{"success":true,"data":{"pressRelease":{"id":"138846","rtpr_id":"nGNX2yPqxs-20260909","ticker":"IRD","exchange":"NASDAQ","all_tickers":["IRD"],"title":"Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027","author":"Globe Newswire","published_at":"2026-09-09T11:00:01.194Z","article_body":"OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no\nserious adverse events or dose-limiting toxicities observed \n\nAll five participants demonstrated clinically meaningful improvement in visual\nfunction, with structural improvements observed in four participants\n\nFDA aligned on ≥3 decibels microperimetry improvement in conjunction with\npatient reported outcomes as a potential pivotal endpoint \n\nClinically meaningful best-corrected visual acuity improvements in 3 of 5\nparticipants and retinal sensitivity improvements on microperimetry observed\nin 3 of 4 evaluable participants\n\nCohort 2 over-enrolled, with dosing expected to be completed in Q4 2026 and\ntopline 3-month data expected in Q2 2027\n\nCash runway into 2029 expected to support multiple clinical inflection\npoints and opportunities for priority review vouchers\n\nWebcast and conference call today at 8:00 a.m. ET with management and Key\nOpinion Leader and retinal specialist, Mark Pennesi, M.D., PhD.\n\nRESEARCH TRIANGLE PARK, N.C., Sept. 09, 2026 (GLOBE NEWSWIRE) -- Opus\nGenetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a\nclinical-stage biopharmaceutical company developing gene therapies to restore\nvision and prevent blindness in patients with inherited retinal diseases\n(IRDs), today announced positive 3- and 6-month results from the low-dose\nCohort 1 of BIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1\nin patients with BEST1-related retinal diseases, including Best vitelliform\nmacular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).\n\nCohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three\nparticipants with BVMD who have reached three months of follow-up and two with\nARB who have reached six months of follow-up. All five participants\ndemonstrated clinically meaningful improvement in visual function, measured as\none or more of the following: best-corrected visual acuity (BCVA),\nlow-luminance visual acuity (LLVA), contrast sensitivity (CS) or\nmicroperimetry, an advanced eye test that maps how well the central part of\nthe retina sees light. Structural improvements were also observed across four\nparticipants. The greatest functional gains were observed in participants with\nless advanced disease, supporting the potential benefit of treating patients\nwhile viable retinal tissue remains. The Company expects to announce 6-month\ndata for the three participants with BVMD in Q2 2027.\n\n“These positive results provide important evidence of OPGx-BEST1’s\npotential to improve both visual function and retinal structure in patients\nwith BEST1-related retinal disease, which we believe has a significantly\nlarger underserved patient population than we previously thought,” said\nGeorge Magrath, M.D., Chief Executive Officer of Opus Genetics. “The\nfunctional and structural improvements across Cohort 1, particularly the\ngreater functional gains observed in patients with viable retinal tissue,\nreinforce our confidence in OPGx-BEST1’s potential to have a positive impact\non the lives of patients with BEST disease. Together with our recent FDA\ninteraction and rapid enrollment of Cohort 2, we believe these data provide a\nclear path toward pivotal development, which we plan to begin next year. We\nwant to recognize the contributions of our investigators, clinical teams, and\nmost importantly, the patients helping advance a potential treatment for this\nblinding disease.”\n\n“These Cohort 1 data provide encouraging evidence that OPGx-BEST1 can be\ndelivered safely and may improve both retinal structure and visual function in\npatients with advanced BEST1-related retinal disease,” said Christine\nNichols Kay, M.D., clinical trial investigator and Director of Clinical\nResearch and Retinal Genetics at Vitreo Retinal Associates.\n\n“We are encouraged by the localization of functional gains to areas of\nviable, but compromised retina and by the opportunity to apply these insights\nprospectively as OPGx-BEST1 is advanced into Cohort 2 and potential pivotal\ndevelopment,” said Mark Pennesi, M.D., Ph.D., clinical trial investigator\nand Chief Medical Officer at the Retina Foundation and Adjunct Professor of\nOphthalmology, Casey Eye Institute, Oregon Health & Science\nUniversity. “From a regulatory perspective, it is particularly exciting to\nalign with the FDA on a >3 decibels change from baseline in\nmicroperimetry anchored to patient reported outcomes as a potential pivotal\nendpoint.”