Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting
MWN-AI** Summary
Opus Genetics, Inc. (Nasdaq: IRD) has announced promising initial results from its Phase 1/2 study of OPGx-BEST1 gene therapy for inherited retinal diseases (IRDs), including Best Vitelliform Macular Dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). The findings were presented at the Macula Society's annual meeting in February 2026, highlighting the positive safety and tolerability profile of the therapy in its sentinel participant—a 63-year-old woman suffering from severe ARB-related vision impairment.
After three months of treatment, no ocular inflammation or dose-limiting toxicities were reported, indicating a favorable safety outcome for OPGx-BEST1. The participant demonstrated early functional improvements, achieving a 12-letter gain in Best Corrected Visual Acuity (BCVA) and a 23% reduction in central subfield thickness (CST), alongside resolution of intraretinal fluid observed as early as one month post-treatment. This signals potential therapeutic benefits for patients suffering from BEST1-related retinal diseases, for which no approved treatments currently exist.
CEO George Magrath expressed optimism regarding these initial results, emphasizing the importance of this milestone for the OPGx-BEST1 program and the patients affected. The study, which is ongoing and currently enrolling participants across various clinical sites in the U.S., aims to assess safety, tolerability, and the biological activity of the therapy through longitudinal outcomes.
This innovative gene therapy seeks to restore function to the defective BEST1 gene, addressing the root cause of retinal degeneration. Opus's development efforts are part of a broader portfolio focused on advanced AAV-based therapies that aim to significantly impact the treatment of severe retinal disorders. Full data from the study is expected by mid-2026.
MWN-AI** Analysis
Opus Genetics recently made significant strides in advancing its OPGx-BEST1 gene therapy, particularly following the promising initial data presented at the Macula Society Annual Meeting. The results from the sentinel participant in this Phase 1/2 study displayed robust safety and tolerability, indicating no ocular inflammation or adverse events, which are critical factors in validating the therapeutic approach for inherited retinal diseases (IRDs). The reported improvements in vision, including a 12-letter gain in Best Corrected Visual Acuity (BCVA) and a 23% reduction in central subfield thickness (CST), suggest that OPGx-BEST1 may not only be safe but also biologically active—enhancing the potential for restoring vision in patients with Bestrophinopathy.
For market participants, Opus Genetics appears to be at a pivotal moment as it navigates early-stage clinical development in a space with substantial unmet medical needs and no approved therapies for BEST1-related IRDs. Investors should closely monitor ongoing enrollment and data from the trial, especially the full cohort data expected by mid-year 2026. This upcoming data will be crucial in determining the therapeutic's efficacy and those insights could play a key role in shaping market expectations.
It is essential to factor in the broader context of pharmaceutical development in this sector; the high-risk, high-reward nature of gene therapies means that while short-term results appear promising, longer-term data will ultimately guide Opus's clinical and commercial viability. Additionally, potential partnerships with larger pharmaceutical companies could arise, driven by the competitive landscape and the growing focus on genetic therapies.
Overall, Opus Genetics presents a compelling investment opportunity, but it comes with inherent risks typical of clinical-stage biotech companies. Careful observation of clinical progress and management updates will be vital for informed decision-making in this dynamic market.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
- Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting toxicities at three months
- Early signals of functional and structural improvement observed at one month and three months
- 12-letter BCVA gain and 23% CST reduction observed in the treated eye at three months
- Full cohort data expected in mid-year 2026
RESEARCH TRIANGLE PARK, N.C., Feb. 27, 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), 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.
The full presentation and video recording will be available on the Opus Genetics website in the Events section.
About OPGx-BEST1 and the Phase 1/2 Trial
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. BEST1-associated IRDs affect an estimated 22,000 patients worldwide and currently have no approved treatments.
The ongoing adaptive, open-label Phase 1/2 study is evaluating single-eye subretinal administration of OPGx-BEST1 up to two dose levels in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts.
The primary objective is to assess safety and tolerability and identify the most appropriate dose for further clinical development, with participants followed longitudinally for long-term outcomes. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.
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 RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional potential indications in presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of 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 Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12, and earlier stage programs, 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our subsequent Quarterly Reports on Form 10-Q, 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.
FAQ**
What specific safety and tolerability metrics were evaluated in the Phase 1/2 study of OPGx-BEST1 gene therapy being developed by Opus Genetics Inc. IRD for patients with BEST1 mutations?
Given the early signals of functional and structural improvements observed, what additional clinical endpoints will be used to evaluate the effectiveness of OPGx-BEST1 in the upcoming full cohort analysis by Opus Genetics Inc. IRD?
How does the safety profile of OPGx-BEST1 compare to other gene therapies targeting inherited retinal diseases developed by Opus Genetics Inc. IRD, especially considering the lack of approved treatments?
Can you elaborate on the significance of the 12-letter BCVA gain and 23% CST reduction reported in the sentinel participant and how that influences the continued recruitment and study design for OPGx-BEST1 by Opus Genetics Inc. IRD?
**MWN-AI FAQ is based on asking OpenAI questions about Ocuphire Pharma Inc Com (NASDAQ: OCUP).
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