Opus Genetics Announces Successful FDA Meeting Supporting Advancement of OPGx-LCA5 Toward Pivotal Trial for LCA5-Related Inherited Retinal Disease
Opus Genetics (Nasdaq: IRD) reported a successful Type B RMAT meeting with the FDA supporting advancement of OPGx-LCA5 toward a pivotal adaptive Phase 3 trial for LCA5-related Leber congenital amaurosis.
The FDA provided constructive feedback on CMC and pivotal trial design, acknowledged unmet need, and affirmed regulatory flexibility; Opus plans to seek the FDA’s new RDEP review. Six late-stage participants in Phase 1/2 showed clinically meaningful vision improvements. The company enrolled the first participant in a planned run-in period, expects dosing after validated commercial CMC in H2 2026, and topline data ~one year later. Recent $23M financing funds operations into H2 2027.
Opus Genetics (Nasdaq: IRD) ha riportato un esito positivo di un incontro di Type B RMAT con la FDA che sostiene l'avanzamento di OPGx-LCA5 verso uno studio adattivo di fase 3 pivotale per la Leber congenita amaurosi correlata a LCA5.
La FDA ha fornito feedback costruttivi su CMC e design dello studio pivotale, ha riconosciuto l'insoddisfatta esigenza e ha confermato flessibilità regolatoria; Opus intende richiedere la nuova revisione RDEP della FDA. Sei partecipanti in fasi avanzate nello studio di fase 1/2 hanno mostrato miglioramenti clinicamente significativi della vista. L'azienda ha arruolato il primo partecipante in un periodo di run-in pianificato, prevede la somministrazione della dose dopo la convalida CMC commerciale in H2 2026, e i dati principali entro circa un anno dopo. Recenti finanziamenti da $23M finanziano le operazioni fino alla seconda metà del 2027.
Opus Genetics (Nasdaq: IRD) informó de una exitosa reunión RMAT de Tipo B con la FDA que respalda el avance de OPGx-LCA5 hacia un ensayo adaptativo pivotal de fase 3 para la amaurosis congénita de Leber relacionada con LCA5.
La FDA proporcionó comentarios constructivos sobre CMC y el diseño del ensayo pivotal, reconoció la necesidad no satisfecha y afirmó la flexibilidad regulatoria; Opus planea solicitar la nueva revisión RDEP de la FDA. Seis participantes en fases tardías de la fase 1/2 mostraron mejoras clínicamente significativas de la visión. La empresa inscribió al primer participante en un periodo de run-in planificado, espera la dosificación tras la validación de CMC comercial en H2 2026, y los datos principales ~ un año después. Financiación reciente de $23M financia las operaciones hasta la H2 de 2027.
Opus Genetics (나스닥: IRD)가 FDA와의 성공적인 타입 B RMAT 회의를 보고했고, LCA5 관련 레버 선천성 망막증에 대한 결정적 적응형 3상 시험으로의 OPGx-LCA5의 진행을 뒷받침했습니다.
FDA는 CMC 및 결정적 시험 설계에 대해 건설적인 피드백을 제공했고, 충족되지 않은 필요를 인정하며 규제 유연성을 확언했습니다; Opus는 FDA의 새로운 RDEP 심사를 신청할 계획입니다. 1/2상 단계의 후기 참가자 6명이 임상적으로 의미 있는 시력 개선을 보였습니다. 회사는 예비 러닝-인 기간에 첫 참가자를 등록했고, 상업 CMC의 검증 후 H2 2026에 투약을 기대하며, 핵심 데이터는 대략 1년 후에 발표될 예정입니다. 최근 $23M의 자금 조달로 2027년 하반기까지 운영 자금을 확보했습니다.
Opus Genetics (Nasdaq: IRD) a annoncé une réussite d'une réunion RMAT de type B avec la FDA soutenant l'avancement de OPGx-LCA5 vers un essai adaptatif pivot de phase 3 pour la rétinite congénitale Leber liée à LCA5.
La FDA a fourni des retours constructifs sur le CMC et le design de l'essai pivot, a reconnu le besoin non satisfait et a affirmé la flexibilité réglementaire; Opus prévoit de demander la nouvelle revue RDEP de la FDA. Six participants en phase tardive des phases 1/2 ont montré des améliorations visuelles cliniquement significatives. La société a recruté le premier participant pendant une période d'épreuve initiale planifiée, s'attend à la dose après la validation du CMC commercial dans H2 2026, et des données de référence environ un an après. Un financement récent de $23M finance les opérations jusqu'au deuxième semestre 2027.
