month results from the low-dose first cohort of its Phase 1/2 BIRD-1 trial of OPGx-BEST1.
OPGx-BEST1 is a gene therapy designed to address the underlying genetic cause of BEST1-related inherited retinal diseases.
All five trial participants demonstrated clinically meaningful improvements in visual function, while four displayed key structural retinal recoveries. Following a successful meeting with federal regulators, the company is preparing for pivotal Phase 3 testing in 2027.
Positive Visual And Structural Trial Outcomes
The low-dose cohort evaluated participants diagnosed with Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).
Functional testing revealed best-corrected visual acuity improvements in 60% of subjects, while 75% of evaluable participants showed gains in retinal sensitivity via microperimetry.
Structural findings included a reduction of vitelliform material in 67% of BVMD patients and complete intraretinal fluid reduction across all ARB patients.
The gene therapy maintained a favorable safety profile, showing no serious adverse events, dose-limiting toxicities, or intraocular inflammation.
Regulatory Alignment and Phase 3 Roadmap
Opus Genetics met with the U.S. Food and Drug Administration in August 2026 to finalize potential pivotal clinical trial endpoints.
The parties aligned on a primary endpoint measuring microperimetry improvement alongside patient-reported outcomes for a randomized controlled trial.
Opus Genetics plans to complete manufacturing requirements in early 2027 before commencing Phase 3 patient dosing later that year.
Cohort Expansion And Broader Market Scope
The clinical-stage biopharmaceutical firm has already advanced to its higher-dose second cohort, over-enrolling eight participants to further evaluate safety and efficacy.
Topline three-month data from this group and six-month data for BVMD participants in the first cohort are expected in the second quarter of 2027.
Additionally, new survey research from Triangle Insights Group indicates that the global target population reaches 45,400 symptomatic BEST1 patients, revealing a significantly larger addressable market than prior estimates suggested.
IRD Price Action: Opus Genetics shares were up 46.66% to $6.36 at the time of publication on Wednesday. The stock is trading at a new 52-week high, according to Benzinga Pro data.
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