Opus Genetics Virtual R&D Science Forum Scheduled for Tuesday, June 16, 2026, at 10:00 am ET

Upcoming Data Readout for Cohort 1 of OPGx-BEST1 Gene Therapy Study Expected in September 2026

LCA5 Accepted to FDA's Rare Disease Evidence Principles (RDEP) Program and Enrolling Pivotal Trial

RDH12, MERTK and RHO Programs Expected to Advance to Clinical Testing in the Next 12 Months

Cash Runway into 2029 Expected to Support Multiple Clinical Inflection Points, Potential Product Approvals

and Opportunities for Priority Review Vouchers

RESEARCH TRIANGLE PARK, N.C., May 12, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the "Company" or "Opus Genetics"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced financial results for the first quarter ended March 31, 2026, and provided a corporate update.

"With encouraging momentum across both our LCA5 and BEST1 gene therapy programs, Opus Genetics is entering a defining stretch where we believe our precision targeted approach can reshape what's possible for patients with inherited retinal diseases," said George Magrath, M.D., Chief Executive Officer of Opus Genetics. "Our upcoming BEST1 Cohort 1 clinical data and June R&D Science Forum will provide key opportunities to showcase the depth and maturity of our science and pipeline. Backed by a strong balance sheet and disciplined capital strategy, we are well-positioned to advance our lead programs toward pivotal trials, and accelerate our earlier--stage RDH12, MERTK, and RHO programs into the clinic."

Pipeline Updates

Research & Development (R&D) Science Forum

   -- Opus Genetics plans to host an R&D Science Forum on Tuesday, June 16, 
      2026, from 10:00 am -- 12:00 pm ET, with management and key opinion 
      leaders (KOLs) highlighting earlier-stage gene therapy programs entering 
      clinical testing and IRD global market opportunities. Registration for 
      the event will be available on the Events page of the Opus Genetics 
      website. 

OPGx-BEST1

   -- Enrollment completed in Cohort 1 of the Phase 1/2 trial (BIRD-1) 
      including participants with both dominant and recessive forms of BEST 
      disease, with the final participant scheduled for dosing this month. 
 
   -- Baseline demographics were presented at the Association for Research in 
      Vision and Ophthalmology (ARVO) 2026 Annual Meeting with the related 
      poster presentation available on the Publications & Presentations page of 
      the Opus Genetics website. Additionally, a slide presentation and video 
      summary recording titled "OPGx-BEST1 Cohort 1 Baseline Demographics and 
      Key Endpoints for IRDs" are provided on the IR Presentations page of the 
      Opus Genetics website. 
 
   -- Opus Genetics expects to announce three-month topline data from Cohort 1 
      of the Phase 1/2 trial in September 2026. 

OPGx-LCA5

   -- Positive six-month pediatric cohort data was presented at ARVO 2026; 
      robust and consistent improvements were observed in cone-mediated 
      function with average gains of approximately 1.5 log unit in cone 
      sensitivity, reaching normal ranges after a single dose. Improvements 
      were also observed in objective measures, visual acuity, and functional 
      vision. The therapy was well tolerated, with most adverse events 
      anticipated, mild, and not related to OPGx-LCA5. 
 
   -- The U.S. Food and Drug Administration (FDA) granted acceptance of the 
      LCA5 program into the Rare Disease Evidence Principles (RDEP) program, 
      which is designed to provide guidance on the types of evidence that can 
      be used to demonstrate substantial evidence of effectiveness, including 
      the potential use of a single adequate and well-controlled study 
      supported by confirmatory evidence. 
 
   -- Recruitment is ongoing in the run-in period for the pivotal Phase 3 
      trial. In parallel, the clinical and commercial batch of drug product is 
      being manufactured. Dosing with OPGx-LCA5 is expected to start in the 
      fourth quarter of 2026. 

OPGx-RDH12

   -- OPGx-RDH12 is being developed to deliver a functional RDH12 gene to 
      photoreceptors using an AAV vector. 
 
   -- This program is expected to enter the clinic in the fourth quarter of 
      2026. Funding is supported by the RDH12 Alliance. 

OPGx-MERTK

   -- OPGx-MERTK is being developed for retinal degeneration caused by 
      mutations in the MERTK gene, which plays a critical role in phagocytosis 
      of photoreceptor outer segments by RPE cells. 
 
