Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410—a Modifier Gene Therapy—in Phase 1/2 ArMaDa Study for Geographic Atrophy
April 05 2024 - 6:02AM
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 the Data and Safety Monitoring Board (DSMB) for the
Phase 1/2 ArMaDa clinical trial for OCU410 recently convened and
approved to proceed dosing with the medium dose of OCU410 in the
dose-escalation phase of the study.
Three subjects with geographic atrophy (GA) were dosed in the
Phase 1/2 clinical trial to date. An additional three subjects will
be dosed with the medium dose (Cohort 2) and three patients with
the high dose (Cohort 3) of OCU410 in the dose-escalation
phase.
“The DSMB has recommended moving forward to medium dose for
dosing subjects with GA,” 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. I believe that
this marks a critical next step towards determining the optimal
dosing regimen and an important milestone for the clinical
development of OCU410.”
“The positive DSMB review for the first modifier gene therapy
for GA significantly builds on the favorable safety and
tolerability profile exhibited by OCU410,” said Huma Qamar, M.D.,
MPH, Chief Medical Officer of Ocugen. “We are very enthusiastic
about the potential of OCU410 as a one-time treatment for life with
a single sub-retinal injection.”
Currently approved products to treat GA have significant
limitations, as they require multiple injections per year and only
target one pathway contributing to GA. OCU410 regulates multiple
pathways involved with the disease including: lipid metabolism,
inflammation, oxidative stress, and membrane attack complex
(complement).
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.
Ocugen is committed to finding solutions for inherited retinal
diseases as well as blindness diseases affecting millions. GA is an
advanced form of dry age-related macular degeneration (dAMD) and
affects approximately 1 million people in the United States.
About dAMDdAMD 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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forward-looking statements that we make in this press release speak
only as of the date of this press release. Except as required by
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press release.
Contact:Tiffany HamiltonHead of
CommunicationsIR@ocugen.com
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