Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Ocugen (NASDAQ: OCGN) announced positive preliminary data from Phase 1 of its OCU410 ArMaDa clinical trial for geographic atrophy (GA). The trial evaluated nine patients across three dose cohorts, with the low-dose group showing 21.4% slower lesion growth compared to untreated eyes. The treatment demonstrated preservation of retinal tissue and improved visual function through low luminance visual acuity (LLVA) tests. OCU410 aims to be a single-dose treatment, potentially offering advantages over current treatments that require multiple injections. The condition affects approximately three million people in the US and Europe combined, with treatment options currently available.
Ocugen (NASDAQ: OCGN) ha annunciato dati preliminari positivi dalla Fase 1 del suo trial clinico OCU410 ArMaDa per l'atrofia geografica (GA). Lo studio ha valutato nove pazienti attraverso tre gruppi di dosaggio, con il gruppo a basso dosaggio che ha mostrato una crescita delle lesioni più lenta del 21,4% rispetto agli occhi non trattati. Il trattamento ha dimostrato di preservare il tessuto retinico e di migliorare la funzione visiva attraverso test di acuità visiva a bassa luminosità (LLVA). OCU410 mira a essere un trattamento monodose, offrendo potenzialmente vantaggi rispetto ai trattamenti attuali che richiedono più iniezioni. Questa condizione colpisce circa tre milioni di persone negli Stati Uniti e in Europa insieme, con opzioni di trattamento attualmente disponibili.
Ocugen (NASDAQ: OCGN) anunció datos preliminares positivos de la Fase 1 de su ensayo clínico OCU410 ArMaDa para la atrofia geográfica (GA). El ensayo evaluó a nueve pacientes en tres grupos de dosis, con el grupo de baja dosis mostrando un crecimiento de lesiones un 21.4% más lento en comparación con los ojos no tratados. El tratamiento demostró preservar el tejido retiniano y mejorar la función visual a través de pruebas de agudeza visual a baja luminosidad (LLVA). OCU410 tiene como objetivo ser un tratamiento de dosis única, lo que podría ofrecer ventajas sobre los tratamientos actuales que requieren múltiples inyecciones. La condición afecta a aproximadamente tres millones de personas en Estados Unidos y Europa en conjunto, con opciones de tratamiento actualmente disponibles.
Ocugen (NASDAQ: OCGN)은 지리적 위축(GA)에 대한 OCU410 ArMaDa 임상 시험 1상에서 긍정적인 초기 데이터를 발표했습니다. 이 시험은 세 개의 용량 그룹에서 아홉 명의 환자를 평가했으며, 저용량 그룹은 치료되지 않은 눈에 비해 21.4% 느린 병변 성장을 보였습니다. 치료는 망막 조직을 보존하고 저광 비주얼 시력(LLVA) 테스트를 통해 시각 기능을 개선했습니다. OCU410은 단일 투여 치료제를 목표로 하고 있으며, 여러 번의 주사가 필요한 현재의 치료보다 잠재적인 이점을 제공할 수 있습니다. 이 질환은 미국과 유럽에서 약 300만 명에게 영향을 미치며, 현재 사용 가능한 치료 옵션이 있습니다.
Ocugen (NASDAQ: OCGN) a annoncé des données préliminaires positives de la phase 1 de son essai clinique OCU410 ArMaDa pour l'atrophie géographique (GA). L'essai a évalué neuf patients dans trois cohortes de dosage, le groupe à faible dose montrant une croissance des lésions 21,4% plus lente par rapport aux yeux non traités. Le traitement a démontré une préservation du tissu rétinien et une amélioration de la fonction visuelle à travers des tests d'acuité visuelle en faible luminosité (LLVA). OCU410 vise à être un traitement à dose unique, offrant potentiellement des avantages par rapport aux traitements actuels nécessitant plusieurs injections. Cette condition affecte environ trois millions de personnes aux États-Unis et en Europe combinés, avec des options de traitement actuellement disponibles.
