Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease

Opus Genetics (Nasdaq: IRD) announced that an Independent Data Monitoring Committee issued a positive recommendation to continue the Phase 1/2 BEST1 (BIRD-1) trial after a pre-specified safety review.

Rhea-AI Summary

Opus Genetics (Nasdaq: IRD) announced that an Independent Data Monitoring Committee issued a positive recommendation to continue the Phase 1/2 BEST1 (BIRD-1) trial after a pre-specified safety review.

The IDMC reviewed one-month safety data from the sentinel participant in the multi-center, adaptive, open-label, dose-exploring study of OPGx-BEST1 in Best disease and recommended advancing enrollment and dosing without modification, enabling dosing of the next four participants.

The company framed the outcome as an encouraging early safety signal that supports continued clinical evaluation of OPGx-BEST1 for Best disease.

Positive

• IDMC issued a positive recommendation to continue the Phase 1/2 BEST1 trial
• Pre-specified one-month safety review of the sentinel participant was favorable
• Authorization to dose the next four participants without protocol changes

Negative

• Safety evidence limited to a single sentinel participant at one month

Argus Dec 9 session

+4.37% close to close Open Argus

Details

News Market Reaction – IRD

On Dec 9, the day this news came out, IRD closed 4.37% above the previous close.

Data tracked by StockTitan Argus for the Dec 9 session.

Key Figures

Trial phase: Phase 1/2 Sentinel follow-up: One-month data Next cohort size: Four participants

Trial phase

Phase 1/2

OPGx-BEST1 clinical trial in Best disease

Sentinel follow-up

One-month data

Safety review period for first trial participant

Next cohort size

Four participants

Planned additional dosing in BEST1 Phase 1/2 trial

Historical Context

5 past events · Latest: Dec 02

5 events

1. Dec 02

   Inducement grants

   24h Move

   -0.5%

   Equity inducement options for new employees under Nasdaq Rule 5635(c)(4).

2. Nov 20

   Conference participation

   24h Move

   +3.8%

   Management presenting at two December healthcare and ophthalmology investor conferences.

3. Nov 13

   BEST1 dosing start

   24h Move

   -3.6%

   First participant dosed in OPGx-BEST1 Phase 1/2 gene therapy trial for Best disease.

4. Nov 12

   Q3 earnings update

   24h Move

   -2.5%

   Q3 2025 financials plus LCA5 clinical progress and RMAT-related regulatory update.

5. Nov 06

   Registered offering

   24h Move

   -4.3%

   Announcement of $23M registered direct equity financing and pre-funded warrants.

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

Key Terms

independent data monitoring committee, phase 1/2, open-label, dose-exploring, +2 more

6 terms

independent data monitoring committee medical

"the Independent Data Monitoring Committee (IDMC) issued a positive recommendation"

A panel of independent medical, statistical and ethical experts who review ongoing clinical trial data to judge participant safety, study integrity and whether the trial should continue, change or stop. Like impartial referees or safety inspectors, their decisions can speed, delay or halt a drug’s development and therefore materially affect a company’s timelines, regulatory chances and investment risk.

phase 1/2 medical

"continue as planned in the Company’s Phase 1/2 BEST1 clinical trial (BIRD-1)"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

open-label medical

"multi-center, adaptive, open-label, dose-exploring study evaluating OPGx-BEST1"

Open-label describes a situation where everyone involved in a study or process knows the full details, such as who is receiving a treatment or intervention. For investors, understanding whether a project or product is open-label helps gauge the level of transparency and potential biases, influencing trust and decision-making. It’s like knowing whether a test or experiment is conducted openly or behind closed doors.

dose-exploring medical

"adaptive, open-label, dose-exploring study evaluating OPGx-BEST1"

Dose-exploring describes early clinical testing that tries different amounts of a drug to find which levels are safe, how the body handles them, and whether higher or lower amounts might work better. For investors, these studies shape the likely size, length and cost of later trials and signal the drug’s chances of success—like trying different oven temperatures to learn which one bakes a cake without burning it.

