NeuroSense Therapeutics Ltd. reported additional positive data from its six-month double-blind Phase 2b PARADIGM trial of NeuroSense's lead drug candidate PrimeC for the treatment of amyotrophic lateral sclerosis. The PARADIGM trial's secondary clinical efficacy outcome measure endpoints included Quality of Life and Survival. Consistent with the previously reported results, PrimeC displayed a clinically meaningful effect on various aspects of patients' quality of life, including mental and physical health.

Moreover, analyses pertaining to survival outcomes emphasized the implications of PrimeC intervention in mitigating disease progression and burden. PrimeC achieved an improvement in complication-free survival compared to placebo (in several methodologies, including MiToS and King's Advanced Stage-free Survival), reducing the risk of ALS disease complications or death by up to 53%. These survival analyses observe time from participant randomization to death from any cause or respiratory insufficiency (defined as tracheostomy or the use of non-invasive mechanical ventilation for over 22 hours per day for 10 or more consecutive days), or time to hospitalization due to ALS-related complications or advancements in MiToS or King's stages.

This news comes on the heels of the recent positive top-line results reported in December 2023 from the PARADIGM trial in 68 participants, which met its primary endpoint of safety and tolerability, establishing a robust safety profile of PrimeC. In addition, PrimeC demonstrated a statistically significant slowing of disease progression with a 37.4% (p=0.03) difference in the gold standard ALS tracking measure, the ALS Functional Rating Scale-Revised ("ALSFRS-R"), in favor of PrimeC vs placebo, and 17.2% (p=0.39) difference in Slow Vital Capacity ("SVC") in favor of PrimeC vs placebo, based on the pre-specified Per Protocol (PP) population analysis. The PP analysis population includes all participants who adhered to the trial protocol and treatment plan without any major protocol deviations and includes 62 patients (43 active and 19 placebo).

PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b (NCT05357950) clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel. 96% of the trial participants who completed the 6-month double-blind portion of the trial chose to receive treatment with PrimeC through a 12-month open label extension.

Furthermore, to date all participants that completed the 18-month trial treatment duration, requested to continue PrimeC, which is provided to them in an Investigator Initiated Trial, not limited with time. An analysis of the ITT top-line data from the 6-month double-blind segment of the trial showed clinically meaningful signs of efficacy with a 29% difference in ALSFRS-R (p=0.12) and a 13% difference in SVC (p=0.5), both in favor of PrimeC vs placebo. These data include all 68 people living with ALS enrolled in Canada, Italy, and Israel, with the exclusion of one patient who was misdiagnosed.

Most patients enrolled in both the active and placebo arms of trial were concurrently treated with Riluzole, the ALS standard of care medication, indicating PrimeC slowed disease progression well beyond the level afforded by the FDA approved ALS drug.