TP-060，5MG，SML4003-5MG，Sigma

Biochem/physiol Actions

Orally active, brain-penetrant, substrate-noncompetitive glucosylceramide synthase (GCS) inhibitor with therapeutic efficacy in a murine Gaucher’s disease (GD) model in vivo.
TP-060 (T-036) is an orally active, brain-penetrant, substrate-noncompetitive, potent and selective glucosylceramide synthase (GCS) inhibitor (human/mouse IC50 = 31/51 nM) that effectively downregulates glucosylceramide (GlcCer) level in GBA mutant human fibroblasts containing mutated glucosylceramidase (IC50 = 7.6 nM). TP-060 is efficacious in lowering both GlcCer and glucosylsphingosine (GlcSph) levels in the plasma and cerebral cortex of Gba D409V KI mice in vivo following a two-month treatment in the murine Gaucher’s disease (GD) model (10 and 30 mg/kg/day p.o.).