Phase II Results Advance NPM1-Mutant AML Ziftomenib Monotherapy, Preparation for Combination Trials

Encouraging topline results have been reported from the KOMET-001 registration-directed phase II clinical trial of ziftomenib monotherapy for relapsed or refractory NPM1-mutant (NPM1-m) acute myeloid leukemia (AML). Kura Oncology, Inc., which is developing and commercializing ziftomenib jointly with Kyowa Kirin Co., Ltd., announced the results in a press release.

In KOMET-001, ziftomenib led to statistically significant achievement of the trial’s primary endpoints of complete response (CR) and CR with partial hematologic recovery, as well as a favorable benefit-risk profile.

“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,” wrote Kura Oncology president and chief executive officer Troy Wilson, PhD, JD.

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

At this point, the two pharmaceutical companies co-developing ziftomenib have submitted the data from KOMET-001 for presentation at a medical conference in the second quarter of 2025. By second quarter of 2025, Kura also expects to submit a New Drug Application to the FDA for ziftomenib.

If end-of-phase I meetings with the FDA are successful, ziftomenib’s co-developers plan to initiate the KOMET-017 protocol for two randomized, double-blind, placebo-controlled, registrational phase III trials to evaluate the agent in intensive and nonintensive combination regimens for patients with NPM1-m and KMT2A-rearranged AML. The two trials would take place in the second half of 2025 and are intended to accelerate US approval of the drug.

“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,” wrote 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.