Opus Genetics Presents Clinical and Preclinical Data at ARVO 2026 Demonstrating Continued Pipeline Advancement in Inherited Retinal Diseases

Rhea-AI Summary

Opus Genetics (Nasdaq: IRD) reported ARVO 2026 clinical and preclinical data showing early functional gains across multiple inherited retinal disease programs. Six-month pediatric LCA5 data show >30-fold cone sensitivity improvements and visual-acuity gains. BEST1 adult data show up to 12-letter acuity improvement and ~23% central subfield thickness reduction. RHO programs report durable retinal preservation and a mutation-independent AAV approach advancing toward clinical translation.

Positive

• OPGx-LCA5: >30-fold improvement in cone sensitivity at six months
• OPGx-LCA5: visual acuity gains vs baseline and untreated eyes
• OPGx-BEST1: up to 12-letter visual acuity improvement at three months
• OPGx-BEST1: ~23% reduction in central subfield thickness
• RHO programs: NOAEL established and dose-dependent structural rescue in canine model
• Mutation-independent AAV: restored rod responses and preserved cone function in swine

Negative

• Clinical data are early-stage and preliminary (primary LCA5 results at six months)
• BEST1 topline 3-month Cohort 1 results pending (expected September 2026)
• Durability beyond reported timepoints is not yet demonstrated in humans

News Market Reaction – IRD

-6.90%

12 alerts

-6.90% Session close to close

+5.1% Peak Tracked

-2.5% Trough Tracked

$356.30M Market Cap

1.1x Rel. Volume

In the May 8 session, IRD declined 6.90%, reflecting a notable negative 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.

Market Context

The stock moved -6.9% in the session following this news. A negative reaction despite detailed posit...

Analysis

The stock moved -6.9% in the session following this news. A negative reaction despite detailed positive ARVO data would fit a pattern where IRD often traded lower after seemingly constructive updates, as seen following multiple March–May news events. With shares still above the $2.68 200-day MA and well off the $0.9011 52-week low, downside moves could partly reflect profit-taking after a strong multi-month run and awareness of registered resale shares rather than a clear repudiation of the LCA5, BEST1, or RHO programs.

Key Figures

Clinical follow-up: 6-month data Cone sensitivity gain: Over 30-fold improvement Visual acuity gain: Up to 12-letter improvement +5 more

8 metrics

Clinical follow-up 6-month data Phase 1/2/3 OPGx-LCA5 pediatric LCA5 study

Cone sensitivity gain Over 30-fold improvement Cone-mediated sensitivity across all OPGx-LCA5 treated patients

Visual acuity gain Up to 12-letter improvement Visual acuity in BEST1 treated eye at 3 months

Retinal thickness change ~23% reduction Central subfield thickness in BEST1 treated eye

Potency range 50–150% Manufacturing lot potency within expected ranges for OPGx-BEST1

Registered resale shares 7,374,632 shares Common stock issuable upon Series B Non-Voting Convertible Preferred conversion

Shares outstanding 71,402,472 shares Common stock outstanding as of March 31, 2026

NOAEL established No-observed-adverse-effect-level Guides clinical dosing for OPGx-RHO canine study

Historical Context

5 past events · Latest: May 04 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
May 04	Regulatory program update	Positive	-0.4%	FDA accepted OPGx-LCA5 into the Rare Disease Evidence Principles program.
Apr 27	Conference participation	Positive	-2.1%	Planned presentations on OPGx-BEST1 data and OPGx-RHO preclinical work.
Apr 10	Conference data update	Positive	-2.5%	Multiple ASCRS abstracts highlighting presbyopia and mesopic-vision programs.
Apr 06	Financing agreement	Positive	+1.3%	Up to $155M Oberland Capital facility and $5M equity investment extending runway.
Mar 24	Corporate recognition	Positive	-1.7%	Named to Fast Company’s 2026 World’s Most Innovative Companies list in Biotech.

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

Pattern Detected

Recent IRD news and conference/data updates have often seen muted or negative next-day moves even when headlines were operationally positive, with only one out of five events showing a positive 24h price reaction.

Recent Company History

Over the past several months, Opus Genetics has reported multiple positive corporate and pipeline milestones. On Mar 24, 2026, it was named to Fast Company’s Most Innovative Companies list, yet the stock fell 1.74%. A strategic financing facility of up to $155 million on Apr 6, 2026 produced a modest 1.32% gain. Subsequent conference and FDA RDEP announcements in April and early May, all supportive of the IRD pipeline, were followed by small share-price declines. Today’s ARVO data add richer clinical detail to the same LCA5, BEST1 and RHO programs highlighted previously.

