Ocugen, Inc. Announces FDA Alignment on Phase 2/3 Pivotal Confirmatory Clinical Trial for Modifier Gene Therapy Candidate OCU410ST for Stargardt Disease
Ocugen (NASDAQ: OCGN) has received FDA alignment to proceed with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, their modifier gene therapy candidate for Stargardt disease. The GARDian trial demonstrated promising results, including:
- 52% slower lesion growth in treated eyes vs untreated eyes at 6-month follow-up
- Statistically significant 2-line improvement in visual function
- Favorable safety profile with no serious adverse events
The upcoming Phase 2/3 trial will involve 51 subjects, with 34 receiving a single subretinal injection of OCU410ST and 17 serving as untreated controls. The company aims for potential BLA filing by 2027. OCU410ST received orphan drug designations from both FDA and EMA in 2023 and 2024. Stargardt disease affects 44,000 patients in the U.S. with no current treatment options.
Ocugen (NASDAQ: OCGN) ha ricevuto l'allineamento della FDA per procedere con uno studio clinico pivotale di fase 2/3 per OCU410ST, il loro candidato terapia genica modificata per la malattia di Stargardt. Lo studio GARDian ha dimostrato risultati promettenti, tra cui:
- Crescita delle lesioni più lenta del 52% negli occhi trattati rispetto a quelli non trattati a 6 mesi di follow-up
- Miglioramento statisticamente significativo di 2 linee nella funzione visiva
- Profilo di sicurezza favorevole senza eventi avversi gravi
Il prossimo studio di fase 2/3 coinvolgerà 51 soggetti, di cui 34 riceveranno un'iniezione subretinica singola di OCU410ST e 17 serviranno come controlli non trattati. L'azienda mira a presentare una domanda di BLA entro il 2027. OCU410ST ha ricevuto designazioni di farmaco orfano sia dalla FDA che dall'EMA nel 2023 e nel 2024. La malattia di Stargardt colpisce 44.000 pazienti negli Stati Uniti senza attuali opzioni di trattamento.
Ocugen (NASDAQ: OCGN) ha recibido la alineación de la FDA para proceder con un ensayo clínico pivotal de fase 2/3 para OCU410ST, su candidato a terapia génica modificada para la enfermedad de Stargardt. El ensayo GARDian mostró resultados prometedores, incluyendo:
- Crecimiento de lesiones un 52% más lento en los ojos tratados en comparación con los ojos no tratados a los 6 meses de seguimiento
- Mejora estadísticamente significativa de 2 líneas en la función visual
- Perfil de seguridad favorable sin eventos adversos graves
El próximo ensayo de fase 2/3 involucrará a 51 sujetos, de los cuales 34 recibirán una única inyección subretiniana de OCU410ST y 17 servirán como controles no tratados. La empresa tiene como objetivo presentar una solicitud de BLA para 2027. OCU410ST recibió designaciones de medicamento huérfano tanto de la FDA como de la EMA en 2023 y 2024. La enfermedad de Stargardt afecta a 44,000 pacientes en EE. UU. sin opciones de tratamiento actuales.
Ocugen (NASDAQ: OCGN)은 스타가르트병에 대한 수정 유전자 치료 후보인 OCU410ST에 대해 FDA의 승인을 받아 2/3상 주요 확인 임상 시험을 진행합니다. GARDian 시험은 다음과 같은 유망한 결과를 보여주었습니다:
- 치료받은 눈에서 비치료 눈에 비해 6개월 후 추적 관찰 시 병변 성장 속도가 52% 느림
- 시각 기능에서 통계적으로 유의미한 2라인 개선
- 심각한 부작용 없이 유리한 안전성 프로필
다가오는 2/3상 시험에는 51명이 참여하며, 34명은 OCU410ST의 단일 망막하 주사를 받고, 17명은 비치료 대조군으로 설정됩니다. 회사는 2027년까지 BLA 제출을 목표로 하고 있습니다. OCU410ST는 2023년과 2024년에 FDA와 EMA로부터 희귀의약품 지정을 받았습니다. 스타가르트병은 현재 치료 옵션이 없는 미국 내 44,000명의 환자에게 영향을 미칩니다.