Ocugen (NASDAQ: OCGN) hat positive vorläufige Daten aus Phase 1 seiner OCU410 ArMaDa-Studie zur geografischen Atrophie (GA) bekannt gegeben. Die Studie bewertete neun Patienten in drei Dosierungsgruppen, wobei die Gruppe mit niedriger Dosierung ein 21,4% langsameres Wachstum von Läsionen im Vergleich zu unbehandelten Augen zeigte. Die Behandlung zeigte eine Erhaltung des retinalen Gewebes und verbesserte die Sehfunktion durch Tests der visuellen Schärfe bei niedriger Helligkeit (LLVA). OCU410 soll eine Einzeldosisbehandlung sein, die potenzielle Vorteile gegenüber aktuellen Behandlungen bietet, die mehrere Injektionen erfordern. Diese Erkrankung betrifft etwa drei Millionen Menschen in den USA und Europa zusammen, mit derzeit verfügbaren Behandlungsoptionen.
- Phase 1 trial showed 21.4% slower lesion growth in treated vs untreated eyes
- 100% of treated eyes showed stabilization of visual function
- No drug-related serious adverse events reported
- Single-dose treatment potential vs current multiple injection requirements
- Only preliminary data from Phase 1 with patient sample (3 patients in low dose cohort)
- Early-stage trial results may not translate to later phases
Insights
The preliminary Phase 1 data for OCU410 shows significant promise in treating geographic atrophy (GA). The key findings demonstrate 21.4% slower lesion growth compared to untreated eyes and 100% stabilization of visual function in treated eyes. This is particularly noteworthy as current treatments require frequent injections and only target the complement pathway, while OCU410 offers a potential one-time treatment addressing multiple disease mechanisms.
The data compares favorably to pegcetacoplan (marketed as SYFOVRE), the only FDA-approved treatment for GA. The preservation of retinal tissue and improvement in low luminance visual acuity (LLVA) are especially significant, as existing treatments haven't shown meaningful benefits in visual function. With a market of 3 million patients in the US and Europe combined and no approved treatments in Europe, OCU410 could capture substantial market share if these results are maintained in larger trials.
MALVERN, Pa., Nov. 19, 2024 (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 positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA).
Currently, there are approximately three million people living with GA in the United States (U.S.) and Europe combined. Patients in the U.S. have only one option available, anti-complement therapy, which requires multiple injections and only addresses one aspect of the disease. There remains no treatment option for GA in Europe.
The OCU410 Phase 1 trial is evaluating nine patients in three dose cohorts (low, medium, and high). The following data was observed for the three patients in the low dose cohort at six months:
- Considerably slower lesion growth (
21.4% ) from baseline in treated vs. untreated fellow eyes that followed the natural history of the disease. This result is favorable when compared to published data on pegcetacoplan injected every month or every other month over six months. - OCU410 treatment showed increasing preservation of retinal tissue around the GA lesions of treated eyes over six months, which also compared favorably to published data on pegcetacoplan given monthly and every other month.
100% of the OCU410 treated eyes showed stabilization of visual function demonstrating treatment benefit as measured by LLVA.
“Currently approved treatments for GA have not shown significant benefit in visual function. More importantly, we often do not realize the logistical challenge and emotional burden both patients and their caregivers must endure for every month or every other month visits,” said Syed M. Shah, MD, FACS, Director of Retina Service, Vice Chair for Research & Digital Health at Emplify Health – La Crosse, Wisconsin. “Based on the science and preliminary data, OCU410 has the potential to improve structural as well as functional outcomes. This ‘one-and-done’ treatment paradigm can be a gamechanger for how we treat patients with GA.”
“OCU410 addresses multiple aspects of the disease beyond the complement pathway,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The latest OCU410 data emphasizes the potential of novel modifier gene therapy as a one-time treatment for dAMD. We remain very encouraged by the latest safety and efficacy data and positive patient outcomes.”
Ocugen also announced promising data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial. All these findings, as well as commentary from study investigators and patient perspectives, were shared at the Company’s recent Clinical Showcase. The data affirms the potential for modifier gene therapy to address both rare inherited retinal diseases and blindness diseases affecting millions.
A full replay of the showcase is available on the Events section of the Ocugen website. For more information about Ocugen’s ongoing clinical trials, please contact clinical.request@ocugen.com
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s 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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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; 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 annual and 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 Communications
Tiffany.Hamilton@ocugen.com
FAQ
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