Syndax SAVE trial data published in JCO showing 88% ORR
Syndax Pharmaceuticals has announced the publication of data from its Phase 1/2 SAVE trial in the Journal of Clinical Oncology, coinciding with a simultaneous presentation at the European Hematology Association 2026 Congress in Stockholm. The trial tested an all-oral regimen of revumenib (Revuforj), decitabine/cedazuridine, and venetoclax in patients with relapsed or refractory AML harbouring NPM1 mutations, KMT2A rearrangements, or NUP98 rearrangements.
Across 42 enrolled patients, the overall response rate was 88%, the composite complete remission rate was 71%, and 60% achieved complete remission or complete remission with partial haematological recovery. Measurable residual disease negativity was recorded in 80% of evaluable patients who achieved composite complete remission, with the majority of NPM1 NGS-evaluable patients falling below the assay's limit of detection. Forty-five per cent of the cohort proceeded to stem cell transplant, and median overall survival after transplant had not been reached at the time of data cut.
Results in venetoclax-exposed patients
One of the more clinically significant findings concerns patients who had already received venetoclax, a population historically associated with median survival of around two to three months. In the SAVE cohort, the CR/CRh rate in these 22 patients was 50%, and median overall survival was at least 12 months, broadly comparable to the venetoclax-naive group. Ghayas C. Issa, Associate Professor of Leukemia at MD Anderson Cancer Center and principal investigator of the trial, said the approximately 12-month median overall survival in prior venetoclax-exposed patients represented a stark contrast to historical benchmarks of under three months, and suggested that menin inhibition may restore sensitivity to BCL2 inhibition after resistance develops.
The combination was generally well tolerated. The most common Grade 3 or higher adverse events were febrile neutropenia (36%), lung infection (21%), thrombocytopenia (21%), and liver enzyme elevation (21%). Rates of Grade 3 or higher differentiation syndrome and QTc prolongation were both 5%, below the rates observed in prior revumenib monotherapy data.
Market context and regulatory path
Revumenib holds FDA approval as monotherapy for relapsed or refractory AML with susceptible NPM1 mutation or KMT2A translocation, making it the only approved menin inhibitor on the market. The SAVE findings add combination evidence in a disease setting where options remain limited, and where the menin inhibitor class is drawing increasing competitive interest. A number of clinical-stage programmes are advancing menin inhibitors from other developers, reflecting a broader consensus that the menin-MLL axis is a durable oncology target.
Syndax is now translating the SAVE signal into two registrational studies. The EVOLVE-2 trial is evaluating revumenib with venetoclax and a hypomethylating agent in newly diagnosed patients unfit for intensive chemotherapy, while the RAVEN trial targets fit newly diagnosed patients. Both studies will be watched closely by the haematology community as potential pivotal datasets that could extend revumenib's label beyond its current relapsed or refractory indication. The timeline for readouts from those trials was not specified in the release, but their initiation underlines Syndax's confidence that the SAVE combination data are reproducible in earlier lines of therapy.
With a 22-month median follow-up, the durability data are maturing, and the publication in the Journal of Clinical Oncology provides a peer-reviewed foundation that should support ongoing investigator-initiated uptake of the regimen ahead of any label expansion.