   -- Clinical development activities are underway. Funding is supported by a 
      consortium led by Abu Dhabi's Healthcare Research and Innovation Fund. 

OPGx-RHO

   -- Preclinical data presented at ARVO 2026 and the Foundation Fighting 
      Blindness Retinal Therapeutics Innovation Summit 2026 provided safety and 
      efficacy data on the use of OPGx-RHO in two large animal models of 
      autosomal-dominant retinitis pigmentosa (adRP). This preclinical work was 
      co-funded by the Foundation Fighting Blindness and the National 
      Institutes of Health. 
 
   -- This program is expected to enter the clinic in 2027. 

Recent Medical Presentations

   -- ARVO 2026 Annual Meeting: 
 
          -- "Restoration of Cone-Mediated Vision After Gene Augmentation in 
             Children with LCA5" 
 
          -- "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" 
 
          -- "Development of Cell-Based Expression and Functional Potency 
             Assays for OPGx-BEST1 Gene Therapy" 
 
          -- "Nonclinical Efficacy and Toxicity Study of GMP-Grade Vector 
             OPGx-RHO (Scaav2/5-RHO820-Shrna820) Delivered by Subretinal 
             Injection in a Canine Model of RHO-adRP" 
 
          -- "Therapeutic Efficacy of a Mutation-Independent AAV Knockdown and 
             Replacement Approach in a Swine Animal Model of Autosomal-Dominant 
             Retinitis Pigmentosa (adRP)" 
 
   -- Foundation Fighting Blindness Retinal Therapeutics Innovation Summit 
      2026: "Safety and Efficacy of OPGx-RHO Silence-and-Replace Gene Therapy 
      for RHO-adRP: Evidence Across Two Large Animal Models" 
 
   -- The Macula Society Annual Meeting: "Preliminary Results from Sentinel 
      Patient in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for 
      the Treatment of BVMD and ARB Due to BEST1 Mutations" 
 
   -- Asia-Pacific Academy of Ophthalmology Congress (APAO): "Gene Therapy for 
      BEST1 Inherited Retinal Disease" 
 
   -- Advanced Therapies Week: "Building Scalable Viral Vector Manufacturing 
      Models" 

Financial Results for the First Quarter Ended March 31, 2026

Cash Position: As of March 31, 2026, Opus Genetics had cash and cash equivalents of $60 million. Subsequent to the end of the period, the Company entered into a strategic financing agreement with Oberland Capital Management, which included funding of a principal amount of $35 million of senior secured notes and a commitment to a $5 million equity investment, which is expected to close on or about May 22, 2026, subject to the satisfaction of customary closing conditions. With approximately $90 million in current cash, and potential future fundings under the facility with Oberland Capital Management, the Company believes its aggregate cash resources will fund operations into 2029. This estimate excludes any potential proceeds from callable warrants or future milestone payments.

Revenue: License and collaborations revenue totaled $2.2 million for the quarter ended March 31, 2026, compared to $4.4 million for the same period in 2025. Revenue in both periods came primarily from reimbursement of research and development (R&D) services based on the Company's collaboration with Viatris, Inc.

Research and Development (R&D) Expenses: R&D expenses were $10.6 million for the quarter ended March 31, 2026, compared to $8.0 million for the same period in 2025. The increase was primarily attributable to higher manufacturing costs associated with the Company's IRD programs, partially offset by lower clinical costs associated with the Company's phentolamine-related programs. R&D expenses included $0.4 million and $0.3 million in stock-based compensation expense for the three months ended March 31, 2026 and 2025, respectively.

General and Administrative (G&A) Expenses: G&A expenses were $5.9 million for the quarter ended March 31, 2026, compared to $6.3 million for the same period in 2025. The decrease was primarily attributable to lower legal costs, partially offset by higher employee compensation-related costs. G&A expenses included $1.1 million and $0.6 million in stock-based compensation expense for the three months ended March 31, 2026 and 2025, respectively.

Net Loss: Net loss for the quarter ended March 31, 2026 was $65.5 million, or ($0.75) per basic and diluted share, compared to a net loss of $8.2 million, or ($0.24) per basic and diluted share, for the same period in 2025. The increase in net loss was primarily due to a non-cash expense, driven by the increase in the fair value change in warrant liabilities associated with the Company's March 2025 warrants, and higher R&D expenses as discussed above.

About Opus Genetics

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