\n\nOPGx-BEST1 demonstrated a favorable safety and tolerability profile, with no\nserious adverse events or dose-limiting toxicities, no intraocular\ninflammation and no vital-sign or safety-laboratory findings of note. All\ntreatment-related adverse events were mild or moderate in severity.\n\nAll five participants demonstrated clinically meaningful improvement in visual\nfunction following treatment with OPGx-BEST1. BCVA improved in 60% of\nparticipants (3/5), LLVA improved in 40% of participants (2/5), and contrast\nsensitivity improved in 40% of participants (2/5). Among evaluable\nparticipants, 75% (3/4) demonstrated clinically meaningful improvement in\nretinal sensitivity by microperimetry. Importantly, these gains were\nconcentrated in the treated retinal pigment epithelial (RPE) Transitional\nZone, where viable photoreceptors remain, with the greatest functional\nimprovements observed in participants with less advanced disease.\n\nStructural improvements were observed in four of five participants, with\nreductions in vitelliform material, the hallmark of BVMD, in 67% of\nparticipants with BVMD (2/3) and reductions in intraretinal fluid in 100% of\nparticipants with ARB (2/2). The third BVMD participant had possible, but not\ndefinitive, reduction in vitelliform material. These findings provide\nproof-of-concept that OPGx-BEST1 may improve visual function and retinal\nstructure in areas where viable retinal tissue remains and are informing\npatient selection and future clinical development.\n\nIn BVMD, vitelliform material accumulates early and is the defining structural\nfeature of the disease, while subretinal fluid appears late and pools in areas\nof established atrophy. Reduction of vitelliform material is therefore the\nmore direct measure of restored RPE function in this population, and it is the\nstructural change that corresponded with functional improvement in those\nparticipants. In ARB, where intraretinal fluid is the dominant structural\nmanifestation, fluid reduction was substantial and consistent in both\nparticipants.\n\nIn August 2026, the Company met with the U.S. Food and Drug Administration\n(FDA) to discuss OPGx-BEST1 development and potential endpoints for a pivotal\nclinical trial. The Company aligned with the FDA on a potential pivotal\nendpoint based on ≥3 dB microperimetry improvement in ≥5 prespecified\nloci, in conjunction with a patient-reported outcome in a randomized,\ncontrolled trial. BCVA, LLVA, and CS may also be acceptable endpoints. The\nCompany also aligned with the FDA on Phase 3 and commercial manufacturing\nrequirements, which it expects to complete in early 2027. The Company expects\nto begin planning for the Phase 3 trial immediately, with participant dosing\nexpected to begin in 2027.\n\nBased on the safety profile and positive proof-of-concept findings from Cohort\n1, the Company has advanced to the higher-dose Cohort 2, evaluating OPGx-BEST1\nat 4.5 x 10⁹ vg/eye, with dosing expected to be completed in Q4 2026 and\ntopline three-month data expected in Q2 2027. Originally designed to enroll\nfive participants, Cohort 2 has been over-enrolled with eight participants,\nmost of whom have BVMD. Data from Cohort 2 are expected to further\ncharacterize the safety, functional and structural responses to OPGx-BEST1 at\nthe higher dose and inform the design of a potential pivotal clinical trial.\n\nNew epidemiology research conducted by Triangle Insights Group, based on a\nsurvey of more than 150 eye care professionals, estimates approximately 23,600\nsymptomatic BEST1 patients in the U.S., including 13,000 diagnosed and 10,600\nundiagnosed patients, and approximately 45,400 symptomatic BEST1 patients\nglobally. These findings suggest a substantially larger addressable patient\npopulation and unmet need than previously estimated.\n\nConference Call & Webcast Details\n\nOpus Genetics will host a webcast and conference call with accompanying slides\ntoday at 8:00 A.M. ET, including comments by management and key opinion\nleader, Mark Pennesi, M.D., PhD., FARVO, a board-certified retinal surgeon at\nthe Retina Foundation of the Southwest. The live and archived webcast may be\naccessed on the Opus Genetics website under the Investors section: Events\n(https://www.globenewswire.com/Tracker?data=vYQ3pfEeKYoAYywP6HOwg4Y3iCn7-mHpvue4KXAf-iv7_4Lf2zlOVPjf7vLbBC9C0TL2zg6S281VeYBWuix9NIPEh4lMvWbuftDrrn5EfMJ48uWhgtX89b4X-2_u_ktv5fsG758sSkX4jyyyY7diqSAJ-yY3pMJXuYckWAwqwear7l1oWm7ESZk0aOg5YVi85lBbN91z8TR1_Tyk8Qw58kwTXA_8YjFcULtDUBx6GYBgslKdo6w3SjNDJ-ZsAU6G_ss2GDo6SyY81RNsI3Ywc5VaN3ek8bvR5cx1SHTRMCCedfD9q9p9MRYWmhonTQnRkRHr88G7JqYQffPU1TXmXXmEZOxczXLO5ooc2MrMwTC25cZ99JvaXI5CqEdBacJgLUjYUKHu3xe7tWuqVrA8Zwphr4JfJvnvBun1wMYoLfXLMQyTkyGoz5s95RElWin1).