Kura Oncology has introduced ziftomenib, a groundbreaking medication aimed at treating patients suffering from relapsed/refractory (R/R) NPM1-mutant acute myeloid leukaemia (AML).
Ziftomenib, an innovative oral investigational drug, targets the menin-KMT2A/MLL protein-protein interaction, addressing the urgent needs of genetically defined AML patients.
During the KOMET-001 phase 1 trial, ziftomenib exhibited a promising safety profile and tolerability, with reported adverse events primarily aligning with the characteristics of the underlying disease.
The monotherapy demonstrated optimal clinical activity at the daily dose of 600 mg, resulting in a 35 percent complete remission rate among 20 NPM1-mutant AML patients treated at the recommended phase 2 dose.
Furthermore, ziftomenib is undergoing assessment in combination with standard treatments such as venetoclax/azacitidine or cytarabine plus daunorubicin (7+3) in both NPM1-mutant and KMT2A-rearranged AML (KOMET-007), as well as with gilteritinib, FLAG-IDA, or LDAC in NPM1-mutant and KMT2A-rearranged AML (KOMET-008).
Given that NPM1-mutant AML accounts for approximately 30 percent of new AML cases annually and lacks approved targeted therapies, ziftomenib's designation as a potential treatment underscores its ability to potentially offer significant advancements over existing options.
AML stands as the predominant form of acute leukaemia in adults, initiating when the bone marrow produces abnormal myeloblasts, which are immature white blood cells, red blood cells, or platelets. Despite the array of treatment options accessible for AML, patient prognosis remains bleak, highlighting a persistent and significant gap in meeting their needs.
