Definition

An orally bioavailable, small molecule inhibitor of menin, with potential antineoplastic activity. Upon oral administration, menin inhibitor DSP-5336 targets and binds to the nuclear protein menin, thereby preventing the interaction between the two proteins menin and menin-mixed lineage leukemia (MLL; myeloid/lymphoid leukemia; KMT2A) and the formation of the menin-MLL complex. This reduces the expression of downstream target genes and results in an inhibition of the proliferation of MLL-rearranged leukemic cells. The menin-MLL complex plays a key role in the survival, growth, transformation and proliferation of certain kinds of leukemia cells.