Ivosidenib is a mutant IDH1 (mIDH1) inhibitor that demonstrated clinical benefit in combination with azacitidine for treatment of mIDH1 acute myeloid leukemia (AML) in the phase 3 AGILE trial. Results from AGILE led to the approval of ivosidenib plus azacitidine for patients with newly diagnosed mIDH1 AML unfit for intensive chemotherapy in Japan. However, data on the use of ivosidenib plus azacitidine in Japanese patients with mIDH1 AML are lacking. Here we present data from six Japanese patients enrolled in AGILE, of whom, three received ivosidenib plus azacitidine and three received placebo plus azacitidine. At data cut-off, Japanese patients in AGILE had an overall survival of 4.4-12.8 months and 24.8-36.5 months in the placebo plus azacitidine and ivosidenib plus azacitidine groups, respectively. Of the Japanese patients enrolled in AGILE, 2 of 3 (66.7%) patients receiving ivosidenib plus azacitidine and none receiving placebo plus azacitidine achieved complete remission within 24 weeks. Results from the Japanese population enrolled in AGILE are aligned with those of the overall study. The efficacy of ivosidenib plus azacitidine in Japanese mIDH1 AML patients enrolled in AGILE who were ineligible for intensive chemotherapy appears to be consistent with the overall study population.
Kiguchi et al. (2026) studied this question.