In animal models, KO-539 was given by the oral route and evidenced weak toxicity in mice and potent leukemic cells differentiation (CD11b-expressing cells in bone marrow of treated mice) and anti-leukemic effects (reduced splenomegaly and increased global survival) in both the MV4-11 MPAL model and patient-derived xenografts from the NPM1c+/FLT3-mut AML subtype [149]. The gene discussed is ITGAM; the disease is acute myeloid leukemia.