End of Phase 2 meeting with the FDA and EMA
expected in Q2 2024 and several biomarker study results anticipated
as early as Q1 2024, as the Company advances partnership
discussions
CAMBRIDGE, Mass., Jan. 9, 2024
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN)
("NeuroSense"), a company developing treatments for severe
neurodegenerative diseases, shares a review of important clinical
achievements in 2023 and provides a road map for anticipated
catalysts in 2024.
Statistically Significant Reduction in ALS Disease
Progression in Phase 2b PARADIGM
Trial
Top-line results in the Phase 2b
PARADIGM trial of PrimeC revealed a statistically
significant slowing of disease progression in ALS patients,
compared to placebo. The study demonstrated a 37.4% (p=0.03)
difference in the FDA-approved ALS functional rating scale revised
(ALSFRS-R), compared to placebo, and a strong safety profile. These
data highlight a novel approach to treating ALS, with potential
implications in other neurodegenerative conditions.
PrimeC is a unique fixed-dose combination of
ciprofloxacin and celecoxib, which may utilize complementary and
synergistic mechanisms to preserve motor neuron health in ALS.
Jeffrey Rosenfeld, MD, PhD,
Professor of Neurology at Loma Linda
University and member of NeuroSense's Scientific Advisory
Board, stated that the magnitude of this apparent improvement is
especially noteworthy considering the natural history of ongoing
disease progression. Furthermore, Prof. Rosenfeld commented that
the most exciting aspects of these data include not only the
statistical differences in ALSFRS-R score at the specified
timepoints, but also that the improved trajectory of the ALSFRS-R
decline was evident from the earliest timepoint, throughout the
entire study period. "These data provide an exciting justification
and indication for a pivotal Phase 3 trial. The complexity of ALS
pathology warrants a multi-drug therapeutic strategy and it is
especially gratifying to see this combination therapy advance. The
pending data on biomarkers of neurodegeneration will also be of
great interest, as we continue to better understand the benefits of
PrimeC and promote this approach."
"The clinical efficacy seen in the topline PARADIGM trial
results demonstrate PrimeC's potential to render a significant and
meaningful clinical benefit to people living with ALS, as any
slowing of progression of this incurable disease is meaningful from
both a clinical and patient perspective. This is perhaps one of the
most significant outcomes seen to date. We believe that these data
in conjunction with hopefully correlative neurofilament
readouts this month could lead to a strategic partnership and an
expedited regulatory pathway for PrimeC towards the market," stated
Alon Ben-Noon, NeuroSense's CEO.
In line with the Phase 2b
findings, an independent study performed at the Ichida Stem Cell
Lab at University of Southern
California in 2023, showed PrimeC performed among the best
in improving motor neuron survival when compared to two FDA
approved ALS drugs as well as several other ALS drugs in
development in an in vitro study utilizing induced
pluripotent stem cells (iPSCs).
Primary Biomarker Endpoints Anticipated in 2024
NeuroSense expects to report results from a strategic collaboration
with Biogen in the second half of January, evaluating the impact of
PrimeC on neurofilament levels in participants enrolled in
PARADIGM. Upon receipt of results, Biogen has the right of first
refusal to co-develop/commercialize PrimeC for the treatment of ALS
for a limited time. The Company also expects to report primary
endpoints of ALS hallmark biomarkers, TDP-43 and ProstaglandinJ2,
to evaluate PrimeC's biological activity and target engagement, in
the first half of 2024.
Next Steps in PrimeC Development Pathway
The FDA has confirmed NeuroSense's chemistry,
manufacturing, and controls (CMC) development plans, in advance of
an expected Phase 3 pivotal trial and potential subsequent
marketing approval. The Company expects End of Phase 2 meetings to
be scheduled with the FDA and the European Medicines Agency for Q2
2024.
Further advancing its platform combination drug therapy
platform, in 2023 NeuroSense also reported positive biomarker
results in Parkinson's and Alzheimer's Disease (AD). The Company
recently commenced enrollment in RoAD, its Phase 2, randomized,
prospective double-blind, single-center, placebo-controlled study
to evaluate safety, tolerability, target engagement, and efficacy
of PrimeC in patients with mild to moderate AD.
Finally, driven by the significant milestones achieved during
the year, NeuroSense has entered into advanced discussions with
fundamental biotech VCs and several big pharma companies focused on
CNS therapeutics, to explore potential strategic partnerships and
business opportunities.
