Opus Genetics Completes Enrollment in Cohort 1 of Phase 1/2 OPGx-BEST1 Gene Therapy Study
Rhea-AI Summary
Opus Genetics (NASDAQ: IRD) completed enrollment in Cohort 1 of its Phase 1/2 study of OPGx-BEST1, enrolling five adults with Best disease (three BVMD, two ARB). Four participants have been dosed and one is scheduled for dosing this month. Baseline demographics were presented at ARVO and three-month topline results for the full cohort are expected in September 2026.
ARVO materials and a summary video are available on the company website. Planned outcomes include OCT, microperimetry, BCVA, low-luminance acuity and contrast sensitivity.
Positive
- Cohort 1 enrollment completed with 5 participants
- Four of five participants have been dosed; fifth scheduled this month
- Baseline demographics and sentinel 3-month tolerability/activity presented at ARVO
- Company plans 3-month topline data in September 2026 across multiple outcome measures
Negative
- Small cohort size of 5 participants limits statistical power
- Only the sentinel participant has reported 3-month results; cohort-level data pending
- Study is open-label and adaptive, which may limit blinded efficacy assessments
News Market Reaction – IRD
In the May 7 session, IRD gained 1.90%, reflecting a mild positive market reaction. Argus tracked a peak move of +5.1% during that session. Argus tracked a trough of -2.5% from its starting point during tracking. Our momentum scanner triggered 12 alerts that day, indicating notable trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Feb 27 | BEST1 data update | Positive | +15.9% | Reported positive 3‑month sentinel OPGx-BEST1 safety and efficacy signals. |
| Feb 25 | sNDA acceptance | Positive | -3.9% | FDA accepted sNDA for phentolamine 0.75% in presbyopia with PDUFA date. |
| Jan 27 | Trial launch | Positive | +0.7% | Launched OPGx-MERTK gene therapy trial for retinitis pigmentosa in 2026. |
| Dec 09 | IDMC safety review | Positive | +4.4% | IDMC gave positive recommendation to continue BEST1 trial without changes. |
| Nov 13 | First patient dosed | Positive | -3.6% | First participant dosed in OPGx-BEST1 Phase 1/2 Best disease trial. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Clinical-trial news has often moved IRD meaningfully, with three positive-aligned reactions and two divergences in the last five tagged events.
Over the past several months, Opus reported multiple clinical milestones, including initial OPGx-BEST1 data with a 12-letter BCVA gain and 23% CST reduction, FDA acceptance of a sNDA for phentolamine 0.75% with an October 17, 2026 PDUFA date, and launch of the OPGx-MERTK trial targeting a 60,000-patient population. Today’s completion of Cohort 1 enrollment for OPGx-BEST1 and the planned three‑month readout connect directly to this ongoing clinical progression.
Key Terms
phase 1/2 medical
gene therapy medical
subretinal medical
optical coherence tomography (oct) medical
microperimetry medical
best-corrected visual acuity medical
low luminance visual acuity medical
contrast sensitivity medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
Target Enrollment Achieved with the Inclusion of Participants with Both Dominant and Recessive Forms of BEST Disease
Baseline Demographics Presented at ARVO Annual Meeting with Summary Audio and Slide Presentation Available on Opus Website
Three-Month Topline Results from Cohort 1 Expected in September 2026
RESEARCH TRIANGLE PARK, N.C., May 07, 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 enrollment in Cohort 1 of its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy.
The adaptive, open-label study is evaluating the safety and efficacy of 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). Five participants have been enrolled in the study, three with BVMD and two with ARB. The first four participants have been dosed and the fifth participant is scheduled for the dosing procedure this month.
“As planned, we enrolled a mix of participants with the dominant and recessive forms of BEST disease to evaluate OPGx-BEST1 in both conditions,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We collaborated closely with our study sites and investigators to identify participants who met our carefully defined entry criteria. In the dominant participants, we completed the added step of using an in vitro platform to confirm that each participant’s disease mutation is amenable to gene augmentation. The promising tolerability data and early efficacy signals we saw in the sentinel participant show the promise for individuals living with this potentially blinding disease. We are grateful to the participants and clinical sites who are participating in the trial, and we look forward to presenting the three-month data on the full cohort later this year.”
In a session today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, the study’s principal investigator, Mark Pennesi, M.D., Ph.D., Chief Medical Officer and Director, Inherited Retinal Degeneration Clinic, Retina Foundation of the Southwest, presented baseline demographics of Cohort 1 (summarized in Table 1) and 3-month results from the first (sentinel) participant treated in the study, highlighting positive tolerability and biological activity following subretinal administration of OPGx-BEST1.
The ARVO poster presentation and a video recording of Dr. Pennesi’s summary of Cohort 1 Baseline Demographics and Key Endpoints for IRDs are available on the Opus Genetics website in the Events section.
Opus expects to announce 3-month topline data from Cohort 1 in September 2026, followed by the presentation of data at an ophthalmology medical conference later this year. Data is expected to be provided on multiple outcome measures including structural optical coherence tomography (OCT) measures, microperimetry, best‑corrected visual acuity, low luminance visual acuity and contrast sensitivity.
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. BEST1-associated IRDs affect an estimated 22,000 patients worldwide and currently have no approved treatments.
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. Opus Genetics 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 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2025, 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
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c8c9006e-0278-4c21-a78c-6da68677c067
