OCU410ST / Ocugen - LARVOL DELTA

Musunuri noted positive 12-month Phase 1 data for OCU410ST, with a "48.2% reduction in lesion growth and a meaningful online 6-letter gain in visual acuity in evaluable treated eyes compared with untreated eyes." (MSN News) - "He described the OCU410 program's interim results showing a 27% reduction in lesion growth at six months and indicated plans to initiate Phase 3 next year."

New P3 trial • P1 data • Ophthalmology

OCU410ST Phase 2/3 pivotal confirmatory trial is on track to complete enrollment in Q1 2026, targeting a biological licensing application (BLA) submission in the first half of 2027. (MSN News)

Enrollment status • FDA filing • Ophthalmology

Guardian: A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease (clinicaltrials.gov) - P2/3 | N=51 | Recruiting | Sponsor: Ocugen | Phase classification: P1/2 ➔ P2/3 | Trial completion date: Oct 2025 ➔ Sep 2026 | Trial primary completion date: Oct 2025 ➔ Sep 2026

Phase classification • Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU410ST—Modifier Gene Therapy for Stargardt Disease (GlobeNewswire) - "EMA provided this opinion based on safety and tolerability that OCU410ST demonstrated in the Phase 1 GARDian trial..."

CHMP • Ophthalmology

Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease (GlobeNewswire) - "Ocugen, Inc...announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies)...The Phase 2/3 clinical trial for OCU410ST 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."

IND • New P2/3 trial • Ophthalmology • Retinal Disorders

Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST—Modifier Gene Therapy for the Treatment of Stargardt Disease (GlobeNewswire) - "Ocugen, Inc...announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy...With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV), if the PRV program is reauthorized by the U.S. Congress....Ocugen is committed to advancing the OCU410ST program through clinical development and plans to initiate the Phase 2/3 pivotal confirmatory trial in the next few weeks with a target Biologics License Application (BLA) filing in 2027."

FDA event • FDA filing • New P2/3 trial • Ophthalmology

Preclinical Safety Evaluation of AAV5-hRORA (OCU410-ST) a Gene Therapy Candidate for Stargardt's disease and other ABCA4 related retinopathies (ASGCT 2025) - "Our results demonstrate that OCU410-ST is safe and tolerable at tested doses. Based on these results, 3.50 ×1011 Vg/eye was identified as the maximum tolerable dose for further planned clinical studies. Disease Focus of Abstract:Eye Disorders"

Gene therapy • Preclinical • Developmental Disorders • Gene Therapies • Hematological Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Ocugen Provides Business Update with First Quarter 2025 Financial Results (GlobeNewswire) - "OCU400 for RP – The EMA provided a positive opinion for ATMP classification for OCU400 and granted eligibility to submit the OCU400 MAA via the centralized procedure as an ATMP based on the current study design and statistical analysis plan. Actively recruiting patients in the U.S. and Canada in the Phase 3 liMeliGhT clinical trial and on track to meet BLA/MAA filing targets in mid-2026; OCU410ST for Stargardt Disease – Received FDA alignment to move forward with Phase 2/3 pivotal confirmatory clinical trial, which can be the basis of a BLA submission. ATMP classification granted by the EMA; OCU410 for GA – Announced that dosing was complete, ahead of schedule, in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410. Received ATMP classification from the EMA."

EMA filing • FDA filing • New P2/3 trial • Trial status • Genetic Disorders • Ophthalmology • Retinitis Pigmentosa

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress (GlobeNewswire) - "Ocugen, Inc...today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025."

Clinical data • Retinitis Pigmentosa

Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update (ARVO 2025) - P1/2 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P1/2 data • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

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 (GlobeNewswire) - "Ocugen, Inc...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...'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...The Company plans to initiate the Phase 2/3 pivotal confirmatory clinical trial for OCU410ST by mid-2025."

EMA filing • European regulatory • New P2/3 trial • New P3 trial • Genetic Disorders • Ophthalmology • Rare Diseases

Ocugen, Inc. Announces FDA Alignment on Phase 2/3 Pivotal Confirmatory Clinical Trial for Modifier Gene Therapy Candidate OCU410ST for Stargardt Disease (GlobeNewswire) - P1/2 | N=42 | NCT05956626 | Sponsor: Ocugen | "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...'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.'"

FDA filing • P1/2 data • Ophthalmology

OCU410ST (AAV5-hRORA) Modifier Gene Therapy Rescues Retinal Degeneration in a Mouse Model of Stargardt Disease (ASGCT 2024) - P1/2 | "AAV5-hRORA-treated retinas demonstrate improvement in multiple clinical, functional, and molecular markers in Abca4-/- mice. This is the first study evaluating the impact of AAV5-hRORA in rescuing retinal degeneration associated with Stargardt disease. Ocugen, Inc."

Gene therapy • Preclinical • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Ophthalmology • Rare Diseases • Retinal Disorders • APP • CD59

Guardian: Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease (clinicaltrials.gov) - P1/2 | N=42 | Recruiting | Sponsor: Ocugen | Not yet recruiting ➔ Recruiting

Enrollment open • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Guardian: Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease (clinicaltrials.gov) - P1/2 | N=42 | Not yet recruiting | Sponsor: Ocugen

New P1/2 trial • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders