- PrimeC demonstrated a clinically meaningful effect on
quality of life and on complication free-survival for patients with
ALS
- Standard ALS Measure ALSFRS-R already
demonstrated a statistically significant effect of PrimeC on
slowing down disease progression
- Further analysis of PARADIGM is on track with
neurofilament results expected in Q1 and TDP-43 and
ProstaglandinJ2 biomarkers expected in the first half of
2024
CAMBRIDGE, Mass., Feb. 21,
2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd.
(Nasdaq: NRSN) ("NeuroSense"), a company developing treatments for
severe neurodegenerative diseases, today reports additional
positive data from its six-month double-blind Phase 2b PARADIGM trial of NeuroSense's lead drug
candidate PrimeC for the treatment of amyotrophic lateral sclerosis
("ALS").
The PARADIGM trial's secondary clinical efficacy outcome measure
endpoints included Quality of Life and Survival. Consistent with
the previously reported results, PrimeC displayed a clinically
meaningful effect on various aspects of patients' quality of life,
including mental and physical health. Moreover, analyses pertaining
to survival outcomes emphasized the implications of PrimeC
intervention in mitigating disease progression and burden. PrimeC
achieved an improvement in complication-free survival compared to
placebo (in several methodologies, including MiToS and King's
Advanced Stage-free Survival), reducing the risk of ALS disease
complications or death by up to 53%. These survival analyses
observe time from participant randomization to death from any cause
or respiratory insufficiency (defined as tracheostomy or the use of
non-invasive mechanical ventilation for over 22 hours per day for
10 or more consecutive days), or time to hospitalization due to
ALS-related complications or advancements in MiToS or
King's stages.
"PrimeC's demonstrated positive effect on quality of life and
survival, aligned with the already positive clinical outcome
measures, in a relatively small Phase 2b clinical trial truly demonstrates its
potential to deliver a meaningful benefit," stated Ferenc Tracik,
M.D., NeuroSense's Chief Medical Officer. "From a clinical
perspective, these parameters are crucial to neurologists, but more
importantly to people living with ALS."
This news comes on the heels of the recent positive top-line
results reported in December 2023
from the PARADIGM trial in 68 participants, which met its
primary endpoint of safety and tolerability, establishing a robust
safety profile of PrimeC. In addition, PrimeC demonstrated a
statistically significant slowing of disease progression with a
37.4% (p=0.03) difference in the gold standard ALS tracking
measure, the ALS Functional Rating Scale-Revised ("ALSFRS-R"), in
favor of PrimeC vs placebo, and 17.2% (p=0.39) difference
in Slow Vital Capacity ("SVC") in favor of PrimeC vs placebo,
based on the pre-specified Per Protocol (PP) population
analysis. The PP analysis population includes all participants
who adhered to the trial protocol and treatment plan without any
major protocol deviations and includes 62 patients (43 active and
19 placebo).
"The positive impact of PrimeC on quality of life and
complication-free survival, together with its demonstrated ability
to meaningfully slow down disease progression, is of great value,
as we see that we have the potential to profoundly affect patients'
lives across the board. We are in great anticipation to soon report
on the status of our collaboration on neurofilaments and how this
may contribute to expediting the development of PrimeC," stated
Alon Ben-Noon, NeuroSense's CEO.
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
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 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.
An analysis of the ITT 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.
These data include all 68 people living with ALS enrolled
in Canada, Italy, and Israel, with the exclusion of
one patient who was misdiagnosed. Most patients enrolled in both
the active and placebo arms of 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 ALS Complication-free and MiTos/King's Advanced
Stage-free Survival Analyses
ALS Complication-free Survival is defined as time from
randomization to death from any cause, or respiratory insufficiency
(defined as tracheostomy or the use of non-invasive mechanical
ventilation for over 22 hours per day for 10 or more consecutive
days), or time to hospitalization due to ALS-related complications.
MiToS Advanced Stage-free Survival or King's Advanced Stage-free
Survival is defined as time from randomization to death from any
cause or respiratory insufficiency similarly, however, also
utilizes time to increase in MiToS stage or King's stage. MiToS
Advanced Stage is based on functional ability (ALSFRS-R) stages
from normal functionality to death, while King's stage is based on
advancements in disease burden (clinical involvement, feeding, or
respiratory failure).
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 the timing for release of neurofilament
results and TDP-43 and ProstaglandinJ2 biomarker results. 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 a delay in the reporting of
additional results from PARADIGM clinical trial, 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 or
its planned Phase 3 pivotal ALS trial of PrimeC; 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. 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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