Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease
April 01 2024 - 5:08PM
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies and vaccines, today
announced that the Data Safety and Monitoring Board (DSMB) for the
OCU410ST clinical trial recently convened and approved to proceed
dosing with the medium dose of OCU410ST in the dose-escalation
phase of the study.
Three patients with Stargardt disease were dosed in the Phase
1/2 clinical trial to date. An additional three patients will be
dosed with the medium dose (Cohort 2) and three patients with the
high dose (Cohort 3) of OCU410ST in the dose-escalation phase.
“The DSMB has recommended moving forward to medium dose for
dosing subjects with Stargardt disease,” said Dr. Peter Chang, MD,
FACS, DSMB Chair for the OCU410ST clinical trial. “No serious
adverse events (SAEs) related to OCU410ST have been reported to
date. I believe that this marks a critical next step towards
determining the optimal dosing regimen for OCU410ST.”
“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,” said
Huma Qamar, M.D., MPH, Chief Medical Officer of Ocugen. “We
recognize the high unmet medical need for Stargardt patients, as
there are no current FDA-approved therapies for the indication, and
we look forward to sharing the interim safety data from our Phase 1
trial in the second quarter of 2024.”
The GARDian clinical trial will assess the safety and efficacy
of unilateral subretinal administration of OCU410ST in subjects
with Stargardt disease and will be conducted in two phases. Phase 1
is a multicenter, open-label, dose ranging study consisting of
three dose levels [low dose (3.75×1010 vg/mL), medium dose
(7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)]. Phase
2 is a randomized, outcome accessor-blinded, dose-expansion study
in which adult and pediatric subjects will be randomized in a 1:1:1
ratio to either one of two OCU410ST dose groups or to an untreated.
The Company will continue to provide clinical updates.
Ocugen is committed to finding solutions for people with
inherited retinal disease for whom no effective treatment options
exist. While an orphan disease, Stargardt affects approximately
40,000 people in the United States alone.
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, compliment 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 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 Twitter and LinkedIn.
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Reform Act of 1995, which are subject to risks and uncertainties.
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factors described in the section entitled “Risk Factors” in the
quarterly and annual reports that we file with the SEC. Any
forward-looking statements that we make in this press release speak
only as of the date of this press release. Except as required by
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press release.
Contact:Tiffany HamiltonHead of
CommunicationsIR@ocugen.com
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