NeuroSense to submit the 12-month data to FDA
to discuss path forward
CAMBRIDGE, Mass., July 9, 2024
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN)
("NeuroSense"), a late-clinical stage biotechnology company
developing novel treatments for severe neurodegenerative diseases,
announces additional positive results from its 12-month
PARADIGM Phase 2b study of PrimeC in
ALS patients. In the study, complication-free survival revealed a
57% difference in favor of PrimeC over placebo after 12 months
of treatment in the Intent to Treat (ITT) population and
demonstrated an outcome of 73% (p=0.02) difference in favor of
PrimeC over placebo after 12 months of treatment in the pre-defined
Per Protocol Population (PP). NeuroSense intends to submit
these 12-month results to regulatory agencies, including the FDA,
to discuss PrimeC's path forward in the short term.
Previously, NeuroSense reported that PrimeC significantly slowed
disease progression by 36% (p=0.009) and improved survival rates by
43% compared to placebo. The Per-Protocol analysis revealed even
greater benefits, with a 40% (p=0.003) improvement in ALSFRS-R
scores and a 63% increase in survival rates, representing a
distinguished advancement in outcomes for a controlled 12-month ALS
study.
Other positive clinical data points included Slow Vital Capacity
(SVC) improvement from a 13% difference after 6 months to a 20%
difference after 12 months in the ITT population, in favor of
PrimeC, and from a 17% difference after 6 months to a 19%
difference after 12 months in the PP population, in favor of
PrimeC. Furthermore, an analysis exploring the rate of decline,
defined as a drop of 4 points or less in the ALSFRS-R after 12
months, revealed a ratio of 4.5 to 1 in favor of PrimeC over
placebo. This outcome underscores the importance of early treatment
and the potential of PrimeC as a disease-modifying drug.
"The latest results from the PARADIGM study are incredibly
encouraging and provide compelling evidence of PrimeC's potential
to significantly benefit people living with ALS. The substantial
improvement in complication-free survival and the consistent
slowing of disease progression highlight PrimeC's promise as an
effective treatment. Given that, for many clinicians and people
with ALS, therapeutic options have recently been significantly
limited; the urgency for effective new therapies is greater than
ever. This development brings renewed hope to patients, and I am
eagerly looking forward to seeing the program enter Phase 3 to
advance the clinical development of this product," said
Jeremy M. Shefner, M.D., Ph.D.,
Professor of Neurology at the Barrow Neurological Institute,
Phoenix, Ariz., and a NeuroSense
advisor.
Alon Ben-Noon, NeuroSense's CEO,
commented: "We believe that these results are unprecedented in a
12-month ALS placebo-controlled clinical study. We are eager to
present them to the FDA and other regulatory agencies to determine
the path forward and to share the outcomes with potential partners
currently conducting due diligence. As a management team, we are
both confident and enthusiastic about the potential for PrimeC in
the market."
"It is equally important to recognize that our clinical team has
accomplished a great deal in a rather short time, and we are
grateful for their involvement in the development program, as well
as the study coordinators and teams, and of course, the study
participants and their families. We look forward to continued
communications on developments, as appropriate, including biomarker
readouts for target engagement, which we expect will shed
additional light on PrimeC's potential to benefit people who suffer
from ALS."
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 the timing of submissions to the
regulatory authorities and 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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