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. Author manuscript; available in PMC: 2019 Dec 1.
Published in final edited form as: J Investig Med. 2018 Aug 19;66(8):1088–1095. doi: 10.1136/jim-2018-000807

Table 3:

Select Novel AML Therapies in Clinical Trials

Therapy Type Target Development
Status		 Potential
Patient
Population
Venetoclax Small molecule inhibitor BCL2 Randomized phase III Newly diagnosed ineligible for standard induction
Sorafenib Small molecule kinase inhibitor Multikinase including FLT3 Randomized phase II FLT3-mutated newly diagnosed, R/R, and possible maintenance
Gilteritinib Small molecule kinase inhibitor FLT3-ITD/FLT3-TKD/AXL Randomized phase III FLT3-mutated, both newly diagnosed and R/R
Crenolanib Small molecule kinase inhibitor FLT3-ITD/FLT3-TKD Randomized phase III FLT3-mutated, both newly diagnosed and R/R
Quizartinib Small molecule kinase inhibitor FLT3-ITD Randomized phase III FLT3-mutated, both newly diagnosed and R/R
Ivosidenib Small molecule IDH1 Randomized Phase III Newly diagnosed IDH1-mutated ineligible for intensive therapy
Pevonedistat Small molecule inhibitor NAE Randomized phase III Newly diagnosed low blast count(20-30%)
CAR-T cells Adoptive cell therapy CD33/CD123/others Phase I R/R
Flotetuzumab DART CD123 Phase I R/R

Abbreviations: BCL2, B-cell Lymphoma 2; FLT3-ITD, fms like tyrosine kinase 3- internal tandem duplication; FLT3-TKD, fms like tyrosine kinase 3- tyrosine kinase domain; IDH1, isocitrate dehydrogenase 1; NAE, Nedd8-activating enzyme; CAR-T, chimeric antigen receptor T-cells; DART, dual-affinity re-targeting antibody.