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Drugs in development · updated 30 October 2026

Acute myeloid leukemia: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:519

As of 30 October 2026 there are 573 active clinical trials (recruiting or ongoing, phases I–III) on 314 drugs for acute myeloid leukemia. 182 are experimental, that is not yet approved in the United States.

573Active trials
182Experimental drugs
19Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
ChidamideTucidinostat, HBI-8000III10NCT07514936
HomoharringtonineOmacetaxineII–III10NCT07651163
CPX-351III7NCT03897127
LisaftoclaxAPG-2575, LisafotoclaxII–III7NCT07651163
AclacinomycinIII4NCT07514936
AclarubicinII–III3NCT07651163
BleximenibIII3NCT07223814
BL-M11D1II–III2NCT07255872
ClifutinibIII2NCT05586074
GM-CSFGranulocyte-Macrophage Colony-Stimulating FactorIII2NCT04229979
PevonedistatIII2NCT04090736
UproleselanII–III2NCT03701308
ASTX030II–III1NCT04256317
GranulocyteIII1NCT07514936
HMPL-306III1NCT06387069
Iomab-BIII1NCT02665065
IPN60340II–III1NCT07623187
MocravimodIII1NCT05429632
VosaroxinQinprezo®II–III1NCT02416388
AnthracyclineII3NCT07060001
CusatuzumabII3NCT04023526
RimiducidAP1903I–II3NCT01744223
AK117I–II2NCT04980885
BG1805I–II2NCT06118788
CAVII2NCT07423104
CD123I–II2NCT06420063
CD19I–II2NCT06158828
CD33I–II2NCT03222674
CD38I–II2NCT03222674
CLN-049I–II2NCT05143996
MezigdomideCC-92480I–II2NCT07356154
RVU120II2NCT06191263
AB8939I–II1NCT05211570
ABD-3001I–II1NCT05601726
AlphaII1NCT05735717
AnthracyclinesII1NCT06451861
AnthraquinonesII1NCT06451861
ARD103I–II1NCT06680752
ArubicinII1NCT07642453
AZD3632I–II1NCT07155226

Sorted by most advanced phase and number of active trials. Showing 40 of 182 drugs.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
VenetoclaxVentoclax, Venclexta®II–III181NCT07581002
AzacitidineAzacytidine, 5-azacytidineII–III161NCT07581002
CytarabineAra-C, Cytosine ArabinosideII–III109NCT07255872
FludarabineFludara, FLUDARABINII–III89NCT04708054
DecitabineDacogen®II–III59NCT03793517
CyclophosphamideCyclophosphamid, CytoxanII–III58NCT07157514
DaunorubicinDaunomycin, Daunoxome®II–III42NCT07255872
BusulfanBusulphan, Busulfex®II–III35NCT04708054
IdarubicinIdamycin Pfs®, Idamycin®II–III32NCT02416388
MelphalanEvomela®, Hepzato®III29NCT05457556
TacrolimusEnvarsus XR, Prograf®III27NCT05457556
GilteritinibXospataII–III25NCT07425808
Mycophenolate MofetilMycophenolate, CellceptIII23NCT05457556
CedazuridineCedaruridineII–III20NCT04256317
IvosidenibAG-120, Tibsovo®III19NCT03839771

Sorted by most advanced phase and number of active trials. Showing 15 of 132 drugs.

All 314 drugs, with the details

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Specific forms

Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D015470 “Leukemia, Myeloid, Acute”. Prevalence: Orphanet.

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