Kyowa Kirin and Kura Oncology have initiated a Japanese Phase II registration-directed trial of ziftomenib in adult patients with relapsed/refractory NPM1-mutated acute myeloid leukemia (AML), the companies announced on April 24, 2026. No efficacy or safety data have been reported from this study; the announcement marks trial initiation only.
The Japan Phase II study is a multicenter, single-arm, open-label trial enrolling adults with relapsed/refractory NPM1-mutated AML. Patient enrollment numbers were not disclosed in the announcement. The companies intend to use data from this trial to support a regulatory filing in Japan following completion. No primary endpoint, dosing details specific to this trial, or monitoring committee statements were included in the announcement.
The Japanese trial is distinct from the global KOMET-001 Phase I/II study (NCT04067336), which formed the basis for the US FDA approval of ziftomenib — marketed as Komzifti — for relapsed/refractory NPM1-mutated AML in adults. KOMET-001 enrolled 92 patients at the 600 mg once-daily oral dose and met its primary endpoint of composite complete remission rate. No efficacy data are available from the newly initiated Japanese study at this time, and the data from KOMET-001 are not directly transferable to this separate registration-directed effort.
Ziftomenib is an oral menin inhibitor. Within the same molecularly defined indication, revumenib — another menin inhibitor developed by Syndax Pharmaceuticals — reported a 64% composite complete remission rate in NPM1-mutated relapsed/refractory AML in the AUGMENT-101 study, according to data published in Blood in 2025. Cross-trial comparisons are limited by differences in study design, duration, and patient populations.
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