{"success":true,"data":{"pressRelease":{"id":"106024","rtpr_id":"nGNX70QsvF","ticker":"IRD","exchange":"NASDAQ","all_tickers":["IRD"],"title":"Opus Genetics Completes Patient Enrollment in Registrational Phase 3 Trial of OPGx-LCA5 for LCA5-Associated Inherited Retinal Disease","author":"Globe Newswire","published_at":"2026-08-03T11:00:00.292Z","article_body":"Topline six-month efficacy data expected by end of 2027\n\nPhase 3 study follows FDA alignment on registrational trial design through\nRare Disease Evidence Principles (RDEP) program\n\nOpus Genetics may submit a BLA based on 6-month efficacy data, with 12-month\ndurability data provided during review\n\nRESEARCH TRIANGLE PARK, N.C., Aug. 03, 2026 (GLOBE NEWSWIRE) -- Opus Genetics,\nInc\n(https://www.globenewswire.com/Tracker?data=wTBGOgcWA5OglKKNYwuSrygHo3lTmmQBitQ10J_adTKdGwpW1Fs8bjqHgjk8cWG29rHTE7mJ8zN1yAdhQl4jLT6aYhpof-jt7jhK4ff-TIg=).\n(Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage\nbiopharmaceutical company developing gene therapies to restore vision and\nprevent blindness in patients with inherited retinal diseases (IRDs), today\nannounced that the last patient has been enrolled in its registrational Phase\n3 clinical trial evaluating OPGx-LCA5, the Company’s investigational gene\ntherapy for LCA5-associated inherited retinal disease.\n\nCompletion of enrollment marks a significant milestone in the development of\nOPGx-LCA5 and follows the program’s acceptance into the U.S. Food and Drug\nAdministration’s (FDA) Rare Disease Evidence Principles (RDEP) program in\nMay 2026. The Phase 3 study was designed in collaboration with the FDA to\nsupport a potential Biologics License Application (BLA) for what could become\nthe first approved therapy for patients living with LCA5-associated inherited\nretinal disease.\n\n“Achieving full enrollment in our registrational Phase 3 trial is a\nsignificant milestone for the OPGx-LCA5 program and reflects the dedication of\nparticipating patients and families, our clinical investigators, and our\nteam,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics.\n“With enrollment now complete, we remain on track to initiate dosing in the\nfourth quarter of 2026, report topline data by the end of 2027, and continue\nadvancing OPGx-LCA5 to bring the first potential treatment to patients with\nLCA5-associated inherited retinal disease.”\n\nThe registrational Phase 3 trial is evaluating the safety and efficacy of a\none-time subretinal administration of OPGx-LCA5 in patients with genetically\nconfirmed LCA5-associated inherited retinal disease. Consistent with the\nCompany’s RDEP discussions with the FDA, the study incorporates innovative\nevidence-generation approaches appropriate for an ultra-rare disease,\nincluding a six-month run-in period in which patients serve as their own\ncontrol. Participants are currently completing the run-in period, and the\nCompany expects to initiate dosing of OPGx-LCA5 in the fourth quarter of 2026,\nwith topline data expected by the end of 2027.\n\nOPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative\nMedicine Advanced Therapy (RMAT) designations from the FDA and has been\naccepted into the FDA’s Rare Disease Evidence Principles (RDEP) program.\n\nThe Company continues to expect that OPGx-LCA5 may qualify for a Rare\nPediatric Disease Priority Review Voucher upon approval, representing a\npotentially significant strategic asset.\n\nAbout OPGx-LCA5\n\nOPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA)\ndue to biallelic mutations in the LCA5 gene (LCA5), which encodes the\nlebercilin protein. LCA5-associated inherited retinal disease is an\nearly-onset severe inherited retinal dystrophy. Studies in patients with this\nmutation have reported evidence for the dissociation of retinal architecture\nand visual function in this disease, suggesting an opportunity for therapeutic\nintervention through gene augmentation. OPGx-LCA5 uses an adeno-associated\nvirus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer\nretina. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and\nRegenerative Medicine Advanced Therapy (RMAT) designations from the FDA and\nhas been accepted into the FDA’s Rare Disease Evidence Principles (RDEP)\nprogram. OPGx-LCA5 is currently being evaluated in a Phase 1/2/3 clinical\ntrial.\n\nAbout Opus Genetics\n\nOpus Genetics is a clinical-stage biopharmaceutical company developing gene\ntherapies to restore vision and prevent blindness in patients with inherited\nretinal diseases (IRDs). The Company is developing durable, one-time\ntreatments designed to address the underlying genetic causes of severe retinal\ndisorders. The Company’s pipeline includes seven AAV-based programs, led by\nOPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal\ndegeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1\nand NMNAT1. The Company is based in Research Triangle Park, NC. For more\ninformation, visit www.opusgtx.com.\n\nForward-Looking Statements\n\nThis press release contains certain statements that are not statements of\nhistorical fact and are forward-looking statements within the meaning of\nSection 27A of the Securities Act of 1933, as amended, Section 21E of the\nSecurities Exchange Act of 1934, as amended, and the Private Securities\nLitigation Reform Act of 1995. Such statements include, but are not limited\nto, statements related to the anticipated timing of topline data from the\nOPGx-LCA5 Phase 3 clinical trial, clinical development, clinical results,\npreclinical data, and future plans for OPGx-LCA5 and expectations regarding\nus, our business prospects, and our results of operations and are subject to\ncertain risks and uncertainties posed by many factors and events that could\ncause our actual business, prospects and results of operations to differ\nmaterially from those anticipated by such forward-looking statements. Factors\nthat could cause or contribute to such differences include, but are not\nlimited to, those described under the heading “Risk Factors” included in\nour most recent Annual Report on Form 10-K for the fiscal year ended December\n31, 2025, our Quarterly Report on Form 10-Q for the quarter ended March 31,\n2026, and in our other filings with the U.S. Securities and Exchange\nCommission. Readers are cautioned not to place undue reliance on these\nforward-looking statements, which speak only as of the date of this press\nrelease. These forward-looking statements are based upon our current\nexpectations and involve assumptions that may never materialize or may prove\nto be incorrect. Actual results and the timing of events could differ\nmaterially from those anticipated in such forward-looking statements as a\nresult of various risks and uncertainties. In some cases, you can identify\nforward-looking statements by the following words: “anticipate,”\n“believe,” “continue,” “could,” “estimate,” “expect,”\n“intend,” “aim,” “may,” “ongoing,” “plan,”\n“potential,” “predict,” “project,” “should,” “strive,”\n“will,” “would” or the negative of these terms or other comparable\nterminology, although not all forward-looking statements contain these words.\nWe undertake no obligation to revise any forward-looking statements in order\nto reflect events or circumstances that might subsequently arise.\n\nContacts:\n\nInvestors\nJenny Kobin\nRemy Bernarda\nIR Advisory Solutions\nir@opusgtx.com\n\nMedia\nKimberly Ha\nKKH Advisors\n917-291-5744\nkimberly.ha@kkhadvisors.com\n\nSource: Opus Genetics, Inc.\n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/468baef1-3b2d-4353-a17e-cb41b45ee6ef)\n\n\n\nGlobeNewswire, Inc. 2026","article_body_html":"","raw_payload":{"data":{"id":"nGNX70QsvF","title":"Opus Genetics Completes Patient Enrollment in Registrational Phase 3 Trial of OPGx-LCA5 for LCA5-Associated Inherited Retinal Disease","author":"Globe Newswire","ticker":"IRD","created":"2026-08-03T11:00:00.292Z","tickers":["IRD"],"exchange":"NASDAQ","article_body":"Topline six-month efficacy data expected by end of 2027\n\nPhase 3 study follows FDA alignment on registrational trial design through\nRare Disease Evidence Principles (RDEP) program\n\nOpus Genetics may submit a BLA based on 6-month efficacy data, with 12-month\ndurability data provided during review\n\nRESEARCH TRIANGLE PARK, N.C., Aug. 03, 2026 (GLOBE NEWSWIRE) -- Opus Genetics,\nInc\n(https://www.globenewswire.com/Tracker?data=wTBGOgcWA5OglKKNYwuSrygHo3lTmmQBitQ10J_adTKdGwpW1Fs8bjqHgjk8cWG29rHTE7mJ8zN1yAdhQl4jLT6aYhpof-jt7jhK4ff-TIg=).\n(Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage\nbiopharmaceutical company developing gene therapies to restore vision and\nprevent blindness in patients with inherited retinal diseases (IRDs), today\nannounced that the last patient has been enrolled in its registrational Phase\n3 clinical trial evaluating OPGx-LCA5, the Company’s investigational gene\ntherapy for LCA5-associated inherited retinal disease.\n\nCompletion of enrollment marks a significant milestone in the development of\nOPGx-LCA5 and follows the program’s acceptance into the U.S. Food and Drug\nAdministration’s (FDA) Rare Disease Evidence Principles (RDEP) program in\nMay 2026. The Phase 3 study was designed in collaboration with the FDA to\nsupport a potential Biologics License Application (BLA) for what could become\nthe first approved therapy for patients living with LCA5-associated inherited\nretinal disease.