Building on our earlier Biology Tangent episode on menin inhibition, this clinical deep-dive explores the pivotal AUGMENT-101 trial results that led to FDA approval of revumenib — the first licensed menin inhibitor for acute leukaemias. Revumenib has shown meaningful activity in relapsed or refractory KMT2A-rearranged and NPM1-mutated acute leukaemias, illustrating how targeting transcriptional dependencies can restore differentiation, even in heavily pretreated patients. We examine key efficacy data, including CR/CRh rates of 23 percent — exceeding historical expectations — with nearly 40 percent of responders proceeding to transplant. The episode also explores biomarkers of response, such as IDH co-mutations; the expected safety profile, marked by differentiation syndrome and QTc prolongation; and emerging resistance mechanisms likely to influence future therapeutic development.

Episode type: Deep Dive suitable for anyone involved in caring for acute leukaemia patients, or interested in menin inhibitors generally.