SNDX-5613 is primarily used in the development of targeted cancer therapies, specifically as an inhibitor of the menin-MLL (mixed lineage leukemia) interaction. This interaction plays a critical role in certain types of acute leukemias, particularly those involving MLL gene rearrangements or NPM1 mutations. By disrupting this protein-protein interaction, SNDX-5613 helps suppress the expression of oncogenic genes that drive the proliferation of leukemia cells.

It is being investigated in clinical trials for the treatment of relapsed or refractory acute leukemia, including acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Its targeted mechanism offers potential for improved selectivity and reduced toxicity compared to conventional chemotherapy. Due to its ability to specifically interfere with leukemia-promoting pathways, SNDX-5613 represents a promising therapeutic option for patients with genetically defined subtypes of acute leukemia who have limited treatment alternatives.