Opus Genetics (Nasdaq: IRD) berichtete von einem erfolgreichen Type-B RMAT-Treffen mit der FDA, das den Fortschritt von OPGx-LCA5 in Richtung einer zentralen adaptiven Phase-3-Studie für LCA5-assoziierte Lebererblindung unterstützt.
Die FDA gab konstruktives Feedback zu CMC und dem Studiendesign, erkannte den ungedeckten Bedarf an und bestätigte regulatorische Flexibilität; Opus plant, die neue FDA-Überprüfung RDEP zu beantragen. Sechs späte Kandidaten in Phase 1/2 zeigten klinisch signifikante Sehverbesserungen. Das Unternehmen rekrutierte den ersten Teilnehmer in einer geplanten Run-In-Periode, erwartet die Verabreichung der Dosis nach validierter kommerzieller CMC in H2 2026 und die Topline-Daten ca. ein Jahr später. Kürzliche Finanzierungen von $23M finanzieren die Operationen bis zur zweiten Hälfte 2027.
Opus Genetics (ناسداك: IRD) أبلغت عن اجتماع RMAT من النوع B ناجح مع FDA يدعم تقدم OPGx-LCA5 نحو تجربة رائدة Adaptive من المرحلة الثالثة لمرض العمى الخلقي المرتبط بـ LCA5.
قدمت FDA ملاحظات بناءة حول CMC وتصميم التجربة الحاسمة، وأقرت بالحاجة غير الملباة وأكدت المرونة التنظيمية؛ تخطط Opus لطلب مراجعة FDA الجديدة RDEP. أظهر ستة مشاركين في المرحلة المتأخرة من المرحلة 1/2 تحسنات بصرية سريرية ذات دلالة. أدرجت الشركة أول مشارك في فترة تشغيل مخطط لها، وتتوقع إعطاء الجرعة بعد التحقق من CMC التجاري في H2 2026، وبيانات رئيسية تقريبا بعد سنة. التمويل الأخير بمقدار $23M يمول العمليات حتى النصف الثاني من 2027.
- FDA RMAT meeting provided CMC and pivotal-design feedback
- Six Phase 1/2 participants showed clinically meaningful vision improvements
- $23 million financing secured to fund operations into H2 2027
- First participant enrolled in run-in for planned adaptive Phase 3
- Company plans dosing after commercial-process CMC in H2 2026
- Phase 3 may enroll as few as 8 participants, limiting sample size
- Dosing requires validated commercial CMC before H2 2026 start
- Program targets patient population 1,000, constraining trial scale
Insights
FDA accepted RMAT feedback and an adaptive Phase 3 path for OPGx-LCA5; dosing planned after commercial-process supply.
Opus Genetics advances the program by converting Phase 1/2 signals and FDA RMAT guidance into a planned adaptive pivotal strategy that may avoid a separate registrational trial. The company reported six treated late-stage participants with clinically meaningful vision improvements and has enrolled the first participant into a run-in period to serve as each subject’s own control.
The near-term path depends on three concrete conditions: availability of validated clinical drug supply manufactured with intended commercial processes, successful completion of the run-in enrollment, and regulatory acceptance of the proposed adaptive design and RDEP eligibility. Each condition is explicit and material to whether dosing occurs as planned in
Watch items and timing: dosing anticipated in
- Outcome of Regenerative Medicine Advanced Therapy (RMAT) meeting provides the potential for an accelerated regulatory pathway to approval of OPGx-LCA5
- First participant enrolled in run-in period for planned adaptive Phase 3 trial
- Company intends to apply for the FDA’s new Rare Disease Evidence Principles (RDEP) review process
- OPGx-LCA5 has the potential to be the first gene therapy and one-time treatment for Leber congenital amaurosis (LCA) type 5
- Recent
$23 million financing led by Perceptive Advisors and Balyasny Asset Management to advance LCA5 and BEST1 programs and fund current operating plans into second half of 2027
RESEARCH TRIANGLE PARK, N.C., Nov. 06, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small-molecule therapies for other ophthalmic disorders, today announced the successful completion of a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene.
