Glucosylceramide synthase-IN-2 is a potent, brain-penetrant, and orally active inhibitor of glucosylceramide synthase (GCS). It demonstrates IC50 values of 15 nM for human GCS and 190 nM for mouse GCS, exhibiting noncompetitive inhibition with C8-ceramide and UDP-glucose. This compound is applicable for research related to Gaucher's disease, showing no significant inhibitory activity against SERT or GCase in vitro. In vivo studies confirm good oral and brain exposure in mice, reducing GlcCer concentrations in plasma and cerebral cortex.
- Potent GCS inhibitor
- Brain-penetrant
- Orally active
- Noncompetitive inhibition type with C8-ceramide and UDP-glucose
- Useful for Gaucher's disease research
- Does not significantly inhibit SERT or GCase activity in vitro
- Shows good oral and brain exposure in vivo