JAMA Neurology Publishes Results from PARADIGM Phase 2b Trial of PrimeC in ALS Demonstrating Meaningful Clinical Outcomes and Biological Activity

Core Insights - The publication in JAMA Neurology presents significant findings from the PARADIGM Phase 2b trial of PrimeC in ALS, indicating meaningful clinical outcomes and biological activity [1][5][6] Study Overview - PARADIGM was a randomized, double-blind, placebo-controlled trial assessing PrimeC's safety and efficacy over a 6-month treatment period followed by a 12-month open-label extension [3][8] - The trial enrolled 68 participants with definite or probable ALS, randomized in a 2:1 ratio to receive PrimeC or placebo [8] Clinical Outcomes - PrimeC treatment resulted in a 64% relative reduction in the risk of ALS-related complications, including respiratory failure and hospitalization (p=0.02) [6][10] - Continuous treatment with PrimeC showed a 7.92-point advantage in ALSFRS-R at 18 months, representing over a 36% benefit in slowing disease progression (p=0.007) [6][9] - Significant improvement in bulbar function was observed (p=0.001) [6][9] Safety Profile - PrimeC demonstrated a safety profile comparable to placebo over 18 months, with most treatment-related adverse events being mild to moderate and transient [6][7] Biomarker Findings - The trial showed significant modulation of ALS-related microRNAs and iron-regulatory biomarkers, supporting PrimeC's biological activity [6][12] - Favorable changes in markers of iron metabolism were observed, including preservation of transferrin levels and stabilization of ferritin [11][12] Future Development - The findings support the advancement of PrimeC to a Phase 3 clinical trial, which has received FDA clearance [6][7][8] - The consistent results across clinical outcomes and biomarkers provide a strong scientific foundation for further evaluation of PrimeC as a potential disease-modifying therapy [6][7][12]