Amir Fathi, MD, MPH, Massachusetts General Hospital Cancer Center, Boston, MA, provides an overview of the Phase I/II first-in-human study (NCT04067336) of KO-539, a selective menin-KMT2A inhibitor, in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML). KO-539 achieves its therapeutic impact by blocking the maturation and differentiation of myeloid malignant cells. Dr Fathi discusses the findings from the study and comments on the impact of genomic profile on outcomes. Notably, out of 12 patients treated so far, two patients achieved robust complete remissions, however, this data is preliminary and further clinical trials are required to confirm the efficacy of KO-539 in AML. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.