Syndax’s Revuforj Shows Up to 97% CRc, 82% ORR and 45% Transplants

In the interim ROAR analysis, 11 heavily pretreated adults with relapsed/refractory NPM1m or KMT2Ar acute leukemia received revumenib monotherapy or in combination, achieving an 82% overall response rate and 64% CR/CRh. 36% proceeded to hematopoietic stem cell transplant, and safety was consistent with known profiles with no Grade ≥3 QTc prolongation or differentiation syndrome.

In the SNDX-5613-0708 phase 1 trial, 35 newly diagnosed NPM1m or KMT2Ar AML patients received revumenib with intensive chemotherapy, yielding a 97% composite complete remission rate and 86% MRD negativity among responders. Both dose level 1 (110/220 mg) and dose level 2 (160/270 mg) cohorts showed similar response rates and safety comparable to chemotherapy alone.

A post-hoc AUGMENT-101 analysis of 19 patients resuming revumenib post-transplant reported a 95% 12-month overall survival rate and 79% relapse-free survival, with median OS and RFS not reached. Patients resumed therapy a median of 65 days post-HSCT and maintained a consistent safety profile with low discontinuation rates.

The phase 1/2 SAVE trial of an all-oral revumenib, decitabine/cedazuridine, and venetoclax regimen in 42 R/R NPM1m, KMT2Ar or NUP98r AML patients demonstrated an 88% ORR, 71% CRc, and 60% CR/CRh overall, including 80% MRD negativity among evaluable responders. 45% proceeded to transplant, 63% resumed revumenib post-HSCT, and the median OS after transplant was not reached with manageable treatment-emergent adverse events.