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

The higher-dose cohort enrolled 11 participants, exceeding its original target of five.

Rhea-AI Summary

Opus Genetics (Nasdaq: IRD) completed dosing in the higher-dose second cohort of its Phase 1/2 BIRD-1 trial evaluating OPGx-BEST1 for BEST1-related retinal diseases. Cohort 2 enrolled 11 participants, versus an original target of five, at 4.5 × 10⁹ vector genomes per eye, three times the Cohort 1 dose. The company expects three-month topline data in the first quarter of 2027 and anticipates dosing in a potential pivotal Phase 3 trial in the second half of 2027.

The open-label trial evaluates a single injection beneath the retina in adults. Previously reported Cohort 1 three- and six-month results showed clinically meaningful improvement in at least one visual-function measure in all five participants, with no serious adverse events. Opus reached FDA alignment on a potential pivotal endpoint and Phase 3 commercial manufacturing requirements; it expects those requirements to be completed in early 2027.

13 points · 0 major

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0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointCohort 2 dosing completed in 11 participants at 4.5 × 10⁹ vector genomes per eye, triple Cohort 1's dose.
• Moderate point. Forward-looking: it has not happened yet and may not happen.Potential pivotal Phase 3 dosing anticipated to begin in the second half of 2027.
• Moderate pointAll five Cohort 1 participants demonstrated clinically meaningful improvement in at least one functional measure.
• Moderate pointFDA alignment reached on a potential pivotal endpoint and Phase 3 commercial manufacturing requirements.
• Minor point. Forward-looking: it has not happened yet and may not happen.Cohort 2 three-month topline data expected in the first quarter of 2027.

8 minor points

• Minor pointCohort 1 safety showed no serious adverse events, dose-limiting toxicities, or intraocular inflammation.
• Minor pointBest-corrected visual acuity improved in 60% (3/5) of Cohort 1 participants.
• Minor pointLow-luminance visual acuity improved in 40% (2/5) of Cohort 1 participants.
• Minor pointContrast sensitivity improved in 40% (2/5) of Cohort 1 participants.
• Minor pointRetinal sensitivity showed clinically meaningful improvement in 75% (3/4) of evaluable Cohort 1 participants.
• Minor pointVitelliform material decreased in 67% (2/3) of Cohort 1 participants with Best vitelliform macular dystrophy.
• Minor pointIntraretinal fluid decreased in both Cohort 1 participants with autosomal recessive bestrophinopathy (2/2).
• Minor point. Forward-looking: it has not happened yet and may not happen.Phase 3 commercial manufacturing requirements expected to be completed in early 2027.

Negative

• Minor pointTreatment-related adverse events occurred in Cohort 1; all were mild or moderate in severity.

Key Figures

Cohort 2 dose: 4.5 × 10⁹ vector genomes per eye Cohort 2 enrollment: 11 participants (original target: 5) Cohort 1 functional improvement: 5 of 5 participants +4 more

Cohort 2 dose

4.5 × 10⁹ vector genomes per eye

Three-fold increase over Cohort 1

Cohort 2 enrollment

11 participants (original target: 5)

Higher-dose Cohort 2

Cohort 1 functional improvement

5 of 5 participants

Clinically meaningful improvement in at least one functional measure

Cohort 1 structural improvement

4 of 5 participants

Structural improvements reported

Cohort 2 topline data

First quarter of 2027

Expected three-month data

Potential pivotal trial dosing

Second half of 2027

Dosing anticipated to begin

Potential pivotal endpoint

At least 3 decibels across 5 or more prespecified retinal loci

FDA alignment also included a patient-reported outcome in a randomized, controlled trial

Previous Clinical trial Reports

2 past events · Latest: Oct 15

Same Type 2 events

1. Oct 15

   OPGx-BEST1 clinical data

   24h Move

   -5.2%

   Presented Cohort 1 findings and reported Cohort 2 enrollment reached 11 participants.

2. Sep 09

   OPGx-BEST1 clinical data

   24h Move

   +32.0%

   Reported Cohort 1 improvements, FDA endpoint alignment, and Cohort 2 at the higher dose.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

vector genomes, microperimetry, best-corrected visual acuity, intraocular inflammation, +1 more

5 terms

vector genomes technical

"4.5 × 10⁹ vector genomes per eye (vg/eye)"

Vector genomes are the genetic payload carried inside a delivery vehicle (often a harmless virus) used to insert specific genes into cells for therapies or vaccines. Think of the vector as a delivery truck and the genome as the package it drops off; the exact contents determine how well a treatment works, how long it lasts, and the safety profile. Investors watch vector genomes because their design and quality drive clinical success, manufacturing complexity, regulatory approval, and therefore the commercial value of biotech programs.

microperimetry medical

"improvement by microperimetry"

Microperimetry is a clinical eye test that maps how well specific spots on the retina detect light while tracking their exact location on an image of the back of the eye. For investors, it matters because it provides precise, location-specific evidence of vision improvement or decline in drug and device trials, affecting regulatory decisions, market potential and reimbursement—think of it as a high-resolution measuring stick for retinal function.

best-corrected visual acuity medical

"best-corrected visual acuity (BCVA) improvement in 60%"

The sharpest level of sight a person can reach when using the best possible glasses or contact lenses; think of it as how well a camera can resolve detail once its lens is perfectly focused. It matters to investors because it is a common clinical measure and regulatory endpoint for eye drugs, procedures and devices, so changes in best-corrected visual acuity indicate whether a treatment works and can drive approval, market size and sales potential.

intraocular inflammation medical

"or intraocular inflammation were observed"

Inflammation occurring inside the eye, affecting internal structures such as the iris, ciliary body, vitreous, retina or choroid. Clinically it is identified by signs like cells or protein (“flare”) in the anterior chamber, vitreous haze, redness, pain, light sensitivity or changes in vision, and it can result from infection, autoimmune disease, surgery, implants or drug reactions; it does not refer to inflammation of external tissues like the eyelids or conjunctiva.

subretinal injection medical

"a single subretinal injection of OPGx-BEST1"

A subretinal injection is a medical procedure that places a drug, gene therapy, or cells directly into the thin space beneath the retina at the back of the eye, delivering treatment precisely where damaged vision cells live. For investors, it matters because this targeted approach can increase effectiveness but also raises surgical complexity, regulatory hurdles, costs and safety considerations that affect clinical success, commercialization timeline and market adoption—think of burying fertilizer under a lawn rather than sprinkling it on top.

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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.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When does Opus Genetics expect OPGx-BEST1 Cohort 2 results and a potential Phase 3 trial?

Opus Genetics expects three-month topline Cohort 2 data in the first quarter of 2027, with dosing in a potential pivotal Phase 3 trial anticipated to begin in the second half of 2027.

What potential OPGx-BEST1 pivotal endpoint did Opus Genetics align on with the FDA?

Opus Genetics reached FDA alignment on a potential endpoint combining at least three decibels of improvement in microperimetry across five or more prespecified retinal loci with a patient-reported outcome in a randomized, controlled trial. Microperimetry measures light sensitivity at specific retinal locations.

Which OPGx-BEST1 Cohort 1 participants had the greatest functional improvements?

The greatest functional improvements occurred in participants with less advanced disease. Opus views this finding as supporting the potential benefit of earlier treatment while viable retinal tissue remains and as informing patient selection for future clinical development.