MALVERN, Pa., Jan. 16, 2025 (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 first patient has been dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME).
“OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “OCU200 holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.”
The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive a total of two intravitreal injections of OCU200 six weeks apart. Patient follow-up will take place up to three months after the last injection.
Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR or wet AMD. Patients affected by these diseases share common symptoms, such as blurriness in vision and progressive vision loss as the disease progresses. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.
“I am seeing an increasing rate of vision-threatening diseases associated with diabetes at my clinic and am eager to provide a new therapeutic option to these patients,” said Dr. David Almedia, Vitreoretinal Surgeon and Clinician Scientist, President and CEO of Erie Retina Research, and Founder and President of Case X Global in Erie, Pennsylvania. “There remains a considerable unmet medical need for DME and DR patients with currently available anti-VEGF treatments.”
OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies.
“We are enthusiastic about getting patients started in the OCU200 Phase 1 clinical trial and sharing not only safety but preliminary efficacy data as the study progresses,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases.”
The Company intends to pursue approval to use OCU200 as a first-line therapy for DME, DR, and wet AMD.
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 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.
Cautionary Note on Forward-Looking Statements
This
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;
the ability of OCU200 to perform in humans in a manner consistent with nonclinical or preclinical study 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
[email protected]
