Kyowa Kirin and Kura initiate Phase II trial of ziftomenib for AML

Ziftomenib is being developed with standard and targeted therapies for front-line AML with NPM1, KMT2A, or FLT3 mutations. Credit: Ekahardiwito / Shutterstock.com. 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). 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). Kyowa Kirin intends to seek regulatory approval in Japan following the completion of the trial. 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. Kyowa Kirin chief medical officer Yoshifumi Torii said: “Patients with r/r NPM1-m AML often face limitations with existing treatment options and have a critical need for new therapeutic alternatives. Ziftomenib has the potential to provide a new treatment approach for these patients. “The initiation of this trial is part of Kyowa Kirin’s patient-centred drug development efforts in our priority area of ‘haematologic malignancies and refractory haematologic disorders’. “We will appropriately advance this trial and work diligently to confirm efficacy and safety, with the goal of ultimately providing a new treatment option to help address unmet needs for patients in Japan as soon as possible.” 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.