Drug class/mechanism | Agent | Suggested patient population | Single/combination | Phase of developmenta | Ref. |
---|---|---|---|---|---|
Cytotoxic chemotherapy | |||||
Liposomal formulation of 7 + 3 | CPX-351 | ≥60 years, sAML, fit for induction therapy | Single agent | 3 | |
Topoisomerase II inhibitor | Vosaroxin | ≥60 years, R/R | With cytarabine | 3 | [19] |
Epigenetic modifiers | |||||
DNMT inhibitor | Guadecitabine | Unfit for intensive therapy or R/R | Single agent | 3 | |
IDH1 inhibitor | AG-120, IDH305, FT-2102 | IDH1 mutated | Single agent/with AZA or induction and consolidation | 1b/2 | |
IDH2 inhibitor | Enasidenib (AG-221) | IDH2 mutated | Single agent/with AZA or induction and consolidation | 3 | [34] |
HDAC inhibitors | Panobinostat | Ongoing investigation | With HMAs or induction and consolidation | 1b/2 | |
Vorinostat | 3 | ||||
Entinostat | 2 | [45] | |||
Pracinostat | 2 | [46] | |||
BET inhibitor | OTX015 | Ongoing investigation | Single agent | 1 | [47] |
DOT1L inhibitor | Pinometostat | MLL-rearranged | Single agent | 1 | [49] |
LSD1 inhibitor | Tranylcypromine, GSK2879552, ORY-1001 | Ongoing investigation | Single agent/with ATRA | 1/2 | – |
FLT3 inhibitors | Sorafenib | FLT3-ITD-mutated R/R | With AZA | 2 | [60] |
Midostaurin | FLT3-ITD or FLT3-TKD, ≤60 years | With induction and consolidation | 3 | [63] | |
Quizartinib | FLT3-ITD-mutated R/R | Single agent | 3 | ||
Crenolanib | FLT3-ITD or FLT3-TKD | Single agent/with induction and consolidation | 2 | ||
Gilteritinib | FLT3-ITD or FLT3-TKD R/R | Single agent | 3 | [74] | |
Antibody-drug conjugates | |||||
Anti-CD33 | Vadastuximab (SGN-CD33A) | CD33+ | Single agent/with HMAs or induction and consolidation | 3 | |
Cell cycle inhibitors | |||||
Polo-like kinase inhibitor | Volasertib | Unfit for intensive therapy | With LDAC or decitabine | 3 | [78] |
Other agents | |||||
BCL-2 inhibitor | Venetoclax (ABT-199) | R/R, or older (≥65 years) and unfit for intensive therapy | Single agent/with HMAs or LDAC | 2 | |
Aminopeptidase inhibitor | Tosedostat | ≥60 year, R/R or unfit for intensive therapy | Single agent/with cytarabine or HMAs | 2 |