gene therapies medical

"a clinical-stage biopharmaceutical company developing gene therapies to restore vision"

Gene therapies are medical treatments that change or replace a person’s genetic instructions to fix or counteract a disease, using biological delivery methods to get the new material into cells — like repairing or swapping pages in a faulty instruction manual. They matter to investors because they can offer one-time or long-lasting cures with large market potential, but carry high development costs, regulatory scrutiny and manufacturing challenges that make outcomes binary and investment risky.

inherited retinal diseases medical

"prevent blindness in patients with inherited retinal diseases (IRDs)"

Inherited retinal diseases are a group of genetic conditions in which errors in the genes that build and maintain the light-sensing cells at the back of the eye cause progressive vision loss or blindness; think of it as faulty wiring in a camera that gradually degrades the picture. They matter to investors because they define a clear patient population with high unmet need, driving demand for diagnostics and novel treatments (gene, cell or drug therapies) that can produce long-term revenue, regulatory milestones and intellectual property value.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Initial favorable safety profile demonstrated in OPGx-BEST1 clinical trial

RESEARCH TRIANGLE PARK, N.C., Dec. 09, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (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 that the Independent Data Monitoring Committee (IDMC) issued a positive recommendation to continue as planned in the Company’s Phase 1/2 BEST1 clinical trial (BIRD-1), which is a multi-center, adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best disease.

The IDMC overseeing the trial completed its pre-specified safety review of the one-month data from the sentinel participant and recommended advancing enrollment and dosing of additional participants in the trial, without modification.

“We are thrilled with this outcome from the first participant, whose encouraging safety results at one month enable us to proceed with dosing the next four participants in our BEST1 Phase 1/2 trial,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “The IDMC’s safety review and recommendation to continue the trial reinforces our confidence as we advance this program. BEST1-associated retinal diseases constitute a substantial unmet medical need, and this progress with OPGx-BEST1 represents an important step toward potentially preserving and restoring visual function for patients with Best disease.”

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.

The multi-center, adaptive, open-label, dose-exploring study, known as BIRD-1, will evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in 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 trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

About BEST1 Inherited Retinal Disease

Best disease, or vitelliform macular dystrophy, is a rare inherited retinal condition caused by mutations in the BEST1 gene, leading to impaired retinal pigment epithelium (RPE) function, progressive vision loss, and, in some cases, blindness. The BEST1 gene is responsible for providing instructions to produce bestrophin, a protein that acts as a channel to manage the movement of charged chloride ions in and out of retinal cells. Variants (mutations) in the BEST1 gene, as well as the PRPH2 gene, can result in the formation of abnormally shaped channels that cannot properly control chloride flow. BEST1 plays a key role in the RPE, which is essential for healthy vision, and such mutations can lead to BEST1-related inherited retinal diseases (bestrophinopathies). These rare conditions affect an estimated 9,000 patients across the United States and can lead to progressive vision loss and blindness.

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, RDH12, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, please 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 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.

FAQ

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

What did Opus Genetics (IRD) announce on December 9, 2025 about the BEST1 trial?

The company said the IDMC issued a positive recommendation to continue the Phase 1/2 BEST1 trial after a one-month sentinel safety review.

What did the IDMC review in the OPGx-BEST1 (IRD) study?

The IDMC completed a pre-specified safety review of the one-month data from the sentinel participant.

How will the IDMC recommendation affect enrollment in IRD’s BEST1 trial?

The recommendation allows the company to advance enrollment and dose the next four participants without modification.

Does the IDMC recommendation mean OPGx-BEST1 is proven safe for Best disease?

No; the recommendation reflects an encouraging early safety signal from one sentinel participant at one month, not a definitive safety or efficacy result.

What type of trial is the BEST1 (BIRD-1) study for Opus Genetics (IRD)?

BEST1 is a multi-center, adaptive, open-label, dose-exploring Phase 1/2 clinical trial evaluating OPGx-BEST1 in participants with Best disease.