MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) this week announced a significant advancement in its ongoing clinical trial for Stargardt disease. The Data Safety and Monitoring Board (DSMB) for the OCU410ST clinical trial has recently given the green light to proceed with dosing patients at a medium dose level in the trial’s dose-escalation phase, marking a pivotal step in the development of this potential treatment.
Stargardt disease, a genetic eye disorder that leads to progressive vision loss, affects approximately 40,000 people in the United States. Currently, there are no FDA-approved therapies for Stargardt disease, leaving a considerable gap in treatment options for those affected. Ocugen’s OCU410ST represents a beacon of hope for patients with this inherited retinal disease.
The DSMB’s recommendation to move forward with medium dose dosing comes after three patients were already dosed at the initial level without reporting any serious adverse events (SAEs) related to OCU410ST. “No serious adverse events related to OCU410ST have been reported to date,” confirmed Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial. “I believe that this marks a critical next step towards determining the optimal dosing regimen for OCU410ST.”
The GARDian clinical trial, designed to assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease, is divided into two phases. The first phase is an open-label, dose-ranging study consisting of three dose levels, with the upcoming cohorts set to receive medium and high doses of OCU410ST. The second phase will be a randomized, outcome accessor-blinded, dose-expansion study involving both adult and pediatric subjects.
Huma Qamar, M.D., MPH, Chief Medical Officer of Ocugen, expressed optimism about the trial’s progress: “We are pleased to see the continued favorable safety and tolerability profile exhibited by OCU410ST, allowing us to evaluate a higher dose in patients with Stargardt retinal dystrophy.”
This advancement is particularly noteworthy as it underscores Ocugen’s commitment to addressing the needs of patients with inherited retinal diseases—a commitment that could potentially lead to the first FDA-approved therapy for Stargardt disease. As the trial progresses, the biopharmaceutical company anticipates sharing interim safety data from the Phase 1 trial in the second quarter of 2024.
In addition to the clinical trial update, Ocugen has announced plans to host a conference call and live webcast to provide a business update and discuss financial results for the year ending December 31, 2023. This event, scheduled for 8:30 a.m. ET on Tuesday, April 2, 2024, promises to offer valuable insights into Ocugen’s operations and future directions.
As Ocugen continues to navigate the complexities of developing treatments for rare diseases, industry watchers and patients alike remain hopeful that OCU410ST will offer a new horizon in the battle against Stargardt disease. With each step forward in the clinical trial, Ocugen moves closer to potentially transforming the landscape of treatment options available to those affected by this challenging condition.
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