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MALVERN, Pa., Aug. 13, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application (MAA).
EMA provided this opinion based on safety and tolerability that OCU410ST demonstrated in the Phase 1 GARDian trial, including 48% slower lesion growth and statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up in evaluable treated eyes compared to untreated eyes. 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 unique adaptive design of this trial includes a masked interim analysis of 24 subjects in the study (16 in treatment group and 8 in control group) at 8 months. 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) and MAA in the EU.
“This positive opinion endorses a single trial as the basis for both BLA and MAA submissions and brings us closer to providing a one-time, modifier gene therapy to approximately 100,000 Stargardt patients in the U.S. and Europe combined,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “We are very encouraged about the prospect of addressing the unmet medical need that exists for these patients who currently have no approved treatment options available to them.”The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. Alignment with the EMA follows recent important milestones for the OCU410ST program, including Rare Pediatric Disease Designation (RPDD) in May, IND clearance in June, and first patient dosing in July. With enrollment scheduled to be complete in the first quarter of 2026 the Company remains on track for a BLA filing in the first half of 2027, aligned with its goal of three BLAs in the next three years.About OCU410STOCU410ST utilizes an AAV delivery platform for the retinal delivery of theRORA(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 DiseaseStargardt 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 atwww.ocugen.comand follow us onXandLinkedIn.
Cautionary Note on Forward-Looking StatementsThispressreleasecontainsforward-lookingstatementswithinthemeaningofThePrivateSecuritiesLitigationReformActof1995,including,butnot limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines,whicharesubjecttorisksanduncertainties.Wemay,insomecases,usetermssuchas“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,butnotlimitedto,therisksthatpreliminary,interimandtop-lineclinicaltrialresultsmaynotbeindicativeof,andmaydifferfrom,finalclinical data;the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data;thatunfavorablenewclinicaltrialdatamayemergeinongoingclinicaltrialsorthroughfurtheranalysesofexistingclinicaltrialdata;thatearlier non-clinicalandclinicaldataandtestingofmaynotbepredictiveoftheresultsorsuccessoflaterclinicaltrials;andthatthatclinicaltrialdataare subject to differing interpretations and assessments, including by regulatory authorities.Theseandotherrisksanduncertaintiesaremorefully describedinourperiodicfilingswiththeSecuritiesandExchangeCommission(SEC), includingtheriskfactorsdescribedinthesectionentitled“Risk Factors”inthequarterlyandannualreportsthatwefilewiththeSEC.Anyforward-lookingstatementsthatwemakeinthispressreleasespeakonlyas ofthedateofthispressrelease.Exceptasrequiredbylaw,weassumenoobligationtoupdateforward-lookingstatementscontainedinthispress release whether as a result of new information, future events, or otherwise, after the date of this press release.