KOMET-001 Phase II Trial Results Encouraging for Ziftomenib Monotherapy in NPM1-Mutant AML

The KOMET-001 registration-directed phase II clinical trial has reported promising results in ziftomenib monotherapy for relapsed or refractory NPM1-mutant (NPM1-m) acute myeloid leukemia (AML). The investigational agent led to statistically significant achievement of the trial’s primary endpoints for complete response (CR) and CR with partial hematologic recovery and featured a favorable benefit-risk profile.

The trial results were announced in a press release from Kura Oncology, Inc. The company is co-developing and commercializing ziftomenib with Kyowa Kirin Co., Ltd.

“We are excited to report positive topline results in R/R [relapsed or refractory] NPM1-m AML patients, underscoring the strong foundation for our ziftomenib program to potentially transform the treatment landscape for these patients,” commented Kura Oncology president and chief executive officer Troy Wilson, PhD, JD.

Ziftomenib, a selective, once-daily oral menin inhibitor, received Breakthrough Therapy Designation from the FDA in April 2024 to treat relapsed or refractory NPM1-m AML. It was granted this designation by the FDA based on data from KOMET-001 and is the only agent of its type to have received this designation.

For their next steps, the two pharmaceutical companies have submitted data from KOMET-001 for presentation at a medical conference to take place in the second quarter of 2025. Kura is also planning for a submission to the FDA of a New Drug Application for ziftomenib in second quarter 2025.

Following successful conclusion of end-of-phase I meetings with the FDA, the co-developers will implement the KOMET-017 protocol for two randomized, double-blind, placebo-controlled, registrational phase III ziftomenib trials. The two trials will evaluate use of this agent in intensive and nonintensive combination regimens for patients with NPM1-m and KMT2A-rearranged AML. These trials, projected to take place in the second half of 2025, are intended by the co-developers to promote the accelerated approval process of the drug in the US.

“The data from the completed KOMET-001 trial and FDA feedback on the planned KOMET-017 protocol strengthens our confidence these trials may offer valuable treatment options for patients throughout the continuum of treatment,” remarked Kyowa Kirin senior managing executive officer and chief medical officer Takeyoshi Yamashita, PhD.

Reference

Kura Oncology and Kyowa Kirin announce positive ziftomenib monotherapy registrational trial and positive FDA feedback for upcoming frontline combination trial designs. Press Release. Kura Oncology, Inc. February 5, 2025. Accessed February 7, 2025.