Ocugen Announces Positive Opinion of EMA’s Committee for Advanced Therapies for ATMP Classification for Novel Modifier Gene Therapy Candidate OCU410 for Geographic Atrophy and OCU410ST for Stargardt Disease
Rhea-AI Summary
Ocugen (NASDAQ: OCGN) has received positive ATMP classification from EMA's Committee for Advanced Therapies for two gene therapy candidates: OCU410 for Geographic Atrophy (GA) and OCU410ST for Stargardt disease.
Key highlights:
- OCU410 Phase 2 dosing is complete, with Phase 3 trials planned for next year, targeting MAA/BLA filings in 2028
- FDA endorsed OCU410ST Phase 2/3 pivotal trial, with potential submissions in 2027
- Preliminary 9-month OCU410 data showed 44% slower lesion growth and 2-line improvement in visual function
- OCU410ST 6-month data demonstrated 52% slower lesion growth and statistically significant visual improvement
Both therapies utilize AAV platform delivering RORA gene through a single subretinal injection. GA affects 2-3 million people in US/Europe, while Stargardt disease impacts 100,000 people, with no approved treatments in Europe.
Positive
- 44% slower lesion growth in OCU410 Phase 1 trial
- 52% slower lesion growth in OCU410ST with statistical significance (p=0.02)
- Single-dose treatment vs current monthly/bi-monthly alternatives
- ATMP classification granted, accelerating regulatory review
- Clear development timeline with BLA/MAA submissions in 2027-2028
Negative
- Several years away from potential market approval (2027-2028)
- Competing against two approved therapies in US for GA treatment
- Early-stage clinical data (Phase 1) still needs confirmation in larger trials
Insights
The positive ATMP classification from the European Commission represents a significant regulatory milestone for Ocugen's gene therapy candidates. This designation accelerates review timelines and permits more frequent EMA interactions, creating a clearer path to market for these potential one-time treatments.
The preliminary efficacy data is compelling:
This pipeline addresses substantial unmet needs. Geographic Atrophy affects 2-3 million people across the US and Europe, with current US-approved treatments requiring monthly/bi-monthly injections and none available in Europe. Stargardt disease affects 100,000 patients with no approved treatments worldwide.
Ocugen's development timeline appears aggressive yet achievable: OCU410ST regulatory submissions targeted for 2027 and OCU410 for 2028. The FDA endorsement for OCU410ST's Phase 2/3 pivotal trial design and completion of OCU410's Phase 2 dosing indicate steady progression.
The RORA gene therapy mechanism targeting all four disease pathways differentiates these candidates from competitors by potentially addressing multiple aspects of disease pathology through a single administration, versus the chronic treatment approach of current GA therapies.
This regulatory advancement significantly strengthens Ocugen's market position in retinal gene therapies. The ATMP classification streamlines European regulatory interactions and potentially shortens approval timelines for both candidates—critical advantages in the competitive ophthalmology space.
Ocugen is pursuing optimal strategic timing between its two lead candidates. OCU410ST (Stargardt) could reach filing by 2027, with OCU410 (Geographic Atrophy) following in 2028. This staggered approach could help manage development resources while building sequential revenue opportunities.
The preliminary efficacy data merits attention: both candidates demonstrating 2-line vision improvements represents functional benefits patients can actually experience in daily activities, not just statistical improvements on retinal imaging. The
The market opportunity is substantial. With no approved treatments for Stargardt disease and options for Geographic Atrophy in Europe, Ocugen is positioned to address significant unmet needs. The one-time treatment approach offers a compelling alternative to current GA therapies requiring 6-13 injections annually.
Investors should monitor upcoming catalysts: initiation of OCU410ST's Phase 2/3 trial by mid-2025, further OCU410 data readouts, and Phase 3 initiation next year. The company's ability to execute these parallel development programs while maintaining adequate funding will be critical success factors moving forward.
MALVERN, Pa., March 03, 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 European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification. OCU410 is a novel, multifunctional modifier gene therapy candidate being developed for the treatment of patients with vision loss due to geographic atrophy (GA)—an advanced stage of dry age-related macular degeneration (dAMD)—and OCU410ST is being developed for Stargardt disease due to ABCA4-related retinopathies.
GA affects 2-3 million people in the United States (U.S.) and Europe combined. There are two approved therapies in the U.S. that require frequent dosing (every month or every other month), however neither therapy has been approved in Europe. Stargardt disease affects 100,000 people in the U.S. and Europe combined, and there are no approved treatments available globally.
“Receiving ATMP classification for OCU410 and OCU410ST is a critical step to potentially address these severely unmet medical needs in the very near future,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Dosing of Phase 2 in the ongoing OCU410 ArMaDa clinical trial is complete, and we are on track to initiate the Phase 3 clinical trial next year to pursue potential Marketing Authorization Application (MAA) and Biologics License Application (BLA) filings in 2028. Last week, the U.S. Food and Drug Administration (FDA) endorsed Ocugen’s plan to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, which can be the basis of BLA and potential MAA submissions in 2027.”
ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life. Additionally, this classification allows Ocugen to interact with EMA more frequently for scientific advice and protocol assistance.
Preliminary 9-month data of OCU410 in GA patients demonstrated considerably slower lesion growth (
6-month data from Phase 1 of the OCU410ST GARDian clinical trial demonstrated considerably slower lesion growth (
“The novel modifier gene in OCU410 and OCU410ST targets all four pathways linked with dAMD and Stargardt and is delivered through a single subretinal injection as a one-and-done treatment,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “We are very pleased with the structural and functional outcomes demonstrated by both of these candidates, along with a stellar safety profile.”
OCU410 and OCU410ST utilize 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.
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 OCU410 and 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
What are the clinical results of Ocugen's OCU410 for Geographic Atrophy?
When will Ocugen (OCGN) submit OCU410 for regulatory approval?
How does OCU410ST perform in treating Stargardt disease?
What is the market size for Ocugen's OCU410 and OCU410ST treatments?
How is Ocugen's OCU410 administered compared to current GA treatments?
