Kyowa Kirin and Kura start Japanese Phase II ziftomenib trial in relapsed or refractory AML

Kyowa Kirin and Kura Oncology have started a Japanese Phase II registrational study of oral menin inhibitor ziftomenib in adult patients with relapsed or refractory (r/r) NPM1-mutated acute myeloid leukaemia (AML). Kyowa Kirin intends to seek regulatory approval in Japan following the completion of the trial.

The single-arm, open-label, multi-centre trial is assessing the efficacy and safety of ziftomenib in these patients. Its primary endpoint is the composite complete remission rate, which includes both complete remission (CR) and complete remission with partial haematologic recovery (CRh).

Ziftomenib received the US Food and Drug Administration (FDA) approval in November 2025, under the brand name Komzifti, for adults with r/r NPM1-mutated AML who have no satisfactory alternative treatment options. Ziftomenib is also in development, along with standard-of-care and targeted therapies for front-line AML with NPM1 mutations, lysine methyltransferase 2A (KMT2A) translocations and FMS-like tyrosine kinase 3 (FLT3) mutations.

Last month, Kyowa Kirin announced the global discontinuation of all ongoing clinical trials for rocatinlimab, an anti-OX40 monoclonal antibody under investigation for moderate-to-severe atopic dermatitis, prurigo nodularis, and moderate-to-severe asthma.