Further preclinical work has demonstrated that pinometostat has increased activity when combined with chemotherapy and hypomethylating agents, leading to two ongoing Phase I/II clinical trials investigating the agent in combination with standard induction chemotherapy or azacitidine in newly diagnosed and R/R AML harboring an MLL rearrangement (Klaus et al., 2014; NCT03724084 and NCT03701295; Table 1). The gene discussed is KMT2A; the disease is acute myeloid leukemia.