MI-463 is a potent and orally bioavailable inhibitor of the menin-mLL interaction (IC50: 15.3 nM).

将含有mLL-AF9癌基因的鼠骨髓细胞（BMC）用MI-463处理可显著抑制其增长（GI50：0.23 μM）。MI-463能有效诱导MLL白血病细胞分化。使用低于微摩尔浓度的MI-463还能显著降低Hoxa9和Meis1的表达。

MI-463在MLL白血病的小鼠模型中显示出显著的生存益处。该化合物具有非常有利的类药性质，包括在小鼠中的代谢稳定性和PK概况。单次静脉或口服给药后，MI-463在外周血中达到高浓度，同时展示高口服生物利用度（45%）。通过每天一次的腹腔内（i.p.）给药，MI-463强烈抑制肿瘤生长。在MI-463处理后，目标基因mLL融合蛋白的表达，包括HOXA9和MEIS1，显著降低。

Leukemia cells are treated with MI-463 or 0.25% DMSO and cultured at 37 °C for 7 days. Media is changed on day 4, viable cell numbers are restored to the original concentration and MI-463 is re-supplied. MTT cell proliferation assay kit is then employed, and plates are read for absorbance at 570 nm using a microplate reader.

For efficacy studies in MV4;11 subcutaneous xenograft mice model, 5×10^6 cells are injected into the 4-6 week old female BALB/c nude mice. Treatment is started when the tumor size reached ~100 mm^3. Vehicle (25% DMSO, 25% PEG400, 50% PBS) or MI-463 are administrated once daily at designated doses using i.p. injections.