Fig. 1. Transcriptional changes following treatment with the menin inhibitor revumenib in patients with relapsed or refractory acute leukaemia with KMT2Ar or mutated NPM1.

RNA-seq before and after treatment with revumenib, showing downregulation of critical leukaemogenic target genes MEIS1, HOXA9 and PBX3 and increase in expression of genes associated with differentiation (CD11b, CD14), with transcriptional suppression of FLT3, a putative transcriptional target of MEIS1. The change in bone marrow blast percentage following treatment is shown. Box plots represent median gene expression or median bone marrow blast percentage, and the 95% CI along with percentage change in gene expression following treatment. Responders are shown in red, nonresponders in black. Results were obtained using a paired t-test with a two-sided P value. Adjustments were not made for multiple comparisons. This analysis included a cohort of 21 evaluable patients. Revumenib was administered in continious 28-day cycles. C2D1, day 1 of treatment cycle 2.