Overview
- Phase 1b data presented at ASH 2025 identified 600 mg daily ziftomenib as the recommended phase 2 dose with venetoclax/azacitidine.
- Responses included a composite complete remission rate of 86%, an objective response rate of 89%, and a complete remission rate of 73% in newly diagnosed NPM1‑mutated AML.
- Central next‑generation sequencing showed MRD negativity in 68% at a 0.1% threshold and 44% at a 0.01% threshold, with first clearance around 9 to 10 weeks.
- At a 26.1‑week median follow‑up, median duration of complete remission and overall survival were not reached, with 70% of patients on study and 55% still receiving ziftomenib.
- Adverse events aligned with expectations for venetoclax/azacitidine, including grade 3 or higher neutropenia in 38% and rare, resolved differentiation syndrome and QTc prolongation, with blood count recovery comparable to the backbone regimen.