In HEK293 cells, MI-2 accessed the protein target menin-MLL and effectively inhibited the menin-MLL-AF9 interaction. MI-2 effectively blocked cell proliferation, and induced cell apoptosis in human MLL leukemia cell lines harboring different MLL translocations MLL-AF9 and MLL-ENL, with the GI50 value of about 5 μM for MI-2. MI-2 showed little effect on the cell growth of E2A-HLF transduced BMC with the GI50 of > 50 μM. MI-2 specifically reduced the immortalization potential of cells transformed with MLL fusion oncoproteins by downregulating the expression of target genes required for MLL fusion protein oncogenic activity [1].

After 7 days of treatment with MI-2, MLL-AF9 transformed BMC showed great morphology changes and the expression of CD11b was greatly increased [1].