Opus Genetics Announces Presentations at Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting
Opus Genetics (Nasdaq: IRD) announced three abstracts acceptance for presentation at the ARVO 2025 Meeting in Salt Lake City. The presentations include 12-month data from their Phase 1/2 trial of OPGx-LCA5, showing persistent efficacy signs in the first three adult patients treated for Leber congenital amaurosis.
The company will also present pre-clinical results for two other gene therapy candidates: OPGx-MERTK for MERTK-related retinitis pigmentosa, tested in a rat model, and OPGx-RDH12 for Leber congenital amaurosis 13, evaluated for tolerability in primates.
Additionally, a subset analysis from the LYNX-1 Phase 3 trial of Phentolamine Ophthalmic Solution 0.75% will be presented, focusing on 25 post-LASIK subjects with dim light disturbances.
Opus Genetics (Nasdaq: IRD) ha annunciato l'accettazione di tre abstract per la presentazione al Meeting ARVO 2025 a Salt Lake City. Le presentazioni includono dati a 12 mesi dal loro studio di Fase 1/2 su OPGx-LCA5, che mostrano segni di efficacia persistente nei primi tre pazienti adulti trattati per l'amaurosi congenita di Leber.
La società presenterà anche risultati preclinici per altri due candidati alla terapia genica: OPGx-MERTK per la retinite pigmentosa correlata a MERTK, testato in un modello di ratto, e OPGx-RDH12 per l'amaurosi congenita di Leber 13, valutato per la tollerabilità nei primati.
Inoltre, sarà presentata un'analisi dei sottogruppi dello studio di Fase 3 LYNX-1 sulla Soluzione Oftalmica di Fentolamina 0,75%, con un focus su 25 soggetti post-LASIK con disturbi in condizioni di scarsa illuminazione.
Opus Genetics (Nasdaq: IRD) anunció la aceptación de tres resúmenes para su presentación en el Meeting ARVO 2025 en Salt Lake City. Las presentaciones incluyen datos a 12 meses de su ensayo de Fase 1/2 de OPGx-LCA5, que muestran signos de eficacia persistente en los primeros tres pacientes adultos tratados por amaurosis congénita de Leber.
La compañía también presentará resultados preclínicos para otros dos candidatos a terapia génica: OPGx-MERTK para la retinitis pigmentosa relacionada con MERTK, probado en un modelo de rata, y OPGx-RDH12 para amaurosis congénita de Leber 13, evaluado por su tolerabilidad en primates.
Además, se presentará un análisis de subgrupo del ensayo de Fase 3 LYNX-1 de la Solución Oftálmica de Fentolamina al 0,75%, centrándose en 25 sujetos post-LASIK con alteraciones en condiciones de poca luz.
Opus Genetics (Nasdaq: IRD)는 솔트레이크시티에서 열리는 ARVO 2025 회의에서 발표할 세 개의 초록이 수락되었다고 발표했습니다. 발표 내용에는 OPGx-LCA5의 12개월 데이터가 포함되어 있으며, 이는 레버 선천성 암맹으로 치료받은 첫 세 명의 성인 환자에서 지속적인 효능의 징후를 보여줍니다.
회사는 또한 MERTK 관련 망막색소변성증에 대한 OPGx-MERTK와 레버 선천성 암맹 13에 대한 OPGx-RDH12의 두 가지 유전자 치료 후보에 대한 전임상 결과를 발표할 예정입니다. 후자는 영장류에서의 내약성을 평가했습니다.
추가로, LASIK 수술 후 25명의 환자를 대상으로 한 조명 저하 장애를 중심으로 한 Phentolamine 안과 용액 0.75%의 LYNX-1 3상 시험의 하위 분석이 발표될 예정입니다.
Opus Genetics (Nasdaq: IRD) a annoncé l'acceptation de trois résumés pour présentation lors de la Réunion ARVO 2025 à Salt Lake City. Les présentations incluent des données à 12 mois de leur essai de Phase 1/2 sur OPGx-LCA5, montrant des signes d'efficacité persistante chez les trois premiers patients adultes traités pour l'amaurose congénitale de Leber.
La société présentera également des résultats précliniques pour deux autres candidats à la thérapie génique : OPGx-MERTK pour la rétinite pigmentaire liée à MERTK, testée dans un modèle de rat, et OPGx-RDH12 pour l'amaurose congénitale de Leber 13, évaluée pour sa tolérabilité chez les primates.
De plus, une analyse de sous-groupe de l'essai de Phase 3 LYNX-1 de la solution ophtalmique de Phentolamine à 0,75 % sera présentée, se concentrant sur 25 sujets post-LASIK présentant des troubles en faible lumière.
