Ocugen, Inc. completes dosing in Phase 2/3 GARDian3 trial of OCU410ST for Stargardt disease, with interim data expected in Q3 2026 and BLA planned for mid-2027.
Written by: Dillikumari A, BPharm
Reviewed by: Pharmacally Editorial Team
Ocugen, Inc. has completed dosing ahead of schedule in its pivotal Phase 2/3 GARDian3 trial evaluating OCU410ST for patients with Stargardt disease.
Dr. Huma Qamar, Chief Medical Officer of Ocugen, stated that the trial enrolled a broad population from pediatric to adult patients across early and advanced disease stages, addressing a significant unmet need in Stargardt disease.
GARDian3 (NCT05956626) is a multicenter, randomized, masked, pivotal confirmatory study that enrolled 63 participants. Patients in the treatment arm received a one-time subretinal injection of OCU410ST (3 × 10¹⁰ vector genomes per eye) in the worse-seeing eye, while the control group remained untreated. The primary endpoint is reduction in atrophic lesion size at 12 months. Key secondary endpoints include best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA), with ellipsoid zone (EZ) preservation as an observational endpoint.
OCU410ST uses an adeno-associated virus serotype 5 (AAV5) vector to deliver the RAR-related orphan receptor A (RORA) gene, targeting disease pathways including lipofuscin accumulation, oxidative stress, inflammation, complement activation, and photoreceptor degeneration, independent of the underlying ABCA4 gene mutation.
An interim analysis is planned in the third quarter of 2026 after 24 patients complete eight months of follow-up. One-year data will support a planned Biologics License Application (BLA) submission in mid-2027.
OCU410ST has shown a favorable safety and tolerability profile, with no serious adverse events or adverse events of special interest reported.
In the Phase 1 GARDian1 trial, treated eyes showed a 54% reduction in atrophic lesion growth versus untreated fellow eyes at 12 months. Treated eyes gained an average of 6 letters in BCVA, while untreated eyes declined by 1.5 letters. The ellipsoid zone (EZ) loss rate was 116% slower in treated eyes, with all treated eyes stabilizing or improving in visual acuity. No drug-related serious adverse events were observed. Results from the Phase 1 GARDian1 trial of OCU410ST have been published in the journal Eye.
Stargardt disease is a genetic retinal disorder caused by mutations in the ABCA4 gene, leading to progressive central vision loss, typically beginning in childhood or adolescence, with no approved treatments available.
Reference
Ocugen Announces Early Completion of Dosing in Phase 2/3 Pivotal Confirmatory Trial of OCU410ST for Stargardt Disease , 01.04.2026 , Ocugen Announces Early Completion of Dosing in Phase 2/3 Pivotal Confirmatory Trial of OCU410ST for Stargardt Disease
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease (GARDian3) , ClinicalTrials.gov ID NCT05956626, https://clinicaltrials.gov/study/NCT05956626
About the Writer
Dilli Kumari is a Pharmacy graduate based in Ariyur, Puducherry. She has a strong interest in pharmacovigilance and is particularly drawn to data analysis within the healthcare field. She is known for her willingness to learn and continuously improve, with a keen attention to detail that sometimes leads her to spend extra time refining her work.