\nOpus Genetics suggests participants join 15 minutes in advance of the event.\n\nAbout BEST1 and OPGx-BEST1\n\nBEST1-related inherited retinal diseases, or bestrophinopathies, are rare\nforms of inherited macular degeneration caused by mutations in the BEST1 gene.\nThese mutations disrupt the normal function of RPE cells, leading to retinal\nlesions, progressive degeneration and vision loss. BEST1-related diseases\ninclude BVMD and ARB, and there are currently no approved therapies that\naddress the underlying genetic cause of these diseases.\n\nOPGx-BEST1 is an investigational gene therapy designed to address the\nunderlying genetic cause of BEST1-related inherited retinal diseases,\nincluding BVMD and ARB. OPGx-BEST1 uses an AAV vector to deliver a functional\ncopy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1\nclinical trial is an adaptive, open-label Phase 1/2 clinical trial evaluating\nthe safety and efficacy of single-eye subretinal administration of OPGx-BEST1\nin adults with BVMD or ARB.\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. In some cases, you can identify forward-looking\nstatements by the following words: “anticipate,” “believe,”\n“continue,” “could,” “estimate,” “expect,” “intend,”\n“aim,” “may,” “ongoing,” “plan,” “potential,”\n“predict,” “project,” “should,” “strive,” “will,”\n“would” or the negative of these terms or other comparable terminology,\nalthough not all forward-looking statements contain these words. Such\nstatements include, but are not limited to, statements related to the\nCompany’s continued clinical development, clinical results, preclinical\ndata, and future plans for OPGx-BEST1, including the anticipated timing of\ndosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase 1/2\nclinical trial; the Company’s patient-selection and development strategy for\nsubsequent clinical trials of OPGx-BEST1; the outcome of the Company’s\nongoing regulatory interactions with the FDA and its expectations regarding\nthe design of, and potential endpoints for, of any pivotal clinical trial of\nOPGx-BEST1; the Company’s expectations regarding the clinical and\ntherapeutic potential of OPGx-BEST1, including with respect to its ability to\nimprove visual function and retinal structure in patients with BEST1-related\nretinal disease; the potential benefit of treating patients with viable\nretinal tissue; the Company’s estimates of the BEST1 symptomatic patient\npopulation in the U.S. and globally; and the Company’s expectations\nregarding its business prospects and results of operations. The clinical trial\nreferenced in this press release is ongoing, and the data described are\ninterim, subject to change, and based on data available as of a specified\ndate. As patient enrollment continues and additional follow-up data is\nobtained, the reported data and other clinical outcomes may change materially.\nThere can be no assurance that the interim results will be predictive of final\nclinical trial results or that additional data will confirm or support these\nobservations. The forward-looking statements contained herein are subject to\ncertain risks and uncertainties posed by many factors and events that could\ncause the Company’s actual business, prospects and results of operations to\ndiffer materially from those anticipated by such forward-looking statements.\nFactors that could cause or contribute to such differences include, but are\nnot limited to, those described under the heading “Risk Factors” included\nin the Company’s most recent Annual Report on Form 10-K for the fiscal year\nended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter\nended June 30, 2026, and in the Company’s other filings with the U.S.\nSecurities and Exchange Commission. Readers are cautioned not to place undue\nreliance on these forward-looking statements, which speak only as of the date\nof this press release. These forward-looking statements are based upon the\nCompany’s current expectations and involve assumptions that may never\nmaterialize or may prove to be incorrect. Actual results and the timing of\nevents could differ materially from those anticipated in such forward-looking\nstatements as a result of various risks and uncertainties. The Company\nundertakes no obligation to revise any forward-looking statements in order to\nreflect events or circumstances that might subsequently arise.