More recently MLL-menin binding inhibitors have entered clinical testing as single agents for AML (K0539, NCT04067336 and SNDX-5613, NCT04065399), with great potential to emerge as important targeted therapies in NPM1-mutated and KMT2A-rearranged AML (Figure 1). This evidence concerns the gene NPM1 and acute myeloid leukemia.