The first-in-human phase I/II clinical trial of AG-221 (NCT01915498) for patients with IDH2-mutant advanced hematologic malignancies reported an objective response rate (ORR) of 40.3% (71/176) in R/R AML patients, with 19.3% CR rate and a median response duration of 5.8 months [54]. Here, IDH2 is linked to acute myeloid leukemia.