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fosgonimeton

Development stage: Discontinued
Lead developer: LeonaBio
Modality: Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration: Subcutaneous Injection

Overview

Fosgonimeton is a small-molecule, first-in-class regenerative therapy developed by Athira Pharma for neurodegenerative diseases, primarily Alzheimer's disease and dementia. It acts as a positive modulator of the hepatocyte growth factor (HGF) system and stimulates the proto-oncogene protein c-Met receptor. By enhancing HGF/MET signaling, fosgonimeton aims to promote synaptic connectivity, neuronal survival, and cognitive function. Preclinical studies demonstrated neurotrophic and pro-cognitive effects; clinical trials have shown it to be generally well tolerated with some biomarker improvements but did not meet primary efficacy endpoints in phase II/III trials for Alzheimer's disease[1][2][5][8].

Other names: fosgonimetonATH 1017ATH1017ATH-1017NDX 1017NDX1017NDX-1017

Targets

MET (Mesenchymal-epithelial transition factor receptor)

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