Opus Genetics Completes Dosing in Cohort 2 of Phase 1/2 Clinical Trial of OPGx-BEST1 Gene Therapy for Inherited Retinal Diseases

Topline three-month data from Cohort 2 expected in the first quarter of 2027, with potential pivotal Phase 3 trial dosing anticipated in the second half of 2027

RESEARCH TRIANGLE PARK, N.C., Nov. 05, 2026 (GLOBE NEWSWIRE) -- 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), today announced the completion of dosing in the higher-dose Cohort 2 of BIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1 in patients with BEST1-related retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).

Cohort 2 is evaluating OPGx-BEST1 at 4.5 × 10⁹ vector genomes per eye (vg/eye), a three-fold increase over the dose administered in Cohort 1. The cohort enrolled 11 participants, exceeding its original target of five participants. The Company expects to report topline three-month data from Cohort 2 in the first quarter of 2027, with dosing in a potential pivotal Phase 3 trial anticipated to begin in the second half of 2027.

“Completing dosing in Cohort 2 represents a key step in advancing OPGx-BEST1 toward potential pivotal development,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “The positive results from Cohort 1, including clinically meaningful improvements in visual function in all five participants and encouraging structural improvements, provide important proof of concept for this program. We expect Cohort 2 data to further characterize the safety, functional, and structural responses to OPGx-BEST1 at the higher dose and inform the design of a potential pivotal trial. We look forward to reporting this additional data in the first quarter of 2027 as we work toward delivering a potential first treatment addressing the underlying genetic cause of BEST1-related retinal diseases.”

OPGx-BEST1 Clinical Development Progress

The BIRD-1 trial is an adaptive, open-label Phase 1/2 clinical trial evaluating the safety and efficacy of a single subretinal injection of OPGx-BEST1 in adults with BVMD or ARB.

In September 2026, Opus Genetics reported positive three- and six-month results from the low-dose Cohort 1, which enrolled five participants treated at 1.5 × 10⁹ vg/eye.

Key findings from Cohort 1 included:

• Favorable safety and tolerability: No serious adverse events, dose-limiting toxicities, or intraocular inflammation were observed. All treatment-related adverse events were mild or moderate in severity.
• Improved visual function: All five participants demonstrated clinically meaningful improvement in at least one functional measure, including best-corrected visual acuity (BCVA) improvement in 60% (3/5), low-luminance visual acuity (LLVA) improvement in 40% (2/5), and contrast sensitivity improvement in 40% (2/5).
• Improved retinal sensitivity: Among evaluable participants, 75% (3/4) demonstrated clinically meaningful improvement by microperimetry.
• Structural improvements: Four of five participants demonstrated structural improvements, including reductions in vitelliform material in 67% of participants with BVMD (2/3) and reductions in intraretinal fluid in both participants with ARB (2/2).

The greatest functional improvements were observed in participants with less advanced disease, supporting the potential benefit of treating patients earlier in their disease course when viable retinal tissue remains and informing patient selection for future clinical development. In August 2026, Opus Genetics reached alignment with the FDA on a potential pivotal endpoint for OPGx-BEST1 based on an improvement of at least three decibels in microperimetry across five or more prespecified retinal loci, together with a patient-reported outcome in a randomized, controlled trial. The Company also aligned with the FDA on Phase 3 commercial manufacturing requirements, which are expected to be completed in early 2027.

About BEST1 and OPGx-BEST1

BEST1-related inherited retinal diseases, or bestrophinopathies, are rare forms of inherited macular degeneration caused by mutations in the BEST1 gene. These mutations disrupt the normal function of RPE cells, leading to retinal lesions, progressive degeneration and vision loss. BEST1-related diseases include BVMD and ARB, and there are currently no approved therapies that address the underlying genetic cause of these diseases.

OPGx-BEST1 is an investigational gene therapy designed to address the underlying genetic cause of BEST1-related inherited retinal diseases, including BVMD and ARB. OPGx-BEST1 uses an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1 clinical trial is an adaptive, open-label Phase 1/2 clinical trial evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adults with BVMD or ARB.

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 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 OPGx-RDH12 cleared to enter the clinic, and additional candidates targeting, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, N.C. 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. In some cases, you can identify forward-looking statements by the following words: “aim,” “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “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. Such statements include, but are not limited to, statements related to the Company’s plans for the clinical development of OPGx-BEST1, including the design, progress and conduct of the BIRD Phase 1/2 clinical trial; the anticipated timing of topline three-month data from Cohort 2 and of other data from the BIRD-1 trial; the expectation that Cohort 2 data will further characterize the safety, functional, and structural responses to OPGx-BEST1 and inform the design of a potential pivotal trial; the Company’s plans for, and the anticipated timing of dosing in, a potential pivotal Phase 3 trial of OPGx-BEST1, and the potential design and endpoints of such trial; the anticipated timing of completion of Phase 3 and commercial manufacturing requirements; the therapeutic potential of OPGx-BEST1, including its potential to be the first treatment addressing the underlying genetic cause of BEST1-related retinal diseases and the potential benefit of earlier intervention; the significance of the results from Cohort 1 of the BIRD-1 trial and whether such results will be predictive of results in Cohort 2 or in any future clinical trial; the Company’s other product candidates; and the Company’s expectations regarding its business prospects and results of operations. There can be no assurance that results from Cohort 1 will be observed in Cohort 2 or in any future clinical trial, that the Company will report data from the BIRD-1 clinical trial on its anticipated timeline, that the FDA will agree that the potential pivotal endpoint or design of any pivotal trial is sufficient to support regulatory approval, or that OPGx-BEST1 will be shown to be safe or effective or receive regulatory approval. Results from Cohort 1 are based on a small number of participants, and interim or preliminary data may change as more patient data become available and are subject to audit and verification procedures. The forward-looking statements contained herein are subject to certain risks and uncertainties posed by many factors and events that could cause the Company’s 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 the Company’s most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, and in the Company’s 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 the Company’s 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. The Company undertakes 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
ir@opusgtx.com

Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com

Source: Opus Genetics, Inc.