"I thank our devoted team, invaluable collaborators, and the
inspiring ALS community for their dedication and support in
reaching this crucial milestone in 2023 and positioning the Company
for success in 2024. We remain, as always, committed to the
development of improved approaches to treat serious neurological
disorders. These efforts are not just about advancing medical
science; they are about improving patient's lives. Our focus
continues to be on delivering optimal value to our shareholders and
stakeholders and making a meaningful difference in the healthcare
landscape," added Ben-Noon.
About ALS
Amyotrophic lateral sclerosis ("ALS") is an incurable
neurodegenerative disease that causes complete paralysis and death
within 2-5 years from diagnosis. Every year, more than 5,000
patients are diagnosed with ALS in the U.S. alone, with an annual
disease burden of $1 billion. The number of patients with ALS
is expected to grow by 24% by 2040 in the U.S. and EU.
About PrimeC
PrimeC, NeuroSense's lead drug candidate, is a novel
extended-release oral formulation composed of a unique fixed-dose
combination of two FDA-approved drugs: ciprofloxacin and celecoxib.
PrimeC is designed to synergistically target several key mechanisms
of ALS that contribute to motor neuron degeneration, inflammation,
iron accumulation and impaired ribonucleic acid ("RNA") regulation
to potentially inhibit the progression of ALS. NeuroSense completed
the 6-month double blind portion of PARADIGM, a prospective,
multinational, randomized, double-blind, placebo-controlled
Phase 2b ALS (NCT05357950) clinical
trial, which met its safety and tolerability endpoints, as well as
showing a statistically significant slowing of disease progression
in the pre-specified Per Protocol (PP) population. Additional data
from the Phase 2b study are expected
H1 2024. PrimeC was granted Orphan Drug Designation by the U.S.
Food and Drug Administration and the European Medicines Agency.
About NeuroSense
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology
company focused on discovering and developing treatments for
patients suffering from debilitating neurodegenerative diseases.
NeuroSense believes that these diseases, which include amyotrophic
lateral sclerosis (ALS), Alzheimer's disease and Parkinson's
disease, among others, represent one of the most significant unmet
medical needs of our time, with limited effective therapeutic
options available for patients to date. Due to the complexity of
neurodegenerative diseases and based on strong scientific research
on a large panel of related biomarkers, NeuroSense's strategy is to
develop combined therapies targeting multiple pathways associated
with these diseases.
For additional information, we invite you to visit
our website and follow us
on LinkedIn and Twitter.
Forward-Looking Statements
This press release contains "forward-looking statements" that
are subject to substantial risks and uncertainties. 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 NeuroSense
Therapeutics' current expectations and are subject to inherent
uncertainties, risks and assumptions that are difficult to predict
and include statements regarding PrimeC as a potential treatment
for people with ALS, the timing for release of additional results
from PARADIGM clinical trial, and other regulatory milestones, the
timing for release of results from the Company's strategic
collaboration with Biogen, the timing for reporting primary
endpoints of ALS hallmark biomarkers to evaluate PrimeC's
biological activity and target engagement, the timing of meetings
to be scheduled with the FDA and the European Medicines Agency, the
timing of a Phase 2 trial for Alzheimer's disease and patient
enrollment regarding a Phase 3 pivotal ALS trial of PrimeC.
Further, certain forward-looking statements are based on
assumptions as to future events that may not prove to be accurate.
The future events and trends may not occur and actual results could
differ materially and adversely from those anticipated or implied
in the forward looking statements. These risks include unexpected
R&D costs or operating expenses, a delay in the reporting of
additional results from PARADIGM clinical trial or the ALS hallmark
biomarkers, a delay in the reporting of results from the Company's
strategic collaboration with Biogen, the timing of expected
regulatory and business milestones, risks associated with meetings
with the FDA and the European Medicines Agency to determine the
best path forward following the results from PARADIGM clinical
trial, including a delay in any such meetings, a delay in patient
enrollment for a Phase 2 trial for Alzheimer's disease or its
planned Phase 3 pivotal ALS trial of PrimeC; the inability to enter
into a strategic partnership; the potential for PrimeC to safely
and effectively target ALS; preclinical and clinical data for
PrimeC; the uncertainty regarding outcomes and the timing of
current and future clinical trials; timing for reporting data; the
development and commercial potential of any product candidates of
the company; and other risks and uncertainties set forth in
NeuroSense's filings with the Securities and Exchange Commission
(SEC). You should not rely on these statements as representing our
views in the future. More information about the risks and
uncertainties affecting the Company is contained under the heading
"Risk Factors" in the Annual Report on Form 20-F filed with the
Securities and Exchange Commission on March
22, 2023 and the Company's subsequent filings with the SEC.
Forward-looking statements contained in this announcement are made
as of this date, and NeuroSense Therapeutics Ltd. undertakes no
duty to update such information except as required under applicable
law.
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