Key Terms

phase 1/2/3, subretinal injection, visual acuity, central subfield thickness, +3 more

7 terms

phase 1/2/3 medical

"reported 6-month results from the ongoing Phase 1/2/3 study of OPGx-LCA5"

Phase 1, 2 and 3 are the main stages of clinical testing for a drug or medical device: Phase 1 checks safety and appropriate dose in a small group, Phase 2 looks for signs the treatment works and gathers more safety data in a larger group, and Phase 3 confirms effectiveness and rare side effects in large, controlled studies. Investors treat these stages as milestone checkpoints because each successful phase lowers development risk and raises a product’s commercial value, while failures or delays can sharply reduce a company’s prospects.

subretinal injection medical

"cone-mediated function following a single subretinal injection."

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.

visual acuity medical

"Early BEST1 Data Show Visual Acuity Gains and Improved Retinal Structure"

Visual acuity is the sharpness or clarity of a person’s vision — how well they can distinguish fine details at a given distance, like the resolution of a camera. Investors care because changes in acuity are a common, measurable outcome for eye drugs, devices and treatments; improvements or declines can drive regulatory approval, reimbursement decisions, market demand and the perceived effectiveness of medical products.

central subfield thickness medical

"~23% reduction in central subfield thickness, indicating improved retinal structure"

Central subfield thickness is a measurement of the retina’s thickness at the very center of the macula, typically taken by an imaging scan of the eye; think of it like measuring the height of the ground at the center of a small target. It matters to investors because changes in this number are a common, objective medical endpoint used to judge whether eye drugs or devices relieve swelling and improve vision, which influences regulatory approval, market potential, and sales forecasts.

no-observed-adverse-effect-level (NOAEL) medical

"Established no-observed-adverse-effect-level (NOAEL) to guide clinical dosing"

The no-observed-adverse-effect-level (NOAEL) is the highest exposure or dose in a safety study at which no harmful effects are seen in the test subjects. Investors care because regulators and companies use this threshold to set safe human exposure limits, guide clinical dosing, and determine whether a product can be approved or requires additional testing — much like a speed limit set after testing where no accidents occurred, it helps predict regulatory risk and liability.

aav medical

"Mutation-Independent AAV Approach Highlights Potential to Address Genetically"

AAV is a small, generally harmless virus repurposed by researchers as a delivery vehicle to insert therapeutic genes into human cells; think of it as a postal service that carries corrective DNA to specific tissues. Investors pay attention because AAV-based treatments can offer durable, potentially one-time cures that command high prices, but they also carry development, manufacturing and regulatory risks that can sharply influence a biotech company’s value.

elisa technical

"assays (RT-dPCR, western blot, ELISA) to quantify gene expression and protein"

A laboratory test that detects specific proteins or antibodies in a sample by producing a measurable color or signal, similar to how a home pregnancy test shows a line when it finds a target substance. Investors care because ELISA results help determine whether a medical product works, meets regulatory standards, or can be manufactured reliably — factors that affect a company’s revenue potential, approval timelines, and risk profile.

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

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Six-Month Clinical Data Demonstrate Restoration of Cone-Mediated Vision in Pediatric LCA5 Patients, with Sensitivity Improvements Reaching Normal Ranges

Early BEST1 Data Show Visual Acuity Gains and Improved Retinal Structure, Supporting Expansion into a Large IRD Population

Preclinical RHO Programs Demonstrate Durable Retinal Preservation and Support Clinical Translation

Mutation-Independent AAV Approach Highlights Potential to Address Genetically Diverse Retinal Diseases with a Single Therapy

Advancements In Potency Assays and Manufacturing Reinforce Scalability and Regulatory Readiness

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 new clinical and preclinical data from multiple programs presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2026 in Denver, Colorado.

The data highlight emerging evidence that Opus Genetics’ gene therapy OPGx-LCA5 may restore daytime vision mediated by cones in pediatric patients with severe, early-onset disease, while also advancing Opus Genetics’ broader pipeline across BEST1 and RHO programs.

“Our ARVO presentations reflect a meaningful shift in what gene therapy can achieve in retinal disease,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We are seeing consistent evidence that we may be able to rapidly restore visual function, with encouraging clinical signals and a growing foundation across multiple programs for patients with genetic blinding diseases.”

Oral Presentation

Restoration of Cone-Mediated Vision After Gene Augmentation in Children with LCA5
The oral presentation, delivered by Tomas S. Aleman, M.D., primary study investigator, reported 6-month results from the ongoing Phase 1/2/3 study of OPGx-LCA5 in pediatric patients with Leber congenital amaurosis type 5 (LCA5), a severe, early-onset, inherited retinal degeneration.

Despite advanced disease and very poor baseline vision, patients treated with OPGx-LCA5 demonstrated robust and consistent restoration of cone-mediated function following a single subretinal injection.

