Phase I/II AUGMENT-101 trial: Post-transplant revumenib in R/R NPM1m, KMT2Ar, or NUP98r AML

Results from a post hoc analysis of the phase I/II AUGMENT-101 (NCT04065399) trial, evaluating the safety and efficacy of revumenib as post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) maintenance in pediatric and adult patients with relapsed/refractory (R/R) NPM1-mutated (NPM1m; n = 4), KMT2A-rearranged (KMT2Ar; n = 14), or NUP98-rearranged (NUP98r; n = 1) acute myeloid leukemia (AML), were presented by Andrius Žučenka at the European Hematology Association (EHA) 2026 Congress, June 11–14, 2026, Stockholm, SE.

Key data: The median RFS and OS were not reached (NR) in patients receiving post-allo-HSCT revumenib, regardless of genotype. The estimated 12-month RFS rates were 50.0%, 85.1%, and not applicable (NA) in the NPM1m, KMT2Ar, and NUP98r groups, respectively. The estimated 12-month OS rates were 75.0%, 100.0%, and NA, respectively. Grade ≥3 treatment-emergent adverse events (TEAEs) occurred in 74% of patients overall; the most common Grade ≥3 TEAEs were thrombocytopenia/platelet count decreased (42%) and neutrophil count decreased (16%). Serious adverse events (SAEs) occurred in 42% of patients. There were no differentiation syndrome (DS) events and one Grade ≥2 corrected QT interval using Fridericia’s formula (QTcF) prolongation event, which was considered unrelated to revumenib.

Key learning: Revumenib demonstrated sustained RFS across genotypes with a tolerable safety profile as post-allo-HSCT maintenance in patients with R/R NPM1m, KMT2Ar, and NUP98r AML, supporting continued evaluation in clinical trials in the post-transplant setting (NCT06575296).