Ocugen (NASDAQ: OCGN) a reçu l'alignement de la FDA pour procéder à un essai clinique pivot de phase 2/3 pour OCU410ST, leur candidat à la thérapie génique modifiée pour la maladie de Stargardt. L'essai GARDian a montré des résultats prometteurs, notamment :
- Croissance des lésions 52% plus lente dans les yeux traités par rapport aux yeux non traités lors du suivi à 6 mois
- Amélioration statistiquement significative de 2 lignes de la fonction visuelle
- Profil de sécurité favorable sans événements indésirables graves
Le prochain essai de phase 2/3 impliquera 51 sujets, dont 34 recevront une injection sous-rétinienne unique d'OCU410ST et 17 serviront de témoins non traités. L'entreprise vise un dépôt potentiel de BLA d'ici 2027. OCU410ST a reçu des désignations de médicament orphelin de la part de la FDA et de l'EMA en 2023 et 2024. La maladie de Stargardt touche 44 000 patients aux États-Unis sans options de traitement actuelles.
Ocugen (NASDAQ: OCGN) hat von der FDA die Genehmigung erhalten, mit einer entscheidenden Phase 2/3-Studie für OCU410ST, ihrem modifizierten Gentherapie-Kandidaten für die Stargardt-Krankheit, fortzufahren. Die GARDian-Studie zeigte vielversprechende Ergebnisse, darunter:
- 52% langsameres Wachstum von Läsionen in behandelten Augen im Vergleich zu unbehandelten Augen nach 6 Monaten Nachbeobachtung
- Statistisch signifikante Verbesserung der Sehfunktion um 2 Zeilen
- Günstiges Sicherheitsprofil ohne schwere unerwünschte Ereignisse
Die bevorstehende Phase 2/3-Studie wird 51 Probanden umfassen, von denen 34 eine einzelne subretinale Injektion von OCU410ST erhalten und 17 als unbehandelte Kontrollen dienen. Das Unternehmen strebt eine mögliche BLA-Einreichung bis 2027 an. OCU410ST erhielt 2023 und 2024 die Orphan-Drug-Designationen von der FDA und der EMA. Die Stargardt-Krankheit betrifft 44.000 Patienten in den USA, für die es derzeit keine Behandlungsmöglichkeiten gibt.
- FDA alignment accelerates development timeline by 2-3 years
- Phase 1 showed 52% slower disease progression
- Statistically significant visual improvement (p=0.02)
- Clean safety profile with no serious adverse events
- Orphan drug designation in both US and EU markets
- Large addressable market of 100,000 patients with no existing treatments
- Phase 2/3 results not expected until 2027
- Small trial size of only 51 patients
- Requires complex subretinal injection procedure
Insights
Ocugen's FDA alignment on a Phase 2/3 pivotal confirmatory trial for OCU410ST represents a significant regulatory acceleration that could transform the company's trajectory. This development potentially shaves 2-3 years off their clinical timeline, positioning them for possible BLA submission by 2027.
The early clinical data is encouraging: 52% slower lesion growth in treated eyes and a statistically significant 10-letter improvement in visual acuity (p=0.02). These results suggest potential disease-modifying effects rather than just symptom management, which could command premium pricing in the orphan drug market.
With 100,000 Stargardt patients in the US and Europe and no approved treatments, OCU410ST addresses a clear unmet need. Orphan designations from both FDA and EMA provide significant benefits including 7 years of US market exclusivity, tax credits, and reduced regulatory fees.
For context, orphan gene therapies typically command prices between
The accelerated timeline will require careful cash management. The streamlined 51-patient Phase 2/3 trial design is capital-efficient compared to traditional larger studies, but Ocugen will need to simultaneously prepare for potential commercialization while completing clinical development.
