ezurpimtrostat

Ezurpimtrostat is an orally bioavailable, quinoline-derived small molecule that acts as a first-in-class inhibitor of palmitoyl-protein thioesterase 1 (PPT1). By inhibiting PPT1, it induces lysosomal disruption, inhibits autophagy, and triggers apoptosis via caspase activation. This mechanism leads to the inhibition of tumor cell proliferation and growth. Ezurpimtrostat has demonstrated high liver tropism and potent anti-tumor activity in preclinical models and is being developed primarily for hepatocellular carcinoma (HCC) and other liver cancers. It has received orphan drug designation from the FDA for HCC. The drug is also under investigation for cholangiocarcinoma, COVID-19 infections, and other cancer indications[1][2][3][4][5].

Other names

ezurpimtrostat [INN]chloroquine analog