Ocugen, Inc. Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 1 of Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410
March 13 2024 - 8:00AM
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 first cohort of its Phase
1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier
gene therapy candidate being developed for geographic atrophy (GA),
an advanced stage of dry age related macular degeneration
(dAMD). GA affects approximately 1 million people in the
United States alone.
“We are very enthusiastic about the potential of OCU410 as a
one-time treatment for life with a single sub-retinal injection,”
said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen.
“While there are currently two recently approved products for the
treatment of GA, both require approximately 6-12 intravitreal
injections annually and target only the complement system. OCU410
addresses multiple pathways causing dAMD, including complement,
lipid metabolism, inflammation, and oxidative stress.”
Up to 13 leading retinal surgery centers across the United
States are participating in the ArMaDa clinical trial. The
enrollment in the first cohort is now complete and 3 subjects
received 200µL single subretinal administration of the low dose
(2.5x1010 vg/mL) of OCU410.
“As a retinal surgeon, I am encouraged by therapeutic options
that can potentially provide long-term benefit to my patients,”
said Lejla Vajzovic, MD, FASRS, Director of Duke Surgical
Vitreoretinal Fellowship Program, Associate Professor of
Ophthalmology with Tenure in Adult and Pediatric Vitreoretinal
Surgery and Diseases, Duke University Eye Center. “OCU410 is a
novel modifier gene therapy approach that could initiate a paradigm
shift in the field of ophthalmology.”
The ArMaDa clinical trial will assess the safety of unilateral
subretinal administration of OCU410 in subjects with GA and will be
conducted in two phases. Phase 1 is a multicenter, open-label,
dose-ranging study consisting of three dose levels [low dose
(2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5
×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded,
dose-expansion study in which subjects will be randomized in a
1:1:1 ratio to either one of two OCU410 treatment groups or to an
untreated control group.
“The American Macular Degeneration Foundation (AMDF) has
supported the research of Dr. Neena Haider, inventor of modifier
gene therapy, and OCU410 in particular, and is pleased that Ocugen
is now spearheading the clinical trials necessary to bring this
therapy closer to patients,” said Matthew Levine, Director of
Grants, Advocacy and Partnerships at AMDF. “The continued
advancement of OCU410 offers hope to those whose vision is already
deteriorating that their remaining vision could be
preserved and could potentially prevent others with an
early dAMD diagnosis from developing any significant
vision loss.”
The Company will continue to provide clinical updates.
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.
About OCU410OCU410 utilizes an AAV delivery
platform for the retinal delivery of the RORA (ROR
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.
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.
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only as of the date of this press release. Except as required by
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Contact: Tiffany Hamilton Head of
Communications Tiffany.Hamilton@ocugen.com
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