NeuroSense Therapeutics Reports Positive Biomarker Data from ALS Phase 2b Trial

NeuroSense Therapeutics Ltd., a biotechnology firm in the late stages of clinical development, has unveiled promising 12-month data from their Phase IIb study (PARADIGM) focused on PrimeC, a treatment for Amyotrophic Lateral Sclerosis (ALS). This data supports earlier findings that indicated PrimeC could decelerate disease progression by 36% and improve survival rates by 43%, underscoring the potential of PrimeC as a groundbreaking ALS therapy.

ALS, a complex neurodegenerative disease, involves the dysregulation of iron and iron-related proteins. The recent results from NeuroSense demonstrate that PrimeC effectively engages with its target by regulating iron metabolism, which is crucial for disease mitigation and enhanced survival. In ALS patients, iron accumulation in the brain is associated with neuronal damage, while elevated ferritin levels, indicative of iron storage, correlate with reduced survival and increased oxidative stress. Conversely, low transferrin levels, critical for iron transport, further exacerbate disease progression.

The 12-month study showed a significant decrease in ferritin and an increase in transferrin levels in patients, suggesting an alleviation of ALS pathology. Throughout the dosing period, iron levels remained stable, with a notable mean difference when compared to placebo groups. These alterations in iron metabolism were in line with improved clinical outcomes; patients on PrimeC maintained better functionality and survival rates than those on placebo.

Dr. Merit Cudkowicz, Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, remarked on the encouraging nature of the study results, emphasizing PrimeC's effectiveness in regulating iron levels. These findings bolster the case for advancing to Phase 3 trials of PrimeC in ALS patients.

NeuroSense Therapeutics is in the process of gathering additional data to present to the FDA for further discussions on the clinical and regulatory pathways. The company is also in advanced discussions with several potential development partners to explore marketing opportunities contingent upon the successful completion and approval of PrimeC.

ALS, known for its devastating impact, typically results in complete paralysis and death within 2-5 years post-diagnosis. Over 5,000 new cases are identified annually in the U.S. alone, with the disease burden estimated at $1 billion per year. The prevalence of ALS is expected to rise by 24% in the U.S. and EU by 2040.

The progression of ALS is commonly measured using the ALS Functional Rating Scale-Revised (ALSFRS-R), which tracks changes in physical abilities over time, impacting functions such as speech, mobility, and breathing. Even a single point change on the ALSFRS-R can significantly affect a patient's quality of life.

The PARADIGM study, a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b trial, included 68 ALS patients across Canada, Italy, and Israel. Notably, 96% of participants who completed the initial 6-month double-blind phase opted to continue with PrimeC in a 12-month open-label extension. Earlier data from the trial indicated a 29% improvement in disease progression in favor of PrimeC compared to placebo.

PrimeC, NeuroSense's leading drug candidate, is an extended-release oral formulation combining ciprofloxacin and celecoxib. This combination targets several key ALS mechanisms, including motor neuron degeneration, inflammation, iron accumulation, and RNA regulation. PrimeC has shown promise in reducing ALS symptoms and altering biological markers in previous Phase 2a trials. It has received Orphan Drug Designation from both the FDA and the European Medicines Agency.

NeuroSense Therapeutics, a clinical-stage biotechnology company, is dedicated to developing treatments for debilitating neurodegenerative diseases such as ALS, Alzheimer's, and Parkinson's. Given the complexity and significant unmet medical needs of these conditions, NeuroSense aims to develop combination therapies targeting multiple disease pathways based on extensive scientific research.

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