Kyowa Kirin and Kura Oncology initiate a Japanese Phase 2 trial of ziftomenib in relapsed or refractory NPM1-mutated AML, advancing global development efforts.
Written By: Pharmacally Medical News Desk
Kyowa Kirin Co., Ltd. and Kura Oncology, Inc. have announced that the first patient has been dosed in a Japanese Phase 2 registrational trial evaluating ziftomenib, an oral menin inhibitor, for adults with relapsed or refractory (R/R) NPM1-mutated acute myeloid leukemia (AML), a molecular subtype that accounts for approximately 30% of AML cases.
The multicenter, single-arm, open-label study is designed to assess both efficacy and safety, with the primary endpoint defined as a composite complete remission rate, including complete remission (CR) and complete remission with partial hematologic recovery (CRh).
The companies intend to use data from this study to support a regulatory submission in Japan.
Ziftomenib previously received approval from the U.S. Food and Drug Administration in November 2025 under the brand name KOMZIFTI™ for adult patients with R/R NPM1-mutated AML who have no satisfactory alternative treatment options.
Yoshifumi Torii, Ph.D., Chief Medical Officer of Kyowa Kirin, stated that patients in this setting face significant limitations with existing therapies and emphasized the need for new treatment options. He added that the company aims to advance the study efficiently to confirm the drug’s efficacy and safety, with the goal of delivering a new therapeutic option to patients in Japan as soon as possible.
Mollie Leoni, M.D., Chief Medical Officer at Kura Oncology, described the trial initiation as an important step in the company’s global development strategy, noting that prior clinical studies conducted outside Japan have demonstrated a favorable efficacy and safety profile, along with the convenience of once-daily oral dosing.
Beyond the relapsed or refractory setting, ziftomenib is being investigated in combination with standard-of-care and targeted therapies for earlier lines of AML treatment, including patients with NPM1 mutations, KMT2A translocations, and FLT3 mutations, reflecting efforts to expand its potential use across a broader patient population.
The development of ziftomenib is supported by a global collaboration established in November 2024 between Kyowa Kirin and Kura Oncology.
Under the terms of the agreement, Kyowa Kirin is responsible for leading development, regulatory activities, and commercialization outside the United States, while Kura Oncology retains responsibility for these functions within the United States and overseas manufacturing.
Both companies collaborate on commercialization activities in the U.S. under a jointly developed strategy, reflecting a coordinated approach to accelerate development timelines and enable broader global access to the therapy.
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