. 2020 Jan 24;8(1):13–32. doi: 10.1007/s40487-019-00108-x

Table 3.

Completed AML clinical trials with enasidenib

Study	Phase	Population	Association	Results (efficacy)	Results (safety)	Reference	Status
Enasidenib
NCT01915498	I-II	R/R AML	–	ORR 40.3% cCR 26%, CR 19%, OS 9.3 months	Hyperbilirubinemia G3/4 18% IDH-DS G3/4 6% Thrombocytopenia G3/4 23% Anemia G3/4 19%	[39]	Active

Study

Phase

Population

Association

Results (efficacy)

Results (safety)

Reference

Status

Enasidenib

I-II

R/R AML

–

ORR 40.3% cCR 26%, CR 19%, OS 9.3 months

Hyperbilirubinemia G3/4 18%

IDH-DS G3/4 6% Thrombocytopenia G3/4 23%

Anemia G3/4 19%

Active

Mechanism: inhibition of IDH2-mutant enzyme

Status: approved by FDA as a single agent in R/R AML patients with proven IDH2 mutation (August 1, 2017)

CR complete response, OS overall survival