Phentolamine Mesylate

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 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. The Company is based in Research Triangle Park, NC. For more information, visit . 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 cash runway and future financing availability, potential future funding under the Oberland facility, potential product approvals, and Priority Review Voucher opportunities, the clinical development, clinical results, preclinical data and future plans for Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, OPGx-MERTK, OPGx-RDH12, OPGx-RHO 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 most recent Annual Report on Form 10-K 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,” “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 Opus Genetics, Inc. Condensed Consolidated Balance Sheets (in thousands, except share amounts and par value) As of March 31, 2026 December 31, 2025 Assets (Unaudited) Current assets: Cash and cash equivalents $ 59,959 $ 45,091 Accounts receivable 1,731 1,995 Contract assets and unbilled receivables (Note 10) 984 1,170 Prepaids and other current assets 3,927 1,788 Total current assets 66,601 50,044 Property and equipment, net 186 199 Total assets $ 66,787 $ 50,243 Liabilities, convertible preferred stock and stockholders’ (deficit) equity Current liabilities: Accounts payable $ 4,422 $ 3,293 Accrued expenses 4,918 4,488 Total current liabilities 9,340 7,781 Warrant liabilities 77,349 25,985 Funding agreement, related party 1,198 1,129 Total liabilities 87,887 34,895 Commitments and contingencies (Note 3 and Note 9) Series B preferred stock, par value $0.0001; 7,374,632 shares and no shares were designated as of March 31, 2026 and December 31, 2025, respectively; 7,374,632 shares and no shares issued and outstanding at March 31, 2026 and December 31, 2025, respectively. 24,736 — Stockholders’ (deficit) equity: Preferred stock, par value $0.0001; 2,625,368 shares and 10,000,000 shares authorized as of March 31, 2026 and December 31, 2025, respectively; no shares issued and outstanding at March 31, 2026 and December 31, 2025. — — Common stock, par value $0.0001; 125,000,000 shares authorized as of March 31, 2026 and December 31, 2025; 71,402,472 and 69,894,507 shares issued and outstanding at March 31, 2026 and December 31, 2025, respectively. 7 7 Additional paid-in capital 208,281 203,930 Accumulated deficit (254,124 ) (188,589 ) Total stockholders’ (deficit) equity (45,836 ) 15,348 Total liabilities, convertible preferred stock and stockholders’ (deficit) equity $ 66,787 $ 50,243 Opus Genetics, Inc. Condensed Consolidated Statements of Comprehensive Loss (in thousands, except share and per share amounts) (Unaudited) For the Three Months Ended March 31, 2026 2025 License and collaborations revenue $ 2,157 $ 4,370 Operating expenses: Research and development 10,577 7,953 General and administrative 5,944 6,346 Total operating expenses 16,521 14,299 Loss from operations (14,364 ) (9,929 ) Fair value change in warrant liabilities (51,364 ) 2,805 Financing costs (582 ) (1,372 ) Interest expense (69 ) — Other income, net 844 302 Loss before income taxes (65,535 ) (8,194 ) Benefit (provision) for income taxes — — Net loss (65,535 ) (8,194 ) Other comprehensive loss, net of tax — — Comprehensive loss $ (65,535 ) $ (8,194 ) Net loss per share: Basic and diluted $ (0.75 ) $ (0.24 ) Number of shares used in per share calculations: Basic and diluted 86,891,352 33,884,920 Source: Opus Genetics, Inc.

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 . 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.