Ocugen, Inc. Announces Dosing Completion in the Phase 2 ArMaDa Clinical Trial for OCU410—a Multifunctional Modifier Gene Therapy for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Ocugen (NASDAQ: OCGN) has completed dosing in the Phase 2 portion of its Phase 1/2 ArMaDa clinical trial for OCU410, a novel gene therapy for geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD). The trial enrolled 51 subjects randomized into treatment and control arms.
The Phase 1/2 study (N=60) showed promising results with a 44% slower lesion growth in treated eyes versus untreated eyes at 9 months, and a clinically meaningful 2-line improvement in visual function. The therapy demonstrated a favorable safety profile with no serious adverse events.
OCU410 is designed as a one-time treatment, targeting multiple disease pathways, unlike current U.S. treatments that require monthly or bi-monthly injections. The trial is being conducted at 14 leading retinal surgery centers across the U.S., with Phase 3 studies planned for 2026 and potential BLA/MAA filings by 2028.
Ocugen (NASDAQ: OCGN) ha completato la somministrazione nella fase 2 del suo studio clinico ArMaDa di fase 1/2 per OCU410, una nuova terapia genica per l'atrofia geografica (GA), una forma avanzata di degenerazione maculare secca legata all'età (dAMD). Lo studio ha arruolato 51 soggetti randomizzati in gruppi di trattamento e controllo.
Lo studio di fase 1/2 (N=60) ha mostrato risultati promettenti con una crescita delle lesioni più lenta del 44% negli occhi trattati rispetto a quelli non trattati a 9 mesi, e un miglioramento clinicamente significativo di 2 linee nella funzione visiva. La terapia ha dimostrato un profilo di sicurezza favorevole senza eventi avversi gravi.
OCU410 è progettato come un trattamento una tantum, mirato a più vie patologiche, a differenza dei trattamenti attuali negli Stati Uniti che richiedono iniezioni mensili o bimestrali. Lo studio viene condotto in 14 centri di chirurgia retinica di punta negli Stati Uniti, con studi di fase 3 previsti per il 2026 e potenziali richieste di BLA/MAA entro il 2028.
Ocugen (NASDAQ: OCGN) ha completado la dosificación en la fase 2 de su ensayo clínico ArMaDa de fase 1/2 para OCU410, una nueva terapia génica para la atrofia geográfica (GA), una forma avanzada de degeneración macular seca relacionada con la edad (dAMD). El ensayo incluyó a 51 sujetos aleatorizados en grupos de tratamiento y control.
El estudio de fase 1/2 (N=60) mostró resultados prometedores con un crecimiento de lesiones un 44% más lento en los ojos tratados en comparación con los no tratados a los 9 meses, y una mejora clínicamente significativa de 2 líneas en la función visual. La terapia demostró un perfil de seguridad favorable sin eventos adversos graves.
OCU410 está diseñado como un tratamiento único, dirigido a múltiples vías de la enfermedad, a diferencia de los tratamientos actuales en EE. UU. que requieren inyecciones mensuales o bimensuales. El ensayo se está realizando en 14 centros de cirugía retiniana líderes en EE. UU., con estudios de fase 3 planificados para 2026 y posibles solicitudes de BLA/MAA para 2028.
Ocugen (NASDAQ: OCGN)은 OCU410의 1상/2상 ArMaDa 임상 시험의 2상 부분에서 투약을 완료했습니다. OCU410은 고급 형태의 나이 관련 건성 황반변성(dAMD)인 지리적 위축(GA)에 대한 새로운 유전자 요법입니다. 이 시험에는 치료군과 대조군으로 무작위 배정된 51명의 피험자가 등록되었습니다.
1상/2상 연구(N=60)는 9개월 동안 치료받은 눈에서 비치료 눈에 비해 병변 성장 속도가 44% 느리다는 유망한 결과를 보여주었으며, 시각 기능에서 임상적으로 의미 있는 2라인 개선을 나타냈습니다. 이 요법은 심각한 부작용 없이 우수한 안전성 프로파일을 보였습니다.
