Drug intelligence / Profile preview

YM201636

Development stage
Preclinical
Lead developer
Astellas Pharma
Modality
Small Molecules
Administration
Oral
01

Overview

YM201636 is a potent and selective small molecule inhibitor of **1-phosphatidylinositol 3-phosphate 5-kinase (PIKfyve)**. Originally developed by Yamanouchi Pharmaceutical (now Astellas Pharma), it is widely utilized as a chemical probe to investigate endosomal trafficking and lysosomal function. By inhibiting PIKfyve, the compound prevents the synthesis of phosphatidylinositol 3,5-bisphosphate (PI(3,5)P2), which in turn triggers the nuclear translocation of **Transcription Factor EB (TFEB)**. This mechanism enhances lysosomal biogenesis and autophagy-mediated clearance of protein aggregates. Recent research has explored its potential in **Parkinson's disease** for the reduction of toxic alpha-synuclein aggregates, as well as its role in inhibiting viral entry (e.g., Ebola, SARS-CoV-2) by disrupting endosomal maturation.

02

Targets

PIKFYVE (Phosphoinositide kinase, FYVE-type zinc finger containing enzyme)PIK3CA (Phosphoinositide 3-kinase alpha)PIP5K1A (PIP5K1α)TPC2 (Two pore segment channel 2)

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