BrainStorm Cell Therapeutics and FDA Agree to Potential NurOwn® Regulatory Pathway for Approval in ALS
February 11 2020 - 6:00AM
BrainStorm Cell Therapeutics, Inc., (NASDAQ:BCLI), a leading
developer of adult stem cell therapies for neurodegenerative
diseases, today announced that the Company recently held a high
level meeting with the U.S. Food and Drug Administration (FDA) to
discuss potential NurOwn® regulatory pathways for approval in ALS.
Repeated intrathecal administration of NurOwn (autologous MSC-NTF
cells) is currently being evaluated in a fully enrolled Phase 3
pivotal trial in ALS (NCT03280056).
In the planned meeting with senior Center for
Biologics Evaluation and Research (CBER) leadership and several
leading U.S. ALS experts, the FDA confirmed that the fully enrolled
Phase 3 ALS trial is collecting relevant data critical to the
assessment of NurOwn efficacy. The FDA indicated that they will
look at the "totality of the evidence" in the expected Phase 3
clinical trial data. Furthermore, based on their detailed data
assessment, they are committed to work collaboratively with
BrainStorm to identify a regulatory pathway forward, including
opportunities to expedite statistical review of data from the Phase
3 trial.
Both the FDA and BrainStorm acknowledged the
urgent unmet need and the shared goal of moving much needed
therapies for ALS forward as quickly as possible.“This is a key
turning point in
our work toward providing ALS patients with
a potential new therapy,” said Chaim Lebovits, President
and CEO of BrainStorm. “We commend the FDA
for its commitment to the ALS
community and to facilitating the development, and
we ultimately hope, the approval of NurOwn. The
entire BrainStorm team is grateful for the ongoing and
conscientious collaboration in the quest to beat ALS.”
Ralph Kern, MD, MHSc, Chief Operating Officer
and Chief Medical Officer, stated, “The entire team at BrainStorm
has collectively worked to ensure that we conduct the finest,
science-based clinical trials. We had the opportunity to
communicate with Senior Leadership at the FDA and discuss how we
can work together to navigate the approval process forward along a
novel pathway. We appreciate their willingness and receptiveness to
consider innovative approaches as we all seek to better serve the
urgent unmet medical needs of the ALS community.”
Brian Wallach, Co-Founder of I AM ALS stated:
“There is nothing more important to those living with ALS than
having access to therapies that effectively combat this fatal
disease. We have been working with BrainStorm for months now
because we believe that NurOwn is a potentially transformative
therapy in this fight. We were privileged to represent the patient
voice at this meeting and are truly grateful to the company and the
FDA for this critical agreement. This is a truly important moment
of hope and we look forward to seeing both the Phase III data and
the hopeful approval of NurOwn as soon as is possible.”
About NurOwn® NurOwn® (autologous MSC-NTF cells)
represent a promising investigational approach to targeting disease
pathways important in neurodegenerative disorders. MSC-NTF cells
are produced from autologous, bone marrow-derived mesenchymal stem
cells (MSCs) that have been expanded and differentiated ex vivo.
MSCs are converted into MSC-NTF cells by growing them under
patented conditions that induce the cells to secrete high levels of
neurotrophic factors. Autologous MSC-NTF cells can effectively
deliver multiple NTFs and immunomodulatory cytokines directly to
the site of damage to elicit a desired biological effect and
ultimately slow or stabilize disease progression. NurOwn® is
currently being evaluated in a Phase 3 ALS randomized
placebo-controlled trial and in a Phase 2 open-label multicenter
trial in Progressive MS.
About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of
innovative autologous adult stem cell therapeutics for debilitating
neurodegenerative diseases. The Company holds the rights to
clinical development and commercialization of the
NurOwn® Cellular Therapeutic Technology Platform used to
produce autologous MSC-NTF cells through an exclusive, worldwide
licensing agreement as well as through its own patents, patent
applications and proprietary know-how. Autologous MSC-NTF cells
have received Orphan Drug status designation from the U.S.
Food and Drug Administration (U.S. FDA) and
the European Medicines Agency (EMA) in ALS. BrainStorm
has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056),
investigating repeat-administration of autologous MSC-NTF cells at
six sites in the U.S., supported by a grant from
the California Institute for Regenerative Medicine (CIRM
CLIN2-0989). The pivotal study is intended to support a BLA filing
for U.S. FDA approval of autologous MSC-NTF cells in ALS.
BrainStorm received U.S. FDA clearance to initiate a
Phase 2 open-label multi-center trial of repeat intrathecal dosing
of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718)
in December 2018 and has been enrolling clinical trial
participants since March 2019. For more information, visit the
company's website.
Safe-Harbor
Statement
Statements in this announcement other than
historical data and information, including statements regarding
future clinical trial enrollment and data, constitute
"forward-looking statements" and involve risks and uncertainties
that could cause BrainStorm Cell Therapeutics
Inc.'s actual results to differ materially from those stated
or implied by such forward-looking statements. Terms and phrases
such as "may", "should", "would", "could", "will", "expect",
"likely", "believe", "plan", "estimate", "predict", "potential",
and similar terms and phrases are intended to identify these
forward-looking statements. The potential risks and uncertainties
include, without limitation, BrainStorm’s need to raise additional
capital, BrainStorm’s ability to continue as a going concern,
regulatory approval of BrainStorm’s NurOwn® treatment candidate,
the success of BrainStorm’s product development programs and
research, regulatory and personnel issues, development of a global
market for our services, the ability to secure and maintain
research institutions to conduct our clinical trials, the ability
to generate significant revenue, the ability of BrainStorm’s
NurOwn® treatment candidate to achieve broad acceptance as a
treatment option for ALS or other neurodegenerative diseases,
BrainStorm’s ability to manufacture and commercialize the NurOwn®
treatment candidate, obtaining patents that provide meaningful
protection, competition and market developments, BrainStorm’s
ability to protect our intellectual property from infringement by
third parties, heath reform legislation, demand for our services,
currency exchange rates and product liability claims and
litigation,; and other factors detailed in BrainStorm's annual
report on Form 10-K and quarterly reports on Form 10-Q available
at http://www.sec.gov. These factors should be considered
carefully, and readers should not place undue reliance on
BrainStorm's forward-looking statements. The forward-looking
statements contained in this press release are based on the
beliefs, expectations and opinions of management as of the date of
this press release. We do not assume any obligation to update
forward-looking statements to reflect actual results or assumptions
if circumstances or management's beliefs, expectations or opinions
should change, unless otherwise required by law. Although we
believe that the expectations reflected in the forward-looking
statements are reasonable, we cannot guarantee future results,
levels of activity, performance or achievements.
CONTACTS
Corporate: Uri Yablonka Chief
Business Officer BrainStorm Cell Therapeutics Inc.
Phone: 646-666-3188 uri@brainstorm-cell.com
Media: Sean Leous Westwicke/ICR PR Phone:
+1.646.677.1839 sean.leous@icrinc.com
Or
Katie Gallagher | Account Director, PR and
MarketingLaVoieHealthScience Strategic CommunicationsO:
617-374-8800 x109M:
617-792-3937kgallagher@lavoiehealthscience.com
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