Ocugen, Inc. Announces Positive 2-Year Data Across Multiple Mutations from Phase 1/2 Clinical Trial of OCU400 —A Novel Modifier Gene Therapy for Retinitis Pigmentosa
Ocugen (NASDAQ: OCGN) announced positive 2-year data from its Phase 1/2 clinical trial of OCU400, a novel modifier gene therapy for retinitis pigmentosa (RP). The trial showed that 100% of treated subjects (9/9) demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years.
Key findings include statistically significant improvement in visual function across multiple mutations (p=0.01), with meaningful improvement of 2-line gain in low-luminance visual acuity. The therapy showed a favorable long-term safety profile with no serious adverse events. OCU400 aims to treat approximately 2 million patients globally (~300,000 in U.S./EU) with a one-time therapy.
The company's Phase 3 OCU400 liMeliGhT clinical trial is ongoing, with BLA submission targeted for first half of 2026.
Ocugen (NASDAQ: OCGN) ha annunciato dati positivi a 2 anni dal suo studio clinico di fase 1/2 su OCU400, una nuova terapia genica modificatrice per la retinite pigmentosa (RP). Lo studio ha mostrato che il 100% dei soggetti trattati (9/9) ha mostrato un miglioramento o una preservazione della funzione visiva rispetto agli occhi non trattati sia dopo un anno che dopo due anni.
Le principali scoperte includono un miglioramento statisticamente significativo della funzione visiva attraverso multiple mutazioni (p=0.01), con un significativo aumento di 2 linee nell'acuità visiva in condizioni di bassa luminosità. La terapia ha mostrato un profilo di sicurezza favorevole a lungo termine senza eventi avversi gravi. OCU400 mira a trattare circa 2 milioni di pazienti a livello globale (~300.000 negli Stati Uniti e in Europa) con una terapia unica.
Il trial clinico di fase 3 OCU400 liMeliGhT è in corso, con la sottomissione della BLA prevista per la prima metà del 2026.
Ocugen (NASDAQ: OCGN) anunció datos positivos a 2 años de su ensayo clínico de fase 1/2 sobre OCU400, una nueva terapia génica modificadora para la retinitis pigmentosa (RP). El ensayo mostró que el 100% de los sujetos tratados (9/9) demostró mejora o preservación de la función visual en comparación con los ojos no tratados tanto a un año como a dos años.
Los hallazgos clave incluyen una mejora estadísticamente significativa en la función visual a través de múltiplesmutaciones (p=0.01), con una mejora significativa de 2 líneas en la agudeza visual en condiciones de baja luminosidad. La terapia mostró un perfil de seguridad a largo plazo favorable sin eventos adversos graves. OCU400 tiene como objetivo tratar aproximadamente a 2 millones de pacientes en todo el mundo (~300,000 en EE.UU./UE) con una única terapia.
El ensayo clínico de fase 3 OCU400 liMeliGhT está en curso, con la presentación de la BLA prevista para la primera mitad de 2026.
Ocugen (NASDAQ: OCGN)은 망막 색소 변성증(RP)을 위한 새로운 변형 유전자 치료제 OCU400의 2년 임상 1/2상 시험에서 긍정적인 데이터를 발표했습니다. 시험 결과 치료를 받은 피험자의 100%(9/9)가 치료되지 않은 눈에 비해 시각 기능이 개선되거나 보존되었다는 것을 보여주었습니다.
주요 발견 사항에는 여러 변이에서 시각 기능의 통계적으로 유의미한 개선(p=0.01)이 포함되며, 낮은 조도에서 2 라인의 시각적 예리함 개선이 나타났습니다. 이 치료제는 심각한 부작용 없이 장기적인 안전성 프로파일이 유리한 것으로 나타났습니다. OCU400은 전 세계적으로 약 200만 명의 환자를 치료하는 것을 목표로 하며(~30만 명은 미국 및 유럽), 단 한 번의 치료로 이루어집니다.
회사의 3상 OCU400 liMeliGhT 임상 시험은 진행 중이며, BLA 제출은 2026년 상반기로 예정되어 있습니다.
Ocugen (NASDAQ: OCGN) a annoncé des données positives sur 2 ans de son essai clinique de phase 1/2 concernant OCU400, une nouvelle thérapie génique modifiée pour la rétinite pigmentaire (RP). L'essai a montré que 100 % des sujets traités (9/9) ont présenté une amélioration ou une préservation de la fonction visuelle par rapport aux yeux non traités, tant à un qu'à deux ans.
Les principales conclusions incluent une amélioration statistiquement significative de la fonction visuelle à travers de multiples mutations (p=0,01), avec une amélioration significative de 2 lignes dans l'acuité visuelle en condition de faible luminosité. La thérapie a montré un profil de sécurité à long terme favorable sans événements indésirables graves. OCU400 vise à traiter environ 2 millions de patients dans le monde (~300 000 aux États-Unis et en Europe) avec une thérapie unique.
L'essai clinique de phase 3 OCU400 liMeliGhT est en cours, avec une soumission de BLA prévue pour le premier semestre 2026.
Ocugen (NASDAQ: OCGN) hat positive 2-Jahres-Daten aus seiner Phase 1/2-Studie zu OCU400, einer neuartigen modifizierenden Gentherapie für Retinitis pigmentosa (RP), bekannt gegeben. Die Studie zeigte, dass 100% der behandelten Probanden (9/9) eine Verbesserung oder Erhaltung der visuellen Funktion im Vergleich zu unbehandelten Augen sowohl nach einem als auch nach zwei Jahren aufwiesen.
