Accordingly, since VTP-50469 and MI-3454 also showed efficacy against NPM1-mutated AML in patient-derived xenograft assays [113,124], further investigations on potential benefit of inhibition of Menin-MLL chromatin complex in patients with frank leukemia or, alternatively to pre-emptively target leukemia-specific NPM1-mutated clones in the setting of persisting MRD, are warranted [5,114]. Here, MEN1 is linked to acute myeloid leukemia.