\n\nContacts:\n\nInvestors\nJenny Kobin\nRemy Bernarda\nIR Advisory Solutions\nir@opusgtx.com\n\nMedia\n\nKimberly Ha\nKKH Advisors\n917-291-5744\nkimberly.ha@kkhadvisors.com\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":"nGNX2yPqxs-20260909","title":"Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027","author":"Globe Newswire","ticker":"IRD","created":"2026-09-09T11:00:01.194Z","tickers":["IRD"],"exchange":"NASDAQ","article_body":"OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no\nserious adverse events or dose-limiting toxicities observed \n\nAll five participants demonstrated clinically meaningful improvement in visual\nfunction, with structural improvements observed in four participants\n\nFDA aligned on ≥3 decibels microperimetry improvement in conjunction with\npatient reported outcomes as a potential pivotal endpoint \n\nClinically meaningful best-corrected visual acuity improvements in 3 of 5\nparticipants and retinal sensitivity improvements on microperimetry observed\nin 3 of 4 evaluable participants\n\nCohort 2 over-enrolled, with dosing expected to be completed in Q4 2026 and\ntopline 3-month data expected in Q2 2027\n\nCash runway into 2029 expected to support multiple clinical inflection\npoints and opportunities for priority review vouchers\n\nWebcast and conference call today at 8:00 a.m. ET with management and Key\nOpinion Leader and retinal specialist, Mark Pennesi, M.D., PhD.\n\nRESEARCH TRIANGLE PARK, N.C., Sept. 09, 2026 (GLOBE NEWSWIRE) -- Opus\nGenetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a\nclinical-stage biopharmaceutical company developing gene therapies to restore\nvision and prevent blindness in patients with inherited retinal diseases\n(IRDs), today announced positive 3- and 6-month results from the low-dose\nCohort 1 of BIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1\nin patients with BEST1-related retinal diseases, including Best vitelliform\nmacular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).\n\nCohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three\nparticipants with BVMD who have reached three months of follow-up and two with\nARB who have reached six months of follow-up. All five participants\ndemonstrated clinically meaningful improvement in visual function, measured as\none or more of the following: best-corrected visual acuity (BCVA),\nlow-luminance visual acuity (LLVA), contrast sensitivity (CS) or\nmicroperimetry, an advanced eye test that maps how well the central part of\nthe retina sees light. Structural improvements were also observed across four\nparticipants. The greatest functional gains were observed in participants with\nless advanced disease, supporting the potential benefit of treating patients\nwhile viable retinal tissue remains. The Company expects to announce 6-month\ndata for the three participants with BVMD in Q2 2027.\n\n“These positive results provide important evidence of OPGx-BEST1’s\npotential to improve both visual function and retinal structure in patients\nwith BEST1-related retinal disease, which we believe has a significantly\nlarger underserved patient population than we previously thought,” said\nGeorge Magrath, M.D., Chief Executive Officer of Opus Genetics. “The\nfunctional and structural improvements across Cohort 1, particularly the\ngreater functional gains observed in patients with viable retinal tissue,\nreinforce our confidence in OPGx-BEST1’s potential to have a positive impact\non the lives of patients with BEST disease. Together with our recent FDA\ninteraction and rapid enrollment of Cohort 2, we believe these data provide a\nclear path toward pivotal development, which we plan to begin next year. We\nwant to recognize the contributions of our investigators, clinical teams, and\nmost importantly, the patients helping advance a potential treatment for this\nblinding disease.”\n\n“These Cohort 1 data provide encouraging evidence that OPGx-BEST1 can be\ndelivered safely and may improve both retinal structure and visual function in\npatients with advanced BEST1-related retinal disease,” said Christine\nNichols Kay, M.D., clinical trial investigator and Director of Clinical\nResearch and Retinal Genetics at Vitreo Retinal Associates.\n\n“We are encouraged by the localization of functional gains to areas of\nviable, but compromised retina and by the opportunity to apply these insights\nprospectively as OPGx-BEST1 is advanced into Cohort 2 and potential pivotal\ndevelopment,” said Mark Pennesi, M.D., Ph.D., clinical trial investigator\nand Chief Medical Officer at the Retina Foundation and Adjunct Professor of\nOphthalmology, Casey Eye Institute, Oregon Health & Science\nUniversity. “From a regulatory perspective, it is particularly exciting to\nalign with the FDA on a >3 decibels change from baseline in\nmicroperimetry anchored to patient reported outcomes as a potential pivotal\nendpoint.”