{"entity":{"id":"komet-001","kind":"trial","name":"KOMET-001","aka":[],"tldr":"KOMET-001 was the registration trial for ziftomenib: a quarter of heavily pretreated patients with NPM1-mutated AML reached complete remission on a once-daily pill.","summary":"KOMET-001, trial NCT04067336 sponsored by Kura Oncology and reported in 2025, was the registration study for ziftomenib, in which about a quarter of heavily pretreated patients with relapsed or refractory NPM1-mutated acute myeloid leukaemia reached complete remission on a once-daily tablet. In 112 adults on 600 mg daily the complete remission rate was 23 percent and the overall response rate 33 percent, including patients who had received venetoclax and FLT3 inhibitors, and the FDA approved the drug on 13 November 2025. OnCo links it to acute myeloid leukaemia, menin and the NPM1 mutation as targets and to ziftomenib. Frontline combinations in KOMET-007 show much higher remission rates, as expected for an earlier line, and whether menin inhibitors belong there is the open question.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04067336","url":"https://clinicaltrials.gov/study/NCT04067336"},{"label":"FDA approval coverage (ASCO Post)","url":"https://ascopost.com/news/november-2025/fda-approves-ziftomenib-for-npm1-positive-aml/"}],"tags":[],"related":["src-asco-post"],"cancers":["aml","aml-npm1-kmt2a"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["menin","npm1"],"drugs":["ziftomenib"],"companies":["kura-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT04067336","phase":"1/2","setting":"Relapsed/refractory NPM1-mutated AML: ziftomenib 600 mg daily monotherapy","sponsor":"Kura Oncology","result":"CR 23%, ORR 33%, median OS 6.6 months.","started":"2019-09-12","startedType":"actual","yearReported":2025,"enrolled":112,"enrolledBasis":"treated","enrolledNote":"ClinicalTrials.gov lists an estimated 263 participants for the phase 1/2 study; 112 is the FDA efficacy population at 600 mg quoted in the approval coverage, while the JCO 2025 phase 2 paper reports 92 patients treated between January 2023 and May 2024.","outcomes":[{"endpoint":"Complete remission","primary":true,"unit":"%","arms":[{"name":"Ziftomenib","n":112,"value":23}],"source":"https://www.appliedclinicaltrialsonline.com/view/fda-approves-komzifti-npm1-mutated-aml-positive-komet-001-trial-data"},{"endpoint":"Overall response rate","unit":"%","arms":[{"name":"Ziftomenib","value":33}]},{"endpoint":"Overall survival (median)","unit":"months","arms":[{"name":"Ziftomenib","value":6.6}]}],"replication":"AUGMENT-101 (revumenib) NPM1 cohort produced a comparable CR+CRh rate, replicating the menin-dependence of NPM1-mutant AML with an independent molecule."},"route":"/trials/komet-001/","neighbours":{"collection":[{"id":"src-asco-post","kind":"collection","name":"The ASCO Post","route":"/collections/src-asco-post/"}],"cancer":[{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"aml-npm1-kmt2a","kind":"cancer","name":"NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia","route":"/cancers/aml-npm1-kmt2a/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"}],"target":[{"id":"menin","kind":"target","name":"Menin","route":"/targets/menin/"},{"id":"npm1","kind":"target","name":"NPM1 mutation","route":"/targets/npm1/"}],"drug":[{"id":"ziftomenib","kind":"drug","name":"Ziftomenib","route":"/drugs/ziftomenib/"}],"company":[{"id":"kura-oncology","kind":"company","name":"Kura Oncology","route":"/companies/kura-oncology/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}