Press release
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Opus Genetics Announces Positive LowDose Cohort 1 Data from Phase 1/2 ClinicalTrial of OPGx-BEST1 and Successful FDAType C Meeting with Potential Phase 3Dosing in 2027 September 09, 2026 7:00am EDT OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no serious adverseevents or dose-limiting toxicities observed All five participants demonstrated clinically meaningful improvement in visual function, withstructural improvements observed in four participants FDA aligned on ≥3 decibels microperimetry improvement in conjunction with patient reportedoutcomes as a potential pivotal endpoint Clinically meaningful best-corrected visual acuity improvements in 3 of 5 participants and retinalsensitivity improvements on microperimetry observed in 3 of 4 evaluable participants Cohort 2 over-enrolled, with dosing expected to be completed in Q4 2026 and topline 3-month dataexpected in Q2 2027 Cash runway into 2029 expected to support multiple clinical inflection points and opportunities forpriority review vouchers Webcast and conference call today at 8:00 a.m. ET with management and Key Opinion Leader andretinal specialist, Mark Pennesi, M.D., PhD. RESEARCH TRIANGLE PARK, N.C., Sept. 09, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc.(Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical companydeveloping gene therapies to restore vision and prevent blindness in patients with inherited retinaldiseases (IRDs), today announced positive 3- and 6-month results from the low-dose Cohort 1 ofBIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1 in patients with BEST1-relatedretinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessivebestrophinopathy (ARB). Cohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three participants with BVMD whohave reached three months of follow-up and two with ARB who have reached six months of follow-up. All five participants demonstrated clinically meaningful improvement in visual function,measured as one or more of the following: best-corrected visual acuity (BCVA), low-luminancevisual acuity (LLVA), contrast sensitivity (CS) or microperimetry, an advanced eye test that mapshow well the central part of the retina sees light. Structural improvements were also observedacross four participants. The greatest functional gains were observed in participants with lessadvanced disease, supporting the potential benefit of treating patients while viable retinal tissueremains. The Company expects to announce 6-month data for the three participants with BVMD inQ2 2027. “These positive results provide important evidence of OPGx-BEST1’s potential to improve bothvisual function and retinal structure in patients with BEST1-related retinal disease, which we believehas a significantly larger underserved patient population than we previously thought,” said George
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Magrath, M.D., Chief Executive Officer of Opus Genetics. “The functional and structuralimprovements across Cohort 1, particularly the greater functional gains observed in patients withviable retinal tissue, reinforce our confidence in OPGx-BEST1’s potential to have a positive impacton the lives of patients with BEST disease. Together with our recent FDA interaction and rapidenrollment of Cohort 2, we believe these data provide a clear path toward pivotal development,which we plan to begin next year. We want to recognize the contributions of our investigators,clinical teams, and most importantly, the patients helping advance a potential treatment for thisblinding disease.” “These Cohort 1 data provide encouraging evidence that OPGx-BEST1 can be delivered safely andmay improve both retinal structure and visual function in patients with advanced BEST1-relatedretinal disease,” said Christine Nichols Kay, M.D., clinical trial investigator and Director of ClinicalResearch and Retinal Genetics at Vitreo Retinal Associates. “We are encouraged by the localization of functional gains to areas of viable, but compromisedretina and by the opportunity to apply these insights prospectively as OPGx-BEST1 is advancedinto Cohort 2 and potential pivotal development,” said Mark Pennesi, M.D., Ph.D., clinical trialinvestigator and Chief Medical Officer at the Retina Foundation and Adjunct Professor ofOphthalmology, Casey Eye Institute, Oregon Health & Science University. “From a regulatoryperspective, it is particularly exciting to align with the FDA on a >3 decibels change from baseline inmicroperimetry anchored to patient reported outcomes as a potential pivotal endpoint.” OPGx-BEST1 demonstrated a favorable safety and tolerability profile, with no serious adverseevents or dose-limiting toxicities, no intraocular inflammation and no vital-sign or safety-laboratoryfindings of note. All treatment-related adverse events were mild or moderate in severity. All five participants demonstrated clinically meaningful improvement in visual function followingtreatment with OPGx-BEST1. BCVA improved in 60% of participants (3/5), LLVA improved in 40% ofparticipants (2/5), and contrast sensitivity improved in 40% of participants (2/5). Among evaluableparticipants, 75% (3/4) demonstrated clinically meaningful improvement in retinal sensitivity bymicroperimetry. Importantly, these gains were concentrated in the treated retinal pigment epithelial(RPE) Transitional Zone, where viable photoreceptors remain, with the greatest functionalimprovements observed in participants with less advanced disease. Structural improvements were observed in four of five participants, with reductions in vitelliformmaterial, the hallmark of BVMD, in 67% of participants with BVMD (2/3) and reductions inintraretinal fluid in 100% of participants with ARB (2/2). The third BVMD participant had possible,but not definitive, reduction in vitelliform material. These findings provide proof-of-concept thatOPGx-BEST1 may improve visual function and retinal structure in areas where viable retinal tissueremains and are informing patient selection and future clinical development. In BVMD, vitelliform material accumulates early and is the defining structural feature of the disease,while subretinal fluid appears late and pools in areas of established atrophy. Reduction of vitelliformmaterial is therefore the more direct measure of restored RPE function in this population, and it isthe structural change that corresponded with functional improvement in those participants. In ARB,where intraretinal fluid is the dominant structural manifestation, fluid reduction was substantial andconsistent in both participants. In August 2026, the Company met with the U.S. Food and Drug Administration (FDA) to discussOPGx-BEST1 development and potential endpoints for a pivotal clinical trial. The Company alignedwith the FDA on a potential pivotal endpoint based on ≥3 dB microperimetry improvement in ≥5prespecified loci, in conjunction with a patient-reported outcome in a randomized, controlled trial.BCVA, LLVA, and CS may also be acceptable endpoints . The Company also aligned with the FDAon Phase 3 and commercial manufacturing requirements, which it expects to complete in early2027. The Company expects to begin planning for the Phase 3 trial immediately, with participantdosing expected to begin in 2027.
