RESEARCH TRIANGLE PARK, N.C., July 09, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company”, “Opus Genetics” or “Opus”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today provided updates on its ongoing OPGx-BEST1 Phase 1/2 clinical trial (BIRD-1) targeting BEST-1 associated IRDs.
Opus expects to announce three-month topline data from Cohort 1 of the Phase 1/2 trial during the second week of September 2026, assuming all participants complete their assessments as scheduled. In addition, the Company plans to present the data at the annual EURETINA Congress taking place in Vienna, Austria from October 1 - 4, 2026.
BIRD-1 is an adaptive, open-label, Phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). The trial is designed as a dose escalation trial to evaluate two doses of OPGx-BEST1: 1.5E9 vg/eye (Cohort 1) and 4.5E9 vg/eye (Cohort 2).
Enrollment in Cohort 1 was completed in May 2026 with five participants in the study, three with BVMD and two with ARB, who were carefully selected to meet the defined entry criteria. In the BVMD participants, the Company completed the added step of using an in vitro platform to confirm that each participant’s disease mutation is amenable to gene augmentation. After the last participant has completed the Month 3 visit, the Independent Data Monitoring Committee (IDMC) will review all Cohort 1 data and determine next steps.
In Cohort 1, the primary endpoint for evaluation is the safety and tolerability of OPGx-BEST1. In addition to safety measures, Opus will be also assessing a number of structural parameters including subretinal fluid as measured by Optical Coherence Tomography (OCT) scans. A reduction in subretinal fluid on OCT would suggest that OPGx-BEST1 has a biological effect demonstrating target engagement. A reduction trending towards 20% may be considered clinically meaningful, and these results, in addition to those on safety, are expected to be used to provide the rationale to advance the trial into Cohort 2 to optimize dose selection, per the trial protocol. Given the high level of patient interest in the trial, potential participants have already been identified to enroll in Cohort 2 if necessary. In the event that OPGx-BEST1 demonstrates a 100% reduction in fluid in the majority of patients in Cohort 1, the trial may be expanded into a potential pivotal trial. Opus expects to present the Month 3 data to the U.S. Food and Drug Administration (FDA) to align on the next steps for clinical development.
In addition to OCT structural assessments, functional endpoints will also be evaluated including microperimetry, best corrected visual acuity (BCVA), low luminance visual acuity (LLVA) and contrast sensitivity. Correlation between functional improvements and structural changes would suggest clinically meaningful target engagement by of functional improvements with structural changes would be suggestive of clinically meaningful target engagement of OPGx-BEST1.
Due to the open-label nature of the trial, the Company plans to enter an investor relations quiet period beginning July 15, 2026, and continuing until the public announcement of these data.
“We continue to work closely with our study sites and investigators to support this trial. We are deeply grateful to the participants and encouraged by the significant interest in this trial from the patient community,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “For the significant number of BEST1 patients in the U.S. and globally, there is a substantial need for treatment options, with no therapies currently available today.”
Additional OPGx-BEST1 resources can be found on the Opus Genetics website:
- Overview, presentations and webcasts can be found here, including:
- Cohort 1 Baseline Demographics and Key Endpoints for IRDs Presentation and Video Recording by Dr. Mark Pennesi (May 2026)
- Preliminary Results from the Sentinel Participant Presentation and Video Recording by Dr. Mark Pennesi (February 2026)
- Publications and medical conference presentations can be found here, including the baseline demographics poster presentation from the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting (May 2026).
About OPGx-BEST1
OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. By restoring BEST1 function, the therapy aims to address the underlying genetic cause of retinal degeneration and support preservation of photoreceptor health and visual function. Estimated global prevalence in BEST1-associated IRDs is approximately 21,800 patients, including roughly 8,400 patients in the U.S. comprised of approximately 8,000 best vitelliform macular dystrophy (BVMD) and approximately 400 autosomal recessive bestrophinopathy (ARB) patients. OPGx-BEST1 is currently being evaluated in an open-label, Phase 1/2 clinical trial. Additional information on the trial can be found by ClinicalTrials.gov Identifier: NCT07185256. There are currently no approved treatments for the disease.
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 anticipated timing of topline data from the OPGx-BEST1 Phase 1/2 clinical trial, clinical development, clinical results, preclinical data, and future plans for OPGx-BEST1 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.
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Source: Opus Genetics, Inc.