Description
A TRPML1 agonist; activates whole-endolysosome IML1 in vacuolin-1-enlarged isolated vacuoles (EC50 = 333 nM); is cytotoxic to MeWo and M12 melanoma cells but not normal melanocytes at 3 µM; induces gastric acid secretion in isolated stomach from wild-type but not TRPML1-/- mice; decreases the necrotic area and percentage of centrally nucleated fibers in the dystrophin-deficient mdx mouse model of Duchenne muscular dystrophy at 2 and 5 mg/kg