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NeuroSense Expands its Phase 2b ALS PARADIGM Trial to Canada and Withdraws Protocol from U.S. IND to Align its Clinical Strategy with the FDA for a Potential Pivotal Phase 3 Study Receives regulatory approval from Health

Key Takeaway: NeuroSense Therapeutics has expanded its Phase 2b ALS PARADIGM trial to Canada following approval from Health Canada, allowing for patient enrollment. As over 50% of patients have been enrolled, topline results are anticipated later this year. However, the company has withdrawn its protocol from the U.S. Investigational New Drug application in response to FDA requests for additional non-clinical data, which will alter its clinical strategy aimed at a future pivotal Phase 3 study. This decision highlights both the progress in Canada and the regulatory challenges faced by the company.

Market Sentiment Analysis

POSITIVE FACTORS

  • Health Canada approved patient enrollment for the ALS PARADIGM trial.
  • Over 50% of patients are already enrolled in the trial.
  • CEO expressed optimism about moving forward with U.S. FDA alignment.

CONCERNS & RISKS

  • The U.S. FDA has requested additional non-clinical data, causing delays.
  • Protocol withdrawal from U.S. IND may affect patient recruitment timelines.

Full Press Release Details

NeuroSense Expands its Phase 2b ALS PARADIGM
Trial to Canada and Withdraws
Protocol from U.S. IND to Align its Clinical Strategy with the FDA for a Potential Pivotal
Mass., Feb. 6, 2023 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a company developing
treatments for severe neurodegenerative diseases, today announced that Health Canada approved the commencement of patient enrollment
in Canada for the Company's Phase 2b PARADIGM study of its combination therapy PrimeC for the treatment of amyotrophic lateral sclerosis
(ALS). Currently, over 50% of patients have been enrolled in PARADIGM with topline results expected in the second half of 2023.
Dr. Christen Shoesmith, Medical Director of the
London Health Sciences Centre ALS Clinic and Principal Investigator of the PARADIGM trial in Canada, commented, "We are very pleased
with Health Canada's approval to recruit and dose people living with ALS in the PARADIGM study. Canada is at the forefront of advancing
promising treatments to provide much needed options for people living with ALS."
The U.S. Food and Drug Administration (FDA) requested
additional non-clinical data from NeuroSense to support the duration of the PARADIGM trial, as PrimeC is intended for long-term administration
for the treatment of ALS. As a result, and following alignment with the agency on this subject, NeuroSense and the FDA have agreed that
NeuroSense will withdraw its study protocol from the Investigational New Drug application (IND). NeuroSense is planning a formal meeting
with the FDA to align its clinical and regulatory strategy for a potential pivotal Phase 3 trial that is intended to support a New Drug
Application (NDA) submission, pending the successful conclusion of the PARADIGM trial.
"Our North American clinical development strategy
got a boost from Health Canada, which approved our Phase 2b PARADIGM study to dose patients," stated NeuroSense CEO Alon Ben-Noon.
"In the interest of treating and completing dosing of people living with ALS in an expedient and safe manner through PARADIGM, we've
focused our North American recruitment in Canada. We look forward to working with the FDA on a path that includes clinical sites
in the U.S. in a future Phase 3 pivotal study of PrimeC to address the dire unmet need of the U.S. ALS community who are eager to receive
PrimeC for long term use."
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 amyotrophic lateral sclerosis (ALS) that contribute to motor neuron
degeneration, inflammation, iron accumulation and impaired RNA regulation to potentially inhibit the progression of ALS. NeuroSense completed
a Phase IIa clinical study which successfully 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. Through a collaboration
with Massachusetts General Hospital in Boston on novel Neuron-Derived Exosomes (NDEs), NeuroSense is working to further determine
the biological changes in ALS-related pathologies and the effect of PrimeC on relevant targets. PrimeC was granted Orphan Drug Designation
by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
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 24% by 2040 in the U.S. and EU.
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
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 patent applications; the company's PrimeC development program; the potential
for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; the timing of current and future clinical trials;
the nature, strategy and focus of the company and further updates with respect thereto; and the development and commercial potential of
any product candidates of the company. Further, certain forward-looking statements are based on assumptions as to future events that may
not prove to be accurate. Such risks and uncertainties include the risk that there will a delay in the timing of the report of topline
results of PARADIGM and other risks and uncertainties set forth in the Company's filings with the Securities and Exchange Commission (SEC),
including the Company's Annual Report on Form 20-F filed with the SEC on April 14, 2022. Further, certain forward-looking statements are
based on assumptions as to future events that may not prove to be accurate. 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
For further information: Email: info@neurosense-tx.com,
Tel: +972 (0)9 799 6183

Frequently Asked Questions

What is the Phase 2b PARADIGM study?

The Phase 2b PARADIGM study evaluates NeuroSense's PrimeC for treating ALS.

Where is the PARADIGM study being conducted?

Patient enrollment for the PARADIGM study has begun in Canada.

What request did the FDA make regarding the trial?

The FDA requested additional non-clinical data for the PARADIGM trial's duration.

What is PrimeC's formulation?

PrimeC is a combination of ciprofloxacin and celecoxib in an extended-release format.

What designation did PrimeC receive?

PrimeC was granted Orphan Drug Designation by both the FDA and EMA.

Last updated: Feb 6, 2023