New miRNA findings unveiled at the AAN, reveal key biological mechanisms behind PrimeC’s clinical benefit in ALS.
NeuroSense Therapeutics, Ltd. (NASDAQ: NRSN), a leading clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, today announced promising new findings from its Phase 2b PARADIGM clinical trial. The data highlights the significant impact of PrimeC, the company’s investigational therapy for the treatment of amyotrophic lateral sclerosis (ALS), on microRNA (miRNA) modulation.
These data are particularly significant because they:
- Represent an important development in understanding how the therapy may slow disease progression in people living with ALS.
- Reveal the consistent effect of PrimeC on altering the expression of miRNAs associated with ALS, reinforcing its potential role in addressing key pathological processes in ALS and underscoring its potential as a transformative, multi-targeted therapeutic.
- Align with the previously reported clinical benefits, including the 33% reduction in disease progression and 58% improvement in survival rates observed with PrimeC treatment.
Key Findings:
- Consistent downregulation of 161 miRNAs in PrimeC-treated patientsIn ALS patients treated with PrimeC, the study observed a profound and consistent downregulation of 161 mature miRNAs across all time points in the double-blind period. In contrast, no significant changes were detected in the placebo arm, emphasizing PrimeC’s targeted biological activity and potential for disease modification.
- Clinical relevance: miRNA modulation and disease progressionPrimeC treatment led to the significant downregulation of ALS-related miRNAs, including miR-199 and miR-181, both of which are associated with ALS disease progression and survival. Notably, both miR-199 and miR-181 are upregulated in ALS. miR-199 is associated with neuroinflammation and reduced neuronal survival, while miR-181 correlates with a higher risk of mortality. This downregulation of both miRNAs in patients corresponds with the already reported improvement in clinical function.
This research, conducted in collaboration with Prof. Noam Shomron of Tel Aviv University’s Faculty of Medical and Health Sciences, a leader in microRNA research and functional genomics, was presented yesterday by Jeffrey Rosenfeld, M.D., Ph.D., Professor of Neurology and Associate Chairman of Neurology at Loma Linda University School of Medicine, during a Late Breaker session at the 77th Annual American Academy of Neurology (AAN) Meeting, in San Diego, CA.