\n\nOPGx-BEST1 demonstrated a favorable safety and tolerability profile, with no\nserious adverse events or dose-limiting toxicities, no intraocular\ninflammation and no vital-sign or safety-laboratory findings of note. All\ntreatment-related adverse events were mild or moderate in severity.\n\nAll five participants demonstrated clinically meaningful improvement in visual\nfunction following treatment with OPGx-BEST1. BCVA improved in 60% of\nparticipants (3/5), LLVA improved in 40% of participants (2/5), and contrast\nsensitivity improved in 40% of participants (2/5). Among evaluable\nparticipants, 75% (3/4) demonstrated clinically meaningful improvement in\nretinal sensitivity by microperimetry. Importantly, these gains were\nconcentrated in the treated retinal pigment epithelial (RPE) Transitional\nZone, where viable photoreceptors remain, with the greatest functional\nimprovements observed in participants with less advanced disease.\n\nStructural improvements were observed in four of five participants, with\nreductions in vitelliform material, the hallmark of BVMD, in 67% of\nparticipants with BVMD (2/3) and reductions in intraretinal fluid in 100% of\nparticipants with ARB (2/2). The third BVMD participant had possible, but not\ndefinitive, reduction in vitelliform material. These findings provide\nproof-of-concept that OPGx-BEST1 may improve visual function and retinal\nstructure in areas where viable retinal tissue remains and are informing\npatient selection and future clinical development.\n\nIn BVMD, vitelliform material accumulates early and is the defining structural\nfeature of the disease, while subretinal fluid appears late and pools in areas\nof established atrophy. Reduction of vitelliform material is therefore the\nmore direct measure of restored RPE function in this population, and it is the\nstructural change that corresponded with functional improvement in those\nparticipants. In ARB, where intraretinal fluid is the dominant structural\nmanifestation, fluid reduction was substantial and consistent in both\nparticipants.\n\nIn August 2026, the Company met with the U.S. Food and Drug Administration\n(FDA) to discuss OPGx-BEST1 development and potential endpoints for a pivotal\nclinical trial. The Company aligned with the FDA on a potential pivotal\nendpoint based on ≥3 dB microperimetry improvement in ≥5 prespecified\nloci, in conjunction with a patient-reported outcome in a randomized,\ncontrolled trial. BCVA, LLVA, and CS may also be acceptable endpoints. The\nCompany also aligned with the FDA on Phase 3 and commercial manufacturing\nrequirements, which it expects to complete in early 2027. The Company expects\nto begin planning for the Phase 3 trial immediately, with participant dosing\nexpected to begin in 2027.\n\nBased on the safety profile and positive proof-of-concept findings from Cohort\n1, the Company has advanced to the higher-dose Cohort 2, evaluating OPGx-BEST1\nat 4.5 x 10⁹ vg/eye, with dosing expected to be completed in Q4 2026 and\ntopline three-month data expected in Q2 2027. Originally designed to enroll\nfive participants, Cohort 2 has been over-enrolled with eight participants,\nmost of whom have BVMD. Data from Cohort 2 are expected to further\ncharacterize the safety, functional and structural responses to OPGx-BEST1 at\nthe higher dose and inform the design of a potential pivotal clinical trial.\n\nNew epidemiology research conducted by Triangle Insights Group, based on a\nsurvey of more than 150 eye care professionals, estimates approximately 23,600\nsymptomatic BEST1 patients in the U.S., including 13,000 diagnosed and 10,600\nundiagnosed patients, and approximately 45,400 symptomatic BEST1 patients\nglobally. These findings suggest a substantially larger addressable patient\npopulation and unmet need than previously estimated.