BrainStorm Cell Therapeutics Announces Publication of NurOwn® ALS Phase 2 Randomized Clinical Trial Data in Neurology
November 20 2019 - 7:00AM
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the
development of innovative autologous cellular therapies for highly
debilitating neurodegenerative diseases, today announced
publication of “NurOwn Phase 2 Randomized Clinical Trial in ALS:
Safety, Clinical and BioMarker Results,” in the international,
peer-reviewed journal Neurology: Volume 93, Number 24 (Published
ahead of print.)
The objective of the randomized
placebo-controlled Phase 2 clinical trial was to determine
the safety and efficacy of a single transplantation of autologous
bone-marrow derived MSC-NTF cells (NurOwn) in participants with
ALS. The clinical trial enrolled 48 participants randomized
3:1 (treatment: placebo) at three leading U.S. investigative sites:
Massachusetts General Hospital, Mayo Clinic and
University of Massachusetts Medical School. After a three-month
pre-transplant run-in period, participants received one dose of
MSC-NTF cells (n=36) or placebo (n=12) and were followed for
six-months. CSF was collected prior to and two-weeks
post-transplantation. The clinical trial confirmed that a single
transplantation of MSC-NTF (NurOwn) cells was safe and
well-tolerated. BrainStorm’s NurOwn cell therapy is the furthest
advanced autologous stem cell treatment in development for
ALS. On October 11, the Company announced that the NurOwn,
Phase 3 clinical trial for ALS was fully enrolled.
Key efficacy findings:
- The rate of ALS disease progression (ALSFRS-R slope) was
stabilized for up to 12-16 weeks in a pre-specified group of
participants with rapid progression (p<0.05) following a single
transplantation
- A higher proportion of MSC-NTF treated study participants
experienced ≥1.5 point/month improvement in ALSFRS-R slope at all
post-treatment time points, and this was statistically significant
at 4 and 12 weeks post-transplantation (p=0.004 and 0.046,
respectively)
- CSF neurotrophic factors increased and inflammatory biomarkers
decreased 2 weeks post-transplantation (p<0.05)
- CSF MCP-1 levels (a marker of microglial activation and
neuroinflammation) significantly decreased post-transplantation and
correlated with ALSFRS-R slope improvement at all time points
(p<0.05)
“The entire investigative team working on the
study are highly encouraged by the promising clinical results,”
said Phase 2 trial manuscript lead author Robert Brown DPhil, Leo
P. and Theresa M. LaChance Chair in Medical Research and Chair of
the Department of Neurology at the University of Massachusetts
Medical School (UMMS) and UMass Memorial Medical Center, “In
addition, we observed a clear biological effect of the treatment on
CSF biomarkers to support its proposed mechanism of action in
ALS.”
“This was a high quality Phase 2 study that
identified dosing, safety, and an important efficacy signal.
These data are exactly what is needed to move forward with the
current Phase 3 testing,” added 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. “These promising results encourage us
to complete the pivotal Phase 3 trial as rapidly as possible,” said
Dr. Anthony Windebank, Judith and Jean Pape Adams Foundation
Professor of Neuroscience at the Mayo Clinic.
“The team at BrainStorm and the clinical
investigators, led by Professors Brown, Cudkowicz and Windebank,
have made a significant contribution to ALS research and to the
peer reviewed-scientific literature with the publication of this
study in Neurology,” said Ralph Kern, MD, MHSc, Chief Operating
Officer and Chief Medical Officer of BrainStorm. He added, “We met
our primary endpoint and demonstrated that a single dose of NurOwn
was safe and well-tolerated while supporting NurOwn’s mechanism of
action on neuroprotection and neuroinflammation pathways in ALS. We
look forward to completing the current Phase 3 study to confirm the
promising Phase 2 findings and expand our understanding of the
potential of MSC-NTF cell therapy in ALS.”
“Having a major study peer-reviewed, accepted
and published by the highly respected journal Neurology is a
significant milestone for BrainStorm, and I thank the entire team
for the many hours they have contributed to the research and
publication process,” said Chaim Lebovits, President and CEO of
BrainStorm. “Results from the study underscore the importance of
conducting a larger Phase 3 clinical trial that will build upon the
data collected in our Phase 2 study. Our Phase 3 study has
recently reached full recruitment. All 200 ALS patients have
been enrolled. We look forward to reporting our clinical results in
the scientific literature and through corporate announcements.”
“We are pleased that the U.S. Phase 2 results of
NurOwn are published, and that the Phase 3 trial enrollment is now
complete,” said Dr. Neil Thakur, PhD, Executive Vice President for
Mission Strategy at the ALS Association. The initial testing of
NurOwn in ALS is promising. We stand ready to support rapid review
by the FDA of BrainStorm’s biologics license application of
NurOwn.”
Brian Wallach, Co-Founder of I AM ALS, said,
“The publication of this important Phase 2 data is a significant
milestone in the clinical development program for BrainStorm’s
NurOwn. I AM ALS supports the development of potentially
transformative therapies like NurOwn and shares in the hope that
when the Phase 3 is completed that it will be the first
disease modifying therapy for
ALS.”
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 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 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 filing for U.S. FDA approval of autologous
MSC-NTF cells in ALS. For more information, visit BrainStorm's
website at www.brainstorm-cell.com.
Safe-Harbor Statements
Statements in this announcement other than
historical data and information 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, risks associated
with BrainStorm's limited operating history, history of losses;
minimal working capital, dependence on its license to Ramot's
technology; ability to adequately protect the technology;
dependence on key executives and on its scientific consultants;
ability to obtain required regulatory approvals; 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.
BRAINSTORM CONTACTS: Investors: Uri Yablonka,
Chief Business Officer BrainStorm Cell Therapeutics Inc Phone: :
+1-201-488-0460 Email: uri@brainstorm-cell.com
Media: Sean Leous Westwicke/ICR PR Phone: +1.646.677.1839
Email: sean.leous@icrinc.com
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