M-525 binds to menin (IC50: 3 nM) and achieves low nanomolar potencies in cell growth inhibition and in the suppression of MLL regulated gene expression in MLL leukemia cells. M-525 is a first-in-class, highly potent, irreversible, and covalent menin-MLL protein-protein interaction inhibitor. It has an Anti-leukemia activity.

M-525 (3-300 nM; 24 h) is potent and effective in suppressing the expression of MEIS1 and HOX genes in MV4 11 cells carrying the MLL-AF4 fusion and in MOLM-13 cells carrying the MLL-AF9 fusion. M-525 achieves an IC50 of 3 nM in the MV4 11 cell line and has an IC50 of 2 μM in the HL-60 cell line. It shows high cellular specificity over non-MLL leukemia cells.