\n\nConference Call & Webcast Details\n\nOpus Genetics will host a webcast and conference call with accompanying slides\ntoday at 8:00 A.M. ET, including comments by management and key opinion\nleader, Mark Pennesi, M.D., PhD., FARVO, a board-certified retinal surgeon at\nthe Retina Foundation of the Southwest. The live and archived webcast may be\naccessed on the Opus Genetics website under the Investors section: Events\n(https://www.globenewswire.com/Tracker?data=vYQ3pfEeKYoAYywP6HOwg4Y3iCn7-mHpvue4KXAf-iv7_4Lf2zlOVPjf7vLbBC9C0TL2zg6S281VeYBWuix9NIPEh4lMvWbuftDrrn5EfMJ48uWhgtX89b4X-2_u_ktv5fsG758sSkX4jyyyY7diqSAJ-yY3pMJXuYckWAwqwear7l1oWm7ESZk0aOg5YVi85lBbN91z8TR1_Tyk8Qw58kwTXA_8YjFcULtDUBx6GYBgslKdo6w3SjNDJ-ZsAU6G_ss2GDo6SyY81RNsI3Ywc5VaN3ek8bvR5cx1SHTRMCCedfD9q9p9MRYWmhonTQnRkRHr88G7JqYQffPU1TXmXXmEZOxczXLO5ooc2MrMwTC25cZ99JvaXI5CqEdBacJgLUjYUKHu3xe7tWuqVrA8Zwphr4JfJvnvBun1wMYoLfXLMQyTkyGoz5s95RElWin1).\nOpus Genetics suggests participants join 15 minutes in advance of the event.\n\nAbout BEST1 and OPGx-BEST1\n\nBEST1-related inherited retinal diseases, or bestrophinopathies, are rare\nforms of inherited macular degeneration caused by mutations in the BEST1 gene.\nThese mutations disrupt the normal function of RPE cells, leading to retinal\nlesions, progressive degeneration and vision loss. BEST1-related diseases\ninclude BVMD and ARB, and there are currently no approved therapies that\naddress the underlying genetic cause of these diseases.\n\nOPGx-BEST1 is an investigational gene therapy designed to address the\nunderlying genetic cause of BEST1-related inherited retinal diseases,\nincluding BVMD and ARB. OPGx-BEST1 uses an AAV vector to deliver a functional\ncopy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1\nclinical trial is an adaptive, open-label Phase 1/2 clinical trial evaluating\nthe safety and efficacy of single-eye subretinal administration of OPGx-BEST1\nin adults with BVMD or ARB.\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. In some cases, you can identify forward-looking\nstatements by the following words: “anticipate,” “believe,”\n“continue,” “could,” “estimate,” “expect,” “intend,”\n“aim,” “may,” “ongoing,” “plan,” “potential,”\n“predict,” “project,” “should,” “strive,” “will,”\n“would” or the negative of these terms or other comparable terminology,\nalthough not all forward-looking statements contain these words. Such\nstatements include, but are not limited to, statements related to the\nCompany’s continued clinical development, clinical results, preclinical\ndata, and future plans for OPGx-BEST1, including the anticipated timing of\ndosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase 1/2\nclinical trial; the Company’s patient-selection and development strategy for\nsubsequent clinical trials of OPGx-BEST1; the outcome of the Company’s\nongoing regulatory interactions with the FDA and its expectations regarding\nthe design of, and potential endpoints for, of any pivotal clinical trial of\nOPGx-BEST1; the Company’s expectations regarding the clinical and\ntherapeutic potential of OPGx-BEST1, including with respect to its ability to\nimprove visual function and retinal structure in patients with BEST1-related\nretinal disease; the potential benefit of treating patients with viable\nretinal tissue; the Company’s estimates of the BEST1 symptomatic patient\npopulation in the U.S. and globally; and the Company’s expectations\nregarding its business prospects and results of operations. The clinical trial\nreferenced in this press release is ongoing, and the data described are\ninterim, subject to change, and based on data available as of a specified\ndate. As patient enrollment continues and additional follow-up data is\nobtained, the reported data and other clinical outcomes may change materially.\nThere can be no assurance that the interim results will be predictive of final\nclinical trial results or that additional data will confirm or support these\nobservations. The forward-looking statements contained herein are subject to\ncertain risks and uncertainties posed by many factors and events that could\ncause the Company’s actual business, prospects and results of operations to\ndiffer materially from those anticipated by such forward-looking statements.\nFactors that could cause or contribute to such differences include, but are\nnot limited to, those described under the heading “Risk Factors” included\nin the Company’s most recent Annual Report on Form 10-K for the fiscal year\nended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter\nended June 30, 2026, and in the Company’s other filings with the U.S.\nSecurities and Exchange Commission. Readers are cautioned not to place undue\nreliance on these forward-looking statements, which speak only as of the date\nof this press release. These forward-looking statements are based upon the\nCompany’s current expectations and involve assumptions that may never\nmaterialize or may prove to be incorrect. Actual results and the timing of\nevents could differ materially from those anticipated in such forward-looking\nstatements as a result of various risks and uncertainties. The Company\nundertakes no obligation to revise any forward-looking statements in order to\nreflect events or circumstances that might subsequently arise.