Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration
April 01 2022 - 7:37AM
Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on
discovering, developing, and commercializing novel gene therapies,
biologicals and vaccines, announced that the first patient has been
dosed in the Phase 1/2 clinical trial of OCU400, a modifier gene
therapy candidate for the treatment of retinitis pigmentosa (RP)
resulting from mutations in the nuclear receptor subfamily 2 group
E member 3 (NR2E3) and Rhodopsin (RHO) genes.
This first patient dosing marks the beginning of the
dose-escalating, observer-blind, Phase 1/2 safety and efficacy
study. This Phase 1/2 study is currently enrolling. More
information can be found on ClinicalTrials.gov, under identifier
number NCT05203939.
“Everyone at Ocugen is excited about this important milestone.
Every day, our teams are working toward developing a therapeutic
for people who have no options when facing inherited retinal
diseases,” said Dr. Shankar Musunuri, Chairman of the Board, Chief
Executive Officer, and Co-Founder of Ocugen. “The first phase of
the study is a safety evaluation of the product, eventually
progressing into an efficacy study in patients. Today’s
announcement signifies a first and monumentally critical step
forward in achieving our mission to cure blindness diseases.”
RP is a group of rare, genetic disorders that involve a
breakdown and loss of cells in the retina (the light-sensitive
tissue that lines the back of the eye). Common symptoms include
difficulty seeing at night and a progressive loss of side
(peripheral) vision. It is generally estimated that RP affects
roughly 1 in 4,000 people – approximately two million people –
globally.1 There is currently no approved therapy intended to stop
the progression of RP based on all of the genetic mutations that
cause the disease.
Ocugen’s modifier gene therapy platform aims to target nuclear
hormone receptors (NHRs) that regulate multiple functions within
the retina, giving it the potential to address many different gene
mutations—and, in turn, multiple retinal diseases—with a single
product. Traditional gene therapy, which transfers a functional
version of a non-functional gene into target cells, addresses only
one individual gene mutation at a time.
“Our premise is that disease progression can be halted at
whatever stage patients are currently at, potentially preventing
further vision loss,” said David Birch, PhD, Scientific Director at
the Rose-Silverthorne Retinal Degenerations Laboratory. “This Phase
1/2 clinical trial targets people who have RP resulting from
mutations in the NR2E3 and RHO genes. Based on the safety and
efficacy outcomes, this study may be expanded to include additional
genetic mutations in a Phase 3 study designed to demonstrate broad
therapeutic applications of OCU400 in people with RP and Leber
congenital amaurosis. If approved, we believe OCU400 may ultimately
impact the lives of people facing retinitis pigmentosa and other
retinal diseases rooted in the mutations of more than 175
genes.”
About Ocugen, Inc. Ocugen, Inc. is a
biotechnology company focused on discovering, developing, and
commercializing novel gene therapies, biologicals and vaccines that
improve health and offer hope for people and global communities. We
are making an impact through courageous innovation, taking science
in new directions in service of patients. Our breakthrough modifier
gene therapy platform has the potential to treat multiple diseases
with one drug and we are advancing research in other therapeutic
areas to offer new options for people with unmet medical needs.
Discover more at www.ocugen.com and follow us on Twitter and
LinkedIn.
Cautionary Note on Forward-Looking
StatementsThis press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995, which are subject to risks and uncertainties.
We may, in some cases, use terms such as “predicts,” “believes,”
“potential,” “proposed,” “continue,” “estimates,” “anticipates,”
“expects,” “plans,” “intends,” “may,” “could,” “might,” “will,”
“should” or other words that convey uncertainty of future events or
outcomes to identify these forward-looking statements. Such
forward-looking statements include information about qualitative
assessments of available data, potential benefits, expectations for
clinical trials, and anticipated timing of clinical trial readouts
and regulatory submissions, including with respect to our planned
Phase 1/2 trial included in our Investigational New Drug
application to the U.S. Food and Drug Administration (FDA) for
OCU400, which was recently accepted by the FDA. This information
involves risks and uncertainties that could cause actual results to
differ materially from those expressed or implied by such
statements. Risks and uncertainties include, among other things,
the uncertainties inherent in research and development, including
the ability to meet anticipated clinical endpoints, commencement
and/or completion dates for clinical trials, regulatory submission
dates, regulatory approval dates and/or launch dates, as well as
risks associated with preliminary and interim data, including the
possibility of unfavorable new clinical trial data and further
analyses of existing clinical trial data; the risk that the results
of in-vitro studies will not be duplicated in human clinical
trials; the risk that clinical trial data are subject to differing
interpretations and assessments, including during the peer
review/publication process, in the scientific community generally,
and by regulatory authorities; and the risk that the Orphan Drug
Designations from the FDA and broad Orphan Medicinal Product
Designation from the European Commission for OCU400 may not result
in a faster approval timeline for OCU400 or increase the likelihood
of any such approvals 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 SEC. Any forward-looking statements
that we make in this press release speak only as of the date of
this press release. Except as required by law, we assume no
obligation to update forward-looking statements contained in this
press release whether as a result of new information, future events
or otherwise, after the date of this press release.
Ocugen Contact: Ken InchaustiHead,
Investor Relations & Communicationsken.inchausti@ocugen.com
Please submit investor-related inquiries
to: IR@ocugen.com
References:
1. Retinitis pigmentosa. National Organization of Rare
Disorders.
https://rarediseases.org/rare-diseases/retinitis-pigmentosa/
(Accessed March 28, 2022)
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