12-month data demonstrated regulation of iron levels, which
aligns with improved ALS survival and disease mitigation
Previous results showed PrimeC slowed disease progression by
36% and improved survival rates, highlighting its potential as a
transformative ALS therapy
CAMBRIDGE, Mass., Aug. 1, 2024
/PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN)
("NeuroSense"), a late-clinical stage biotechnology company
developing novel treatments for severe neurodegenerative diseases,
announces positive 12-month iron biomarker data from its Phase IIb
study (PARADIGM), which evaluated the safety and efficacy of PrimeC
in people living with Amyotrophic Lateral Sclerosis (ALS). This
data provides additional insights that align with the Company's
recent announcements of improved survival by 43% and slowed disease
progression by 36%.
ALS is a multifactorial disease in which iron and its related
proteins play a critical role in its pathophysiology. These new
results demonstrate the target engagement of PrimeC in iron
regulation, which is linked to disease mitigation and improved
survival. Iron metabolism is crucial in ALS pathology, with
transferrin and ferritin being significant contributors to the
progress of the disease. Iron accumulation in various brain regions
of ALS patients has been linked to neuronal damage. Elevated
ferritin levels, an indicator of iron storage, are consistently
observed in ALS patients and are associated with reduced survival
and potential oxidative stress. Conversely, lower levels of
transferrin, the primary iron transport protein, contribute to iron
dysregulation and disease progression.
The 12-month study results show a significant decrease in
ferritin levels and a corresponding increase in transferrin levels,
both indicating alleviation of the pathology. Iron levels remained
stable over the 12-month dosing period, with a mean difference of
4.536 µmol/L (95% CI [1.143, 7.929], p=0.01) compared to those who
started on placebo and transitioned to PrimeC after the initial
6-month double-blind phase.
These positive changes in iron metabolism align with improved
clinical outcomes. Patients on PrimeC maintained better
functionality and survival rates compared to those on placebo.
"The 12-month results from the PARADIGM Phase IIb study are
encouraging, showing slowing of disease progression and improved
survival outcomes," said Merit Cudkowicz, M.D., M.Sc., Chair of
Neurology and Director of the Sean M. Healey & AMG Center for
ALS at Massachusetts General Hospital. "This new analysis
highlights PrimeC's ability to regulate the iron panel in people
living with ALS, underscoring the drug's target engagement. These
findings strongly support the proceeding to phase 3 testing of
PrimeC in ALS."
The Company is currently compiling additional data to share with
the FDA for discussion to determine the clinical and regulatory
path forward. Additionally, the Company is continuing advanced
discussions with several potential development partners to explore
marketing opportunities following the potential completion and
approval of PrimeC 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.
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 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 the risk
that the Company will not enter into an agreement with a
development partner at all or on favorable terms; unexpected
R&D costs or operating expenses, a delay in the reporting of
additional results from PARADIGM clinical trial, a delay in a Phase
3 trial for PrimeC in ALS, 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.
Logo -
https://mma.prnewswire.com/media/1707291/NeuroSense_Therapeutics_Logo.jpg
View original
content:https://www.prnewswire.com/news-releases/neurosense-therapeutics-announces-positive-biomarker-data-from-als-phase-2b-clinical-trial-302212321.html
SOURCE NeuroSense