- PrimeC slowed disease progression by 43% (p=0.02) in
pre-specified high-risk ALS patients
- Consistent data across subgroups underscore the potential
of PrimeC to redefine the ALS treatment paradigm
CAMBRIDGE, Mass., May 7, 2024
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (Nasdaq: NRSN)
("NeuroSense"), a company developing novel treatments for severe
neurodegenerative diseases, announces new analyses from its
Phase 2b PARADIGM clinical trial,
demonstrated a statistically significant slowing of disease
progression in high-risk ALS patients treated with PrimeC by
43% (p=0.02) as compared to placebo in the
pre-specified per protocol (PP) population analysis after 6 months
of treatment. The slowing of disease progression demonstrated
by PrimeC versus placebo translates to a 5.04 points difference in
the ALSFRS-R in favor of PrimeC (Confidence Interval: 0.862, 9.214;
n=38).
High-risk patients, defined by the European Network for the Cure
of ALS (ENCALS) Risk Factor as those with a higher risk for rapid
disease progression, comprise approximately 50% of the total ALS
population.
"The impressive results of these new analyses raise further
enthusiasm for the potential impact of PrimeC on people with ALS.
With the magnitude of its effect and consistency across subgroups,
PrimeC, if approved following a Phase 3 trial, could significantly
improve the standard of care for people with ALS," commented
Jeremy M. Shefner M.D., Ph.D.,
Professor of Neurology at the Barrow Neurological Institute,
Phoenix, Ariz., and a NeuroSense
advisor.
In addition to the subgroup of high-risk patients, patients
treated with PrimeC who had symptoms for up to 12 months prior to
the baseline visit (newly diagnosed patients), showed a 52%
slowing of disease progression (p=0.008) versus placebo in
the PP population analysis. This translates to a 7.76-point
difference in the ALSFRS-R in favor of PrimeC (Confidence Interval:
2.27, 13.25; n=22). These subgroup analyses provide data suggesting
consistent, statistically significant effect of PrimeC on the range
of participants within the PARADIGM study.
In the Intent-to-treat (ITT) population, high-risk ALS patients
treated with PrimeC experienced a 31% (p=0.13) reduction in disease
progression, which translates to a 3.2 points difference in the
ALSFRS-R in favor of PrimeC (Confidence Interval: -1.03, 7.43;
n=41). In addition, in the group of participants who had symptoms
for up to 12 months prior to l the baseline visit (newly diagnosed
patients) experienced a 36% reduction (p=0.14), which translates to
a 4.56-point difference in the ALSFRS-R in favor of PrimeC
(Confidence Interval: -1.6, 10.72; n=25).
Additional results of the subgroup analysis of the effect of
PrimeC versus placebo on ALSFRS-R showed:
- Participants with a disease duration of up to 18 months (PP
analysis), PrimeC demonstrated a 38% change (p=0.054)
- Participants with a disease duration of up to 24 months (PP
analysis), PrimeC demonstrated 37% reduction in symptom scores
(p=0.047)
The Company plans to utilize these subgroup analyses to help
inform the design of the upcoming pivotal trial, which the Company
believes will increase its probability of success and its
cost-effectiveness.
Alon Ben-Noon, NeuroSense's CEO
commented, "We believe this is one of the most compelling results
seen to-date in an advanced, double-blind clinical trial in ALS,
demonstrating slowing the progression of ALS in patients in earlier
stages with an aggressive disease. Seeing PrimeC's
significant impact in the general PARADIGM population in all
various clinical aspects, and even more in multiple sub-groups on
the gold-standard ALSFRS-R, is truly gratifying, and we are looking
forward to analyzing and reporting on the 12-month study survival
data in the next few weeks."
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 people are diagnosed with ALS in the U.S. alone, with an
annual disease burden of $1 billion. The number of people
living with ALS is expected to grow by 24% by 2040 in the U.S. and
EU.
About ALSFRS-R
Disease progression is measured by the
ALS Functional Rating Scale-Revised (ALSFRS-R), which is the most
widely used ALS tracking tool accepted by the FDA, utilized by
neurologists treating ALS patients, in clinical trials, and by
other regulators to determine disease progression. It tracks 12
changes in a person's physical abilities over time including
functions such as: speech, walking, climbing stairs,
dressing/hygiene, handwriting, turning in bed, cutting food,
salivation, swallowing, and breathing. A single point change
on the ALSFRS-R has a significant impact on ALS patients, such as
the transition from independent feeding to requiring assistance or
independent breathing to needing to use a machine ventilator.
About PARADIGM
PARADIGM is a prospective,
multinational, randomized, double-blind, placebo-controlled
Phase 2b (NCT05357950) clinical trial
of PrimeC in ALS. The trial included 68 participants living with
ALS in Canada, Italy, and Israel. 96% of the trial participants who
completed the 6-month double-blind portion of the trial chose to
receive treatment with PrimeC through a 12-month open label
extension. Furthermore, to date all participants that completed the
18-month trial treatment duration, requested to continue PrimeC,
which is provided to them in an Investigator Initiated Trial, not
limited with time.
As previously reported, top-line data from the 6-month
double-blind segment of the trial showed clinically meaningful
signs of efficacy with a 29% difference in ALSFRS-R (p=0.12) and a
13% difference in SVC (p=0.5), both in favor of PrimeC vs placebo
in analysis of the intent to treat (ITT) population. These data
include all 68 people living with ALS enrolled
in Canada, Italy, and Israel, with the exclusion of
one patient who was misdiagnosed. In the PP top-line
analysis from PARADIGM, a statistically significant slowing of
disease progression was observed with a 37.4% (p=0.03) difference
in ALSFRS-R in favor of PrimeC vs placebo. Most patients enrolled
in both the active and placebo arms of the trial were concurrently
treated with Riluzole, the ALS standard of care medication,
indicating PrimeC slowed disease progression well beyond the level
afforded by the FDA approved ALS drug.
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 a Phase 2a clinical trial which met its safety
and efficacy endpoints including reducing functional and
respiratory deterioration and statistically significant changes in
ALS-related biological markers indicating PrimeC's biological
activity. 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 X.
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 and regarding the success and
cost-effectiveness of a pivotal trial. 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 the risk that PrimeC will not
improve the standard of care for people with ALS, that the pivotal
trials will occur and if it will occur will be successful or
cost-effective, unexpected R&D costs or operating expenses, the
timing of expected regulatory and business milestones, risks
associated with meeting with the FDA to determine the best path
forward following the results from PARADIGM clinical trial,
including a delay in any such meeting, a delay in patient
enrollment for a Phase 2 trial for Alzheimer's disease; the
potential for PrimeC to safely and effectively target AD;
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. 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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