Related Experiment Video
Updated: Jan 17, 2026
04:36
Comet Assay to Quantify DNA Damage in FLT3 Mutant-expressing 32D Cells after Exposure to Type I and Type II FLT3 Inhibitors
Published on: October 17, 2025
845
Ziftomenib in Relapsed or Refractory NPM1-Mutated AML
Eunice S Wang1, Pau Montesinos2, James Foran3
1Roswell Park Comprehensive Cancer Center, Buffalo, NY.
Summary
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.
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.
Related Concept Videos
02:56Treatment Resistant Cancers
3.7K
Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.7K
02:50Combination Therapies and Personalized Medicine
5.9K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
5.9K
02:23Abnormal Proliferation
5.1K
Under normal conditions, most adult cells remain in a non-proliferative state unless stimulated by internal or external factors to replace lost cells. Abnormal cell proliferation is a condition in which the cell's growth exceeds and is uncoordinated with normal cells. In such situations, cell division persists in the same excessive manner even after cessation of the stimuli, leading to persistent tumors. The tumor arises from the damaged cells that replicate to pass the damage to the...
5.1K