Ocugen, Inc. Announces First Patient Dosed in Phase 2/3 GARDian3 Pivotal Confirmatory Trial for OCU410ST—Novel Modifier Gene Therapy Candidate for Stargardt Disease
Ocugen (NASDAQ: OCGN) has announced the dosing of its first patient in the Phase 2/3 GARDian3 pivotal confirmatory trial for OCU410ST, a modifier gene therapy candidate for Stargardt disease. The trial follows encouraging Phase 1 results that showed 48% slower lesion growth and a statistically significant 2-line gain in visual acuity at 12-month follow-up.
The Phase 2/3 study will enroll 51 participants, with 34 receiving a one-time subretinal injection of OCU410ST and 17 serving as untreated controls. The therapy targets ABCA4-associated retinopathies, affecting approximately 100,000 patients in the U.S. and Europe. Ocugen plans to submit a Biologics License Application (BLA) in 2027, based on one-year follow-up data.
Ocugen (NASDAQ: OCGN) ha annunciato la somministrazione della prima dose al primo paziente nello studio pivotale di conferma di Fase 2/3 GARDian3 per OCU410ST, un candidato terapia genica modificatrice per la malattia di Stargardt. Lo studio segue risultati incoraggianti di Fase 1 che hanno mostrato una riduzione del 48% nella crescita delle lesioni e un significativo miglioramento di 2 linee nell'acuità visiva a 12 mesi di follow-up.
Lo studio di Fase 2/3 arruolerà 51 partecipanti, con 34 soggetti che riceveranno un'iniezione subretinica unica di OCU410ST e 17 che fungeranno da controlli non trattati. La terapia è rivolta alle retinopatie associate al gene ABCA4, che colpiscono circa 100.000 pazienti negli Stati Uniti e in Europa. Ocugen prevede di presentare una domanda di autorizzazione biologica (BLA) nel 2027, basandosi sui dati di follow-up a un anno.
Ocugen (NASDAQ: OCGN) ha anunciado la dosificación del primer paciente en el ensayo pivotal confirmatorio de Fase 2/3 GARDian3 para OCU410ST, un candidato a terapia génica modificadora para la enfermedad de Stargardt. El ensayo sigue resultados alentadores de Fase 1 que mostraron una reducción del crecimiento de lesiones del 48% y una ganancia estadísticamente significativa de 2 líneas en agudeza visual a los 12 meses de seguimiento.
El estudio de Fase 2/3 inscribirá a 51 participantes, con 34 recibiendo una inyección subretiniana única de OCU410ST y 17 como controles no tratados. La terapia está dirigida a retinopatías asociadas con ABCA4, que afectan aproximadamente a 100,000 pacientes en EE. UU. y Europa. Ocugen planea presentar una Solicitud de Licencia Biológica (BLA) en 2027, basada en datos de seguimiento a un año.
Ocugen (NASDAQ: OCGN)은 스타가르트병 치료 후보인 OCU410ST에 대한 2/3상 GARDian3 결정적 확인 임상시험에서 첫 환자 투여를 발표했습니다. 1상 결과에서는 병변 성장률이 48% 느려지고 12개월 추적 관찰 시 시력에서 통계적으로 유의한 2줄 개선이 확인되었습니다.
2/3상 연구는 51명의 참여자를 모집하며, 이 중 34명은 OCU410ST 단회 망막하 주사를 받고 17명은 치료받지 않는 대조군으로 구성됩니다. 이 치료법은 ABCA4 관련 망막병증을 대상으로 하며, 미국과 유럽에서 약 10만 명의 환자가 영향을 받습니다. Ocugen은 1년 추적 데이터에 근거하여 2027년 생물의약품 허가 신청(BLA)을 계획하고 있습니다.
Ocugen (NASDAQ : OCGN) a annoncé la première administration chez un patient dans l'essai pivot de phase 2/3 GARDian3 confirmatoire pour OCU410ST, un candidat en thérapie génique modificatrice pour la maladie de Stargardt. L'essai fait suite à des résultats encourageants de phase 1 montrant une ralentissement de 48 % de la croissance des lésions et un gain statistiquement significatif de 2 lignes d'acuité visuelle au suivi à 12 mois.
L'étude de phase 2/3 recrutera 51 participants, dont 34 recevront une injection sous-rétinienne unique d'OCU410ST et 17 serviront de témoins non traités. La thérapie cible les rétinopathies associées à ABCA4, touchant environ 100 000 patients aux États-Unis et en Europe. Ocugen prévoit de soumettre une demande d'autorisation de mise sur le marché biologique (BLA) en 2027, basée sur les données de suivi à un an.
