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 U.S. Food and Drug Administration (FDA) has
cleared the Company’s Investigational New Drug (IND) amendment to
initiate a Phase 3 clinical trial of OCU400, a modifier gene
therapy product candidate being developed for retinitis pigmentosa
(RP).
“The initiation of the OCU400 Phase 3 clinical trial is a
significant milestone for patients with RP and a pivotal event for
Ocugen as a company,” said Dr. Shankar Musunuri, Chairman, CEO and
Co-Founder of Ocugen. “OCU400 is the first gene therapy program to
enter Phase 3 with a broad RP indication. Until now, there has been
only one marketed product to treat one of the 100 gene mutations
associated with RP. Now there is real hope for all RP patients who
haven’t had a treatment option.”
The Phase 3 study will have a sample size of 150
participants—one arm of 75 participants with the RHO gene mutation
and the other arm with 75 participants that are gene agnostic. In
each arm, participants will be randomized 2:1 to the treatment
group (2.5 x 1010 vg/eye of OCU400) and untreated control group,
respectively.
In the Phase 1/2 OCU400 clinical trial, a Multi-Luminance
Mobility Testing (MLMT) scale was the primary functional endpoint.
For the Phase 3 OCU400 clinical trial, an updated mobility course
will be used—Luminance Dependent Navigation Assessment (LDNA)— that
includes a wider range of light intensity (0.04-500 Lux) and Lux
Levels (0-9) with a uniform correlation between Lux level and Lux
intensity.
“A sensitive mobility course, LDNA, was developed by Ocugen in
collaboration with FDA for the Phase 3 clinical trial to allow
enrollment of patients with early to advanced stages of disease,”
said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are
extremely encouraged that with this Phase 3 study design more than
50% of intent to treat RHO patients would meet the responder
criteria, which is demonstrating 2 or greater Lux level of
improvement after one year of treatment based on Phase 1/2 study
results.”
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.
“We believe that the gene-agnostic clinical trial design
provides an appropriate therapeutic option to include patients who
have greater potential of benefiting from treatment,” said Dr. Huma
Qamar, Chief Medical Officer at Ocugen. “We are looking forward to
working with our selected trial sites and leading retinal surgeons
to deliver this novel modifier gene therapy to potentially address
unmet medical needs.”
Ocugen previously announced that OCU400 has received orphan drug
and RMAT designations from the FDA. With the initiation of the
Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA
approval target.
About OCU400OCU400 is the
Company’s gene-agnostic modifier gene therapy product based on NHR
gene, NR2E3. NR2E3 regulates diverse physiological
functions within the retina—such as photoreceptor development and
maintenance, metabolism, phototransduction, inflammation and cell
survival networks. Through its drive functionality, OCU400 resets
altered/affected cellular gene-networks and establishes
homeostasis—a state of balance, which has the potential to improve
retinal health and function in patients with inherited retinal
diseases.
About RPRP is a group of rare, genetic
disorders that involve a breakdown and loss of cells in the retina,
leading to vision loss and blindness. Currently, RP is associated
with mutations in more than 100 genes.
There are no approved treatment options that
slow or stop the progression of multiple forms of RP. Proposed
treatments for RP include gene-replacement therapy, retinal implant
devices, retinal transplantation, stem cells, vitamin therapy, and
other pharmacological treatments. Current gene-replacement
therapies are promising but are limited to treating just a single
mutation. In addition, while gene therapies may provide a new
functional gene, they do not necessarily eliminate the underlying
genetic defect, which may still cause stress and toxic effects
leading to retina degeneration. Therefore, the development of
gene-specific replacement therapy is highly challenging, especially
when multiple and unknown genes are involved. Thus, novel
therapeutic approaches targeting broader RP disease in a gene
agnostic manner offer greater hope for patients.
About Ocugen, Inc.Ocugen, Inc. is a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies, biologics, 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.
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 benefits, expectations for ongoing
clinical trials, anticipated regulatory filings and anticipated
development timelines, 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 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; and that that clinical
trial data are subject to differing interpretations and
assessments, including by regulatory authorities. 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.
Contact:Tiffany HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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