NEW
YORK, Feb. 23, 2024 /PRNewswire/ -- BrainStorm
Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult
stem cell therapeutics for neurodegenerative diseases, today
announced that it has submitted a Special Protocol Assessment (SPA)
request to the U.S. Food and Drug Administration (FDA) for a Phase
3b study of NurOwn®, its
investigational treatment for amyotrophic lateral sclerosis
(ALS).
The submission follows an in-person meeting that BrainStorm held
with the FDA in December 2023. The
interactions leading up to this submission have provided guidance
on advancing the planned Phase 3b
trial. Participating in an SPA program would allow BrainStorm to
reach consensus with the FDA on its clinical trial design,
including the proposed statistical analysis, before the study is
initiated. This would help ensure that the study would be
considered adequate to support a future marketing evaluation. The
response is expected within 45 days of submission.
"Having an SPA in place is an important next step in advancing
our development program for NurOwn," said Chaim Lebovits, President and Chief Executive
Officer of BrainStorm. "We believe that the SPA will potentially
de-risk certain regulatory aspects of the program. We look forward
to working with the Agency to complete the SPA."
Stacy Lindborg, Ph.D., Co-CEO of
BrainStorm added, "BrainStorm remains deeply committed to the ALS
community. We continue to believe that NurOwn, if approved, has the
potential to make a substantial difference in the lives of those
affected by this devastating disease. We are grateful for the FDA's
ongoing guidance and look forward to finalizing the SPA of this
registration enabling trial."
Special Protocol Assessment
A Special Protocol Assessment (SPA) is a process in which drug
developers may ask to meet with the FDA to reach agreement on the
design and size of certain clinical trials to determine if they
adequately address scientific and regulatory requirements for a
study that could support marketing approval. An SPA agreement
indicates concurrence by FDA with the adequacy and acceptability of
specific critical elements of overall protocol design for a study
intended to support a future marketing application. These elements
are critical to ensuring that the trial conducted under the
protocol can be considered an adequate and well-controlled study
that can support marketing approval. Feedback on these issues
provides the greatest benefit to companies in planning late-phase
development strategy. An SPA agreement does not indicate FDA
concurrence on every protocol detail.
About NurOwn®
The NurOwn® technology platform (autologous MSC-NTF cells)
represents a promising investigational therapeutic approach to
targeting disease pathways important in neurodegenerative
disorders. MSC-NTF cells are harvested from each person with ALS
and are manufactured using an innovative and proprietary process to
secrete neurotrophic factors to target specific neurodegenerative
diseases. The lead program for NurOwn is for the treatment of
ALS. BrainStorm's long-term commitment to ALS is demonstrated
in preclinical research and a series of clinical studies, all of
which have been published in peer-reviewed journals.
The NurOwn clinical program has generated valuable insights into
the pathology of ALS, as well as disease progression and treatment.
Since the initial Phase 3 readout, BrainStorm has shared the full
dataset through rigorous peer-reviewed analysis, including:
quantification of Floor Effect, which had been noted, but never
before explored in depth; evaluation of multiple pre-specified
biomarkers, collected at seven different points across 20 weeks
during the trial, allowing a longitudinal view; and analysis of
genetic data, which represents one of the first ALS trials to
prospectively invoke pharmacogenomic analysis of clinical outcome,
offering great promise for the development of future treatments for
ALS.
About BrainStorm Cell Therapeutics
Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of
innovative autologous adult stem cell therapeutics for debilitating
neurodegenerative diseases. BrainStorm 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 designation status from the U.S. Food and
Drug Administration (FDA) and the European Medicines Agency (EMA)
for the treatment of amyotrophic lateral sclerosis (ALS).
BrainStorm has completed a Phase 3 trial in ALS (NCT03280056); this
trial investigated the safety and efficacy of repeat-administration
of autologous MSC-NTF cells and was supported by a grant from the
California Institute for Regenerative Medicine (CIRM CLIN2-0989),
and another grant from the ALS Association and I AM ALS. BrainStorm
completed under an investigational new drug application a Phase 2
open-label multicenter trial (NCT03799718) of autologous MSC-NTF
cells in progressive MS and was supported by a grant from the
National MS Society (NMSS).
Notice Regarding Forward-Looking
Statements
This press release contains "forward-looking statements" that are
subject to substantial risks and uncertainties, including
statements regarding meetings with the U.S. Food and Drug
Administration (FDA), Special Protocol Assessment (SPA), ADCOM
meeting related to NurOwn, the timing of a PDUFA action date for
the BLA for NurOwn, the clinical development of NurOwn as a therapy
for the treatment of ALS, the future availability of NurOwn to
patients, and the future success of BrainStorm. All statements,
other than statements of historical fact, contained in this press
release are forward-looking statements. Forward-looking statements
contained in this press release may be identified by the use of
words such as "anticipate," "believe," "contemplate," "could,"
"estimate," "expect," "intend," "seek," "may," "might," "plan,"
"potential," "predict," "project," "target," "aim," "should,"
"will" "would," or the negative of these words or other similar
expressions, although not all forward-looking statements contain
these words. Forward-looking statements are based on BrainStorm's
current expectations and are subject to inherent uncertainties,
risks and assumptions that are difficult to predict. These
potential risks and uncertainties include, without limitation,
management's ability to successfully achieve its goals,
BrainStorm's ability to raise additional capital, BrainStorm's
ability to continue as a going concern, prospects for future
regulatory approval of NurOwn, whether BrainStorm's future
interactions with the FDA will have productive outcomes, 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
Media:
Lisa Guiterman
Phone: +1 202-330-3431
lisa.guiterman@gmail.com
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SOURCE BrainStorm Cell Therapeutics Inc.