ATH-1001

**ATH-1001** is a small molecule and the active metabolite of the prodrug fosgonimeton (ATH-1017). It acts as a highly potent, specific **positive modulator of the hepatocyte growth factor (HGF)/MET neurotrophic system**, enhancing HGF binding and activation of the MET tyrosine kinase receptor. This leads to downstream signaling via the PI3K/Akt and MAPK pathways, augments NMDA receptor-mediated long-term potentiation through protein kinase C, and is believed to promote synaptic regeneration and neuronal survival. ATH-1001 readily crosses the blood-brain barrier and is under investigation primarily for neurodegenerative disorders, notably Alzheimer's disease, with the intent to both improve cognitive symptoms and potentially slow or reverse neurodegeneration[1][3].