Opus Genetics (Nasdaq: IRD) gab die Annahme von drei Abstracts zur Präsentation auf dem ARVO 2025 Meeting in Salt Lake City bekannt. Die Präsentationen umfassen 12-Monats-Daten aus ihrer Phase 1/2-Studie zu OPGx-LCA5, die anhaltende Wirksamkeitszeichen bei den ersten drei erwachsenen Patienten zeigen, die wegen der Leber kongenitalen Amaurose behandelt wurden.
Das Unternehmen wird auch präklinische Ergebnisse für zwei weitere Gentherapiekandidaten präsentieren: OPGx-MERTK für MERTK-assoziierte Retinitis pigmentosa, getestet in einem Rattenmodell, und OPGx-RDH12 für die Leber kongenitale Amaurose 13, die auf Verträglichkeit bei Primaten bewertet wurde.
Darüber hinaus wird eine Subgruppenanalyse aus der LYNX-1 Phase 3-Studie zur Phentolamin-Augentropfenlösung 0,75 % präsentiert, die sich auf 25 Post-LASIK-Patienten mit Dunkelheitsstörungen konzentriert.
- 12-month efficacy persistence in Phase 1/2 trial of OPGx-LCA5
- All three adult patients showed visual improvement
- OPGx-LCA5 demonstrated good tolerability
- Advancing to Phase 3 trial pending continued positive results
- Small patient sample size (only 3 patients) in Phase 1/2 trial
Insights
Opus Genetics has reported encouraging durability data for its lead gene therapy candidate OPGx-LCA5, with efficacy persisting for one full year in the first three adult patients from its Phase 1/2 trial. This durability milestone is particularly significant for gene therapies, where long-term effectiveness is a critical consideration for both clinical and commercial success.
The company has signaled confidence in these results by announcing plans to advance OPGx-LCA5 into a pivotal Phase 3 trial, representing a material progression in their clinical development timeline. For a rare disease like Leber congenital amaurosis (LCA), this advancement represents a substantial derisking event in the pathway to potential regulatory approval.
Beyond their lead program, Opus will be presenting pre-clinical data on two additional candidates – OPGx-MERTK for retinitis pigmentosa and OPGx-RDH12 for LCA13 – demonstrating pipeline depth with multiple assets targeting different genetic retinal disorders. For a clinical-stage biotech with a
The persistent efficacy in this small patient cohort potentially establishes proof-of-concept for their AAV-based gene therapy approach in treating inherited retinal diseases, which could have broader implications across their development pipeline.
The one-year durability data for OPGx-LCA5 represents a clinically meaningful development in the treatment landscape for Leber congenital amaurosis. LCA is among the most severe inherited retinal disorders, typically causing significant visual impairment from early childhood, with LCA5 specifically associated with mutations in the lebercillin gene that affect photoreceptor function.
The persistence of both subjective and objective efficacy signals at the 12-month mark is particularly encouraging from a mechanistic perspective. Gene therapies for inherited retinal diseases must demonstrate not just initial transgene expression but sustained functional improvement, as the goal is to provide long-term correction of the underlying genetic defect in retinal cells.
While the current data comes from only three adult patients, these early signals in a progressive disorder like LCA5 suggest the potential for disease modification rather than just symptomatic improvement. The planned progression to Phase 3 indicates the company believes the risk-benefit profile supports further development, which is significant given the invasive nature of subretinal delivery required for these therapies.
The company's broader pipeline targeting MERTK-related retinitis pigmentosa and RDH12-associated LCA13 demonstrates a comprehensive approach to addressing different genetic mutations causing inherited retinal degeneration. These genetically defined populations represent the frontier of precision medicine in ophthalmology, with potential for transformative outcomes in previously untreatable conditions.
New Data from First Three Adult Patients in Phase 1/2 Trial with OPGx-LCA5 Showed that Subjective and Objective Signs of Efficacy Persisted for One Year
Durham, NC., March 05, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmologic disorders, today announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting, to take place May 4-8, 2025 in Salt Lake City, UT. The abstracts feature 12-month data from the first three adult patients in our ongoing Phase 1/2 trial of OPGx-LCA5, as well as pre-clinical results on OPGx-MERTK and OPGx-RDH12.
An abstract summarizing a subset analysis from the previously completed LYNX-1 Phase 3 trial of Phentolamine Ophthalmic Solution
“We are pleased to have the opportunity to share data on our gene therapy candidates and to engage with the global ophthalmology community at ARVO 2025,” said George Magrath, M.D., Chief Executive Officer at Opus Genetics. “We look forward to presenting the 12-month data on adult patients being treated in the ongoing Phase 1/2 trial of our most advanced gene therapy candidate OPGx-LCA5. Assuming continued safety and efficacy in the current study, we plan to advance OPGx-LCA5 into a pivotal Phase 3 trial and we are hopeful that, if successful in Phase 3, and approved, OPGx-LCA5 may offer a potentially life-changing therapeutic option for individuals living with LCA5.”
