NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) (“NeuroSense”), a company developing treatments for severe neurodegenerative diseases, today announced it has completed dosing of the last patient in the double-blind segment of its Phase 2b amyotrophic lateral sclerosis (ALS) trial of PrimeC (PARADIGM). PrimeC is designed to synergistically target several key ALS mechanisms that contribute to motor neuron degeneration, inflammation, iron accumulation, and impaired RNA regulation to inhibit the progression of ALS. PrimeC has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Sixty-nine people living with ALS in Canada, Italy, and Israel were enrolled in the double-blind segment of PARADIGM (NCT05357950), a multinational, randomized, double-blind, placebo-controlled Phase 2b clinical trial of PrimeC in ALS, wherein trial participants were dosed for 6 months after being randomized 2:1 to receive PrimeC or placebo, respectively. After completion of the double-blind segment, the participants had the option to enroll in a 12-month open label extension (OLE), during which they all receive treatment with PrimeC. 96% of the participants chose to continue into the OLE.
The Company expects to release clinical efficacy results (secondary endpoints) from the double-blind segment of the trial in December 2023. These secondary endpoints include clinical outcome measures: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R), Slow Vital Capacity (SVC), quality of life and overall survival. ALSFRS-R is recognized and widely used as an endpoint in ALS clinical trials that are evaluated by the FDA. This scale aids in providing a measurement of the impact of the disease on various functional activities and the overall quality of life of patients with ALS. The Company also expects to report the safety and tolerability results (primary endpoints) of the double-blind segment of the trial in December 2023.
The Company expects to report on another primary endpoint, the assessment of ALS-biomarkers, TDP-43 and Prostagladin2, to evaluate PrimeC’s biological activity, in H1 2024 following the completion of the ongoing analysis of patients’ plasma. PrimeC was previously observed to have a statistically significant impact in TDP-43 and Prostagladin2, in NeuroSense’s previous Phase 2a trial.
Additionally, in the first quarter of 2024, the Company expects to report results from a strategic collaboration with Biogen that is evaluating the impact of PrimeC on patients enrolled in PARADIGM. Under this collaboration, Biogen is to meaningful biomarker analysis and upon receipt of results, has the right of first refusal to co- fund this develop/commercialize PrimeC for the treatment of ALS for a limited time following the results.
Recent findings from independent studies at the University of Southern California showed PrimeC significantly increased the survival rate of induced motor neurons in an in vitro ALS study and PrimeC performed among the best in improving motor neuron survival when compared to several other ALS drugs in development and two U.S. FDA approved ALS drugs.
“We are excited to soon begin sharing the results of the double-blind segment of our Phase 2b trial,” stated Alon Ben Noon, CEO of NeuroSense. “We would like to thank the trial participants, their caregivers and families, as well as the sites’ Principal Investigators and study coordinators for their tremendous contribution to PARADIGM.”