NeuroSense’s novel drug, PrimeC, is a combination therapy, which aims to inhibit the progression of ALS, by mitigating the degeneration and inflammation of motor neurons. The disease-modifying drug is designed to target multiple ALS pathways, including regulation of microRNA synthesis, reduction of neuroinflammation, and modulation of iron accumulation. PrimeC has received orphan drug status from the FDA and EMA. NeuroSense is currently preparing to commence a pivotal Phase 3 study.

PrimeC demonstrated a 33% functional improvement (p=0.007) and a 58% improvement in survival rates, in PARADIGM, a Phase 2b clinical study. Results revealed a significant difference of nearly 8 points in ALSFRS-R between the group treated with PrimeC for 18 months and the group that received placebo for 6 months before transitioning to PrimeC for 12 months. 68 people living with ALS were enrolled in PARADIGM, an 18-month, randomized, double-blind, multi-national, placebo-controlled study. Participants were administered PrimeC or placebo (ratio of 2:1, respectively), twice daily, for 6 months. 96% of people who complete the 6 months dosing joined the 12-month open label extension study, in which all participants received PrimeC.

NeuroSense has also completed a Phase 2a clinical study, which met its safety and efficacy endpoints. In people living with ALS, PrimeC attenuated functional and respiratory deterioration, as well as induced statistically significant changes in ALS-related biological markers, indicating PrimeC’s biological activity.

Additional pre-clinical models of ALS, demonstrate the potential of PrimeC to become a first-line therapy. In collaboration with the University of Southern California’s Ichida Stem Cell Lab, a world-renowned stem cell research lab, PrimeC was shown to significantly increase survival rate of induced motor neurons in an in vitro study, utilizing induced pluripotent stem cells (iPSCs) generated from people living with ALS. In pre-clinical zebrafish models of ALS, PrimeC significantly outperformed conventional treatments, demonstrating improvement in motor performance, and recovery of the morphology of motor neurons, neuromuscular junction structures, and microglial cells.