Ocugen, Inc. Announces Positive DSMB Recommendation for OCU400-101 Clinical Trial
April 25 2022 - 7:33AM
Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on
discovering, developing, and commercializing novel gene therapies,
biologicals and vaccines, announced today that the independent Data
and Safety Monitoring Board (DSMB) for its Phase 1/2 clinical trial
of OCU400, the Company’s flagship modifier gene therapy candidate
for the treatment of Retinitis Pigmentosa (RP), reviewed safety
data based on dosing to date and recommended that the study proceed
with enrolling additional subjects.
The OCU400-101 clinical study to assess the safety and efficacy
of modifier gene therapy candidate OCU400 for RP resulting from
mutations in the nuclear receptor subfamily 2 group E member 3
(NR2E3) and Rhodopsin (RHO) genes recently dosed its first patient.
The DSMB recommended that the Company continue enrolling the
remaining study subjects in this current cohort at the target dose
level.
Ocugen’s modifier gene therapy platform targets 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.
“It’s a positive first step that the DSMB review of the current
OCU400-101 study results identified no serious adverse events and
recommended that the study proceed with enrollment,” said Mark
Pennesi, MD, PhD, Professor of Ophthalmology and Chief of the Paul
H. Casey Ophthalmic Genetics Division, Oregon Health & Science
University, and member of Ocugen’s Retina Scientific Advisory
Board. “We’re looking forward to understanding how this modifier
gene therapy platform could treat inherited retinal degeneration,
potentially bringing an option to people affected with this
disease.”
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 Phase 1/2
trial included in our Investigational New Drug application to the
U.S. Food and Drug Administration (FDA) for OCU400, which is
actively enrolling patients following review of preliminary safety
data by the independent Data and Safety Monitory Board. 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
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