Planned Phase 3b trial Designed
to Confirm Efficacy and Safety of NurOwn in
mild-to-moderate ALS patients
NEW
YORK, Feb. 27, 2024 /PRNewswire/ -- BrainStorm
Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of
adult stem cell therapeutics for neurodegenerative diseases, today
announced that it will present a poster (#M201) outlining the
proposed design for a Phase 3b trial
of NurOwn® in amyotrophic lateral sclerosis (ALS) at the MDA
Clinical and Scientific Conference, on Monday, March 4 between 6-8 p.m. at the Hilton Orlando Hotel,
Florida. The design is for a
two-part, multicenter, Phase 3b study
to assess the efficacy and safety of NurOwn in participants with
ALS. Eligible patients will initially enter a 24-week randomized,
double-blind, placebo-controlled period (Part A), followed by a
24-week open-label extension period (Part B). The entry criteria
will enroll people living with mild-to-moderate ALS. The primary
endpoint will be based on the Revised Amyotrophic Lateral Sclerosis
Functional Rating Scale (ALSFRS-R).
BrainStorm recently submitted a Special Protocol Assessment
(SPA) request to the U.S. Food and Drug Administration (FDA) for
this Phase 3b study of NurOwn. A SPA
agreement would indicate concurrence by FDA with the adequacy and
acceptability of the overall protocol design for the planned Phase
3b study, intended to support a
future marketing application. The details are currently under
review by the FDA and the company plans to finalize the details
following the receipt of input from the Agency.
"We are committed to doing everything we can to make NurOwn
available to ALS patients in need and we are pleased to present an
outline of our planned confirmatory Phase 3b trial," said Chaim Lebovits, President
and Chief Executive Officer of BrainStorm. "Together with our
advisors, we have designed a trial that we believe will generate
robust and conclusive data. Our goal is to develop an
efficacious treatment option that, if approved, can help patients
and generate value for our stakeholders."
Bob Dagher, MD, commented, "We
have given careful consideration to a Phase 3b trial that will conclusively demonstrate the
efficacy and safety of NurOwn and support a new NDA. Our
previous studies have provided us with valuable insight into the
NurOwn treatment effect. We believe that by incorporating these
learnings into the design of a confirmatory study we can maximize
the prospects of a successful outcome. The planned study will
enroll mild-to-moderate ALS patients, as we believe that this is
the appropriate patient population in which the course of the
disease can be accurately measured using ALSFRS-R. We are grateful
for the FDA's ongoing engagement and look forward to its feedback
and to finalizing the SPA."
The abstract for this presentation can be viewed here.
Brainstorm will post a copy of the poster to its corporate website
at the conclusion of the presentation.
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 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.
CONTACT:
Media:
Lisa Guiterman
Phone: +1 202-330-3431
lisa.guiterman@gmail.com
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SOURCE BrainStorm Cell Therapeutics Inc.