LDC4297 is primarily used in research settings as a potent and selective inhibitor of the menin-MLL (mixed lineage leukemia) protein-protein interaction. This interaction plays a critical role in certain types of leukemia, particularly MLL-rearranged (MLL-r) leukemias, which are aggressive forms of acute leukemia affecting both children and adults. By disrupting the menin-MLL binding, LDC4297 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 and induction of apoptosis, making LDC4297 a valuable tool compound in the development of targeted leukemia therapies. Its high selectivity and cellular activity also make it useful for probing the biological functions of the menin pathway in epigenetic regulation and gene expression. Due to its role in blocking oncogenic transcriptional programs, LDC4297 serves as a lead molecule for the design of clinical candidates in oncology drug discovery.