Related Experiment Video
Updated: Jun 17, 2025
10:49
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
18.1K
Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101)
Ghayas C Issa1, Ibrahim Aldoss2, Michael J Thirman3
1Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX.
Summary
Revumenib demonstrated significant efficacy in treating relapsed/refractory KMT2A-rearranged acute leukemia, achieving high remission rates with a manageable safety profile in a large patient cohort.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- KMT2A-rearranged (KMT2Ar) acute leukemia is a challenging diagnosis with limited treatment options.
- Revumenib targets the menin-KMT2A interaction, a key driver in KMT2Ar leukemias.
Purpose of the Study:
- To evaluate the efficacy and safety of revumenib in patients with relapsed/refractory (R/R) KMT2Ar acute leukemia.
- To assess the complete remission (CR) or CR with partial hematologic recovery (CR + CRh) rate in this patient population.
Main Methods:
- Phase I/II, open-label, dose-escalation and expansion study (AUGMENT-101).
- Enrolled patients aged ≥30 days with R/R KMT2Ar acute leukemia.
- Revumenib administered orally every 12 hours in 28-day cycles.
Main Results:
- 94 patients treated; 57 were efficacy-evaluable.
- CR + CRh rate was 22.8% (95% CI, 12.7 to 35.8), exceeding the null hypothesis (P = .0036).
- Overall response rate was 63.2%, with 68.2% achieving no detectable residual disease.
Conclusions:
- Revumenib shows high remission rates and a predictable safety profile in R/R KMT2Ar acute leukemia.
- This study represents the largest evaluation of a targeted therapy for KMT2Ar acute leukemia.
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