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NeuroSense Receives U.S. FDA Confirmation of CMC Strategy for PrimeC in Preparation for Pivotal Phase 3 in ALS and Commercial Readiness Clinical efficacy top-line results of PARADIGM, a Phase 2b trial in ALS, expected in

Key Takeaway: NeuroSense Therapeutics has received confirmation from the U.S. FDA regarding its chemistry, manufacturing, and controls strategy for the drug PrimeC, which is being developed for amyotrophic lateral sclerosis (ALS). This confirmation is pivotal as it allows NeuroSense to progress toward a Phase 3 clinical trial. The current Phase 2b trial, PARADIGM, involves 69 ALS patients across multiple countries, and the company anticipates results for secondary endpoints soon. PrimeC has also been granted Orphan Drug Designation, enhancing its regulatory support as NeuroSense continues to focus on the urgent need for effective ALS treatments.

Market Sentiment Analysis

POSITIVE FACTORS

  • FDA confirmation of CMC strategy is a significant milestone.
  • NeuroSense is moving towards a pivotal Phase 3 clinical trial.
  • PrimeC showing potential with high participant interest in open-label extension.
  • Orphan Drug Designation received, indicating regulatory support.

CONCERNS & RISKS

  • Potential delays in reporting Phase 2b trial results.
  • Risks of failing to meet clinical trial endpoints.
  • Commercial potential and effectiveness of PrimeC still unproven.

Full Press Release Details

NeuroSense Receives U.S. FDA Confirmation of CMC Strategy for PrimeC in Preparation for Pivotal Phase 3 in ALS and Commercial Readiness
CAMBRIDGE, Mass., Nov. 13, 2023 /PRNewswire/ -- NeuroSense
Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a company developing treatments for severe neurodegenerative diseases, today
announced it has concluded a successful Type D meeting with the U.S. Food and Drug Administration (FDA) for PrimeC in the treatment of
amyotrophic lateral sclerosis (ALS). FDA Type D meetings are focused on a narrow set of issues at key decision points to provide timely
feedback critical to move a drug development program forward. The purpose of NeuroSense's meeting with the FDA was to discuss
PrimeC's chemistry, manufacturing, and controls (CMC) development plans in advance of an expected Phase 3 pivotal study and potential
subsequent marketing approval. The FDA agreed with NeuroSense's proposed CMC development plan. PrimeC has already been granted Orphan
Drug Designation by the FDA and the European Medicines Agency (EMA).
"This confirmation from the FDA of our CMC strategy for a future
pivotal Phase 3 clinical trial and commercial launch of PrimeC in the treatment of ALS marks a critical milestone in our drug development
program. It sets the stage for a smooth transition, particularly as we anticipate clinical efficacy top-line results (secondary endpoints)
from our Phase 2b trial very soon," stated Alon Ben-Noon, CEO of NeuroSense.
NeuroSense is currently conducting PARADIGM (NCT05357950), a multinational,
randomized, double-blind, placebo-controlled Phase 2b clinical trial of PrimeC in ALS. Sixty-nine people living with ALS in Canada, Italy,
and Israel have been enrolled into PARADIGM, which aims to assess PrimeC's efficacy, as well as safety and tolerability. Primary
and secondary endpoints of the study include assessment of ALS-biomarkers, evaluation of clinical efficacy, and improvement in quality
of life. In the double-blind segment trial participants were dosed for 6 months after being randomized 2:1 to receive PrimeC or placebo,
respectively. After completion of the 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. 96% of the participants chose to continue into the OLE.
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.
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. PrimeC was granted Orphan Drug
Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
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
For additional information, we invite you to visit our website and
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 for release of results from the double-blind segment
of the Company's Phase 2b trial and regarding an expected Phase 3 pivotal study. 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
a delay in the reporting of clinical top-line results from PARADIGM clinical trial, the failure to meet the primary or secondary endpoints
of the trial, 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 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.

Frequently Asked Questions

What is PrimeC's role in treating ALS?

PrimeC targets multiple mechanisms contributing to ALS progression, aiming to inhibit motor neuron degeneration.

What recent FDA confirmation did NeuroSense receive?

The FDA confirmed NeuroSense's CMC strategy for PrimeC before its Phase 3 study.

What is the Orphan Drug Designation for PrimeC?

PrimeC has been granted Orphan Drug Designation by the FDA and EMA for ALS treatment.

How many participants are in the Phase 2b trial?

The PARADIGM trial has enrolled 69 ALS patients from Canada, Italy, and Israel.

What is the focus of NeuroSense Therapeutics?

NeuroSense focuses on developing therapies for neurodegenerative diseases like ALS.

Last updated: Nov 13, 2023