Key findings include:

• Well tolerated, with no dose-limiting toxicities and adverse events that were mild, expected, and resolved
• Over 30-fold improvements in cone sensitivity across all treated patients, representing recovery of primary photoreceptor function
• Improvement in visual acuity in treated eyes relative to baseline and untreated control eyes
• Objective confirmation of efficacy across multiple independent readouts, including dark-adapted pupillary light responses showing improved amplitude and latency thresholds
• Early and durable improvements in functional vision in an orientation and mobility test
• Consistent patient-reported improvements in daily functional vision

“These results indicate that even in severe, early-onset disease, there remains a possible window of opportunity to restore cone function and meaningfully improve vision,” said Dr. Tomas S. Aleman. “Gene augmentation in LCA5 demonstrated dramatic recovery of daytime vision in adolescents, paving the path for use in younger patients with theoretically greater treatment potential. Importantly, these results build on prior adult data and demonstrate that in severe pediatric disease, viable cone photoreceptors may be rescued, supporting a broader therapeutic window.”

Poster Presentations

Preliminary Results from Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for ARB and BVMD Due to BEST1 Mutations

• Well tolerated through three months with no ocular inflammation or treatment-related adverse events
• Up to 12-letter improvement in visual acuity in the treated eye
• ~23% reduction in central subfield thickness, indicating improved retinal structure
• Early patient-reported improvements, including reduced perception of progressive vision dimming

These findings provide early clinical evidence for BEST1 gene augmentation in a large inherited retinal disease population. Opus Genetics has completed enrollment in Cohort 1 of its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, and expects to announce 3-month topline data from Cohort 1 in September 2026.

Development of Cell-Based Expression and Functional Potency Assays for OPGx-BEST1 Gene Therapy

• Developed robust, reproducible assays (RT-dPCR, western blot, ELISA) to quantify gene expression and protein production
• Demonstrated consistent potency across manufacturing lots within expected ranges (50–150%)
• Established a functional assay to measure BEST1 channel activity, supporting mechanism-driven validation
  

These capabilities support scalable development and regulatory readiness.

Nonclinical Efficacy and Toxicity Study of GMP-Grade Vector OPGx-RHO Delivered by Subretinal Injection in a Canine Model of RHO-adRP

• Established no-observed-adverse-effect-level (NOAEL) to guide clinical dosing
• Demonstrated preservation of retinal structure and function in treated regions
• Observed dose-dependent structural rescue, supporting clinical translation
  

Therapeutic Efficacy of a Mutation-Independent AAV Knockdown and Replacement Approach in a Swine Animal Model of Autosomal-Dominant Retinitis Pigmentosa (RHO)

• Mutation-independent approach demonstrated:
  • Restoration of rod-driven visual responses
  • Maintenance of cone function over time
  • Preservation of retinal structure, including outer retinal thickness and photoreceptor morphology
• Identified minimal effective dose and evidence of durability across timepoints
• Data also suggest interocular vector transfer, indicating potential systemic distribution dynamics

These results support a differentiated strategy to treat genetically heterogeneous retinal diseases with a single therapeutic approach.

The presentations and posters will be available on the Opus Genetics website here.

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 Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12, MERTK, RHO, CNGB1 and NMNAT1, 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, 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.

FAQ

What did Opus Genetics announce about OPGx-LCA5 six-month results (IRD)?

Six-month pediatric data showed >30-fold cone sensitivity improvements and visual-acuity gains in treated eyes. According to Opus Genetics, independent readouts included pupillary responses and orientation-and-mobility tests confirming early functional recovery.

What were the early clinical findings for OPGx-BEST1 reported at ARVO 2026 (IRD)?

Early adult data reported up to a 12-letter improvement in treated-eye acuity and ~23% central subfield thickness reduction. According to Opus Genetics, Cohort 1 enrollment is complete and 3-month topline data are expected September 2026.

What preclinical RHO program results did Opus Genetics present (IRD)?

Preclinical studies showed preservation of retinal structure and function with an established NOAEL in canine models. According to Opus Genetics, results support clinical translation and identified dose-dependent structural rescue signals.

How does Opus Genetics' mutation-independent AAV approach perform in models of RHO-related RP (IRD)?

The mutation-independent knockdown-and-replace AAV restored rod-driven responses and maintained cone function over time in a swine model. According to Opus Genetics, minimal effective dose and durability signals were observed across timepoints.

Are the ARVO 2026 results from Opus Genetics sufficient to change clinical guidance or approval timelines (IRD)?

No definitive guidance or approval timelines were provided; data are early and intended to support further development. According to Opus Genetics, the findings advance regulatory readiness but do not report regulatory approvals or final clinical outcomes.