Table 1

Emerging and promising agents for the treatment of AML

AgentMechanism of actionSuggested patient populationNotes
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. 
AgentMechanism of actionSuggested patient populationNotes
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.

Close Modal

or Create an Account

Close Modal
Close Modal