OCU410은 한 번의 치료로 설계되었으며, 현재 미국에서 매월 또는 격월로 주사가 필요한 치료법과 달리 여러 질병 경로를 목표로 합니다. 이 시험은 미국 전역의 14개 주요 망막 수술 센터에서 진행되고 있으며, 2026년에 3상 연구가 계획되어 있고 2028년까지 BLA/MAA 제출이 가능할 것으로 보입니다.
Ocugen (NASDAQ: OCGN) a terminé l'administration dans la phase 2 de son essai clinique ArMaDa de phase 1/2 pour OCU410, une thérapie génique novatrice pour l'atrophie géographique (GA), une forme avancée de dégénérescence maculaire liée à l'âge (dAMD). L'essai a inclus 51 sujets randomisés dans des groupes de traitement et de contrôle.
L'étude de phase 1/2 (N=60) a montré des résultats prometteurs avec une croissance des lésions 44% plus lente dans les yeux traités par rapport aux yeux non traités après 9 mois, et une amélioration cliniquement significative de 2 lignes dans la fonction visuelle. La thérapie a présenté un profil de sécurité favorable sans événements indésirables graves.
OCU410 est conçu comme un traitement unique, ciblant plusieurs voies de la maladie, contrairement aux traitements actuels aux États-Unis qui nécessitent des injections mensuelles ou bimensuelles. L'essai est réalisé dans 14 centres de chirurgie rétinienne de premier plan aux États-Unis, avec des études de phase 3 prévues pour 2026 et des dépôts potentiels de BLA/MAA d'ici 2028.
Ocugen (NASDAQ: OCGN) hat die Dosierung in der Phase 2 seiner Phase 1/2 ArMaDa-Studie für OCU410 abgeschlossen, eine neuartige Gentherapie gegen geografische Atrophie (GA), eine fortgeschrittene Form der altersbedingten trockenen Makuladegeneration (dAMD). In die Studie wurden 51 Probanden randomisiert in Behandlungs- und Kontrollgruppen aufgenommen.
Die Phase 1/2-Studie (N=60) zeigte vielversprechende Ergebnisse mit einem 44% langsameren Läsionswachstum in behandelten Augen im Vergleich zu unbehandelten Augen nach 9 Monaten und einer klinisch bedeutenden Verbesserung von 2 Linien in der Sehfunktion. Die Therapie wies ein günstiges Sicherheitsprofil ohne schwerwiegende unerwünschte Ereignisse auf.
OCU410 ist als einmalige Behandlung konzipiert und zielt auf mehrere Krankheitswege ab, im Gegensatz zu den aktuellen Behandlungen in den USA, die monatliche oder zwei-monatliche Injektionen erfordern. Die Studie wird in 14 führenden Zentren für Netzhautchirurgie in den USA durchgeführt, mit geplanten Phase-3-Studien für 2026 und möglichen BLA/MAA-Anträgen bis 2028.
- Phase 1/2 study showed 44% slower lesion growth in treated eyes vs untreated eyes at 9 months
- Demonstrated 2-line improvement in visual function compared to untreated eyes
- Favorable safety profile with no serious adverse events
- Single injection treatment compared to current monthly/bi-monthly treatments
- Successfully completed Phase 2 enrollment ahead of schedule
- Phase 3 trials not starting until 2026
- Potential regulatory approvals still years away (2028)
- Faces competition from existing FDA-approved treatments
Insights
The completion of Phase 2 dosing for OCU410 marks a pivotal advancement in treating Geographic Atrophy (GA), with several compelling differentiators that position it as a potential market leader. The Phase 1/2 data reveals two critical advantages: 44% slower lesion growth and a clinically meaningful 2-line improvement in visual function compared to untreated eyes.
The therapy's unique value proposition lies in its multifunctional modifier approach, targeting multiple disease pathways simultaneously - a significant advancement over current FDA-approved treatments that focus solely on complement inhibition. This broader mechanism of action could potentially address the complex nature of GA more effectively, particularly given the disease's multifactorial etiology involving both genetic and environmental factors.
The market opportunity is substantial, with 266 million dry AMD patients globally and approximately 2-3 million GA patients in the US and Europe. Notably, European patients currently have no approved treatments, representing an untapped market segment. The therapy's one-time administration model could dramatically improve the treatment paradigm, eliminating the burden of monthly or bi-monthly injections required by current treatments.
