Ocugen announces dosing completion in second cohort of GARDian clinical trial for Stargardt disease

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Ocugen has announced dosing has been completed in the second cohort of its Phase 1/2 GARDian clinical trial (NCT05956626) evaluating OCU410ST (AAV-hRORA) for Stargardt disease.

OCU410ST, is a modifier gene therapy candidate that utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and is being developed as a one-time treatment for life.¹

"The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” said Huma Qamar, MD, MPH, chief medical officer of Ocugen in a press release from the company.¹ “We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from Phase 1 of the clinical trial.”

To date, 6 patients have been dosed with OCU410ST in the Phase1/2 clinical trial with the low dose (3.75×1010 vg/mL), and medium dose (7.5×1010 vg/mL). An additional 3 patients will be dosed with a high dose (2.25×10E11 vg/mL) in the dose-escalation phase.²

The company stated in a press release that a Data and Safety Monitoring Board meeting will convene in June to review the 4-week safety data of the medium dose cohort before proceeding with Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study.

The GARDian clinical trial is a multicenter study, conducted in 2 phases and plans to enroll up to a total of 42 subjects. Phase 1 is a multicenter, open-label, dose-ranging/dose escalation study with a 3+3 design enrolling up to 18 subjects. While Phase 2 is a randomized, dose-expansion cohort in which 24 subjects will be randomized in a 1:1:1 ratio in to either one of two treatment groups or to an untreated control group.²

According to Ocugen, clinical trial updates for OCU410ST are expected to be provided in the third quarter of 2024.

References:

1. Ocugen Announces Dosing Completion of Subjects with Stargardt Disease in Cohort 2 of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy. Press release; May 15, 2024. Accessed May 15, 2024. https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-dosing-completion-subjects-stargardt-disease

2. Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease (GARDian). NCT05956626. https://clinicaltrials.gov/study/NCT05956626