\n\n“Achieving full enrollment in our registrational Phase 3 trial is a\nsignificant milestone for the OPGx-LCA5 program and reflects the dedication of\nparticipating patients and families, our clinical investigators, and our\nteam,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics.\n“With enrollment now complete, we remain on track to initiate dosing in the\nfourth quarter of 2026, report topline data by the end of 2027, and continue\nadvancing OPGx-LCA5 to bring the first potential treatment to patients with\nLCA5-associated inherited retinal disease.”\n\nThe registrational Phase 3 trial is evaluating the safety and efficacy of a\none-time subretinal administration of OPGx-LCA5 in patients with genetically\nconfirmed LCA5-associated inherited retinal disease. Consistent with the\nCompany’s RDEP discussions with the FDA, the study incorporates innovative\nevidence-generation approaches appropriate for an ultra-rare disease,\nincluding a six-month run-in period in which patients serve as their own\ncontrol. Participants are currently completing the run-in period, and the\nCompany expects to initiate dosing of OPGx-LCA5 in the fourth quarter of 2026,\nwith topline data expected by the end of 2027.\n\nOPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative\nMedicine Advanced Therapy (RMAT) designations from the FDA and has been\naccepted into the FDA’s Rare Disease Evidence Principles (RDEP) program.\n\nThe Company continues to expect that OPGx-LCA5 may qualify for a Rare\nPediatric Disease Priority Review Voucher upon approval, representing a\npotentially significant strategic asset.\n\nAbout OPGx-LCA5\n\nOPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA)\ndue to biallelic mutations in the LCA5 gene (LCA5), which encodes the\nlebercilin protein. LCA5-associated inherited retinal disease is an\nearly-onset severe inherited retinal dystrophy. Studies in patients with this\nmutation have reported evidence for the dissociation of retinal architecture\nand visual function in this disease, suggesting an opportunity for therapeutic\nintervention through gene augmentation. OPGx-LCA5 uses an adeno-associated\nvirus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer\nretina. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and\nRegenerative Medicine Advanced Therapy (RMAT) designations from the FDA and\nhas been accepted into the FDA’s Rare Disease Evidence Principles (RDEP)\nprogram. OPGx-LCA5 is currently being evaluated in a Phase 1/2/3 clinical\ntrial.\n\nAbout Opus Genetics\n\nOpus Genetics is a clinical-stage biopharmaceutical company developing gene\ntherapies to restore vision and prevent blindness in patients with inherited\nretinal diseases (IRDs). The Company is developing durable, one-time\ntreatments designed to address the underlying genetic causes of severe retinal\ndisorders. The Company’s pipeline includes seven AAV-based programs, led by\nOPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal\ndegeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1\nand NMNAT1. The Company is based in Research Triangle Park, NC. For more\ninformation, visit www.opusgtx.com.\n\nForward-Looking Statements\n\nThis press release contains certain statements that are not statements of\nhistorical fact and are forward-looking statements within the meaning of\nSection 27A of the Securities Act of 1933, as amended, Section 21E of the\nSecurities Exchange Act of 1934, as amended, and the Private Securities\nLitigation Reform Act of 1995. Such statements include, but are not limited\nto, statements related to the anticipated timing of topline data from the\nOPGx-LCA5 Phase 3 clinical trial, clinical development, clinical results,\npreclinical data, and future plans for OPGx-LCA5 and expectations regarding\nus, our business prospects, and our results of operations and are subject to\ncertain risks and uncertainties posed by many factors and events that could\ncause our actual business, prospects and results of operations to differ\nmaterially from those anticipated by such forward-looking statements. Factors\nthat could cause or contribute to such differences include, but are not\nlimited to, those described under the heading “Risk Factors” included in\nour most recent Annual Report on Form 10-K for the fiscal year ended December\n31, 2025, our Quarterly Report on Form 10-Q for the quarter ended March 31,\n2026, and in our other filings with the U.S. Securities and Exchange\nCommission. Readers are cautioned not to place undue reliance on these\nforward-looking statements, which speak only as of the date of this press\nrelease. These forward-looking statements are based upon our current\nexpectations and involve assumptions that may never materialize or may prove\nto be incorrect. Actual results and the timing of events could differ\nmaterially from those anticipated in such forward-looking statements as a\nresult of various risks and uncertainties. In some cases, you can identify\nforward-looking statements by the following words: “anticipate,”\n“believe,” “continue,” “could,” “estimate,” “expect,”\n“intend,” “aim,” “may,” “ongoing,” “plan,”\n“potential,” “predict,” “project,” “should,” “strive,”\n“will,” “would” or the negative of these terms or other comparable\nterminology, although not all forward-looking statements contain these words.\nWe undertake no obligation to revise any forward-looking statements in order\nto reflect events or circumstances that might subsequently arise.\n\nContacts:\n\nInvestors\nJenny Kobin\nRemy Bernarda\nIR Advisory Solutions\nir@opusgtx.com\n\nMedia\nKimberly Ha\nKKH Advisors\n917-291-5744\nkimberly.ha@kkhadvisors.com\n\nSource: Opus Genetics, Inc.\n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/468baef1-3b2d-4353-a17e-cb41b45ee6ef)\n\n\n\nGlobeNewswire, Inc. 2026"},"type":"article","timestamp":"2026-08-03T11:00:00.488291308Z","server_sent_at_ms":1785754800488},"received_at":"2026-08-03T11:00:00.540Z","source_url":null},"analysis":{"id":"95033","press_release_id":"106024","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":[],"eventType":"clinical_trial","narrative":"Opus Genetics completed patient enrollment in its registrational Phase 3 trial of OPGx-LCA5 for LCA5-associated inherited retinal disease, marking a key de-risking event.\n\nThe study incorporates innovative evidence-generation approaches aligned with the FDA's Rare Disease Evidence Principles program, with topline efficacy data expected by the end of 2027.\n\nDosing is set to begin in the fourth quarter of 2026, and the therapy holds potential for a Biologics License Application and a Rare Pediatric Disease Priority Review Voucher.","sentiment":"bullish","agentHooks":{"shouldPost":false,"suggestedAngle":"Phase 3 enrollment complete for lead asset OPGx-LCA5; data readout targeted for end of 2027."},"keyFigures":{"drugName":"OPGx-LCA5","phaseOfTrial":"Phase 3","customDimensions":{"designations":["Rare Pediatric Disease","Orphan Drug","Regenerative Medicine Advanced Therapy (RMAT)"],"dosing_initiation":"fourth quarter of 2026","topline_data_timing":"end of 2027"}},"quotedText":"Achieving full enrollment in our registrational Phase 3 trial is a significant milestone for the OPGx-LCA5 program and reflects the dedication of participating patients and families, our clinical investigators, and our team","namedEntities":{"people":[{"name":"George Magrath, M.D.","role":"Chief Executive Officer"}],"products":["OPGx-LCA5"],"companies":[{"name":"Opus Genetics, Inc.","ticker":"IRD"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"materialImpact":{"score":3,"reasoning":"Completion of enrollment in the registrational Phase 3 trial is a critical de-risking milestone for the company's lead program. It confirms execution capability and establishes a clear timeline for topline data and potential regulatory submission, though the binary readout is still over a year away."},"tickerRelevance":{"others":[],"primary":"IRD"},"globalImportance":30,"audienceRelevance":20,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"small-cap","eventGravity":"clinical-trial-milestone","sectorWeight":"biotech"}},"event_type":"clinical_trial","event_type_secondary":null,"sentiment":"bullish","material_impact_score":3,"narrative":"Opus Genetics completed patient enrollment in its registrational Phase 3 trial of OPGx-LCA5 for LCA5-associated inherited retinal disease, marking a key de-risking event.\n\nThe study incorporates innovative evidence-generation approaches aligned with the FDA's Rare Disease Evidence Principles program, with topline efficacy data expected by the end of 2027.\n\nDosing is set to begin in the fourth quarter of 2026, and the therapy holds potential for a Biologics License Application and a Rare Pediatric Disease Priority Review Voucher.","key_figures":{"drugName":"OPGx-LCA5","phaseOfTrial":"Phase 3","customDimensions":{"designations":["Rare Pediatric Disease","Orphan Drug","Regenerative Medicine Advanced Therapy (RMAT)"],"dosing_initiation":"fourth quarter of 2026","topline_data_timing":"end of 2027"}},"named_entities":{"people":[{"name":"George Magrath, M.D.","role":"Chief Executive Officer"}],"products":["OPGx-LCA5"],"companies":[{"name":"Opus Genetics, Inc.","ticker":"IRD"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"model_name":"glm-4.7","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-08-03T11:02:13.610Z","global_importance":30,"audience_relevance":20,"importance_components":{"tickerTier":"small-cap","eventGravity":"clinical-trial-milestone","sectorWeight":"biotech"}},"durationMs":76859,"modelName":"glm-4.7"}}