\n\nContacts:\n\nInvestors\nJenny Kobin\nRemy Bernarda\nIR Advisory Solutions\nir@opusgtx.com\n\nMedia\n\nKimberly Ha\nKKH Advisors\n917-291-5744\nkimberly.ha@kkhadvisors.com\n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/468baef1-3b2d-4353-a17e-cb41b45ee6ef)\n\n\n\nGlobeNewswire, Inc. 2026"},"type":"article","timestamp":"2026-09-09T11:00:01.236695017Z","server_sent_at_ms":1788951601236},"received_at":"2026-09-09T11:00:01.288Z","source_url":"https://www.globenewswire.com/news-release/2026/09/09/3358458/0/en/opus-genetics-announces-positive-low-dose-cohort-1-data-from-phase-1-2-clinical-trial-of-opgx-best1-and-successful-fda-type-c-meeting-with-potential-phase-3-dosing-in-2027.html"},"analysis":{"id":"127695","press_release_id":"138846","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":["Interim, open-label data from only five participants with no control arm -- natural disease fluctuation or investigator bias cannot be excluded","Company's own forward-looking caveats note interim results are subject to change materially as follow-up matures","Clinical-stage small-cap with no approved products; the Phase 3 path still depends on higher-dose Cohort 2 safety and successful pivotal execution"],"eventType":"clinical_trial","narrative":"Opus Genetics reported positive 3- and 6-month Cohort 1 data from its Phase 1/2 BIRD-1 trial of OPGx-BEST1 in BEST1-related retinal diseases, with all five participants showing clinically meaningful visual function improvement and no serious adverse events or dose-limiting toxicities.\n\nA successful August 2026 FDA Type C meeting produced alignment on a potential pivotal endpoint of at least 3 decibels of microperimetry improvement in five or more prespecified loci, paired with a patient-reported outcome, with Phase 3 participant dosing expected to begin in 2027.\n\nThe higher-dose Cohort 2 has been over-enrolled to eight participants, with dosing expected to complete in Q4 2026 and topline three-month data expected in Q2 2027; 6-month data for the three BVMD participants is also expected in Q2 2027.\n\nNew epidemiology from Triangle Insights Group estimates roughly 23,600 symptomatic U.S. BEST1 patients and about 45,400 globally -- a larger addressable population than previously estimated -- and management says cash runway extends into 2029, funding multiple clinical inflection points.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"Clean Phase 1/2 safety plus FDA endpoint alignment hands OPGx-BEST1 a defined Phase 3 path with dosing in 2027 -- the key gene-therapy catalyst timeline for this small-cap."},"keyFigures":{"drugName":"OPGx-BEST1","phaseOfTrial":"Phase 1/2","customDimensions":{"trial_name":"BIRD-1","cash_runway":"into 2029","cohort1_dose":"1.5 x 10⁹ vg/eye","cohort2_dose":"4.5 x 10⁹ vg/eye","bcva_improvement":"60% (3/5)","bvmd_6month_data":"Q2 2027","llva_improvement":"40% (2/5)","phase3_dosing_start":"2027","cohort1_participants":5,"cohort2_participants":8,"cohort2_topline_data":"Q2 2027","fda_pivotal_endpoint":"≥3 dB microperimetry improvement in ≥5 prespecified loci plus patient-reported outcome","us_diagnosed_patients":13000,"serious_adverse_events":0,"structural_improvement":"4/5 participants","us_symptomatic_patients":23600,"us_undiagnosed_patients":10600,"dose_limiting_toxicities":0,"cohort2_dosing_completion":"Q4 2026","microperimetry_improvement":"75% (3/4 evaluable)","global_symptomatic_patients":45400,"contrast_sensitivity_improvement":"40% (2/5)"}},"quotedText":"These positive results provide important evidence of OPGx-BEST1's\npotential to improve both visual function and retinal structure in patients\nwith BEST1-related retinal disease","namedEntities":{"people":[{"name":"George Magrath, M.D.","role":"CEO of Opus Genetics"},{"name":"Christine Nichols Kay, M.D.","role":"clinical trial investigator; Director of Clinical Research and Retinal Genetics at Vitreo Retinal Associates"},{"name":"Mark Pennesi, M.D., Ph.D.","role":"clinical trial investigator; Chief Medical Officer at the Retina Foundation; Adjunct Professor of Ophthalmology, Casey Eye Institute, OHSU"}],"products":["OPGx-BEST1","BIRD-1","OPGx-LCA5"],"companies":[{"name":"Opus Genetics, Inc.","ticker":"IRD","relationship":"filer"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"},{"name":"Triangle Insights Group","relationship":"third-party epidemiology researcher"},{"name":"Vitreo Retinal Associates","relationship":"clinical trial site"},{"name":"Retina Foundation of the Southwest","relationship":"investigator affiliation"},{"name":"Oregon Health & Science University","relationship":"investigator affiliation"}],"dollarAmounts":[]},"materialImpact":{"score":4,"reasoning":"Positive proof-of-concept from Cohort 1 (all 5 participants improved visual function, no serious adverse events or DLTs) combined with FDA alignment on a potential pivotal endpoint and planned Phase 3 dosing in 2027 defines a clear regulatory path -- a major catalyst for a clinical-stage small-cap, though data are early, open-label, and interim."