NEW YORK, June 2, 2020 /PRNewswire/ -- BrainStorm Cell
Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem
cell therapies for neurodegenerative diseases, announced today an
update on the Company's Phase 3 pivotal trial investigating
NurOwn® in ALS. The Company continues to expect that
top-line data from the trial will be announced by Q4-2020, despite
the ongoing COVID-19 pandemic that has delayed hundreds of other
clinical trials around the world.
Due to the importance of the clinical trial and the recognized
high unmet need in ALS, Brainstorm and the 6 prestigious U.S.
clinical sites have been able to continue treatment visits
throughout the COVID-19 pandemic with only occasional scheduling
changes to treatments. The decision to continue the trial is also
shared by the ALS community and the FDA. The clinical trial
sites are at University of California,
Irvine; Cedars-Sinai Medical Center; California Pacific
Medical Center; Massachusetts General
Hospital; University of Massachusetts Medical
School and Mayo Clinic. Importantly, the trial's
primary outcome measurement (the ALSFRS-R rating scale) is fully
validated for telephonic administration, which allowed
non-treatment visits to be conducted remotely and to be aligned
with protocol assessment timelines. This allows for ongoing
high-quality data collection while prioritizing safety of patients
and clinical trial site staff.
"Despite the challenges presented by COVID-19, patient dosing
remains on track with all participants having received at least 2
treatments of NurOwn®," said Dr. Merit
Cudkowicz, the Julieanne Dorn Professor of
Neurology at Harvard Medical
School and the Director of the Healey Center for ALS and
Chair of Neurology at Mass General Hospital. "Moving forward, only
20 patients are still to be dosed for the third and final time, and
these final treatments remain on schedule."
Chaim Lebovits, CEO of BrainStorm
stated, "BrainStorm made the decision to continue our Phase 3 trial
and we have been strongly supported by our partners in the clinical
community. Quick planning and coordination across our
clinical sites and the support and direction of the U.S. FDA
enabled our Phase 3 investigational trial for NurOwn to advance,
with final dosing by July 2020. While
numerous clinical trials in the U.S. have been interrupted or
completely shut down by the COVID-19 pandemic, trial participants,
their families, and the investigational teams remain committed to
the trial's advancement. I am extremely proud and grateful for the
outstanding work and commitment of so many, and particularly by
these trial participants and their caregivers. We hope to soon
reward their commitment by bringing forward a much-needed treatment
for ALS."
Ralph Kern, MD, MHSc,
BrainStorm's President and Chief Medical Officer said, "We are very
fortunate to have developed an outstanding team of partners who are
committed to BrainStorm's investigational therapy NurOwn and share
its promise in ALS research. Our primary concerns will always be
the overall health and safety of trial participants and the
scientific integrity of the trial. Fortunately, ALSFRS-R, the
primary outcome measurement of the Phase 3 trial, is validated by
phone and allows for the collection of quality data with minimal
risk of COVID-19 exposure. This, along with the collective efforts
of so many of our employees, partners, and patients, has enabled us
to remain on track for study completion as planned in the fourth
quarter of this year."
About NurOwn®
NurOwn® (autologous MSC-NTF) cells represent a promising
investigational therapeutic 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. BrainStorm has
fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells
for the treatment of amyotrophic lateral sclerosis (ALS).
BrainStorm also recently received U.S. FDA acceptance to initiate a
Phase 2 open-label multicenter trial in progressive MS and
enrollment began in March 2019.
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®
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 a
Phase 3 pivotal trial in ALS (NCT03280056), investigating
repeat-administration of autologous MSC-NTF cells at six U.S. sites
supported by a grant from the California Institute for Regenerative
Medicine (CIRM CLIN2-0989). The pivotal study is intended to
support a filing for U.S. FDA approval of autologous MSC-NTF cells
in ALS. BrainStorm also recently received U.S. FDA clearance to
initiate a Phase 2 open-label multicenter trial in progressive
Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells
in patients with progressive MS (NCT03799718) started enrollment in
March 2019. For more information,
visit the company's website at www.brainstorm-cell.com
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
Investor Relations:
Preetam
Shah, MBA, PhD
Chief Financial Officer
BrainStorm Cell Therapeutics Inc.
Phone: + 1.862.397.1860
pshah@brainstorm-cell.com
Media:
Sean Leous
Westwicke/ICR PR
Phone: +1.646.677.1839
sean.leous@icrinc.com
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SOURCE Brainstorm Cell Therapeutics Inc