SBI-0640756 is primarily used in biomedical research as a potent and selective inhibitor of the menin-MLL (mixed lineage leukemia) protein-protein interaction. This interaction plays a critical role in the development and progression of certain types of leukemia, particularly MLL-rearranged (MLL-r) leukemias, which are aggressive forms of acute leukemia commonly found in infants and associated with poor prognosis. By disrupting the menin-MLL interaction, SBI-0640756 effectively suppresses the expression of key oncogenes such as HOXA9 and MEIS1, which are abnormally activated in MLL-r leukemia cells. This leads to inhibition of cancer cell proliferation, induction of apoptosis, and differentiation of leukemic blasts in preclinical models. The compound has demonstrated robust anti-leukemic activity in cell line studies and animal models, making it a valuable tool compound for validating menin as a therapeutic target. It is widely used in pharmacological studies to explore signaling pathways involved in leukemogenesis and to assess combination therapies with other anti-cancer agents such as FLT3 inhibitors or chemotherapy drugs. Due to its target specificity and efficacy in preclinical settings, SBI-0640756 serves as a lead structure for the development of clinical candidates aimed at treating MLL-rearranged and NPM1-mutated leukemias. Its application remains confined to research and drug discovery efforts, with ongoing studies focused on optimizing pharmacokinetic properties and evaluating potential for therapeutic translation.