Ocugen (NASDAQ: OCGN) hat die Dosierung des ersten Patienten in der Phase 2/3 GARDian3 entscheidenden Bestätigungsstudie für OCU410ST bekanntgegeben, einem gentherapeutischen Kandidaten zur Modifikation der Stargardt-Krankheit. Die Studie folgt auf vielversprechende Phase-1-Ergebnisse, die ein 48% langsameres Wachstum der Läsionen und einen statistisch signifikanten 2-Zeilen-Gewinn der Sehschärfe nach 12 Monaten zeigten.
Die Phase-2/3-Studie wird 51 Teilnehmer einschließen, von denen 34 eine einmalige subretinale Injektion von OCU410ST erhalten und 17 als unbehandelte Kontrollgruppe dienen. Die Therapie richtet sich gegen ABCA4-assoziierte Retinopathien, die etwa 100.000 Patienten in den USA und Europa betreffen. Ocugen plant, basierend auf den Einjahres-Follow-up-Daten, im Jahr 2027 einen Biologika-Zulassungsantrag (BLA) einzureichen.
- None.
- BLA submission timeline extends to 2027, indicating a lengthy development process
- Relatively small trial size of 51 participants for a pivotal study
Insights
Ocugen achieves key milestone with first patient dosed in pivotal OCU410ST trial for Stargardt disease, backed by promising Phase 1 data.
Ocugen has reached a significant clinical milestone with the first patient dosed in its Phase 2/3 GARDian3 trial for OCU410ST, their modifier gene therapy for Stargardt disease. This trial advancement follows encouraging Phase 1 results showing 48% slower lesion growth and a statistically significant 2-line gain in visual acuity (p=0.031) at 12-month follow-up in treated versus untreated eyes.
The pivotal trial employs an adaptive design with 51 participants - 34 receiving treatment and 17 serving as controls. A planned interim analysis at 8 months on 24 subjects should provide early efficacy and safety signals. This efficient design could accelerate the path to potential approval for a currently untreatable condition affecting 100,000 patients in the US and Europe.
Notably, OCU410ST has maintained a favorable safety profile with no serious adverse events reported, addressing a critical hurdle for ocular gene therapies. If successful, this one-time therapy could transform treatment for the estimated 1 million patients worldwide with Stargardt disease, representing substantial market potential.
This trial marks Ocugen's second late-stage program, supporting their strategic goal of filing three BLAs within three years. The planned 2027 BLA submission for OCU410ST suggests the company is building a robust pipeline of potentially transformative therapies for inherited retinal diseases.
MALVERN, Pa., July 18, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies).
“Dosing the first patient is an especially significant milestone and brings us closer to our goal of addressing the unmet medical need that exists for all Stargardt patients—100,000 in the U.S. and Europe and 1 million worldwide,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Progressing our second modifier gene therapy candidate into a registration clinical trial is a pivotal step in potentially providing a one-time therapy for life for the millions of patients affected by inherited retinal diseases.”
The Phase 2/3 clinical trial for OCU410ST builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated
“Initiating dosing in this pivotal Phase 2/3 study is an important advancement for Ocugen and more importantly for the Stargardt community," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “The adaptive design of this trial, including a masked interim analysis at 8 months on 24 subjects, enables us to efficiently evaluate early signals of efficacy and safety while optimizing study conduct. This ensures we generate robust and meaningful data to support our regulatory submissions for approvals.”
"Treating the first patient with this novel gene therapy in the GARDian3 trial is a proud and hopeful moment for our team and for families affected by Stargardt disease," said Victor H. Gonzalez, MD, Principal Investigator and retinal surgeon at Valley Retina Institute, McAllen, Texas. "For decades, patients have faced the progressive loss of central vision with no approved treatment options. The encouraging Phase 1 results give us confidence that OCU410ST could meaningfully slow disease progression and help preserve vision. This trial brings us closer to the possibility of a one-time gene therapy that could transform patients’ quality of life for years to come."
OCU410ST maintains a favorable safety and tolerability profile with no serious adverse events or adverse events of special interest, including ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization.
The Phase 2/3 study will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company’s planned Biologics License Application (BLA).
The OCU410ST Phase 2/3 pivotal confirmatory trial represents Ocugen’s second late-stage clinical program. Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases 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 address significant unmet medical need for large patient populations through our gene-agnostic approach. 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 OCU410ST 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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