The meeting provided constructive feedback from the FDA on key elements of Opus’s registration strategy, including Chemistry, Manufacturing and Controls (CMC), and the pivotal trial design. The FDA acknowledged the significant unmet medical need for individuals with LCA5-related blindness and reaffirmed its commitment to regulatory flexibility for rare genetic diseases.
“The FDA’s guidance provides confidence in our path to approval for OPGx-LCA5,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “Importantly, we expect to be able to advance our ongoing trial using an adaptive design that includes a Phase 3 portion which will avoid the requirement for a separate registrational trial. Given the severe nature of the disease, we are actively identifying patients who may qualify for the Phase 3 and enrolling them into the planned run-in period to monitor their disease. This productive RMAT interaction represents an important milestone as we continue working closely with the FDA to bring sight-restoring gene therapies to patients who currently have no approved treatment options. We look forward to meeting with the FDA again in the coming months.”
To date in the Phase 1/2 portion of the trial, six late-stage participants have been treated with OPGx-LCA5, and all six have experienced clinically meaningful improvements in vision, providing evidence of biological activity with the potential for functional restoration of vision in individuals with advanced disease.
Opus will incorporate the FDA’s feedback into its updated clinical development and CMC plans for the Phase 3 portion of the study to include enrolling as few as 8 participants in a single arm, 12-month study utilizing an adaptive design, which provides flexibility on endpoints and number of participants, reflective of LCA5 as a rare condition with an urgent medical need.
The Company expects the Phase 3 portion of the trial will include a run-in period prior to dosing to evaluate the natural history of each participant to serve as their own control in the study. Opus is actively identifying patients for this segment and has enrolled the first participant. Efficacy and safety will be assessed using measures such as visual acuity, full-field stimulus testing, microperimetry, and Multi-Luminance Orientation and Mobility Test (MLoMT). Following availability of validated clinical drug supply manufactured with the intended commercial processes, dosing with OPGx-LCA5 is anticipated in the second half of 2026, with topline clinical data expected approximately one year later.
“With recent financing secured, we are strongly positioned to advance our LCA5 program with the rigor and urgency this patient community deserves. As we progress into our LCA5 pivotal trial and initiate clinical testing in our second gene therapy program for the treatment of BEST1-related disease, we are entering the next stage of growth as we build the world’s leading portfolio of gene therapies for inherited retinal diseases,” concluded Dr. Magrath.
In September 2025, the FDA introduced the Rare Disease Evidence Principles (RDEP) review process to facilitate the approval of drugs to treat rare diseases with very small patient populations with significant unmet medical need and with a known genetic defect that is the major driver of the pathophysiology. With a patient population of less than 1,000, Opus believes that its LCA5 program meets the eligibility criteria for the RDEP process and plans to submit an application.
About OPGx-LCA5
OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.
About Leber Congenital Amaurosis (LCA) and LCA5
Leber congenital amaurosis (LCA) is a group of inherited retinal diseases characterized by severely impaired vision or blindness at birth. Some retinal experts consider LCA to be a severe form of retinitis pigmentosa (RP). The condition is caused by degeneration and/or dysfunction of photoreceptors, the cells in the retina that make vision possible. Mutations in one of more than two dozen genes can cause LCA.
LCA5 is an ultra-rare disease caused by mutations in the LCA5 gene, which encodes lebercilin, a protein essential for photoreceptor structure and function. LCA5 accounts for roughly
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. The Company’s pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its lead gene therapy candidates are OPGx-LCA5, which is in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration. Opus Genetics is also advancing Phentolamine Ophthalmic Solution
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, the clinical development of, and clinical results and future plans for, OPGx-LCA5, potential meetings with the FDA regarding our OPGx-LCA5 program, 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, 2024 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
FAQ
What did Opus Genetics announce about OPGx-LCA5 on November 6, 2025?
When does Opus expect to dose participants in the OPGx-LCA5 Phase 3 portion (IRD)?
What clinical data supports moving OPGx-LCA5 toward a pivotal trial (IRD)?
How large will the adaptive Phase 3 for OPGx-LCA5 (IRD) be and what is the design?
Does Opus have funding to support OPGx-LCA5 development (IRD)?
Will Opus apply for the FDA Rare Disease Evidence Principles review for OPGx-LCA5 (IRD)?
