- Meeting discussion to focus on finalizing Phase 3 study design
and NDA readiness, ensuring a clear regulatory path for
submission based on trial success
- Parallel preparations in Canada: NeuroSense plans to
submit its dossier in Q2 2025, with a regulatory decision on
commercialization expected by Q1 2026
CAMBRIDGE, Mass., Oct. 28,
2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd.
(NASDAQ: NRSN)
("NeuroSense"), a late-clinical stage biotechnology
company developing novel treatments for severe
neurodegenerative diseases, today announced it is scheduled
for a Type C meeting with the U.S. Food and Drug
Administration (FDA) to discuss the design of its upcoming Phase 3
clinical trial for PrimeC, a potential treatment
for Amyotrophic Lateral Sclerosis
(ALS).
The FDA Type C meeting, scheduled for November 6, 2024,
will focus on finalizing the design of the Phase 3 trial, a pivotal
step toward securing approval for the drug. NeuroSense will also
review its readiness for a future New Drug Application (NDA)
submission, ensuring its regulatory strategy aligns with the FDA's
requirements. NeuroSense plans to give an update on the results of
the meeting following receipt of the meeting minutes which are
expected approximately one month after the meeting.
PrimeC, NeuroSense's lead product, has shown promising results
in clinical trials, including a statistically significant reduction
in disease progression clinical and biological markers. With an
urgent unmet need in ALS, NeuroSense is positioning PrimeC as a
potential breakthrough therapy.
In parallel, NeuroSense plans to submit its regulatory dossier
to Health Canada in Q2 2025, with a regulatory decision anticipated
by Q1 2026. The Company estimates the potential market opportunity
for PrimeC in Canada alone to be
approximately $100 million to
$150 million in annual revenue.
This decision follows the recommendations of Canadian regulatory
experts and recent clinical findings, which demonstrated that
PrimeC significantly reduced disease progression (p=0.009) and
improved survival rates by 43% compared to placebo,
highlighting its potential as a breakthrough therapy for 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 regulatory filings and
regulatory decisions, the market opportunity in Canada and securing regulatory approval in
global markets. 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 of a delay in submission by the Company of
its regulatory dossier, that regulatory approvals for PrimeC
will be delayed or not obtained in Canada or elsewhere; that the market
opportunity in Canada will not be
as currently estimated; unexpected R&D costs or operating
expenses, insufficient capital to complete development of PrimeC, 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 and Health Canada 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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