{"entity":{"id":"idea-menin-infant-all","kind":"idea","name":"Menin inhibitors for infant KMT2A-rearranged ALL","aka":[],"tldr":"Infant leukaemia is driven almost entirely by KMT2A fusions, which menin inhibitors were built to attack. Add them to the new blinatumomab-containing backbone.","summary":"Infant ALL EFS plateaued below 50% for two decades; blinatumomab (Interfant-21) is the first step forward. Revumenib is approved for KMT2Ar acute leukaemia including children ≥1 year and has paediatric formulation data; combining with chemotherapy and blinatumomab in the first year of life is the obvious next trial.","asOf":"2026-09-07","links":[{"label":"Issa et al., The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia (Nature 2023)","url":"https://doi.org/10.1038/s41586-023-05812-3"}],"tags":[],"related":[],"cancers":["all-leukemia","all-infant"],"sections":[],"technologies":[],"targets":["kmt2a","menin"],"drugs":["revumenib","ziftomenib","blinatumomab"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["interfant-06","augment-101"],"people":[],"bottlenecks":[],"keyPapers":["paper-blinatumomab-all-leukemia-n-engl-j-med-2017","paper-blinatumomab-all-leukemia-lancet-oncol-2015","paper-blinatumomab-all-leukemia-j-clin-oncol-2011"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"Adding a menin inhibitor to Interfant-21-type therapy raises 2-year EFS above 80% in KMT2A-rearranged infants without excess differentiation syndrome or hepatotoxicity.","rationale":"KMT2A fusion is the sole driver in most infant ALL; menin inhibition produces MRD-negative remissions in relapsed KMT2Ar leukaemia.","test":"International single-arm pilot then randomised addition of revumenib or ziftomenib to Interfant-21 backbone, with pharmacokinetics and CYP3A4 interaction monitoring in infants.","maturity":"early-clinical"},"route":"/ideas/idea-menin-infant-all/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"all-infant","kind":"cancer","name":"Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)","route":"/cancers/all-infant/"}],"target":[{"id":"kmt2a","kind":"target","name":"KMT2A (MLL) rearrangement","route":"/targets/kmt2a/"},{"id":"menin","kind":"target","name":"Menin","route":"/targets/menin/"}],"drug":[{"id":"blinatumomab","kind":"drug","name":"Blinatumomab","route":"/drugs/blinatumomab/"},{"id":"revumenib","kind":"drug","name":"Revumenib","route":"/drugs/revumenib/"},{"id":"ziftomenib","kind":"drug","name":"Ziftomenib","route":"/drugs/ziftomenib/"}],"trial":[{"id":"augment-101","kind":"trial","name":"AUGMENT-101","route":"/trials/augment-101/"},{"id":"interfant-06","kind":"trial","name":"Interfant-06","route":"/trials/interfant-06/"}],"paper":[{"id":"paper-blinatumomab-all-leukemia-n-engl-j-med-2017","kind":"paper","name":"Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia","route":"/key-papers/paper-blinatumomab-all-leukemia-n-engl-j-med-2017/"},{"id":"paper-blinatumomab-all-leukemia-lancet-oncol-2015","kind":"paper","name":"Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study","route":"/key-papers/paper-blinatumomab-all-leukemia-lancet-oncol-2015/"},{"id":"paper-blinatumomab-all-leukemia-j-clin-oncol-2011","kind":"paper","name":"Targeted therapy with the T-cell-engaging antibody blinatumomab of chemotherapy-refractory minimal residual disease in B-lineage acute lymphoblastic leukemia patients results in high response rate and prolonged leukemia-free survival","route":"/key-papers/paper-blinatumomab-all-leukemia-j-clin-oncol-2011/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}]}}