NeuroSense ALS Trial Shows 73% Better Complication-Free Survival After Slowing Disease Progression

NeuroSense Therapeutics Ltd., a biotechnology company at the forefront of developing treatments for severe neurodegenerative diseases, has shared promising additional results from its 12-month PARADIGM Phase 2b study of PrimeC in Amyotrophic Lateral Sclerosis (ALS) patients. The study revealed a significant improvement in complication-free survival, showing a 57% difference in favor of PrimeC compared to placebo after 12 months of treatment in the Intent to Treat (ITT) population. In the Per Protocol (PP) population, the outcome was even more pronounced, with a 73% difference favoring PrimeC over placebo.

NeuroSense plans to submit these encouraging 12-month results to regulatory bodies, including the FDA, to discuss the forward path for PrimeC. Previously, the company reported that PrimeC slowed disease progression by 36% and improved survival rates by 43% compared to placebo. The Per Protocol analysis showed even more significant benefits, with a 40% improvement in ALSFRS-R scores and a 63% increase in survival rates.

Further positive clinical data included an improvement in Slow Vital Capacity (SVC), which increased from a 13% difference after 6 months to a 20% difference after 12 months in the ITT population, favoring PrimeC. In the PP population, the SVC improvement went from 17% after 6 months to 19% after 12 months. An analysis of the rate of decline, defined by a drop of 4 points or less in ALSFRS-R after 12 months, showed a ratio of 4.5 to 1 in favor of PrimeC over placebo, underscoring the drug's potential as a disease-modifying treatment.

Dr. Jeremy M. Shefner, a Professor of Neurology and NeuroSense advisor, highlighted the excitement surrounding these results, noting the substantial improvement in complication-free survival and the consistent slowing of disease progression. He emphasized the urgent need for effective new therapies for ALS, given the limited options currently available.

NeuroSense's CEO, Alon Ben-Noon, expressed confidence in the unprecedented results of the 12-month ALS placebo-controlled clinical study. The company is eager to present these findings to the FDA and other regulatory agencies to determine the path forward and share the outcomes with potential partners. Ben-Noon also acknowledged the significant contributions of the clinical team, study coordinators, participants, and their families.

ALS, a neurodegenerative disease that causes complete paralysis and death within 2–5 years of diagnosis, affects over 5,000 people annually in the U.S. alone, with the number expected to grow by 24% by 2040 in the U.S. and EU. Disease progression in ALS is measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a widely used tracking tool that monitors changes in physical abilities.

The PARADIGM trial, a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b study, included 68 ALS patients from Canada, Italy, and Israel. Of those who completed the 6-month double-blind portion of the trial, 96% chose to continue treatment with PrimeC through a 12-month open-label extension. As of June 2024, all participants who completed the 18-month trial requested to continue PrimeC treatment.

PrimeC, NeuroSense's lead drug candidate, is an extended-release oral formulation combining ciprofloxacin and celecoxib. It targets key mechanisms of ALS, such as motor neuron degeneration, inflammation, iron accumulation, and impaired RNA regulation. This combination aims to inhibit ALS progression and has shown promise in previous Phase 2a clinical trials, meeting safety and efficacy endpoints.

NeuroSense Therapeutics focuses on developing treatments for debilitating neurodegenerative diseases, including ALS, Alzheimer's, and Parkinson's. Given the limited therapeutic options available, NeuroSense's strategy involves developing combined therapies targeting multiple pathways associated with these diseases.