Revumenib combination therapy associated with high response rates in relapsed/refractory AML

An all-oral treatment regimen of revumenib, decitabine/cedazuridine, and venetoclax displayed an acceptable safety profile and was associated with high efficacy rates in paediatric and adult patients with relapsed or refractory acute myeloid leukaemia (AML) who were susceptible to menin inhibitors.

The phase 1/2 SAVE study (NCT05360160) enrolled patients with relapsed or refractory AML and KMT2A rearrangement, NPM1 mutation, or NUP98 rearrangement and exposed them to revumenib (a menin inhibitor), decitabine/cedazuridine, and venetoclax combination therapy. Dr Ghayas Issa (MD Anderson Cancer Center, TX, USA) discussed the results of 5 paediatric and 28 adult participants [1].

The most frequently observed grade 3 or higher adverse events (AEs) were febrile neutropenia (33%), lung infection (33%), elevated AST/ALT levels (18%), sepsis (18%), respiratory failure (18%), and decreased platelet counts (18%). “We also saw 3 cases of QT prolongation and 1 case of differentiation syndrome,” said Dr Issa.

“The overall response rate was very high, at 82%,” stressed Dr Issa. The complete response (CR) rate was 39%. Moreover, 14 of the 16 participants who achieved a CR/CRh reached MRD negativity (10^-4), and 13 participants proceeded to haematopoietic stem cell transplantation (see Table). The median time to the first morphologic response was 28 days. Finally, the 6-month overall survival rate was 68%.

Table: High response rates of revumenib combination therapy in relapsed/refractory AML [1]



CR/CRh, complete response and complete response with partial hematologic recovery; HSCT, haematopoietic stem cell transplantation; MLFS, morphological leukaemia-free state; MRD, minimal residual disease; NE, not estimable; ORR, objective response rate.

“The experimental treatment regimen delivered an acceptable safety profile and high efficacy rates in this population of patients with relapsed or refractory AML,” decided Dr Issa. “The study is currently enrolling untreated patients with AML who are susceptible to menin inhibitors and are ineligible for intensive chemotherapy.”

1. Issa GC, et al. Phase I/II study of the all-oral combination of revumenib with decitabine/cedazuridine and venetoclax in R/R AML (SAVE). Abstract 216, 66^th ASH Annual Meeting, 7–10 December 2024, San Diego, CA, USA.

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Posted on 29 January 202529 January 2025