DS-3201 is primarily investigated for its use in oncology, specifically as a menin-MLL (mixed lineage leukemia) interaction inhibitor. It shows promising application in the treatment of certain types of acute leukemias, particularly those involving MLL rearrangements or mutations in the FLT3 gene. By disrupting the menin-MLL protein-protein interaction, DS-3201 helps suppress the expression of leukemogenic genes, leading to inhibition of cancer cell proliferation and induction of apoptosis in malignant cells. It is being studied in clinical trials for relapsed or refractory acute myeloid leukemia (AML) and other hematological malignancies, offering a targeted therapeutic approach with potential for improved outcomes in genetically defined patient populations.