Ocugen, Inc. Announces Completion of Dosing in Subjects with Geographic Atrophy Secondary to dAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy
July 25 2024 - 6:30AM
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 third cohort of its Phase
1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel 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.
“Currently, there are two FDA approved anti-complement therapies
for GA, which targets only one pathway for a disease that has a
multifactorial and complex etiology,” said Majda Hadziahmetovic,
MD, Associate Professor of Ophthalmology, Vitreoretinal Diseases
and Surgery, Duke University Eye Center, and the lead investigator
for the OCU410 Phase 1/2 trial. “Though these treatments are a
significant milestone, they provide limited benefits and involve a
continuous regimen of multiple intravitreal injections over several
years. This modifier gene therapy could potentially transform the
landscape of GA treatment.”
Three subjects received a single subretinal administration of
the highest dose (200 µL of 1.5x1011 vg/mL) being tested, which
completed the dosing for the Phase 1 stage of the trial. The ArMaDa
clinical trial for OCU410 is being performed at 14 leading retinal
surgery centers across the United States.
“We are very encouraged about the potential of OCU410 as a
one-time treatment option for GA,” said Dr. Huma Qamar, Chief
Medical Officer of Ocugen.
We have initiated a Phase 2 clinical trial that will assess the
safety and efficacy of OCU410 in a larger group of patients who
will be randomized into either of two treatment groups (medium- or
high-dose) or a control group.
The Company will continue to provide clinical updates on a
periodic basis.
About the Phase 1/2 ArMaDa clinical trialThe
ArMaDa Phase 1/2 clinical trial will assess the safety of
unilateral subretinal administration of OCU410 in subjects with
geographic atrophy and will be conducted in two phases. Phase
1 is a multicenter, open-label, dose-escalation 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.
About dry age-related macular
degeneration (dAMD) and geographic
atrophy (GA)Dry age-related macular
degeneration (dAMD) affects approximately 10 million Americans and
more than 266 million people worldwide. It is characterized by the
thinning of the macula, the portion 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 all AMD
cases.
About OCU410OCU410 utilizes an
adeno-associated virus (AAV) 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 both in vitro and in vivo (animal model) studies. These results
demonstrate the ability of OCU410 to target multiple pathways
linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as
a one-time gene therapy for the treatment of geographic
atrophy.
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.
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press release contains forward-looking statements within the
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preliminary, interim and top-line clinical trial results may not be
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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
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and that that clinical trial data are subject to differing
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Contact:Tiffany HamiltonHead of Communications
Tiffany.Hamilton@ocugen.com
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