Kura Oncology, Inc. (Nasdaq: KURA) announced the publication of a manuscript in Blood, the journal of the American Society of Hematology, detailing the preclinical development of ziftomenib, a menin inhibitor approved by the U.S. Food and Drug Administration in 2025 for adult patients with relapsed or refractory NPM1-mutated acute myeloid leukemia (AML).

The publication, based on a collaboration between Kura Oncology and the University of Michigan initiated in 2014, describes ziftomenib's binding profile, its inhibition of the menin-KMT2A interaction, and its activity across multiple genetically defined leukemia models.

In preclinical studies across KMT2A-rearranged, NPM1-mutated, and NUP98-rearranged leukemia models, ziftomenib showed suppression of KMT2A-regulated genes including MEIS1 and HOXA9, induction of differentiation, and reduced leukemia cell viability. The compound also induced leukemia regression and extended survival in xenograft models, including durable responses after treatment discontinuation in a patient-derived model.

Researchers also evaluated ziftomenib against treatment-emergent MEN1 mutations associated with resistance to menin inhibition, finding that ziftomenib retained activity against certain resistance-associated mutations where other menin inhibitors did not. In the KOMET-001 clinical trial, the MEN1-M327I resistance mutation emerged in only 1 of 29 evaluable patients.

Kura Oncology is continuing to study ziftomenib, marketed as KOMZIFTI, in combination with standards of care across newly diagnosed and relapsed or refractory NPM1-mutated AML, KMT2A-rearranged AML, FLT3-mutated AML, and additional indications including advanced gastrointestinal stromal tumors.