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Based on the safety profile and positive proof-of-concept findings from Cohort 1, the Company hasadvanced to the higher-dose Cohort 2, evaluating OPGx-BEST1 at 4.5 x 10⁹ vg/eye, with dosingexpected to be completed in Q4 2026 and topline three-month data expected in Q2 2027. Originallydesigned to enroll five participants, Cohort 2 has been over-enrolled with eight participants, most ofwhom have BVMD. Data from Cohort 2 are expected to further characterize the safety, functionaland structural responses to OPGx-BEST1 at the higher dose and inform the design of a potentialpivotal clinical trial. New epidemiology research conducted by Triangle Insights Group, based on a survey of more than150 eye care professionals, estimates approximately 23,600 symptomatic BEST1 patients in theU.S., including 13,000 diagnosed and 10,600 undiagnosed patients, and approximately 45,400symptomatic BEST1 patients globally. These findings suggest a substantially larger addressablepatient population and unmet need than previously estimated. Conference Call & Webcast Details Opus Genetics will host a webcast and conference call with accompanying slides today at 8:00 A.M.ET, including comments by management and key opinion leader, Mark Pennesi, M.D., PhD.,FARVO, a board-certified retinal surgeon at the Retina Foundation of the Southwest. The live andarchived webcast may be accessed on the Opus Genetics website under the Investorssection: Events. Opus Genetics suggests participants join 15 minutes in advance of the event. About BEST1 and OPGx-BEST1 BEST1-related inherited retinal diseases, or bestrophinopathies, are rare forms of inherited maculardegeneration caused by mutations in the BEST1 gene. These mutations disrupt the normal functionof RPE cells, leading to retinal lesions, progressive degeneration and vision loss. BEST1-relateddiseases include BVMD and ARB, and there are currently no approved therapies that address theunderlying genetic cause of these diseases. OPGx-BEST1 is an investigational gene therapy designed to address the underlying genetic causeof BEST1-related inherited retinal diseases, including BVMD and ARB. OPGx-BEST1 uses an AAVvector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The ongoingBIRD-1 clinical trial is an adaptive, open-label Phase 1/2 clinical trial evaluating the safety andefficacy of single-eye subretinal administration of OPGx-BEST1 in adults with BVMD or ARB. About Opus Genetics Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restorevision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company isdeveloping durable, one-time treatments designed to address the underlying genetic causes ofsevere retinal disorders. The Company’s pipeline includes seven AAV-based programs, led byOPGx-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 isbased in Research Triangle Park, NC. For more information, visit www.opusgtx.com. Forward-Looking Statements This press release contains certain statements that are not statements of historical fact and areforward-looking statements within the meaning of Section 27A of the Securities Act of 1933, asamended, Section 21E of the Securities Exchange Act of 1934, as amended, and the PrivateSecurities Litigation Reform Act of 1995. In some cases, you can identify forward-lookingstatements 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. Such statements include, but are not limited to, statementsrelated to the Company’s continued clinical development, clinical results, preclinical data, and futureplans for OPGx-BEST1, including the anticipated timing of dosing completion and topline data from
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Cohort 2 of the OPGx-BEST1 Phase 1/2 clinical trial; the Company’s patient-selection anddevelopment strategy for subsequent clinical trials of OPGx-BEST1; the outcome of the Company’songoing regulatory interactions with the FDA and its expectations regarding the design of, andpotential endpoints for, of any pivotal clinical trial of OPGx-BEST1; the Company’s expectationsregarding the clinical and therapeutic potential of OPGx-BEST1, including with respect to its abilityto improve visual function and retinal structure in patients with BEST1-related retinal disease; thepotential benefit of treating patients with viable retinal tissue; the Company’s estimates of theBEST1 symptomatic patient population in the U.S. and globally; and the Company’s expectationsregarding its business prospects and results of operations. The clinical trial referenced in this pressrelease is ongoing, and the data described are interim, subject to change, and based on dataavailable as of a specified date. As patient enrollment continues and additional follow-up data isobtained, the reported data and other clinical outcomes may change materially. There can be noassurance that the interim results will be predictive of final clinical trial results or that additional datawill confirm or support these observations. The forward-looking statements contained herein aresubject to certain risks and uncertainties posed by many factors and events that could cause theCompany’s actual business, prospects and results of operations to differ materially from thoseanticipated by such forward-looking statements. Factors that could cause or contribute to suchdifferences include, but are not limited to, those described under the heading “Risk Factors”included in the Company’s most recent Annual Report on Form 10-K for the fiscal year endedDecember 31, 2025, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, and inthe Company’s other filings with the U.S. Securities and Exchange Commission. Readers arecautioned not to place undue reliance on these forward-looking statements, which speak only as ofthe date of this press release. These forward-looking statements are based upon the Company’scurrent expectations and involve assumptions that may never materialize or may prove to beincorrect. Actual results and the timing of events could differ materially from those anticipated insuch forward-looking statements as a result of various risks and uncertainties. The Companyundertakes no obligation to revise any forward-looking statements in order to reflect events orcircumstances that might subsequently arise. Contacts: InvestorsJenny KobinRemy BernardaIR Advisory Solutionsir@opusgtx.com Media Kimberly HaKKH Advisors917-291-5744kimberly.ha@kkhadvisors.com Source: Opus Genetics, Inc.Released September 9, 2026