S64315 (MIK665) was evaluated in a phase 1 study (NCT02992483) and has subsequently progressed to phase II studies in hematological malignancies in combination with VOB560, a novel Bcl-2 inhibitor ((Novartis, Cambridge, MA, USA) (NCT04702425) and azacytidine (NCT04629443) and venetoclax (NCT03672695) in acute myeloid leukemia (AML). The gene discussed is BCL2; the disease is acute myeloid leukemia.