Ocugen Completes Dosing for Stargardt Disease in Phase 1/2 GARDian Trial of OCU410ST

May 15, 2024 – Ocugen, Inc. (NASDAQ: OCGN), a biotechnology firm dedicated to the development of innovative gene and cell therapies and vaccines, has announced the completion of dosing in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA). This one-time treatment, targeting Stargardt disease, marks a significant step forward in the company's research efforts.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, highlighted the importance of this achievement, stating, "The completion of dosing for Cohort 2 participants signifies a vital clinical milestone for our pioneering modifier gene therapy. We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, which enables 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, six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial. An additional three patients will receive the high dose (Cohort 3) of OCU410ST in the ongoing dose-escalation phase.

Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix, commented on the significance of this progress. He stated, “There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting central vision and currently lacks any FDA-approved treatment options. OCU410ST offers hope to these patients. I am excited that we completed dosing of the last patient in Cohort 2 at Associated Retina Consultants in Phoenix, AZ, with the surgical team led by Dr. Mark Kwong, Medical Director of ARC."

A Data and Safety Monitoring Board meeting is scheduled next month to review the 4-week safety data of the medium dose cohort before moving forward with Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study.

The GARDian clinical trial aims to evaluate the safety and effectiveness of unilateral subretinal administration of OCU410ST in patients with Stargardt disease. The trial is divided into two phases. Phase 1 is a multicenter, open-label, dose-ranging study involving three dose levels: low, medium, and high. Phase 2 will be a randomized, outcome assessor-blinded, dose-expansion study where adult and pediatric subjects will be randomized in a 1:1:1 ratio to one of two OCU410ST dose groups or an untreated group.

Ocugen remains dedicated to finding solutions for individuals with inherited retinal diseases who currently have no effective treatment options. Stargardt disease, although classified as an orphan disease, affects approximately 100,000 people across the United States and Europe combined.

The company plans to provide a clinical trial update for OCU410ST in the third quarter of 2024.

Stargardt disease is a genetic eye condition that causes degeneration of the retina and loss of vision. It is the most common form of inherited macular degeneration, leading to progressive vision loss due to the degeneration of photoreceptor cells in the central part of the retina known as the macula. This condition typically begins in childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), which supports photoreceptors, is also affected in those with Stargardt disease.

OCU410ST employs an AAV delivery platform to deliver the RORA gene to the retina. This modifier gene therapy approach, based on the Nuclear Hormone Receptor RORA, addresses pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, inflammation, and cell survival networks.

Ocugen, Inc. is focused on discovering, developing, and commercializing innovative gene and cell therapies and vaccines. The company aims to make a significant impact on patient lives through bold innovation and scientific exploration. Their modifier gene therapy platform holds potential for treating multiple retinal diseases with a single product. Additionally, Ocugen is advancing research in infectious diseases to support public health and orthopedic diseases to meet unmet medical needs.

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