Opus Genetics Reveals New Clinical Data From Its Ongoing Phase 1/2 Study Of OPGx-BEST1 Gene Therapy; Sentinel Participant Showed OPGx-BEST1 Was Well Tolerated With No Ocular Inflammation, Treatment-related Adverse Events, Or Dose-limiting Toxicities At Three Months;

Opus Genetics, Inc. (NASDAQ:IRD) ("Opus Genetics" or the "Company"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), announced today new clinical data from its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, presented at the 49th Annual Meeting of the Macula Society, in San Diego, California.

The presentation, titled "Preliminary Results from an Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1 Mutations," reported 3-month results from the first (sentinel) adult participant treated in the study, highlighting positive safety, tolerability, and biological activity following subretinal administration of OPGx-BEST1.

The sentinel participant is a 63-year-old female with Autosomal-Recessive Bestrophinopathy (ARB) disease with severe functional impairment. The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities. Early signals of functional vision improvement were observed, including an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA) in the treated study eye. In addition, structural improvement in central subfield thickness (CST) was observed with a 23% decrease in the study eye. Resolution of intraretinal fluid was also seen as early as 1-month in areas with less atrophy.

"We are encouraged by these results from our sentinel participant, showing OPGx-BEST1 was well-tolerated and demonstrated promising initial efficacy at three months," said George Magrath, M.D., Chief Executive Officer, Opus Genetics. Although early, this data represents an important milestone for our OPGx-BEST1 program and for patients with BEST1-related retinal diseases."

"BEST1-related retinal diseases represent a significant unmet medical need, with no approved treatments currently available," said Mark Pennesi, M.D., Ph.D., study investigator at the Retina Foundation of the Southwest in Dallas, Texas. "The preliminary results from this study, including the early favorable safety profile and initial signals of functional and structural improvement, are encouraging and support continued evaluation of OPGx-BEST1 as a gene augmentation approach for patients with BEST1-associated disease."

Recruitment in the Phase 1/2 study is ongoing at two clinical sites in the U.S., with additional sites expected to open in Florida, Cincinnati and New York. Two participants have been enrolled to date, with 3-month results from the full Cohort 1 expected in mid-year 2026.