Wichtige Ergebnisse umfassen einen statistisch signifikanten Anstieg der visuellen Funktion bei mehreren Mutationen (p=0.01), mit einem signifikanten Verbesserung von 2 Zeilen der Sehschärfe bei schwachem Licht. Die Therapie zeigte ein günstiges langfristiges Sicherheitsprofil ohne schwerwiegende unerwünschte Ereignisse. OCU400 zielt darauf ab, ungefähr 2 Millionen Patienten weltweit (~300.000 in den USA/EU) mit einer einmaligen Therapie zu behandeln.
Die Phase-3-Studie OCU400 liMeliGhT des Unternehmens ist noch im Gange, mit geplanter Einreichung der BLA für die erste Hälfte des Jahres 2026.
- 100% efficacy rate in treated subjects for visual function improvement/preservation
- Statistically significant improvement in visual function (p=0.01)
- Favorable long-term safety profile with no serious adverse events
- Clear timeline for BLA submission in H1 2026
- Large addressable market of 2 million patients globally
- Small sample size of only 9 evaluable subjects
- Phase 3 trials still needed before potential commercialization
- BLA submission not expected until 2026
Insights
The two-year data from OCU400's Phase 1/2 trial represents a significant clinical milestone in gene therapy for retinitis pigmentosa. The 100% response rate across all treated subjects and statistically significant improvement (p=0.01) in visual function validate the mutation-agnostic approach. The two-line gain in low-luminance visual acuity demonstrates meaningful clinical benefit, particularly relevant for RP patients who typically struggle in low-light conditions.
The mutation-agnostic mechanism is particularly groundbreaking - traditional gene therapies target specific mutations, limiting their applicability. OCU400's ability to work across multiple mutations could potentially address the entire RP patient population (
This data release significantly strengthens OCU400's commercial potential. The addressable market of 2 million patients globally (300,000 in US/EU) represents a substantial commercial opportunity. Traditional gene therapies typically target specific mutations, fragmenting the market, but OCU400's mutation-agnostic approach could capture the entire RP market with a single product.
The one-time therapy model suggests premium pricing potential, typical for gene therapies. Comparable rare disease gene therapies are priced between
The durability of effect at two years is important for gene therapy validation. The improvement in low-luminance visual acuity is particularly meaningful for RP patients, as night blindness is often the first symptom of disease progression. The 100% response rate in mobility testing up to one year suggests real-world functional benefits, which could support favorable reimbursement decisions.
The clean safety profile with no serious adverse events related to OCU400 is essential for regulatory approval prospects. The advancement to Phase 3 with a clear BLA timeline indicates confidence in the data package. If approved, OCU400 would be the first mutation-agnostic gene therapy for RP, potentially revolutionizing treatment paradigms for inherited retinal diseases.
100% (9/9) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years100% (9/9) of treated evaluable subjects demonstrated improvement or stabilization in mobility testing, which was only performed up to one year- Improvement in visual function was statistically significant (p=0.01, treated vs untreated eyes), regardless of mutation at two years
- Favorable long-term safety and tolerability profile with no serious adverse events related to OCU400
MALVERN, Pa., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced a positive 2-year safety and efficacy update for the Phase 1/2 OCU400 clinical trial. OCU400 is intended for the treatment of early to advanced retinitis pigmentosa (RP) in pediatric and adult populations. Currently, about 2 million patients globally (~300,000 in U.S./EU) desperately need rescue, otherwise they can potentially become legally blind at some point in their life. OCU400 is intended to treat all these patients with a one-time therapy.
OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes. This treatment effect was statistically significant (p=0.01) in all subjects, regardless of mutation at two years, validating the gene-agnostic mechanism of action for OCU400.
This data highlights the unique therapeutic potential of OCU400. To date, Ocugen’s groundbreaking modifier gene therapy platform provides long-term safety and meaningful improvements in visual function for patients suffering from RP—a condition that leads to progressive vision loss.
“We are thrilled to share these compelling results from the Phase 1/2 OCU400 trial,” said Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-Founder of Ocugen. “These findings represent a significant step forward in our mission to bring life-changing therapies to patients with inherited retinal disorders and confirm the transformative potential of our modifier gene therapy platform.”
"It is truly remarkable to see the significant improvements in visual acuity in patients treated with OCU400 sustained at two years,” said Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin. “The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases."
“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer at Ocugen. “These 2 year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”
The Phase 3 OCU400 liMeliGhT clinical trial is currently ongoing and on target for BLA submission in the first half of 2026.
About OCU400
OCU400 is the Company’s modifier gene therapy product based on a nuclear hormone receptor (NHR) gene called NR2E3. This gene regulates diverse physiological functions within the retina, such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival. Retinal cells in RP patients have a dysfunctional gene network, and OCU400 resets this network to reestablish a healthy cellular homeostasis—which has the potential to improve vision in patients with RP.
About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP and Stargardt disease, as well as multifactorial diseases like dry age-related macular degeneration (dAMD). Our modifier gene therapy platform is based on the use of NHRs, master gene regulators, which have the potential to restore homeostasis—the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently, Ocugen has three modifier gene therapy programs in the clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400 Phase 3 liMeliGhT clinical trial, the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA) secondary to dAMD and the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease are currently underway. GA affects approximately two to three million people in the U.S. and EU combined, and Stargardt disease affects nearly 100,000 people in the U.S. and EU combined.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU400 to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact:
Tiffany Hamilton
Head of Corporate Communications
Tiffany.Hamilton@ocugen.com
FAQ
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