ATH-1105

ATH-1105 is an investigational, orally administered, brain-penetrant small molecule drug developed by Athira Pharma for the treatment of amyotrophic lateral sclerosis (ALS). It acts as a positive modulator of the neurotrophic hepatocyte growth factor (HGF) system by enhancing signaling through the MET receptor. Preclinical studies have shown that ATH-1105 improves motor and nerve function, preserves axon and myelin integrity, reduces inflammation and neurodegeneration biomarkers (such as TDP-43 and neurofilament light chain), and extends survival in ALS models. The drug demonstrates consistent neuroprotective and anti-inflammatory effects across central nervous system compartments relevant to ALS pathogenesis. As of 2024, it is in Phase 1 clinical trials assessing safety, tolerability, and pharmacokinetics in healthy volunteers[2][3][4][5][6].