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Ziftomenib in Relapsed or Refractory NPM1-Mutated AML.

Eunice S Wang1, Pau Montesinos2, James Foran3

  • 1Roswell Park Comprehensive Cancer Center, Buffalo, NY.

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology
|September 25, 2025
PubMed
Summary
This summary is machine-generated.

Ziftomenib shows significant clinical benefit for relapsed/refractory NPM1-mutated AML. This oral menin inhibitor was well-tolerated, demonstrating manageable side effects and deep responses in heavily pretreated patients.

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Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Relapsed/refractory Acute Myeloid Leukemia (AML) with NPM1 mutations (NPM1-m) presents a significant unmet need.
  • Targeting menin offers a novel therapeutic strategy for AML.

Purpose of the Study:

  • To evaluate the efficacy and safety of ziftomenib, a potent oral menin inhibitor, as monotherapy for relapsed/refractory NPM1-m AML.
  • To determine the complete remission (CR) or CR with partial hematologic recovery (CRh) rate in patients treated with ziftomenib.

Main Methods:

  • Phase II of the KOMET-001 trial enrolled patients with relapsed/refractory NPM1-m AML.
  • Ziftomenib was administered at 600 mg once daily.
  • The primary endpoint was the CR/CRh rate.

Main Results:

  • The CR/CRh rate was 22% (95% CI, 14-32), meeting the primary endpoint.
  • 61% of patients achieved measurable residual disease negativity.
  • The overall response rate was 33%, with a median duration of 4.6 months. Common adverse events included febrile neutropenia, anemia, and thrombocytopenia. Differentiation syndrome occurred in 25% of patients and was manageable.

Conclusions:

  • Ziftomenib demonstrated significant clinical benefit and deep responses in heavily pretreated patients with relapsed/refractory NPM1-m AML.
  • The drug was well-tolerated, with a manageable safety profile including differentiation syndrome.
  • Ziftomenib represents a promising targeted therapy for this patient population.