FDA Accepts Priority Review for Ziftomenib in NPM1-Mutant AML

Kura Oncology, Inc., in partnership with Kyowa Kirin Co., Ltd., has announced that the FDA has accepted its New Drug Application (NDA) for ziftomenib. This application seeks approval for ziftomenib as a treatment for adult patients suffering from relapsed or refractory acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The FDA has granted a Priority Review and set a Prescription Drug User Fee Act (PDUFA) target action date for November 30, 2025.

Troy Wilson, President and CEO of Kura Oncology, emphasized the significance of the FDA's acceptance, marking a major achievement for the companies and offering hope to patients who currently have limited treatment options. This milestone is underscored by robust clinical data supporting ziftomenib and reflects the dedication of the teams involved. With Kyowa Kirin, Kura is keen to collaborate closely with the FDA throughout the review process and prepare for the potential launch of ziftomenib, which could significantly affect patients' lives.

The NDA's foundation lies in the positive findings from the Phase 2 KOMET-001 trial, which targeted R/R NPM1-mutant AML (NCT #04067336). This trial successfully met its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh), achieving statistical significance. Moreover, ziftomenib demonstrated a favorable safety and tolerability profile, with limited myelosuppression and only 3% of patients discontinuing due to treatment-related reasons.

Takeyoshi Yamashita, Executive Vice President and Chief Medical Officer at Kyowa Kirin, noted the critical need for innovative treatments for adult R/R NPM1-m AML patients, who often face a bleak prognosis. The NDA acceptance is a pivotal step in ongoing efforts to develop new therapeutic strategies for AML. Kyowa Kirin and Kura are dedicated to ensuring that ziftomenib is accessible to patients swiftly upon approval, aiming to make a substantial impact on those affected by this challenging condition.

The KOMET-001 trial is designed to evaluate ziftomenib's clinical efficacy, safety, and tolerability. It is notably the only investigational treatment to receive Breakthrough Therapy Designation from the FDA for R/R NPM1-mutant AML. Ziftomenib has also been granted Fast Track and Orphan Drug Designations. Detailed results from the KOMET-001 study are scheduled for presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, with an encore presentation planned for the 2025 European Hematology Association (EHA) Congress.

Acute myeloid leukemia (AML) is a prevalent acute leukemia in adults, characterized by the abnormal production of myeloblasts, red blood cells, or platelets in the bone marrow. Despite the availability of numerous treatments, patients with AML continue to face dismal prognoses and significant unmet needs. The menin pathway is a key factor in many genetic alterations associated with AML, with NPM1 mutations being among the most common. These mutations account for approximately 30% of AML cases. Despite high initial response rates to frontline therapies, relapse rates in NPM1-mutant AML patients are significant, resulting in poor survival outcomes. Currently, there are no FDA-approved therapies specifically targeting NPM1-mutant AML.

Ziftomenib represents a promising development as a potent and selective oral menin inhibitor. It is currently under investigation for the treatment of genetically defined AML patients with significant unmet needs. It received Breakthrough Therapy Designation from the FDA in April 2024, based on data from Kura's KOMET-001 trial. Kura Oncology and Kyowa Kirin have been working closely to evaluate ziftomenib in various clinical settings, including its combination with existing therapies for newly diagnosed and R/R NPM1-mutant and KMT2A-rearranged AML.