Groundbreaking PARADIGM Trial Offers New Hope for ALS
Treatment
CAMBRIDGE, Mass., Oct. 24,
2024 /PRNewswire/ -- NeuroSense Therapeutics
Ltd. (Nasdaq: NRSN) ("NeuroSense"), a late-clinical stage
biotechnology company developing novel treatments for severe
neurodegenerative diseases, today announced transformative findings
from its PARADIGM clinical trial of PrimeC. These results
illuminate a new frontier in the treatment of ALS (Amyotrophic
Lateral Sclerosis), a disease long considered untreatable at its
core. The trial demonstrating the significant impact of PrimeC on
the regulation of microRNAs (miRNAs), which play a key role in ALS
progression, providing compelling evidence of the drug's potential
to alter the underlying mechanisms of the disease.
ALS is a fatal disease, known for its relentless destruction of
motor neurons, leading to loss of muscle function, speech, and
eventually, the ability to breathe. Understanding the molecular
drivers of ALS is key to finding a way to slow or stop its
progression. These findings represent a breakthrough in that
understanding.
Key Findings:
- Regulation ofCritical miRNAs: The PARADIGM trial
revealed that PrimeC regulates specific miRNAs - key genetic
markers that control gene expression involved in ALS progression.
These miRNAs were unchanged in the placebo group, underscoring the
profound impact of PrimeC on ALS's pathological pathways. MicroRNAs
play a crucial role in regulating how genes express themselves, and
their dysregulation has long been linked to ALS. By restoring
balance to these genetic regulators, PrimeC offers a new method of
combating this devastating disease.
- Restoring miRNA Balance in ALS Treatment: PrimeC
enhances microRNA (miRNA) maturation, addressing the underlying
mechanisms of ALS. By influencing Dicer, the endonuclease that
processes precursor miRNA into active forms, PrimeC may restore the
balance of dysregulated miRNAs in ALS patients. This modulation is
thought to facilitate the production of functional miRNAs that
regulate gene expression, allowing PrimeC to target disrupted
genetic pathways in ALS and potentially slow disease progression
and improve patient outcomes. Clear Differentiation from
Placebo: Patients treated with PrimeC demonstrated consistent
effect in miRNA manifestation, with no similar changes seen in the
placebo group. This clear distinction offers compelling evidence of
PrimeC's potential as a disease modifying treatment, rather than a
symptomatic one.
Conclusion:
In conclusion, PrimeC demonstrated beneficial regulation of key
miRNAs, supporting its potential to engage critical genetic targets
involved in ALS progression. The 2-fold reduction of several miRNAs
following PrimeC treatment is particularly striking, offering both
a powerful biomarker for tracking ALS and a potential pathway for
new therapeutic strategies. This regulation of miRNAs underscores
PrimeC's capability to influence ALS at the regulatory level, where
previous treatments have struggled.
This work was done in collaboration with Professor Noam Shomron who is a world-leading scientist in
the field of genetics.
The results from the PARADIGM trial reflect NeuroSense
Therapeutics' dedication to developing treatments that go beyond
surface-level symptom management, aiming instead to address the
root causes of neurodegenerative diseases. PrimeC's ability to
regulate miRNAs represents an important step toward a future where
ALS patients have more therapeutic options.
For more information about NeuroSense Therapeutics and its
ongoing research, visit www.neurosense-tx.com.
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. 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 Prime C's potential to treat 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 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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