Syndax Publishes SAVE Trial Data Supporting Revuforj Combination Regimen in Relapsed/Refractory AML

Phase 1/2 data published in the Journal of Clinical Oncology position Syndax Pharmaceuticals to pursue label expansion for Revuforj® (revumenib) beyond its current FDA-approved monotherapy indication, with SAVE trial results demonstrating an all-oral triplet regimen producing deep remissions in a heavily pretreated relapsed/refractory AML population. For regulatory affairs leads tracking the supplemental BLA pathway, the dataset now carries peer-reviewed weight alongside its simultaneous presentation at the European Hematology Association 2026 Congress.

Across 42 enrolled patients with NPM1-mutated, KMT2A-rearranged, or NUP98-rearranged R/R AML, the combination of revumenib, decitabine/cedazuridine, and venetoclax achieved an 88% overall response rate (37/42), a 71% composite complete remission rate (30/42), and 80% MRD negativity among evaluable CRc responders. The recommended Phase 2 dose was confirmed as the existing FDA-approved monotherapy dose, a finding that simplifies CMC consistency arguments for any supplemental submission and reduces the manufacturing scale-up burden for the all-oral regimen.

The venetoclax-exposed subgroup warrants particular attention from clinical operations and medical affairs teams: 50% CR/CRh (11/22) and approximately 12-month median overall survival in a population where historical median survival has been reported at under three months. Venetoclax-naïve patients achieved 70% CR/CRh (14/20). Forty-five percent of patients (19/42) proceeded to stem cell transplant, with 63% of those resuming revumenib post-transplant and median overall survival after transplant not yet reached.

The Phase 1/2 SAVE trial was a single-center, open-label study; the enrolled population included five adolescents, with a median age of 40 years and a median of two prior lines of therapy. Syndax's CMO cited the SAVE dataset as direct support for two ongoing pivotal programs: EVOLVE-2, evaluating the combination in newly diagnosed patients unfit for intensive chemotherapy, and RAVEN, targeting fit patients. Both trials will generate the controlled, multi-center data that a supplemental BLA for a new combination indication would require under 21 CFR Part 314 standards.

Process validation and supply readiness for a three-drug all-oral regimen will become a near-term operational checkpoint as EVOLVE-2 and RAVEN advance toward pivotal readouts.

Source: Syndax Pharmaceuticals via GlobeNewswire, 11 June 2026.