Ocugen Gains FDA Alignment on Key Aspects of OCU400—Modifier Gene Therapy—Pivotal Phase 3 Study Design
December 21 2023 - 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 the Company received alignment from FDA on key
aspects of the Phase 3 clinical trial design to assess the safety
and efficacy of OCU400 in patients with RHO and other gene
mutations associated with Retinitis Pigmentosa (RP).
“This news brings us even closer to fulfilling our mission to
bring our first-in-class, gene-agnostic therapies to market and
provide access to patients globally,” said Dr. Shankar Musunuri,
Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We
look forward to beginning the Phase 3 clinical trial, which we plan
to initiate in early 2024.”
During a multidisciplinary meeting with FDA, based on
preliminary results from an ongoing Phase 1/2 study, Ocugen
received alignment on key aspects of the Phase 3 study
design—including the study endpoint, patient enrollment strategy,
and study duration of one year. The Phase 3 clinical trial will
enroll a broader group of RP patients, including patients with the
most common RHO gene mutation, based on OCU400’s potentially
gene-agnostic mechanism of action.
With orphan drug and RMAT designations in place for OCU400,
FDA’s alignment on key aspects of the Phase 3 study design
positions Ocugen to confidently move forward in pursuing product
development and licensure for OCU400.
Currently there are approximately 110,000 patients in the United
States with RP and 1.6 million patients globally. Of these
patients, more than 10% have the RHO genetic mutation. Advancing
OCU400 to Phase 3 clinical development will be an important step
toward addressing unmet needs in the RP patient community.
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.
Cautionary Note on Forward-Looking
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
therapeutic and clinical benefits of our product candidates,
expectations for clinical trial timing and results, anticipated
timing of clinical trial updates and expectations for timing and
outcome of regulatory interactions, which are subject to risks and
uncertainties. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
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of future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks, and uncertainties that may cause actual events or
results to differ materially from our current expectations,
including, but not limited to, the risks that preliminary, interim
and top-line clinical trial results may not be indicative of, and
may differ from, final clinical data; that 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 be predictive of the
results or success of later clinical trials; that that clinical
trial data are subject to differing interpretations and
assessments, including by regulatory authorities; that receipt of
orphan drug and RMAT designations may not lead to faster
development or regulatory review; and that regulatory authorities
may disagree with additional aspects of our clinical trial designs
or may not approve our future IND applications on the anticipated
timeline or at all. 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
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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.
Contact:Tiffany HamiltonHead of
CommunicationsIR@ocugen.com
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