Abstract details
| Title: | Recovery of cone mediated vision in severe ciliopathy after gene augmentation; One year results from a Phase I/II trial of LCA5-LCA (OPGx-LCA5) |
| Author: | Tomas Aleman, M.D., Schele Eye Institute, University of Pennsylvania et al |
| Presentation time: | May 4, 2025 from 4:30 PM to 4:45 PM MT |
| Location: | Room 255E |
OPGx-LCA5 is an investigational gene therapy for the treatment of Leber congenital amaurosis (LCA). The candidate is being evaluated in an ongoing non-randomized single ascending Phase 1/2 dose escalation study. Previously announced results showed OPGx-LCA5 to be well tolerated, with all three adult patients showing visual improvement at six months. New data from the study, to be presented at ARVO, show that subjective and objective signs of efficacy persisted for a year.
| Title: | Evaluation of MERTK gene therapy in RCS rats following a single bilateral subretinal injection |
| Author: | Mayur Choudhary, PhD., Opus Genetics et al |
| Presentation time: | May 8, 2025 from 11:45 AM to 1:30 PM MT |
| Posterboard Number: | 5944 - A0009 |
OPGx-MERTK is a gene therapy being developed by Opus Genetics to treat patients impacted by MERTK-related retinitis pigmentosa (RP). Results from a pre-clinical study which evaluated OPGx-MERTK in a rat model of RP will be presented at ARVO.
| Title: | Evaluation of ocular tolerability of OPGx-RDH12 by subretinal delivery in cynomolgus primates |
| Author: | Ash Jayagopal, PhD., Opus Genetics et al |
| Presentation time: | May 5, 2025 from 8:30 AM to 10:15 AM MT |
| Posterboard Number: | 1621 - B0205 |
OPGx-RDH12 is a gene therapy being developed to treat Leber congenital amaurosis 13 (LCA13), a genetic retinal dystrophy caused by mutations in the RDH12 gene. This pre-clinical study was conducted to evaluate the tolerability of OPGx-RDH12 in primates. Results from this pre-clinical study will be presented at ARVO.
| Title: | LYNX-1 Phase 3 trial of the safety and efficacy of phentolamine ophthalmic solution for the treatment of reduced mesopic low contrast vision: A subset analysis of keratorefractive subjects |
| Author: | Kostas Charizanis, PhD., Opus Genetics et al |
| Presentation time: | May 4, 2025 from 8:00 AM to 9:45 AM MT |
| Posterboard Number: | 173 - A0289 |
LYNX-1 was a randomized, double-masked, placebo-controlled trial of the safety and efficacy of Phentolamine Ophthalmic Solution
About Opus Genetics
Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our most advanced investigational gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. BEST1 investigational gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; we expect that a Phase 1/2 study will be initiated in 2025. The pipeline also includes 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, statements concerning data from and future enrollment for our clinical trials and our pipeline of additional indications.
These forward-looking statements relate to 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 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. 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.
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, including, without limitation:
- Our ability to successfully integrate the business of former Opus Genetics Inc. and manage our expanded combined product pipeline;
- Our ability to develop and obtain regulatory approval for newly acquired gene therapies to treat inherited retinal diseases;
- Our ability to obtain and maintain orphan drug designation or rare pediatric disease designation for our current and future product candidates;
- The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;
- Regulatory requirements or developments;
- Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;
- Delays or difficulties in the enrollment of patients in clinical trials;
- Substantial competition, including from generic versions of our product candidates;
- Rapid technological change;
- Our development of sales and marketing infrastructure;
- Future revenue losses and profitability;
- Changes in capital resource requirements;
- Risks related to our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs;
- Domestic and worldwide legislative, regulatory, political and economic developments;
- Our dependency on key personnel;
- Changes in market opportunities and acceptance;
- Reliance on third parties to conduct our clinical trials and supply and manufacture drug supplies;
- Future, potential product liability and securities litigation;
- System failures, unplanned events, or cyber incidents;
- The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;
- Risks that our licensing or partnership arrangements may not facilitate the commercialization or market acceptance of our product candidates;
- Future fluctuations in the market price of our common stock;
- Actions by activist stockholders;
- The success and timing of commercialization of any of our product candidates;
- Obtaining and maintaining our intellectual property rights; and
- The success of mergers and acquisitions.
The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the Securities and Exchange Commission that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Contacts
| Corporate | Investor Relations |
| Nirav Jhaveri CFO ir@opusgtx.com | Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com |
FAQ
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