Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410—Modifier Gene Therapy—for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
December 13 2023 - 7:30AM
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 ArMaDa Phase 1/2 clinical trial of OCU410 (AAV-RORA), a
modifier gene therapy product candidate being developed for dry AMD
(dAMD).
“OCU410, our first-in-class modifier gene therapy for dAMD,
addresses gaps among other therapies available and in development
for dAMD as a potential one-time treatment for life,” said Dr.
Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “We are
very pleased to continue advancing our ophthalmic gene therapy
pipeline, which remains the Company’s primary focus.”
This Phase 1/2 trial will assess the safety and efficacy of
OCU410 for geographic atrophy (GA) secondary to dAMD and will be
conducted in two phases. Phase 1 is a multicenter, open-label,
dose-ranging study. Phase 2 is a randomized expansion phase in
which subjects will be randomized in a 1:1:1 ratio to either one of
two OCU410 dose groups or to an untreated control group.
OCU410 is a potential curative therapy with a single sub-retinal
injection that targets multiple pathways causing dAMD, including
lipid metabolism, inflammation, oxidative stress, and complement
activation. Currently, the other therapeutic options available
target only complement activation and require approximately 6-12
intravitreal injections annually.
“Breaking new ground in the pursuit of vision restoration, our
pioneering modifier gene therapy candidate, OCU410, achieves
another major milestone by dosing a GA patient in a Phase 1/2
clinical trial,” said Arun Upadhyay, PhD, Chief Scientific Officer,
Head of R&D at Ocugen. “OCU410 offers hope for those battling
GA that are faced with limited treatment options and the real
prospect of ultimately losing their vision.”
“There remains a great unmet need for novel durable and
effective treatments for GA, which remains one of the most common
causes of vision loss globally,” said Benjamin Bakall, MD, PhD,
director of clinical research at Associated Retina Consultants
(ARC) and clinical assistant professor at University of Arizona,
College of Medicine – Phoenix. “I am excited that we performed the
first surgery with this novel therapeutic approach—designed to
restore homeostasis and slow disease progression following a single
treatment—at ARC in Phoenix, AZ, with the surgical team led by Dr.
Mark Kwong, medical director of ARC.”
The first surgery was successful in delivering the new gene
underneath the retina; the light sensitive nerve tissue lining the
inside of the eye.
About dAMD and GAdAMD affects approximately 10
million Americans and more than 266 million people worldwide. It is
characterized by the thinning of the macula. The macula is the part
of the retina responsible for clear vision in one’s direct line of
sight.
dAMD involves the slow deterioration of the retina with
submacular drusen (small white or yellow dots on the retina),
atrophy, loss of macular function and central vision impairment.
dAMD accounts for 85-90% of the total AMD population.
GA, an advanced form of dry age-related macular degeneration,
affects approximately 1 million people in the United States alone.
About OCU410OCU410 utilizes an AAV delivery
platform for the retinal delivery of the RORA (RAR
Related Orphan Receptor A) gene. The RORA protein plays
an important role in lipid metabolism, reducing lipofuscin deposits
and oxidative stress, and demonstrates an anti-inflammatory role
in-vitro and in-vivo (animal model) studies. These results
demonstrate the ability for OCU410 to target multiple pathways
linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as
a one-time gene therapy for the treatment of GA. Currently, the
other therapeutic options available target only complement
activation and require approximately 6-12 intravitreal injections
annually.
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.
About Associated Retina ConsultantsAssociated
Retina Consultants is the largest independent retina practice in
the state of Arizona, taking part in groundbreaking clinical trials
to bring new treatments for the benefit of patients with diseases
affecting the retina. In addition to collaborating with Ocugen on
the OCU410 clinical trial, in October 2023, Associated Retina
Consultants—with a surgical team led by Dr. Mark Kwong—performed
gene therapy with OCU400 on a pediatric CEP290 patient. This was
the first retinal gene therapy of its kind performed on a child in
Arizona.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995, 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. 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 HamiltonHead of
CommunicationsIR@ocugen.com
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