Data and Safety Monitoring Board Approves Initiation of Phase 2 of OCU410ST GARDian Clinical Trial for Stargardt Disease
Source: GlobeNewswire- Determined the high dose of OCU410ST to be the maximum tolerated dose
- No serious adverse events have been reported
- Approved proceeding to Phase 2 using high and medium doses of OCU410ST
MALVERN, Pa., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved enrollment for the second phase of the Phase 1/2 clinical trial. OCU410ST (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for Stargardt disease.
“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,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas & Retina Consultants of America, and a lead study investigator. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial has progressed and continues to bring hope to patients with Stargardt disease, which still has no FDA-approved treatments.”
The first phase of the Phase 1/2 clinical trial was an open-label, dose-ranging study that enrolled nine patients to receive either a low (3.75 x 1010 vg/mL), medium (7.5 x 1010 vg/mL), or high (2.25 x 1011 vg/mL) dose of OCU410ST administered via subretinal injection. No serious adverse events (SAEs) have been reported, and the DSMB determined the high dose to be the maximum tolerated dose (MTD).
Stargardt disease is the most common form of inherited macular dystrophy. Symptoms of bilateral central vision loss typically begin in childhood and gradually worsen over time.
“We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the prospect of addressing a substantial unmet medical need for the estimated 100,000 Stargardt patients in the U.S. and Europe.”
The GARDian clinical trial is currently being performed at 6 leading retinal surgery centers across the U.S.
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents 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.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
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Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
