AGI-6780 is a selective small molecule inhibitor primarily used in research settings to target mutant forms of the isocitrate dehydrogenase 2 (IDH2) enzyme, particularly the R140Q mutation. It is employed to study the biological effects of blocking the production of the oncometabolite 2-hydroxyglutarate (2-HG), which accumulates in certain cancers and contributes to dysregulated cell differentiation.

In preclinical models, AGI-6780 has demonstrated the ability to reverse aberrant epigenetic modifications and promote differentiation of immature myeloid cells, making it a valuable tool for investigating acute myeloid leukemia (AML) and other IDH2-mutant malignancies. Its application supports the development of targeted therapies by providing insights into the reversibility of oncogenic metabolic effects and the potential for epigenetic reprogramming in cancer treatment.