Data and Safety Monitoring Board Approves Initiation of Phase 2 of OCU410ST GARDian Clinical Trial for Stargardt Disease
22 Octubre 2024 - 6:02AM
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 DiseaseStargardt 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 OCU410STOCU410ST 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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StatementsThis press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995, including, but not limited to, strategy,
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and timelines for the preclinical and clinical development of
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Contact:Tiffany HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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