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December 8, 2025Blood

Phase 1/2, multi-center, open-label clinical study to evaluate the safety, pharmacokinetics, and efficacy of Zefamenib combined with chemotherapy or targeted Agents in patients with Acute Myeloid Leukemia

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Authors

HDHaiping DaiDWDepei WuSCSuning Chen

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Implication

Phase 1/2 trial assesses the safety and efficacy of zefamenib with chemotherapy in acute myeloid leukemia patients, indicating promising outcomes.

Key Points

  • Zefamenib demonstrated promising anti-leukemia efficacy in patients with acute myeloid leukemia, achieving an overall response rate of 89.3%.
  • Complete remission rates between 45-75% were noted with standard treatments, underscoring the need for effective combinations like zefamenib plus venetoclax.
  • Phase 1/2 study design explores safety and pharmacokinetics of zefamenib combined with intensive chemotherapy or venetoclax/azacitidine.
  • Findings may improve treatment strategies for Chinese acute myeloid leukemia patients with specific genetic mutations.

Cite This Study

Dai et al. (2025) studied this question.

synapsesocial.com/papers/69362f714fa91c937236e17ehttps://doi.org/10.1182/blood-2025-8257
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-0072025 · 9 citations
  2. 2Ziftomenib in combination with venetoclax and azacitidine in newly diagnosed NPM1-m acute myeloid leukemia: Phase 1b results from KOMET-0072025 · 13 citations
  3. 3Preliminary data from the ongoing Phase 1 study of the menin-MLL inhibitor enzomenib (DSP-5336) in combination with venetoclax and azacitidine in patients with relapsed or refractory Acute Myeloid Leukemia2025 · 8 citations
  4. 4The role of menin inhibitors in acute myeloid leukemia2025 · 2 citations
  5. 5A pre-emptive bridge-to-transplant therapy for measurable residual disease with venetoclax and azacitidine in NPM1-mutated Acute Myeloid Leukemia: Updates from the ongoing GIMEMA AML2521 phase 2 trial2025