- Top-Line Results from 6-month double-blind Phase
2b PARADIGM trial with
NeuroSense's lead drug candidate for ALS, PrimeC,
include:
- PrimeC achieved primary safety and tolerability endpoints
with a safety and tolerability profile comparable to
placebo
- Meaningful slowing of disease progression: 29% difference
(P=0.12) in favor of ALSFRS-R outcome for patients treated with
PrimeC vs placebo
- Slow Vital Capacity: observed a positive trend in favor
of patients treated with PrimeC compared to placebo
- Neurofilament biomarker results from Biogen collaboration
expected in January 2024
- Primary biomarker endpoints and exploratory biomarkers
from this trial, expected in H1 2024
- Company to host webcast conference call to present data
today at 8:30 AM EST
CAMBRIDGE, Mass., Dec. 5, 2023
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (Nasdaq: NRSN)
("NeuroSense"), a company developing treatments for severe
neurodegenerative diseases, today reported that it met its primary
safety and tolerability endpoints and achieved secondary clinical
efficacy endpoints in the top-line results of its randomized,
placebo-controlled, double-blind segment the Company's Phase
2b amyotrophic lateral sclerosis
("ALS") trial of PrimeC ("PARADIGM"). The trial's secondary
clinical efficacy outcome measure endpoints included Amyotrophic
Lateral Sclerosis Functional Rating Scale-Revised ("ALSFRS-R") and
Slow Vital Capacity ("SVC"), a measure of respiratory function.
NeuroSense expects to report an additional primary biomarker
endpoint of the assessment of ALS hallmarks, TDP-43 and
Prostagladin2, to evaluate PrimeC's biological activity, in the
first half of 2024 following the completion of the analysis of
participants' plasma.
The PARADIGM (NCT05357950) trial is a prospective,
multinational, randomized, double-blind, placebo-controlled
Phase 2b clinical trial of PrimeC in
ALS. Participants living with ALS in Canada, Italy, and Israel were enrolled and dosed for 6 months
after being randomized 2:1 to receive PrimeC or placebo,
respectively. After completion of the 6-month double-blind segment,
the participants had the option to enroll in a 12-month open label
extension (OLE), during which they all receive treatment with
PrimeC.
Over the course of 6 months, patients treated with PrimeC
experienced a slowing of disease progression, including preserved
daily function. The clinical efficacy results showed a 29%
difference in favor of PrimeC in ALSFRS-R (p=0.12) and a 13%
difference in favor of PrimeC in SVC (p=0.5) based on data
from 68 out of 69 patients, due to 1 misdiagnosed participant.
These data include 45 patients randomized to the PrimeC arm and 23
patients randomized to the placebo arm, from the 6-month
double-blind segment. The safety and tolerability profile of PrimeC
was comparable to the placebo. Nearly all participants, 96%, who
completed the 6-month double-blind portion of the trial chose to
receive treatment with PrimeC through the 12-month open label
extension. To date, all the participants who already completed the
18-month trial have opted to continue treatment of PrimeC by
joining a subsequent investigator-initiated trial.
NeuroSense expects to report results from a strategic
collaboration with Biogen in January
2024, evaluating the impact of PrimeC on neurofilament
levels in participants enrolled in PARADIGM. Upon receipt of
results, Biogen has the right of first refusal to
co-develop/commercialize PrimeC for the treatment of ALS for a
limited time following the results.
"The clinical advancement of a new therapy that helps slow down
the progression of ALS, with the potential to preserve quality of
life, has the capacity to significantly positively impact people
living with ALS and their families. While the ALSFRS-R and SVC
results are Phase 2 data and were not powered for statistical
significance, the positive results support moving forward to a
Phase 3 pivotal trial. The biomarker data will also be very
informative, and I look forward to seeing those results in early
2024," stated Merit Cudkowicz, M.D., Chair of Neurology at
Massachusetts General Hospital, the Director of the Healey &
AMG Center for ALS, Julieanne Dorn Professor of Neurology at
Harvard Medical School, and member of
NeuroSense's Scientific Advisory Board. "I am excited by the
top-line clinical data from PARADIGM, as this is an important
milestone for the patients I care for and for the entire ALS
community."
"The release of this portion of the top-line results of the
PARADIGM trial marks an exciting milestone for NeuroSense as we
take another step toward helping people suffering from this dire
disease. We look forward to meeting with the FDA to determine the
best path forward and to advancing discussions with strategic
partners who share our vision for PrimeC to benefit people living
with ALS," stated Alon Ben Noon, CEO
of NeuroSense. "I would like to thank my devoted team and everyone
who made this possible, the trial participants, their caregivers
and families, as well as the sites' principal investigators and
trial coordinators for their tremendous contribution to
PARADIGM."
"In addition to the safety and tolerability profile observed, we
believe the 29% difference observed in ALSFRS-R in favor of PrimeC
as well as the 13% decline in SVC compared to the placebo arm,
illustrate PrimeC's potential to render a meaningful clinical
benefit to people living with ALS," stated Dr. Ferenc Tracik,
NeuroSense's Chief Medical Officer. "With the trial powered to
determine statistically significant changes in ALS biomarkers, we
look forward to the additional primary endpoint data in the first
half of 2024."
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 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.
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, the timing for release of additional results
from PARADIGM clinical trial, the timing for release of results
from the Company's strategic collaboration with Biogen, the cash
runway of the Company, the timing of a Phase 2 trial for
Alzheimer's disease and patient enrollment regarding a Phase 3
pivotal ALS trial of PrimeC. 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, a delay in the reporting of results from the
Company's strategic collaboration with Biogen, 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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