Description
A UAE inhibitor (IC50 = 1 nM); selective for UAE over NAE, SAE, and ATG7 (IC50s = 28, 850, and >10,000 nM, respectively); inhibits proliferation in a panel of 30 cancer cell lines (EC50s = 0.006-1.1 µM); activates the UPR and expansion of the endoplasmic reticulum surface area in HCT116 cells; reduces tumor growth in various mouse xenograft models and PDX mouse models from 12.5-25 mg/kg