Data and Safety Monitoring Board Approves Simultaneous Enrollment in Cohort 3 and Phase 2 Initiation in OCU410 ArMaDa study for Geographic Atrophy
31 Maio 2024 - 9:36AM
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 a positive outcome of the Data and Safety Monitoring
Board (DSMB) Review for its Phase 1/2 ArMaDa clinical trial for
OCU410 (AAV5-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
2-3 million people in the U.S. and Europe combined.
Six subjects with GA were dosed in the Phase 1/2 clinical trial
to date—three subjects were dosed with the low dose and three
subjects were dosed with the medium dose. An additional three
patients will be dosed with the high dose of OCU410 in the
dose-escalation phase.
“The DSMB has recommended to proceed with dosing subsequent GA
subjects with the high dose of OCU410 in the dose-expansion phase
of the study and concurrently initiate Phase 2 dosing,” said Dr.
Peter Chang, MD, FACS, DSMB Chair for the OCU410 clinical trial.
“No serious adverse events (SAEs) related to OCU410 have been
reported to date in both low- and medium-dose cohorts. I believe
that this marks a critical next step towards determining the
maximum tolerated dose for OCU410 and is an important milestone for
its clinical development.”
"We are delighted to report a second positive DSMB
recommendation for the treatment of GA, which significantly builds
on the favorable safety and tolerability profile exhibited by
OCU410," said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of
Ocugen. "We are very enthusiastic about the potential of OCU410 as
a potential one-time treatment for GA with a single sub-retinal
injection. The currently approved treatments for GA target only the
complement pathway and require approximately 6-12 intravitreal
injections annually. OCU410 addresses multiple pathways causing
dAMD, including complement, lipid metabolism, inflammation, and
oxidative stress, providing long-term benefit to patients."
The ArMaDa clinical trial will assess the safety and efficacy 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.
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 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.
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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
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Contact:Tiffany HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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