Ocugen Inc. has announced the progression of its OCU410ST gene therapy into Phase 2 clinical trials for Stargardt disease, following a positive review by the Data and Safety Monitoring Board (DSMB). The decision was based on encouraging safety data from the Phase 1 portion of the GARDian clinical trial, indicating that OCU410ST was safe and well-tolerated among the initial patient cohort.
The Phase 1/2 clinical trial is being conducted at six retinal surgery centers across the U.S. The initial phase was an open-label, dose-ranging study involving nine patients who received a subretinal injection of OCU410ST at varying doses: low (3.75 x 10^10 vg/mL), medium (7.5 x 10^10 vg/mL), or high (2.25 x 10^11 vg/mL). The DSMB determined the highest dose to be the maximum tolerated dose (MTD).
Encouraging Safety Profile
According to Dr. Charles Wykoff, Director of Research at Retina Consultants of Texas & Retina Consultants of America, the safety and tolerability profile of OCU410ST is promising. "The DSMB has recommended moving forward with Phase 2 enrollment, as safety data indicates that OCU410ST appears to be safe and well-tolerated to date," he stated. No serious adverse events (SAEs) were reported during Phase 1.
Addressing Unmet Needs in Stargardt Disease
Stargardt disease, the most common form of inherited macular dystrophy, leads to progressive vision loss due to the degeneration of photoreceptor cells in the macula. The disease typically manifests in childhood or adolescence, with symptoms of bilateral central vision loss that worsen over time. Currently, there are no FDA-approved treatments for Stargardt disease, leaving approximately 100,000 patients in the U.S. and Europe with limited options.
OCU410ST: A Novel Modifier Gene Therapy
OCU410ST is designed as a one-time novel modifier gene therapy. It utilizes an adeno-associated virus (AAV) delivery platform to introduce the RORA (RAR Related Orphan Receptor A) gene into the retina. This gene regulates pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.
"We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. The company is focusing on advancing gene and cell therapies, biologics, and vaccines for various diseases.
The advancement of OCU410ST to Phase 2 represents a significant step forward in addressing the unmet medical need for effective treatments for Stargardt disease. The trial will continue to evaluate the safety and efficacy of OCU410ST in a larger patient population.
