RESEARCH TRIANGLE PARK, N.C. — September 30, 2025 — Leads & Copy — Opus Genetics, Inc. (Nasdaq: IRD) has announced positive three-month data from the pediatric cohort of its Phase 1/2 clinical trial (OPGx-LCA5-1001) evaluating OPGx-LCA5, a gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5).
The trial included three pediatric participants aged 16-17 with severe baseline vision impairment who received a single subretinal injection of OPGx-LCA5. Early data showed a group average of a 0.3 logMAR improvement which is greater than was observed in the adult cohort. All three participants had improvements across multiple measures of visual function from one month; showing greater than one (>1) log unit improvement in cone sensitivity to both red and blue light. Two out of the three participants had greater improvement in the treated eye compared to the control eye.
Combined adult data support that improvements in visual acuity were sustained through 18 months. OPGx-LCA5 has been well-tolerated in all six participants treated to date. No ocular serious adverse events or dose-limiting toxicities have been observed.
The company expects to meet with the U.S. Food and Drug Administration (FDA) in the fourth quarter of this year to discuss these results and the next steps for its LCA5 program.
Tomas S. Aleman, M.D., of the Scheie Eye Institute, University of Pennsylvania and principal investigator of the study, stated that this is important evidence supporting that gene augmentation therapy can potentially restore cone function in patients with LCA5.
Opus Genetics management will host a webcast and conference call today at 8:30 a.m. Eastern Time to discuss the OPGx-LCA5 clinical trial data. The live and archived webcast may be accessed on the Opus Genetics website under the Investors section: Events. The live call can be accessed by dialing 888-506-0062 (domestic) or 973-528-0011 (international) and entering conference code: 906168.
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.
