NurOwn® Data Safety Monitoring Board Recommends ALS Phase 3 Clinical Trial Continue
October 28 2019 - 7:45AM
BrainStorm Cell Therapeutics, Inc. (NASDAQ: BCLI), a leading
developer of adult stem cell therapies for neurodegenerative
diseases, today announced that the Phase 3 clinical trial
independent Data Safety Monitoring Board (DSMB) has completed the
second, pre-specified interim analysis, of safety outcomes for the
first 106 patients who received repeat dosing of NurOwn® in the
Phase 3 trial for ALS patients (NCT03280056). The DSMB indicated
that the Phase 3 clinical trial should continue as planned.
“After reviewing all of the safety data as of
September 30th, the DSMB has recommended the study continue without
any changes in the protocol. We did not identify any significant
safety concerns,” said Carlayne Jackson, MD, FAAN Professor of
Neurology and Otolaryngology UT Health San Antonio, and the DSMB
Chairperson.
"We are very pleased with the DSMB
recommendation that the Phase 3 clinical trial continue without any
protocol modification. This represents an important clinical trial
advancement for BrainStorm and for the development of NurOwn as an
innovative cellular therapy approach for ALS patients. Enrollment
is now completed and top line data is expected in Q4 2020, which
will be used to support an FDA Biologics License Application," said
Ralph Kern MD, MHSc, BrainStorm Chief Operating Officer and Chief
Medical Officer.
BrainStorm is conducting a repeat dose,
randomized, placebo-controlled Phase 3 trial of NurOwn (200 ALS
participants randomized 1:1 to NurOwn or placebo) at 6 major US
clinical sites, supported by a grant from the California Institute
for Regenerative Medicine (CIRM CLIN2-0989). The study is now fully
enrolled and will generate top line data in Q4 2020.
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. 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
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