- The Type C meeting with the FDA, combined with the recent
18-month Phase 2b PARADIGM study
readout, has the Company on track to commence a Phase 3 study in
mid-2025
- PrimeC has already demonstrated a significant impact on
slowing disease progression and increasing survival rates in people
living with ALS
CAMBRIDGE, Mass., Dec. 11,
2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd.
(NASDAQ: NRSN) ("NeuroSense"), a late-clinical stage biotechnology
company developing novel treatments for severe
neurodegenerative diseases, today announced it has concluded a Type
C meeting with the U.S. Food and Drug Administration (FDA) for
PrimeC in the treatment of amyotrophic lateral sclerosis (ALS). The
purpose of the meeting was to discuss the design of a proposed
Phase 3 clinical study and the plan for submission of an eventual
505(b)(2) marketing application. The Company had a productive
discussion with the FDA regarding the design of the planned Phase 3
pivotal study with PrimeC, including efficacy and safety
measurements.
The FDA's positive feedback and guidance on overall trial design
marks a noteworthy achievement for NeuroSense, as alignment on the
design is a critical step in enabling the study to meet regulatory
expectations and potentially provide sufficient data for the drug's
approval.
In light of the FDA's feedback, NeuroSense plans to submit a
final protocol to the FDA during the first half of 2025 with the
aim of commencing enrollment of the pivotal Phase 3 study in
mid-2025, which would include approximately 300 patients divided by
a ratio of 2:1, PrimeC to placebo. The Phase 3 study is expected to
be a randomized, multi-center, multinational, prospective,
double-blind, placebo-controlled study, with an open label
extension (OLE), to evaluate the efficacy and safety of PrimeC in
people living with ALS. Following 12 months of treatment, it is
expected that all participants will transition to PrimeC for a
12-month OLE.
"The feedback from the FDA regarding our clinical strategy for
the planned pivotal Phase 3 study and the plan for submission of an
eventual marketing application, represents a significant milestone
in our drug development program. It validates the progress we've
made and reinforces our commitment to advancing a potential
therapeutic option for people living with ALS, whose need for
innovative treatments is urgent. This step brings us closer to
delivering a much-needed solution to the ALS community," stated
Alon Ben-Noon, CEO of
NeuroSense.
NeuroSense has already completed PARADIGM (NCT05357950), a
multinational, randomized, double-blind, placebo-controlled Phase
2b clinical trial of PrimeC in
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 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.
During the first 6 months of the trial, 45 participants were
randomized to receive PrimeC, and 23 participants were randomized
to receive placebo. This was followed by a 12-month open-label
extension with all participants receiving PrimeC in a blinded
manner, where neither the participants nor the clinical staff were
aware of the initial treatment allocation.
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 submission of
regulatory submissions to the FDA and the timing of
commencement of enrollment for clinical trials, if any, and
anticipated trial design. 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 commencement of
enrollment for the Phase 3 trial, if any, or a delay in regulatory
submissions with the FDA, the risk that the trial will not be
completed, meet regulatory expectations or provide sufficient data
for drug approval, unexpected changes in trial design, delay in
submission by the Company of its regulatory dossier, that
regulatory approvals for PrimeC will be delayed or not obtained in
the U.S., Canada or elsewhere;
unsuccessful results of the Phase 3 trial, 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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