Ocugen announces Data and Safety Monitoring Board approves enrollment in high dose cohort 3 in GARDian Study for Stargardt disease


OCU410ST is a modifier gene therapy candidate being developed for Stargardt disease, which affects approximately 100,000 people in the United States and Europe combined.

(Image Credit: AdobeStock/Jouni) man in a board room with a thumbs up

(Image Credit: AdobeStock/Jouni)

Ocugen Inc announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved to proceed with dosing the high dose of OCU410ST in the dose-escalation phase of the study.

OCU410ST (AAV5-hRORA) is a modifier gene therapy candidate being developed for Stargardt disease, which affects approximately 100,000 people in the US and Europe combined. It is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration.1

The company’s candidate, OCU410ST, utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. This is a part of Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

There currently are no FDA-approved therapeutic options for patients diagnosed with Stargardt disease.

According to the company’s news release, 6 patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date in the low dose cohort and medium dose cohort. An additional 3 patients will be dosed with the high dose in cohort 3.

Peter Y. Chang, MD, FACS, DSMB chairman for the OCU410ST clinical trial, noted the DSMB has recommended moving forward to dose subsequent subjects with Stargardt disease at the targeted high dose.

“No serious adverse events (SAEs) related to OCU410ST have been reported to date,” Chang said. “This is an important next step in the clinical progress for OCU410ST and encouraging for patients living with this most common form of inherited retinal disease.”

Huma Qamar, MD, MPH, chief medical officer of Ocugen, said the company reported a second positive DSMB recommendation for the treatment of Stargardt disease and build upon the favorable safety and tolerability profile exhibited by OCU410ST.

“We recognize the high unmet medical need for Stargardt patients as there is no approved product,” Qamar said. “We are enthusiastic about OCU410ST as a potential one-time treatment for life with a single sub-retinal injection. We look forward to sharing a clinical trial update later this year."

According to the news release, the Phase 1/2 GARDian clinical trial will include up to 42 subjects—30 adults and 12 children with Stargardt disease—who exhibit mild to moderate disease symptoms and will assess the safety of unilateral subretinal administration of OCU410ST.

The company noted its clinical trial is being conducted in two phases. The first is a multicenter, open-label, dose-ranging/dose-escalation study consisting of t3 dose levels [low dose (3.75× 1010 vg/mL), medium dose (7.5× 1010 vg/mL), and high dose (2.25× 1011 vg/mL)].1

The second, Phase 2, is a randomly assigned, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be enrolled in a 1:1:1 ratio to randomize subjects into two different treatment groups at varying dose levels, or a control (untreated group), allowing for an assessment of the treatment's efficacy across different dosages.1

  1. Ocugen. Ocugen Announces Data and Safety Monitoring Board Approves Enrollment in High Dose Cohort 3 in GARDian Study for Stargardt Disease. GlobeNewswire News Room. Published June 21, 2024. Accessed June 21, 2024. https://www.globenewswire.com/news-release/2024/06/21/2902220/0/en/Ocugen-Announces-Data-and-Safety-Monitoring-Board-Approves-Enrollment-in-High-Dose-Cohort-3-in-GARDian-Study-for-Stargardt-Disease.html
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