{"entity":{"id":"augment-101","kind":"trial","name":"AUGMENT-101","aka":[],"tldr":"The trial that turned menin inhibition from an idea into the first approved drug for KMT2A-rearranged leukaemia.","summary":"KMT2Ar cohort (n=57 efficacy population): CR+CRh 22.8%, ORR 63.2%, most CR/CRh MRD-negative; median OS ~8 months. JCO 2024. Led to November 2024 approval in KMT2Ar acute leukaemia (adults and children ≥1 year) and, with the NPM1 cohort, to the October 2025 NPM1 approval. Differentiation syndrome and QT prolongation were the key toxicities; MEN1 resistance mutations emerged in about a third of relapsing patients.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04065399","url":"https://clinicaltrials.gov/study/NCT04065399"}],"tags":[],"related":[],"cancers":["aml","all-leukemia","aml-npm1-kmt2a"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["menin","kmt2a","npm1"],"drugs":["revumenib"],"companies":["syndax"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":[],"people":["eytan-stein","ghayas-issa"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT04065399","phase":"1/2","setting":"Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy","sponsor":"Syndax","result":"CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort.","started":"2019-11-05","startedType":"actual","yearReported":2024,"enrolled":94,"enrolledBasis":"treated","enrolledNote":"ClinicalTrials.gov lists an estimated 447 participants for the whole phase 1/2 programme, which is still recruiting; the JCO 2024 KMT2A-rearranged analysis treated 94 patients between October 2021 and July 2023 (57 efficacy-evaluable).","outcomes":[{"endpoint":"CR + CRh (KMT2Ar cohort)","primary":true,"unit":"%","arms":[{"name":"Revumenib","n":57,"value":22.8}],"source":"https://ascopubs.org/doi/10.1200/JCO.24.00826"},{"endpoint":"Overall response rate","unit":"%","arms":[{"name":"Revumenib","value":63.2}],"source":"https://doi.org/10.1200/JCO.24.00826"}],"replication":"KOMET-001 (ziftomenib, a different menin inhibitor) produced a similar CR rate in NPM1-mutated AML, confirming the target."},"route":"/trials/augment-101/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"aml-paediatric","kind":"cancer","name":"Acute myeloid leukaemia in children","route":"/cancers/aml-paediatric/"},{"id":"all-infant","kind":"cancer","name":"Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)","route":"/cancers/all-infant/"},{"id":"aml-npm1-kmt2a","kind":"cancer","name":"NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia","route":"/cancers/aml-npm1-kmt2a/"},{"id":"all-paediatric-relapsed","kind":"cancer","name":"Relapsed and refractory acute lymphoblastic leukaemia in children","route":"/cancers/all-paediatric-relapsed/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"}],"target":[{"id":"kmt2a","kind":"target","name":"KMT2A (MLL) rearrangement","route":"/targets/kmt2a/"},{"id":"menin","kind":"target","name":"Menin","route":"/targets/menin/"},{"id":"npm1","kind":"target","name":"NPM1 mutation","route":"/targets/npm1/"}],"drug":[{"id":"revumenib","kind":"drug","name":"Revumenib","route":"/drugs/revumenib/"}],"company":[{"id":"syndax","kind":"company","name":"Syndax Pharmaceuticals","route":"/companies/syndax/"}],"term":[{"id":"differentiation-syndrome","kind":"term","name":"Differentiation syndrome","route":"/terms/differentiation-syndrome/"}],"person":[{"id":"eytan-stein","kind":"person","name":"Eytan M. Stein","route":"/people/eytan-stein/"},{"id":"ghayas-issa","kind":"person","name":"Ghayas C. Issa","route":"/people/ghayas-issa/"}],"idea":[{"id":"idea-menin-infant-all","kind":"idea","name":"Menin inhibitors for infant KMT2A-rearranged ALL","route":"/ideas/idea-menin-infant-all/"}],"paper":[{"id":"paper-augment-101-revumenib-menin-nature-2023","kind":"paper","name":"AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation","route":"/key-papers/paper-augment-101-revumenib-menin-nature-2023/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}