AGPI

AGPI is an investigational small molecule drug candidate being developed by Agyany Pharma for the treatment of Parkinson's disease and Gaucher disease. The drug is designed to target the biological link between these two conditions, which is primarily centered on the GBA1 gene and the activity of the enzyme glucocerebrosidase (GCase). Mutations in GBA1 are the most common genetic risk factor for Parkinson's disease and the direct cause of Gaucher disease. AGPI aims to modulate GCase activity, potentially acting as a pharmacological chaperone or activator to restore lysosomal function and reduce the accumulation of toxic protein aggregates such as alpha-synuclein. As of early 2024, the candidate is in the human toxicology phase, which corresponds to Phase 1 clinical development.