},"tickerRelevance":{"others":[],"primary":"IRD"},"globalImportance":35,"audienceRelevance":28,"eventTypeSecondary":["regulatory"],"importanceComponents":{"tickerTier":"small-cap","eventGravity":"positive-interim-clinical-data-plus-fda-endpoint-alignment","sectorWeight":"gene-therapy-biotech","dataLimitation":"n=5, open-label, interim","deRiskingFactor":"regulatory path defined, Phase 3 dosing 2027"}},"event_type":"clinical_trial","event_type_secondary":["regulatory"],"sentiment":"bullish","material_impact_score":4,"narrative":"Opus Genetics reported positive 3- and 6-month Cohort 1 data from its Phase 1/2 BIRD-1 trial of OPGx-BEST1 in BEST1-related retinal diseases, with all five participants showing clinically meaningful visual function improvement and no serious adverse events or dose-limiting toxicities.\n\nA successful August 2026 FDA Type C meeting produced alignment on a potential pivotal endpoint of at least 3 decibels of microperimetry improvement in five or more prespecified loci, paired with a patient-reported outcome, with Phase 3 participant dosing expected to begin in 2027.\n\nThe higher-dose Cohort 2 has been over-enrolled to eight participants, with dosing expected to complete in Q4 2026 and topline three-month data expected in Q2 2027; 6-month data for the three BVMD participants is also expected in Q2 2027.\n\nNew epidemiology from Triangle Insights Group estimates roughly 23,600 symptomatic U.S. BEST1 patients and about 45,400 globally -- a larger addressable population than previously estimated -- and management says cash runway extends into 2029, funding multiple clinical inflection points.","key_figures":{"drugName":"OPGx-BEST1","phaseOfTrial":"Phase 1/2","customDimensions":{"trial_name":"BIRD-1","cash_runway":"into 2029","cohort1_dose":"1.5 x 10⁹ vg/eye","cohort2_dose":"4.5 x 10⁹ vg/eye","bcva_improvement":"60% (3/5)","bvmd_6month_data":"Q2 2027","llva_improvement":"40% (2/5)","phase3_dosing_start":"2027","cohort1_participants":5,"cohort2_participants":8,"cohort2_topline_data":"Q2 2027","fda_pivotal_endpoint":"≥3 dB microperimetry improvement in ≥5 prespecified loci plus patient-reported outcome","us_diagnosed_patients":13000,"serious_adverse_events":0,"structural_improvement":"4/5 participants","us_symptomatic_patients":23600,"us_undiagnosed_patients":10600,"dose_limiting_toxicities":0,"cohort2_dosing_completion":"Q4 2026","microperimetry_improvement":"75% (3/4 evaluable)","global_symptomatic_patients":45400,"contrast_sensitivity_improvement":"40% (2/5)"}},"named_entities":{"people":[{"name":"George Magrath, M.D.","role":"CEO of Opus Genetics"},{"name":"Christine Nichols Kay, M.D.","role":"clinical trial investigator; Director of Clinical Research and Retinal Genetics at Vitreo Retinal Associates"},{"name":"Mark Pennesi, M.D., Ph.D.","role":"clinical trial investigator; Chief Medical Officer at the Retina Foundation; Adjunct Professor of Ophthalmology, Casey Eye Institute, OHSU"}],"products":["OPGx-BEST1","BIRD-1","OPGx-LCA5"],"companies":[{"name":"Opus Genetics, Inc.","ticker":"IRD","relationship":"filer"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"},{"name":"Triangle Insights Group","relationship":"third-party epidemiology researcher"},{"name":"Vitreo Retinal Associates","relationship":"clinical trial site"},{"name":"Retina Foundation of the Southwest","relationship":"investigator affiliation"},{"name":"Oregon Health & Science University","relationship":"investigator affiliation"}],"dollarAmounts":[]},"model_name":"glm-5.3-flash","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-09T11:06:25.353Z","global_importance":35,"audience_relevance":28,"importance_components":{"tickerTier":"small-cap","eventGravity":"positive-interim-clinical-data-plus-fda-endpoint-alignment","sectorWeight":"gene-therapy-biotech","dataLimitation":"n=5, open-label, interim","deRiskingFactor":"regulatory path defined, Phase 3 dosing 2027"}},"durationMs":null,"modelName":"glm-5.3-flash"}}