Ocugen Gets DSMB Approval to Enroll High Dose Cohort in Stargardt Study

Ocugen, Inc., a biotechnology firm dedicated to innovative gene and cell therapies, has announced a significant development in their ongoing OCU410ST GARDian clinical trial. The Data and Safety Monitoring Board (DSMB) has given the green light to proceed to the high dose phase of the dose-escalation study. OCU410ST, a gene therapy candidate targeting Stargardt disease, shows promise in addressing this hereditary eye disorder that affects around 100,000 individuals in the U.S. and Europe.

Up to this point, six patients with Stargardt disease have received treatment in the initial low and medium dose phases of the Phase 1/2 clinical trial. The next step involves administering the high dose to three additional patients. Dr. Peter Y. Chang, DSMB Chair for the clinical trial, noted that no serious adverse events related to OCU410ST have been observed so far. He emphasized that this milestone is crucial for the progression of the therapy and brings hope to those suffering from this common inherited retinal disease.

Dr. Huma Qamar, Chief Medical Officer of Ocugen, expressed enthusiasm about the DSMB's positive recommendation and highlighted the urgent need for effective treatments for Stargardt disease, given the lack of approved therapies. Dr. Qamar emphasized the potential of OCU410ST as a one-time treatment through a single subretinal injection and mentioned plans to provide further updates on the clinical trial later in the year.

The Phase 1/2 GARDian trial aims to enroll up to 42 subjects, comprising both adults and children with mild to moderate symptoms of Stargardt disease. The study is split into two phases: Phase 1 involves a multicenter, open-label, dose-ranging study with three different dose levels (low, medium, and high) of OCU410ST, while Phase 2 is a randomized, outcome assessor-blinded, dose-expansion study. In Phase 2, participants will be divided into two treatment groups receiving different doses or a control group, facilitating a thorough evaluation of the therapy’s efficacy across varying dosages.

Currently, there are no FDA-approved treatments for Stargardt disease. Ocugen is committed to developing a gene-agnostic approach to treat inherited retinal diseases and is optimistic about the ongoing progress of the OCU410ST trial.

Stargardt disease is a genetic condition leading to retinal degeneration and vision loss, often developing during childhood or adolescence. This disorder is the most common form of inherited macular degeneration. It primarily affects the photoreceptor cells in the macula, a part of the retina responsible for sharp, central vision, while peripheral vision usually remains intact. The retinal pigment epithelium (RPE), a support layer for photoreceptors, is also impacted in those with Stargardt disease.

OCU410ST employs an AAV delivery system for the RORA gene, targeting multiple pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, and inflammation. This approach is part of Ocugen’s broader strategy to utilize modifier gene therapy to treat retinal diseases.

Ocugen, Inc. is at the forefront of developing groundbreaking therapies to enhance patient health and address unmet medical needs. Their efforts span across various fields, including gene and cell therapies for retinal and infectious diseases, as well as orthopedic conditions. The company's innovative approach aims to deliver transformative treatments, harnessing unique intellectual and human capital to make a significant impact on global health.