NeuroSense Therapeutics' PrimeC, a novel oral formulation, has shown promising results in a Phase 2b clinical trial (PARADIGM) for Amyotrophic Lateral Sclerosis (ALS), demonstrating positive biomarker data and improved clinical outcomes. The 12-month data revealed significant regulation of iron levels, aligning with improved survival and disease mitigation in ALS patients.
Iron Regulation and Clinical Outcomes
The PARADIGM trial results indicated a significant decrease in ferritin levels and a corresponding increase in transferrin levels in patients treated with PrimeC. Specifically, iron levels remained stable over the 12-month dosing period, with a mean difference of 4.536 μmol/L (95% CI [1.143, 7.929], p=0.01) compared to those who transitioned from placebo to PrimeC after the initial 6-month double-blind phase. These changes in iron metabolism correlated with improved clinical outcomes, as patients on PrimeC maintained better functionality and survival rates compared to those on placebo.
Impact on Disease Progression and Survival
Previous results from the PARADIGM trial showed that PrimeC slowed disease progression by 36% and improved survival rates by 43%. Further analysis revealed a 57% difference in complication-free survival in favor of PrimeC over placebo in the Intent to Treat (ITT) population, and a 73% difference (p=0.02) in the Per Protocol Population (PP) after 12 months of treatment. These findings suggest that PrimeC has the potential to significantly impact the course of ALS.
Expert Commentary
"The latest results from the PARADIGM study are incredibly encouraging and provide compelling evidence of PrimeC's potential to significantly benefit people living with ALS," said Jeremy M. Shefner, M.D., Ph.D., Professor of Neurology at the Barrow Neurological Institute, Phoenix, Ariz., and a NeuroSense advisor. Merit Cudkowicz, M.D., M.Sc., Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, added, "This new analysis highlights PrimeC's ability to regulate the iron panel in people living with ALS, underscoring the drug's target engagement. These findings strongly support proceeding to phase 3 testing of PrimeC in ALS."
Future Directions
NeuroSense is compiling additional data to share with the FDA to determine the clinical and regulatory path forward for PrimeC. The company is also in discussions with potential development partners to explore marketing opportunities following potential approval of PrimeC for ALS.
About PrimeC
PrimeC is an extended-release oral formulation combining ciprofloxacin and celecoxib, designed to synergistically target key mechanisms of ALS, including motor neuron degeneration, inflammation, iron accumulation, and impaired RNA regulation.
