brelgometon

Brelgometon (ATH-1105) is an orally available, brain-penetrant, next-generation small molecule drug candidate developed by LeonaBio. It acts as a positive modulator of the neurotrophic Hepatocyte Growth Factor (HGF) system, designed to enhance neuroprotective signaling. The drug is primarily being developed for the treatment of amyotrophic lateral sclerosis (ALS), with potential future applications in other neurodegenerative conditions such as Alzheimer's and Parkinson's diseases. Clinical data from a Phase 1 trial in healthy volunteers demonstrated a favorable safety profile, dose-proportional pharmacokinetics, and confirmed central nervous system penetration. A Phase 2 proof-of-concept study in ALS patients is targeted for initiation in the second half of 2026.