生产企业：LeonaBio, Inc.
Brelgometon (ATH-1105) is an investigational oral, brain-penetrant small molecule in clinical development by LeonaBio, Inc. for the potential treatment of neurodegenerative diseases, with ALS as the lead indication. LeonaBio completed the first-in-human Phase 1 double-blind, placebo-controlled clinical trial (NCT06432647) in November 2024, enrolling 80 healthy volunteers. Results demonstrated favorable safety and tolerability, dose-proportional pharmacokinetics, and confirmed central nervous system penetration. Preclinical evidence showed statistically significant improvements in nerve and motor function, biomarkers of inflammation and neurodegeneration, and survival across multiple ALS models. A Phase 2 proof-of-concept and dose-ranging study is planned as a randomized, double-blind, placebo-controlled trial, with initiation targeted for the second half of 2026.
Brelgometon (ATH-1105) is an investigational oral, brain-penetrant small molecule in clinical development by LeonaBio, Inc. for the potential treatment of neurodegenerative diseases, with ALS as the lead indication. LeonaBio completed the first-in-human Phase 1 double-blind, placebo-controlled clinical trial (NCT06432647) in November 2024, enrolling 80 healthy volunteers. Results demonstrated favorable safety and tolerability, dose-proportional pharmacokinetics, and confirmed central nervous system penetration. Preclinical evidence showed statistically significant improvements in nerve and motor function, biomarkers of inflammation and neurodegeneration, and survival across multiple ALS models. A Phase 2 proof-of-concept and dose-ranging study is planned as a randomized, double-blind, placebo-controlled trial, with initiation targeted for the second half of 2026.
Brelgometon (ATH-1105) is a novel, orally available, brain-penetrant, next-generation small molecule designed to positively modulate the neurotrophic HGF (hepatocyte growth factor) system. It acts as a positive modulator of the HGF system, promoting neurotrophic support for the potential treatment of neurodegenerative diseases.
Brelgometon (ATH-1105) is currently in clinical development for the potential treatment of amyotrophic lateral sclerosis (ALS) as the lead indication. It is also being explored for other neurodegenerative diseases including Alzheimer's disease and Parkinson's disease.
