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 it has received notification from FDA to begin its
expanded access program (EAP) for the treatment of adult patients,
aged 18 and older, with retinitis pigmentosa (RP) with OCU400—a
modifier gene therapy product candidate.
“Each clinical milestone achieved by OCU400 brings us closer to
providing a potential one-time treatment for life to patients
living with RP,” said Dr. Shankar Musunuri, Chairman, CEO, and
Co-founder of Ocugen. “With positive Phase 1/2 study data and an
ongoing Phase 3 liMeliGhT (pronounced “limelight”) clinical trial,
we now plan to work with clinicians, patients, and the RP community
to provide access to OCU400 for eligible patients through our EAP.
The EAP strengthens our commitment to serving RP patients—300,000
in the U.S. and Europe and 1.6 million globally.”
EAP allows patients who have unmet medical needs with serious or
life-threatening conditions to access treatments outside of a
clinical trial that are not yet approved by the FDA.
The OCU400 EAP is available for patients with early,
intermediate to advanced RP with at least minimal retinal
preservation who may benefit from the mechanism of action of OCU400
prior to approval of the Biologics License Application
(BLA). Ocugen is actively dosing patients in the Phase 3
liMeliGhT clinical trial.
“RP patients with mutations in multiple genes currently have no
therapeutic options. As a retinal surgeon, I am encouraged by the
therapeutic potential of OCU400 to provide long-term benefit,” said
Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal
Fellowship Program, Associate Professor of Ophthalmology with
Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke
University Eye Center, and Retina Scientific Advisory Board Chair
of Ocugen. “The OCU400 EAP gives RP patients access to this novel
modifier gene therapy outside of the ongoing Phase 3 study.”
“We are pleased to make OCU400 available to
patients beyond our Phase 3 liMeliGhT clinical trial through this
EAP,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. "We are
excited to expand our enrollment to include patients representing a
diverse array of RP gene mutations. This program reflects our
ongoing commitment to develop a safe and effective therapy for RP
patients who may not have other treatment options.”
Ocugen previously announced that OCU400 has received orphan drug
and Regenerative Medicine Advanced Therapy (RMAT) designations from
FDA and that the European Medicines Agency (EMA) accepted the
U.S.-based trial for submission of a Marketing Authorization
Application (MAA). With the dosing of patients in the Phase 3
clinical trial program underway, OCU400 remains on track for
targeted BLA and MAA approval in 2026.
About OCU400
EAPThe OCU400 EAP is a U.S.-only protocol for (1) eligible
adult RP patients, 18 years and older, with early, intermediate to
advanced disease with at least minimal retinal preservation, (2)
patients who participated in the OCU400 Phase 1/2 study and who
qualify for dosing in the contralateral eye, (3) patients who
failed to meet inclusion criteria in the Phase 1/2 trial and
ongoing Phase 3 liMeliGhT clinical trial who could benefit from
OCU400, and (4) RP patients who can benefit from the mechanism of
action of OCU400 prior to BLA approval.
Additional information on the OCU400 EAP will be available on
www.clinicaltrials.gov.
About OCU400 Phase 3 (liMeliGhT) for RPThe
Phase 3 liMeliGhT clinical trial, with a duration of one year, will
have a sample size of 150 participants. One arm will include 75
participants with RHO gene mutations, and the other arm will
include 75 participants who have mutations in other genes. Within
each arm, participants will be randomized 2:1 to the treatment
group (2.5 x1010 vector genomes/eye of OCU400) and untreated
control group, respectively. Patients eight years of age and older
with early to late-stage RP are being recruited to participate in
the liMeliGhT study.
About OCU400OCU400 is the Company’s modifier
gene therapy product based on a nuclear hormone receptor (NHR) gene
called NR2E3. This gene regulates diverse physiological functions
within the retina, such as photoreceptor development and
maintenance, metabolism, phototransduction, inflammation, and cell
survival. Retinal cells in RP patients have a dysfunctional gene
network, and OCU400 resets this network to reestablish a healthy
cellular homeostasis—which has the potential to improve vision in
patients with RP.
About Modifier
Gene TherapyModifier gene therapy
is designed to fulfill unmet medical needs related to retinal
diseases, including IRDs, such as RP, Leber congenital amaurosis
(LCA) and Stargardt disease, as well as multifactorial diseases
like dry age-related macular degeneration (dAMD). Our modifier gene
therapy platform is based on the use of NHRs, master gene
regulators, which have the potential to restore homeostasis — the
basic biological processes in the retina. Unlike single-gene
replacement therapies, which only target one genetic mutation, we
believe that our modifier gene therapy platform, through its use of
NHRs, represents a novel approach that has the potential to address
multiple retinal diseases caused by mutations in multiple genes
with one product, and to address complex diseases that are
potentially caused by imbalances in multiple gene networks.
Currently, Ocugen has three modifier gene therapy programs in the
clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400
Phase 3 liMeliGhT clinical trial, the OCU410 Phase 1/2 ArMaDa
clinical trial for geographic atrophy (GA) secondary to dAMD and
the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease
are currently underway. GA affects approximately two to three
million people in the U.S. and EU combined and Stargardt disease
affects nearly 100,000 people in the U.S. and EU combined.
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 patients’
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 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 Corporate
Communications Tiffany.Hamilton@ocugen.com
Ocugen (NASDAQ:OCGN)
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Ocugen (NASDAQ:OCGN)
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