Ocugen Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene Therapy
April 19 2024 - 6:13AM
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 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, gene-agnostic option for the treatment of GA,” said Dr.
Huma Qamar, Chief Medical Officer of Ocugen. “OCU410 regulates
multiple pathways involved with the disease, including lipid
metabolism, inflammation, oxidative stress, and membrane attack
complex (complement) with a single sub-retinal injection.”
Dosing in the second cohort is complete and 3 subjects received
200 mL single subretinal administration of the medium dose (5x1010
vg/mL) of OCU410. Up to 13 leading retinal surgery centers
across the United States are participating in the ArMaDa
clinical trial.
“Currently we have two FDA approved, anti-complement therapies
for GA targeting a single pathway of the disease, which has
multifactorial and complex etiology,” said Syed M. Shah, MD, Vice
Chair of Research and Digital Medicine and Director of Retina
Service at Gundersen Health System, La Crosse, WI, and the lead
investigator for the OCU410 Phase 1/2 trial. “The limited benefit
comes with the burden of continued multiple intravitreal injections
spanning over several years. This novel modifier gene therapy has
the potential to transform the therapeutic landscape in GA
treatment.”
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 high dose, which is the final dose in the
Phase 1 dose-escalation study.
The ArMaDa Phase 1/2 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 Company will continue to provide clinical updates on an
ongoing basis.
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 as well as inhibiting the complement system in
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.
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
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of future events or outcomes to identify these forward-looking
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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
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release speak only as of the date of this press release. Except as
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this press release.
Contact: Tiffany Hamilton Head of
Communications Tiffany.Hamilton@ocugen.com
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