The planned 2026 Phase 3 study and targeted 2028 BLA/MAA filings present a clear path to commercialization. The favorable safety profile, with no serious adverse events related to OCU410, positions it advantageously against existing treatments that carry risk of complications affecting vision. This safety aspect, combined with the convenience of single administration, could drive significant market adoption and potentially establish OCU410 as a first-line treatment option for GA.
- Completed Phase 2 enrollment with randomization of 51 subjects into treatment and control arms
- Phase 1/2 study (N=60) demonstrated favorable safety and tolerability profile with no serious adverse events related to OCU410, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization
- Subjects showed considerably slower lesion growth (
44% ) from baseline in treated eyes versus untreated fellow eyes at 9 months in follow-up data from the Phase 1 study - Clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes was noted in the Phase 1 portion of the trial
- Preservation of retinal tissue at 9 months around GA lesions of treated eyes with a single injection of OCU410 in Phase 1 compared favorably to published data on a leading FDA-approved complement inhibitor given monthly or every other month at the same time points
MALVERN, Pa., Feb. 12, 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 that dosing is complete, ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410—a novel multifunctional modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). Age-related macular degeneration (AMD) affects 1 in 8 people 60 years and older. The global prevalence of dAMD is 266 million worldwide and by 2050 more than 5 million Americans may suffer from this incurable condition. Today, GA – the later stage of dAMD – affects approximately 2-3 million people in the United States (U.S.) and Europe.
There are limited options for patients with dAMD in the U.S. and current therapies involve frequent (monthly or every other month) injections and have unwanted side effects that can affect vision. These therapies are not approved in Europe, leaving approximately 2 million patients with no therapeutic option.
“Dosing completion is a major accomplishment for our OCU410 program,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Based on the multifunctional effect of our modifier gene therapy, the profound unmet medical need, limited treatment options, and the fact that it is designed as a one and done treatment, we believe OCU410 can be a potential blockbuster therapy and the gold standard for treating GA worldwide. The data from this trial will help us design a future pivotal Phase 3 study planned for 2026 and enable our commercial strategy for Biologics License Application (BLA) and Marketing Authorization Application (MAA) filings as soon as 2028.”
“The preliminary efficacy and safety data from the Phase 1/2 study are highly encouraging, demonstrating the potential of OCU410 to improve both structural and functional outcomes,” said Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics and Biomedical Engineering with Tenure at Duke University Eye Center. “I look forward to the Phase 2 results and believe a one-time gene therapy could reshape the treatment landscape, offering a transformative option for patients.”
GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. are limited to those targeting a single mechanism—the complement pathway—requiring frequent intravitreal injections, either monthly or every other month. By contrast, OCU410 is a multifunctional modifier gene therapy, which targets multiple pathways associated with GA.
“Given the safety concerns associated with currently approved GA treatments, the encouraging safety and tolerability profile of OCU410 offers a promising treatment option,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “With Phase 2 enrollment now complete, OCU410 has the potential to be a one-time treatment, reducing the burden of frequent injections, improving patient compliance, and ultimately enhancing quality of life.”
In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dAMD will be assessed. Fifty-one (51) patients were randomized 1:1:1 into either of two treatment groups (medium or high dose) or a control group. In the treatment groups, subjects received a single subretinal 200-µL administration of 5 x 1010 vector genomes (vg)/mL (medium dose) or 1.5 x 1011 vg/mL (high dose), while the control group remained untreated.
The ArMaDa clinical trial for OCU410 is being performed at 14 leading retinal surgery centers across the U.S.
About the Phase 1/2 ArMaDa clinical trial
The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open label, dose-escalation study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome assessor-blinded, dose-expansion study in which subjects were randomized in a 1:1:1 ratio to either the medium dose or high dose OCU410 treatment groups or to an untreated control group.
About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function, and central vision impairment. dAMD accounts for 85
About OCU410
OCU410 utilizes an adeno-associated virus (AAV) platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in both in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.
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 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 OCU410 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com
FAQ
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