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Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia (first-in-human phase 1/2 study)

Phase 1/2NCT01915498

Population
Relapsed or refractory AML with a mutant IDH2 (the largest subgroup of the phase 1 dose-escalation and expansion cohorts; 176 patients)
Design
First-in-human phase 1/2 dose-escalation and expansion study in advanced myeloid malignancies with an IDH2 mutation (doses 50 to 650 mg daily; 100 mg daily chosen for expansion). Efficacy reported for the relapsed or refractory AML subgroup. Single-arm; no randomized comparator.
Arms
  • Enasidenib (experimental, n = 176): Oral enasidenib once daily; 100 mg daily selected for the expansion phase
People
Stein EM
Show the numbers
Results reported for Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia (first-in-human phase 1/2 study)
EndpointAnalysisEnasidenibControlEffect
ORRRelapsed or refractory AML subgroup (n = 176)Overall response rate in relapsed or refractory AML, assessed by investigators using the modified 2003 International Working Group criteria (71 of 176 patients responded)primaryStein 201740.3% (33–48%)—Median response duration 5.8 months, as stated in the abstract.
OSRelapsed or refractory AML subgroup (n = 176)primaryStein 2017median 9.3 months—Median OS was 19.7 months for the 34 patients (19.3%) who attained complete remission, a response-selected subgroup.
Results reported for Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia (first-in-human phase 1/2 study)
EndpointAnalysisEnasidenibControlEffect
ORRRelapsed or refractory AML subgroup (n = 176)Overall response rate in relapsed or refractory AML, assessed by investigators using the modified 2003 International Working Group criteria (71 of 176 patients responded)primaryStein 201740.3% (33–48%)—Median response duration 5.8 months, as stated in the abstract.
OSRelapsed or refractory AML subgroup (n = 176)primaryStein 2017median 9.3 months—Median OS was 19.7 months for the 34 patients (19.3%) who attained complete remission, a response-selected subgroup.
Illustrative — modelled from reported values, not trial data. OS, primary.
0%25%50%75%100%05101520Months (modelled)
  • Enasidenib (experimental)

Median-only curves assume a constant hazard (exponential) and fit plateaus, as seen with some immunotherapies, poorly.

Data table (modelled values)
MonthsEnasidenib
0100.0%
663.9%
1240.9%
1728.2%
2318.0%
Primary publication: Stein EM, DiNardo CD, Pollyea DA, Fathi AT, Roboz GJ, Altman JK, et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017;130(6):722-731. PubMedDOI

Appears in

Sources

  1. Stein EM, DiNardo CD, Pollyea DA, Fathi AT, Roboz GJ, Altman JK, et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017;130(6):722-731.

    PubMedDOI