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 dosing is complete in the second cohort of its Phase
1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier
gene therapy candidate being developed for Stargardt disease as a
one-time treatment for life.
"The completion of dosing for Cohort 2 participants signifies an
important clinical milestone for our pioneering modifier gene
therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer of
Ocugen. “We are encouraged by the ongoing positive safety and
tolerability profile demonstrated by OCU410ST, enabling us to
consider higher doses in patients as we progress with the
dose-escalation study. We look forward to sharing preliminary
safety and efficacy data from Phase 1 of the clinical trial.”
Six patients with Stargardt disease have been dosed in the Phase
1/2 clinical trial to date. An additional three patients will be
dosed with the high dose (Cohort 3) of OCU410ST in the
dose-escalation phase.
“There remains a great unmet medical need for patients with
Stargardt disease, which is the most common inherited retinal
disease affecting the center of the vision and does not have any
FDA-approved treatment options. OCU410ST is a novel modifier gene
therapy that provides hope to these patients,” said Benjamin
Bakall, MD, PhD, Director of Clinical Research at Associated Retina
Consultants and Clinical Assistant Professor at University of
Arizona, College of Medicine – Phoenix. “I am excited that we
completed dosing of the last patient in Cohort 2, who received
medium dose of this novel therapeutic leveraging a gene-agnostic
approach, at Associated Retina Consultants (ARC) in Phoenix, AZ
with the surgical team led by Dr. Mark Kwong, Medical Director of
ARC.”
A Data and Safety Monitoring Board meeting will convene next
month to review the 4-week safety data of the medium dose cohort
before proceeding with Cohort 3 (high dose), which is the final
dose in the Phase 1 dose-escalation study.
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
group.
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
100,000 people in the United States and Europe combined.
The Company expects to provide a clinical trial update for
OCU410ST in the third quarter of 2024.
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, 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 patients’ 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.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995, including,
but not limited to, statements regarding qualitative assessments of
available data, potential benefits, expectations for ongoing
clinical trials, anticipated regulatory filings and anticipated
development timelines, which are subject to risks and
uncertainties. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should,” or other words that convey uncertainty
of future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks, and uncertainties that may cause actual events or
results to differ materially from our current expectations,
including, but not limited to, the risks that preliminary, interim
and top-line clinical trial results may not be indicative of, and
may differ from, final clinical data; that unfavorable new clinical
trial data may emerge in ongoing clinical trials or through further
analyses of existing clinical trial data; that earlier non-clinical
and clinical data and testing of may not be predictive of the
results or success of later clinical trials; and that that clinical
trial data are subject to differing interpretations and
assessments, including by regulatory authorities. These and other
risks and uncertainties are more fully described in our periodic
filings with the Securities and Exchange Commission (SEC),
including the risk 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 law, we assume no obligation to update forward-looking
statements contained in this press release whether as a result of
new information, future events, or otherwise, after the date of
this press release.
Contact: Tiffany Hamilton Head of
Communications Tiffany.Hamilton@ocugen.com
Ocugen (NASDAQ:OCGN)
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