NeuroSense's PrimeC Shows Significant Efficacy and Survival Benefits in ALS from PARADIGM Study

NeuroSense Therapeutics, a biotechnology company in the late stages of clinical development, has announced promising results from their PARADIGM Phase 2b study of PrimeC for Amyotrophic Lateral Sclerosis (ALS). The 12-month data analysis demonstrated a significant improvement in the rate of decline in ALS Functional Rating Scale-Revised (ALSFRS-R) scores and survival rates among subjects who received PrimeC compared to those who started on a placebo. These results suggest that PrimeC could be a groundbreaking treatment for ALS, prompting NeuroSense to plan a Phase 3 clinical study in the U.S. and Europe.

The PARADIGM study's intent-to-treat (ITT) analysis revealed a 36% improvement in ALSFRS-R scores, equating to a difference of 6.5 points, with a highly significant P value of 0.009. Additionally, participants who received PrimeC from the start showed a 43% better survival rate compared to those initially on placebo. A pre-defined analysis of the Per-Protocol Population indicated an even more impressive result, with a 40% improvement in ALSFRS-R scores and a 63% increase in survival rates for participants who began treatment with PrimeC.

Merit Cudkowicz, M.D., M.Sc., a prominent figure in neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, emphasized the importance of these findings. Cudkowicz highlighted the urgent need for new ALS treatments and underscored PrimeC's potential based on its mechanism of action and the promising Phase 2b trial results. The drug's efficacy warrants further evaluation in an expedited Phase 3 trial.

Vivian Drory, MD, who leads the ALS clinic at Tel-Aviv Sourasky Medical Center, echoed this sentiment, noting that the 12-month study results underline the significant potential of PrimeC as a disease-modifying drug for ALS. Drory stressed the importance of early intervention for maximizing benefits, which will be critical in designing the upcoming Phase 3 study.

ALS, a devastating neurodegenerative disease, leads to complete paralysis and death within 2-5 years of diagnosis. Annually, more than 5,000 people in the U.S. are diagnosed with ALS, with the disease burden costing approximately $1 billion. Projections indicate a 24% increase in the number of ALS patients in the U.S. and EU by 2040.

The ALS Functional Rating Scale-Revised (ALSFRS-R) is the most widely used tool for tracking ALS progression, assessing 12 changes in physical abilities over time. These include speech, walking, climbing stairs, and breathing. A single point change on the ALSFRS-R can significantly impact patients' lives, such as the need for assisted feeding or mechanical ventilation.

PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b clinical trial (NCT05357950). It involved 68 ALS patients across Canada, Italy, and Israel. Of the participants who completed the 6-month double-blind part of the trial, 96% chose to continue with PrimeC treatment through a 12-month open-label extension. To date, all participants who completed the 18-month trial have requested to continue PrimeC.

Previously reported data from the 6-month double-blind segment of the trial showed a 29% improvement in favor of PrimeC. The Per-Protocol (PP) analysis revealed a statistically significant 37.4% improvement in ALSFRS-R scores compared to placebo. Notably, most patients in both the active and placebo arms were also treated with Riluzole, the standard ALS medication, indicating that PrimeC's benefits extend beyond current treatment options.

PrimeC, NeuroSense's leading drug candidate, is an extended-release oral formulation combining ciprofloxacin and celecoxib. It targets key ALS mechanisms, including motor neuron degeneration, inflammation, iron accumulation, and RNA regulation. PrimeC has demonstrated safety and efficacy in a Phase 2a trial and received Orphan Drug Designation from the FDA and the European Medicines Agency.

NeuroSense Therapeutics is committed to developing treatments for severe neurodegenerative diseases, with a focus on ALS, Alzheimer's, and Parkinson's disease. Their strategy involves developing combined therapies targeting multiple disease pathways, based on robust scientific research and biomarker analysis.

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