OCU410 / Ocugen - LARVOL DELTA

Ocugen…announced that the first patient was dosed in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA), its first-in-class modifier gene therapy candidate for GA secondary to dry age-related macular degeneration (dAMD) (GlobeNewswire) - "The Phase 3 trial is a global, multicenter, randomized, controlled study enrolling 237 subjects with GA secondary to dAMD, randomized 2:1 to a single 200 µL subretinal injection of OCU410 (5×1010 vg/mL) or an untreated control arm, with sites in the United States, Canada, Europe, and Latin America....A single, adequate and well-controlled Phase 3 trial, aligned with FDA feedback, is intended to support a BLA filing anticipated in 2028."

FDA filing • Trial status • Dry Age-related Macular Degeneration

OCU410-301: Phase 3 Study to Assess Efficacy, Safety & Tolerability of OCU410 For Geographic Atrophy Secondary to Dry AMD (clinicaltrials.gov) - P3 | N=237 | Recruiting | Sponsor: Ocugen

New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Ocugen Provides Business Update with Second Quarter 2026 Financial Results (The Manila Times) - "Received U.S. Food and Drug Administration (FDA) clearance to initiate OCU410 Phase 3 trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (AMD); on track to initiate Phase 3 this quarter....Phase 2/3 GARDian3 trial evaluating OCU410ST in patients with all mutations of Stargardt disease...Topline results anticipated in the second quarter of 2027, with a BLA submission to follow mid-2027."

FDA filing • New P3 trial • P2/3 data • Dry Age-related Macular Degeneration • Ophthalmology

ArMaDa: Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (clinicaltrials.gov) - P1/2 | N=60 | Active, not recruiting | Sponsor: Ocugen | Trial completion date: May 2026 ➔ Feb 2030

Trial completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Ocugen Announces Topline 12-month Data from Phase 2 ArMaDa Clinical Trial Evaluating OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (Ocugen Press Release) - "31% reduction in lesion growth in the optimal dose (medium) group compared to control (p< 0.05); 27% slower rate of ellipsoid zone (EZ) loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function; 55% of treated patients demonstrated ≥30% lesion size reduction vs. control; Subgroup analysis (subjects with baseline GA lesions ≥5 mm2 and ≤17.5 mm2) showed 33% reduction in lesion growth compared to control in medium dose OCU410 with similar reductions in the high dose group...Ocugen plans to initiate the OCU410 Phase 3 registrational trial in the third quarter of 2026 in line with the Company’s goal of three BLA filings in three years."

FDA filing • New P3 trial • P2 data • Dry Age-related Macular Degeneration

Ocugen Announces that the U.S. Food and Drug Administration Has Granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Geographic Atrophy, Secondary to Dry Age-Related Macular Degeneration (The Manila Times) - "The RMAT designation for OCU410 was supported by Phase 2 clinical data demonstrating clinically meaningful efficacy and a favorable safety profile, with no reported serious adverse events related to drug...In early July 2026, Ocugen reached alignment with the FDA on the design of the OCU410 Phase 3 registrational trial, with study initiation expected in the third quarter of 2026, and a Biologics License Application (BLA) filing anticipated in 2028."

FDA event • FDA filing • New P3 trial • Dry Age-related Macular Degeneration

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

ArMaDa: Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (clinicaltrials.gov) - P1/2 | N=60 | Active, not recruiting | Sponsor: Ocugen | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2025 ➔ May 2026 | Trial primary completion date: Sep 2025 ➔ Feb 2026

Enrollment closed • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

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

Title: Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) 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 • Dry Age-related Macular Degeneration • 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 Dosing Completion in the Phase 2 ArMaDa Clinical Trial for OCU410—a Multifunctional Modifier Gene Therapy for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (GlobeNewswire) - "Ocugen, Inc...today announced that dosing is complete, ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410—a novel multifunctional modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD)...'The data from this trial will help us design a future pivotal Phase 3 study planned for 2026 and enable our commercial strategy for Biologics License Application (BLA) and Marketing Authorization Application (MAA) filings as soon as 2028.'"

EMA filing • FDA filing • New P3 trial • Trial status • Age-related Macular Degeneration • Ophthalmology

Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (GlobeNewswire) - P1/2 | N=63 | ArMaDa (NCT06018558) | Sponsor: Ocugen | "Ocugen, Inc...announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA)....Considerably slower lesion growth (21.4%) from baseline in treated vs. untreated fellow eyes that followed the natural history of the disease. This result is favorable when compared to published data on pegcetacoplan injected every month or every other month over six months."

P1/2 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

Gene Therapy for Non-Hereditary Retinal Disease: Age-Related Macular Degeneration, Diabetic Retinopathy, and Beyond. (PubMed, Genes (Basel)) - "This manuscript reviews ongoing gene therapy clinical trials for these disorders, including ABBV-RGX-314, ixoberogene soroparvovec (ixo-vec), and 4D-150. ABBV-RGX-314 utilizes an adeno-associated virus (AAV) vector to deliver a transgene encoding a ranibizumab-like anti-VEGF antibody fragment, demonstrating promising results in Phase 1/2a and ongoing Phase 2b/3 trials...Other therapies reviewed include EXG102-031, FT-003, KH631, OLX10212, JNJ-1887, 4D-175, and OCU410. These therapies offer potential advantages of reduced treatment frequency and enhanced safety profiles, representing a paradigm shift in management towards durable and efficacious cellular-based biofactories. These advancements in gene therapy hold promise for improving outcomes in AMD and addressing the complex challenges of DME and DR, providing new avenues for the treatment of diabetic eye diseases."

Gene therapy • Journal • Review • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration • VEGFC

Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD. (PubMed, Gene Ther) - "AAV5-hRORA-treated mice showed reduced deposits, restored CD59 expression and attenuated amyloid precursor protein (APP) expression compared with untreated eyes...This is the first study evaluating the impact of RORA modifier gene therapy on rescuing retinal degeneration. Our studies demonstrate efficacy of RORA in improving STGD and dry AMD-like disease."

Journal • Preclinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders • APP • CD59

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

ArMaDa: Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (clinicaltrials.gov) - P1/2 | N=63 | Recruiting | Sponsor: Ocugen | Trial completion date: May 2026 ➔ Sep 2025 | Trial primary completion date: Mar 2026 ➔ Sep 2025

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (clinicaltrials.gov) - P1/2 | N=63 | Recruiting | Sponsor: Ocugen

New P1/2 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders