• First randomized, double-blind, placebo-controlled trial to demonstrate treatment-associated reduction of TDP-43 in people living with ALS
  • TDP-43 pathology is present in more than 97% of ALS cases and is widely recognized as a central driver of disease progression
  • PrimeC demonstrates target engagement with consistent effects across clinical outcomes, survival, and biomarkers, supporting its potential as a disease-modifying therapy