Key risks remain: the need to replicate early results in the larger confirmatory trial, manufacturing scale-up challenges typical of gene therapies, and potential competition from other emerging treatments for retinal diseases.
Ocugen's FDA alignment for OCU410ST represents a potential breakthrough for Stargardt disease, an inherited macular degeneration that typically begins in childhood or adolescence, progressively robbing patients of central vision during their most productive years.
The modifier gene therapy approach employed by OCU410ST is scientifically elegant. Rather than attempting to replace the mutated ABCA4 gene (which at 6.8 kilobases exceeds AAV vector capacity), OCU410ST delivers the NREP gene. This acts as a molecular bypass, reducing toxic vitamin A byproduct accumulation that damages photoreceptors regardless of which specific ABCA4 mutation a patient carries.
The 10-letter visual acuity improvement observed in the Phase 1 trial translates to meaningful real-world benefits - potentially enabling patients to read standard print, recognize faces, or maintain driving privileges. For progressive conditions like Stargardt, even stabilization would be considered successful, making improvement particularly noteworthy.
The trial design warrants attention: the 51-patient Phase 2/3 study is relatively small for a pivotal trial but appropriate for a rare disease with objective anatomical endpoints. The primary endpoint of atrophic lesion size can be precisely measured using spectral domain optical coherence tomography, reducing variability and increasing statistical power.
The subretinal delivery approach, while surgically complex, ensures precise targeting of the therapeutic vector to photoreceptors and retinal pigment epithelium. This likely contributes to the favorable safety profile observed to date, avoiding the inflammation seen with intravitreal delivery of some gene therapies.
For retina specialists and patients who have had no treatment options beyond vitamin A avoidance and sun protection, OCU410ST represents the most advanced therapeutic candidate for Stargardt disease, potentially offering a one-time treatment that could alter the natural history of this blinding condition.
MALVERN, Pa., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that alignment has been reached with the U.S. Food and Drug Administration (FDA) to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a biologics license application (BLA) submission.
The GARDian trial for OCU410ST demonstrated:
- A favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest
- Considerably slower lesion growth (
52% ) from baseline in treated eyes versus untreated fellow eyes at 6-month follow-up from the Phase 1 study
- Clinically meaningful 2-line (10-letter) improvement in visual function (BCVA) at 6-month follow-up from the Phase 1 study, which is statistically significant (p=0.02) in treated eyes
“I am very pleased that the FDA has recognized the promise of Ocugen’s modifier gene therapy for Stargardt disease and accelerated the regulatory pathway for OCU410ST,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This new development allows us to initiate a pivotal confirmatory trial for this game-changing, one-time treatment for life in the next few months and prepare for a potential BLA filing by 2027. Now patients suffering from Stargardt disease have a new hope where previously none existed. This achievement furthers our mission to cure blindness diseases.”
Stargardt disease affects 100,000 people in the U.S. and Europe combined, and there is no treatment. OCU410ST received orphan drug designations from the FDA and the European Medicines Agency (EMA) in 2023 and 2024, respectively.
“Getting approval for a Phase 2/3 trial is a pivotal milestone, as this approach has never been explored in clinical trials for Stargardt disease. The FDA’s decision underscores the potential of OCU410ST to meet a critical unmet medical need for the approximately 44,000 Stargardt patients in the U.S.,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Professor of Ophthalmology, Pediatrics and Biomedical Engineering with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen.
The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) compared to untreated controls. One-year data will be utilized for the BLA filing.
“This approval pathway, established in collaboration with the FDA, has made it possible to expedite the clinical development of OCU410ST by two to three years and has aided in bringing an innovative gene therapy to patients desperate for a treatment option,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “Recent data from the OCU410ST clinical trial have shown significant improvements in both structural and functional outcomes. Additionally, OCU410ST has consistently demonstrated a very favorable safety and tolerability profile.”
Accelerating the clinical timeline of OCU410ST will save significant costs in addressing disease burden even sooner than anticipated.
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 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, 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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