Table 1.

Emerging and promising agents for the treatment of AML

Agent	Mechanism of action	Suggested patient population	Notes
CPX-351	Liposomal formulation of 7+3 in 5:1 molar ratio	sAML fit for induction chemotherapy	Phase 2 study showed OS benefit in sAML
			Phase 3 study fully accrued and waiting for final analysis
Vosaroxin	Novel topoisomerase II inhibitor	Relapsed/refractory AML	OS benefit when censored for allogeneic transplant; mucositis notable as toxicity
Guadecitabine	Hypomethylating agent resistant to deamination	Unfit for intensive chemotherapy	May supplant LDC, decitabine, 5-azacitadine
SGN-CD33A	ADC against CD33 with stable linker	Being explored as a combination with hypomethylating and traditional induction	Next-generation ADC against CD33
Volasertib	Novel PLK1 inhibitor	Being explored as a combination with hypomethylating and traditional induction	OS benefit in small randomized phase 2 study when combined with LDC
Quizartinib	FLT3 inhibitor	FLT3 + AML	Impressive single-agent activity against FLT3-ITD; resistance emerges in most patients
Crenolanib	FLT3 inhibitor with activity against TKD-resistance mutation	FLT3-ITD or FLT3-TKD	Active against TKD mutations
ASP-2215	FLT3 inhibitor with activity against TKD-resistance mutation	FLT3-ITD or FLT3-TKD	Impressive single-agent activity with CRc rate of 43%
AG-221	IDH2 inhibitor	IDH2 mutated	Impressive single-agent activity (41% overall response rate) in relapsed or refractory AML
AG-120	IDH1 inhibitor	IDH1 mutated	Impressive single-agent activity in relapsed or refractory AML
EPZ-5676	DOT1L inhibitor	MLL rearranged	Combinations with standard of care should be explored
ABT-199	BCL2 inhibitor	Ongoing investigation	May have increased activity in patients with IDH mutations
OTX-015	BET inhibitor	Ongoing investigation	Combinations with standard of care should be explored
Pracinostat	HDAC inhibitor	Ongoing investigation	Impressive activity in combination with 5-azacitidine; awaiting survival data.

7 + 3, 7 days of cytarabine and 3 days of daunorubicin.