- Disease progression was slowed by 36% (p=0.009) in
participants who received PrimeC for 12 months compared to those
who initially received a placebo
- Consistent data across multiple endpoints underscore the
potential of PrimeC to redefine the ALS treatment
paradigm
- NeuroSense planning for Phase 3 clinical study in the
U.S. and Europe
CAMBRIDGE, Mass., July 1, 2024
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a
late-clinical stage biotechnology company developing novel
treatments for severe neurodegenerative diseases, today reported
statistically significant results from the 12-month data analysis
of the PARADIGM Phase 2b study
evaluating PrimeC in people living with Amyotrophic Lateral
Sclerosis (ALS).
The data show a significant improvement in the rate of decline
of ALS Functional Rating Scale-Revised (ALSFRS-R) scores and
survival rates for subjects who received PrimeC from the start of
the trial compared to those who started on placebo. Specifically,
the intent to treat (ITT) analysis of the study at 12 months
revealed a difference of 6.5 points in the ALSFRS-R, which
represents a 36% improvement and a highly statistically significant
P value of 0.009.
In addition, at 12 months participants on PrimeC demonstrated
better survival than those initially on placebo, by 43%.
In an additional pre-defined analysis of the Per-Protocol
Population at 12 months, the results showed an even greater effect,
with a difference of approximately 7.7 points (p=0.003) between the
groups, translating to more than 40% improvement for participants
who received PrimeC from the start compared to those on placebo.
Furthermore, this analysis indicated that the survival rate of
participants on PrimeC improved by 63% compared to participants who
received a placebo.
"These exciting long-term results demonstrate how study
participants experienced more slowing of progression over time with
PrimeC as measured against ALSFRS-R, which is the current
gold-standard scale used in ALS drug development," said Merit
Cudkowicz, M.D., M.Sc., chair of neurology and Director of the Sean
M. Healey & AMG Center for ALS at Massachusetts General
Hospital. "The need for new treatments for people living with ALS
has never been greater. PrimeC has great potential based on its
mode of action and the phase 2 trial results and warrants further
evaluation in a Phase 3 trial in an expeditious manner."
Vivian Drory, MD, Head of the ALS
clinic at Tel-Aviv Sourasky Medical Center, added: "The promising results from the
12-month PARADIGM study highlight the significant potential of
PrimeC as a disease-modifying drug for ALS. These findings
underscore the importance of early intervention, which can lead to
more substantial benefits, and provide valuable insights that will
inform the design of the Company's Phase 3 study, increasing the
likelihood of success."
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 (June
2024) 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, in the 6-month double-blind segment of
the trial, the data showed clinically meaningful signs of efficacy
with a 29% difference in favor of PrimeC vs placebo in analysis of
the intent to treat (ITT) population. 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,
YouTube and X. Information that may be important to
investors may be routinely posted on our website and these social
media channels.
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. 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, 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; 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
Neurosense; the ability of NeuroSense to remain listed on Nasdaq;
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
NeuroSense is contained under the heading "Risk Factors" in the
Annual Report on Form 20-F filed with the Securities and Exchange
Commission on April 4, 2024 and
NeuroSense's subsequent filings with the SEC. Forward-looking
statements contained in this announcement are made as of this date,
and NeuroSense undertakes no duty to update such information except
as required under applicable law.
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