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NeuroSense Therapeutics (NASDAQ: NRSN) announced groundbreaking results from its Phase 2b PARADIGM clinical trial for PrimeC, an investigational treatment for amyotrophic lateral sclerosis (ALS), at the 77th Annual American Academy of Neurology (AAN) Meeting in San Diego. The data, presented by Dr. Jeffrey Rosenfeld of Loma Linda University School of Medicine and Dr. Jeremy Shefner of the Barrow Neurological Institute, showcased significant microRNA modulation and a marked reduction in ALS progression.
Dr. Rosenfeld's presentation, delivered during a highly selective "Late Breaker" session, focused on PrimeC's impact on microRNA (miRNA) modulation. The study revealed a consistent downregulation of 161 miRNAs in PrimeC-treated patients, including ALS-related miR-199 and miR-181, which are associated with disease progression and survival. No significant changes were observed in the placebo group. This miRNA modulation offers a mechanistic explanation for the previously reported 33% reduction in disease progression and 58% improvement in survival rates observed in the trial. This data, according to Professor Noam Shomron of Tel Aviv University, who collaborated on the research, "speaks to the potential of PrimeC to directly influence key molecular mechanisms in ALS."
Dr. Shefner's presentation further highlighted PrimeC's safety, efficacy, and target engagement, based on 18 months of data from the Phase 2b trial. The results indicated a marked reduction in ALS progression and positive changes across multiple biomarker endpoints, confirming PrimeC's biological activity.
NeuroSense CEO Alon Ben-Noon expressed excitement over these findings, stating that the results "provide further compelling support of PrimeC's potential to modify disease progression in people living with ALS," and position PrimeC as a promising therapeutic candidate. The company, which reported a net loss of $10.2 million in 2024, alongside reduced R&D ($5.7 million) and G&A expenses ($4.2 million), has already aligned its Phase 3 study design with the FDA and anticipates initiating the trial in 2025. NeuroSense is also actively pursuing partnership discussions to facilitate PrimeC's advancement and plans early commercialization in Canada, projecting peak annual revenues of $100 million to $150 million. The company ended 2024 with $3.4 million in cash. The Phase 3 trial will require additional funding. These developments suggest that PrimeC is a potential disease-modifying treatment for ALS. The complete Phase 2b dataset will be released on April 8th.