Multinational phase 3 study of addition of ivosidenib to azacitidine for newly diagnosed AML with IDH1 mutation

Somatic mutations in isocitrate dehydrogenase 1 (IDH1) occur in 6-10% of patients with acute myeloid leukemia. Ivosidenib (IVO) is an oral inhibitor of the mutant IDH1 enzyme. The multinational phase 3 AGILE study compared IVO plus azacitidine (AZA) versus placebo (PBO) plus AZA for newly diagnosed IDH1 mutated AML in patients ineligible for intensive chemotherapy. IVO + AZA, compared with PBO + AZA, resulted in improved EFS, OS, and clinical response. The safety of IVO + AZA was favorable and consistent with previous studies.