Mice: effects of BAG-956 were investigated in vivo alone (at a dose of 100 mg/kg) and in combination with nilotinib (at a dose of 20 mg/kg) using mice IV injected with 32D.p210-luc+ cells. Overall tumor burden, as assessed by measured levels of bioluminescence in vehicle- and drug-treated mice, was observed to be the lowest in the BAG-956 (100 mg/kg) + nilotinib (20 mg/kg)-treated group, compared with mice treated with vehicle or either agent alone.

BAG-956 was randomly screened against a panel of human leukemia cell lines, including AML, ALL, and CML. All of the cell lines showed appreciable sensitivity to increasing concentrations of BAG-956 after approximately 3 to 4 days of treatment, with a dose-dependent decline in cell viability and an average IC50 less than or equal to 100 nM for most cell lines analyzed. Most cells were killed at 500 to 1000 nM BAG-956. A group of AML patient samples (AML1, AML2, AML4, and AML5) showed various degrees of sensitivity to BAG-956 after 24 hours of treatment, with inhibition of cellular proliferation observed at concentrations of BAG-956 between 100 and 1000 nM.