NeuroSense Therapeutics to Present New Data from PrimeC's Phase 2b Trial in ALS at the Annual American Academy of Neurology Meeting

Rhea-AI Summary

NeuroSense Therapeutics (NASDAQ: NRSN) will present new data from its Phase 2b trial of PrimeC, a novel ALS treatment, at the 77th Annual American Academy of Neurology Meeting on April 8, 2025, in San Diego.

Two key presentations are scheduled: Dr. Jeremy Shefner will present at 2:24 PM, focusing on PrimeC's safety, efficacy, and target engagement data from an 18-month Phase 2b trial. The results show marked reduction in ALS progression and multiple biomarker endpoints confirming biological activity.

Dr. Jeffrey Rosenfeld will present at 6:15 PM during a late breaker session, discussing microRNA modulation and iron-related pathways in ALS treatment with PrimeC. The presentations highlight PrimeC's potential as a disease-modifying therapy for ALS patients.

Positive

• Phase 2b trial showed marked reduction in ALS progression
• PrimeC demonstrated safety and tolerability in 18-month trial
• Multiple biomarker endpoints confirmed biological activity
• Advancing to Phase 3 trial

Negative

• None.

Insights

Clinical Neurologist

The Phase 2b trial results for PrimeC represent a potentially significant advancement in ALS therapeutics. While the article doesn't provide specific efficacy metrics, the reported "marked reduction in ALS progression" is noteworthy in a disease with treatment options. Most current ALS therapies only modestly slow progression or manage symptoms rather than addressing disease mechanisms.

PrimeC's multi-targeted approach addressing microRNA modulation and iron-related pathways aligns with our current understanding of ALS pathophysiology, which involves multiple cellular and molecular mechanisms. The biomarker endpoints demonstrating target engagement provide objective evidence the therapy is affecting intended biological pathways.

What's particularly compelling is the suggestion of disease-modifying potential. In ALS treatment, true disease modification rather than symptomatic relief represents the gold standard we've struggled to achieve. The statements from both Dr. Shefner and Dr. Rosenfeld suggest the results exceeded expectations, though we should await the full data presentation for detailed efficacy measures, statistical significance, and patient numbers.

The progression to a planned Phase 3 trial indicates sufficient confidence in these results to advance development. This preliminary evidence suggests PrimeC could potentially address the complex pathology of ALS more effectively than existing options.

Biotechnology Analyst

These Phase 2b results potentially represent a significant milestone for NeuroSense. With a micro-cap valuation of just $21.4 million, positive clinical data in a high-unmet-need indication like ALS could drive substantial valuation reassessment as the company progresses toward Phase 3.

The ALS therapeutic market commands premium pricing due to effective treatment options. Current approved therapies like riluzole and edaravone provide modest benefits, leaving substantial opportunity for more effective treatments that can demonstrate disease modification.

PrimeC's dual mechanism targeting multiple disease pathways provides potential competitive differentiation. The biomarker data showing target engagement strengthens the scientific rationale and potentially reduces Phase 3 risk by confirming the mechanism is working as intended.

The advancement to Phase 3 will require significant capital, likely exceeding NeuroSense's current resources, suggesting potential financing events or partnership discussions may be forthcoming. For investors, the key value inflection point will be the complete Phase 2b data set presentation on April 8th, which should provide the specific efficacy metrics and statistical analyses not detailed in this announcement.

This news materially derisks the PrimeC development program, though considerable challenges remain in executing a successful Phase 3 program and navigating the regulatory approval process.

Two presentations in the General Neurology and Late Breaker sessions will respectively cover new clinical and microRNA data from NeuroSense

CAMBRIDGE, Mass., April 4, 2025 /PRNewswire/ -- NeuroSense Therapeutics, Ltd. (NASDAQ: NRSN), a leading clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, today announced that two members of its Scientific Advisory Board will present new data from the Company's Phase 2b trial during the General Neurology and Late Breaker sessions at the 77^th Annual American Academy of Neurology (AAN) Meeting. The presentations and conference will be taking place in San Diego, CA on April 8, 2025.

Jeremy Shefner, M.D., Ph.D., Chair of the Department of Neurology and Senior Vice President at the Barrow Neurological Institute, will present new, compelling data from the Phase 2b trial of PrimeC, NeuroSense's novel investigational therapy for amyotrophic lateral sclerosis (ALS), at 2:24 p.m. His presentation, "Shifting the Paradigm: PrimeC, an Oral Candidate for ALS, Demonstrates Safety, Efficacy, and Target Engagement in an 18-Month Phase 2b Trial," will highlight PrimeC's disease-modifying potential in ALS treatment.

"This exciting Phase 2b trial data underscores PrimeC's potential to redefine ALS treatment," says Shefner. "By demonstrating safety and tolerability, an indication of marked reduction in ALS progression, and multiple biomarker endpoints establishing PrimeC's biological activity, we move closer to offering a disease-modifying therapy that could lead to significantly improved clinical outcomes for people with ALS."

Additionally, Jeffrey Rosenfeld, M.D., Ph.D., Professor of Neurology and Associate Chairman of Neurology at Loma Linda University School of Medicine, will present insights during a late breaker session at 6:15 p.m. on the role of microRNA modulation in ALS treatment, depicting the multi-targeted approach of PrimeC. His presentation, "MicroRNA Profiling and Iron-Related Modulation as Key Markers for Target Engagement in ALS Treatment with PrimeC," will shed new light on the understanding of PrimeC's mechanism of action in ALS.

"PrimeC represents great potential to fundamentally change ALS treatment, with its multi-targeted approach revealing profound insights into disease mechanisms," says Rosenfeld. "By exploring microRNA modulation and iron-related pathways, we deepen our understanding of how PrimeC engages key targets of disease, offering new hope for tackling the complexities of ALS. These significant changes in such relevant biomarker endpoints reflecting target engagement, along with the clinical outcomes reported earlier, heighten our anticipation and enthusiasm for the forthcoming phase 3 trial of PrimeC."

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 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 the potential 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 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; 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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SOURCE NeuroSense

FAQ

What are the key findings from NRSN's Phase 2b trial of PrimeC for ALS treatment?

The trial demonstrated PrimeC's safety, tolerability, marked reduction in ALS progression, and multiple biomarker endpoints confirming biological activity.

When and where will NeuroSense (NRSN) present its Phase 2b PrimeC trial results?

The results will be presented on April 8, 2025, at the 77th Annual American Academy of Neurology Meeting in San Diego.

What is the mechanism of action of NRSN's PrimeC in treating ALS?

PrimeC employs a multi-targeted approach, focusing on microRNA modulation and iron-related pathways to engage key disease targets.

What are the next steps for NRSN's PrimeC development program?

Following the positive Phase 2b results, NeuroSense is preparing for a Phase 3 trial of PrimeC.