Successful Type B RDEP Meeting confirms FDA alignment on pivotal Phase 3 study design
FDA indicates Opus Genetics may submit a BLA based on 6-month efficacy data, with 12-month durability data provided during review
Company expects to initiate Phase 3 dosing in 4Q 2026

RESEARCH TRIANGLE PARK, N.C., July 06, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc . (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) in a Type B Rare Disease Evidence Principles (RDEP) meeting on the design of its registrational Phase 3 clinical trial evaluating OPGx-LCA5 for LCA5-associated IRD, an early-onset severe inherited retinal dystrophy.

Opus Genetics has received the meeting minutes from the Type B meeting confirming several key elements of the trial. The Phase 3 study is expected to enroll eight participants who are able to complete microperimetry testing with both eyes treated. The study is also expected to include a six-month run-in period, allowing each participant to serve as their own natural history control prior to receiving treatment. Seven of the eight planned participants have already been enrolled and are currently completing the run-in period, and the Company expects to initiate dosing in the fourth quarter of 2026.

The primary efficacy endpoint is a mean improvement of at least 7 decibels (dB) in retinal sensitivity across the central 16 test loci, a clinically meaningful measure of visual function. The Phase 3 study is designed with greater than 90% statistical power to detect a treatment effect of at least seven decibels. The Phase 1/2 trial supports this outcome measure as those participants able to complete microperimetry demonstrated an average improvement of approximately 10.5 dB.

Importantly, the FDA indicated that Opus Genetics may submit a Biologics License Application (BLA) based on compelling efficacy at the six-month primary endpoint, with 12-month durability data submitted during the BLA review process.

“We believe this alignment with the FDA provides a clear roadmap toward a potential BLA submission and, most importantly, brings us one step closer to delivering a treatment for patients living with LCA5-associated blindness,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We have had a very positive experience working with the FDA through the RDEP program to develop a registrational pathway for this ultra-rare inherited retinal disease. With enrollment nearly complete, we anticipate initiating dosing in the Phase 3 study during the fourth quarter of 2026. In addition, OPGx-LCA5 may qualify to receive a Priority Review Voucher, representing a potentially significant strategic asset.”

Jean Bennett, M.D., Ph.D., Co-Founder and Board Member, Opus Genetics added, “LCA5-associated inherited retinal disease is among the most severe forms of childhood blindness, with patients experiencing profound vision loss at an early age and no approved treatment options. We are encouraged by the FDA's continued engagement and look forward to advancing this important program as rapidly as possible for patients and families.”

About OPGx-LCA5

OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and has been accepted into the FDA’s Rare Disease Evidence Principles (RDEP) program.

About Opus Genetics

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com .

Forward Looking Statements
This press release contains certain statements that are not statements of historical fact and are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to the clinical development, clinical results, preclinical data, and future plans for OPGx-LCA5 and expectations regarding us, our business prospects, and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, and in our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “strive,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.

Contacts:

Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
[email protected]

Media

Kimberly Ha
KKH Advisors
917-291-5744
[email protected]

Source: Opus Genetics, Inc.