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Miltefosine

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Also known as D-18506ImpavidoMiltefosinaMiltexNSC-605583NSC-758968Hexadecylphosphocholinen-hexadecylphosphocholinemiltefosinMiltefocinSID26749044SID484338SID144205019SID170465622MMV688990MiltofosineMILTEFOSINE [5MM]MiltefosineC0237431

Summary

Miltefosine (CHEMBL125) is an approved small-molecule antineoplastic agent (ATC P01CX04) targeting AKT1; indicated across 7 conditions including leishmaniasis and visceral leishmaniasis.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: P01CX04
  • Targets: 1 (AKT1)
  • Indications: 7 conditions
  • Clinical trials: 36
  • Chemistry: 407.6 Da · C21H46NO4P

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL125
NameMiltefosine
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID3599
ChEBICHEBI:75283
ATCP01CX04
Molecular formulaC21H46NO4P
Molecular weight407.6
InChIKeyPQLXHQMOHUQAKB-UHFFFAOYSA-N

SMILES: CCCCCCCCCCCCCCCCOP(=O)([O-])OCC[N+](C)(C)C

IUPAC name: hexadecyl 2-(trimethylazaniumyl)ethyl phosphate

ChEBI definition: A phospholipid that is the hexadecyl monoester of phosphocholine.

Pharmacological roles (ChEBI): antineoplastic agent, antiprotozoal drug, antifungal agent, immunomodulator, anti-inflammatory agent, apoptosis inducer, protein kinase inhibitor, anticoronaviral agent.

Also known as: D-18506, Impavido, Miltefosina, Miltefosine, Miltex, NSC-605583, NSC-758968, Hexadecylphosphocholine, n-hexadecylphosphocholine, miltefosin, miltefosine, hexadecylphosphocholine

Patent coverage: 6,078 distinct patent families (24,203 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

GeneTargetActionpAffinityCancer dependencyUniProt
AKT1AKT serine/threonine kinase 1Inhibition5.023.4%P31749

Broader ChEMBL bioactivity targets: 10 (assay-derived). Sample: Prelamin-A/C, Ferritin light chain, Estrogen receptor, Type-1 angiotensin II receptor, Alpha-1A adrenergic receptor, Voltage-gated inwardly rectifying potassium channel KCNH2, Adenosine receptor A3, Cruzipain, M-phase inducer phosphatase 1, RAC-alpha serine/threonine-protein kinase.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 10 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
LMNA5.45Potency3548nMCHEMBL_ACT_3646559
AKT15.02IC509600nMCHEMBL_ACT_12713908
ADRA1A5AC5010000nMCHEMBL_ACT_25217770

Target pathways

Aggregated over 1 target gene(s): AKT1.

Top Reactome pathways

113 total, by targets touching each:

PathwayTargetsGenes
Apoptosis1AKT1
Hemostasis1AKT1
Intrinsic Pathway for Apoptosis1AKT1
Activation of BAD and translocation to mitochondria1AKT1
Activation of BH3-only proteins1AKT1
Signaling by ERBB21AKT1
PIP3 activates AKT signaling1AKT1
Developmental Biology1AKT1
Cytokine Signaling in Immune system1AKT1
Adaptive Immune System1AKT1
Downregulation of ERBB2:ERBB3 signaling1AKT1
Metabolism1AKT1
Translocation of SLC2A4 (GLUT4) to the plasma membrane1AKT1
Tetrahydrobiopterin (BH4) synthesis, recycling, salvage and regulation1AKT1
Signaling by NOTCH1AKT1
Signal Transduction1AKT1
Cell Cycle1AKT1
Disease1AKT1
MTOR signalling1AKT1
Inhibition of TSC complex formation by AKT (PKB)1AKT1
Immune System1AKT1
Regulation of beta-cell development1AKT1
Signaling by VEGF1AKT1
Signaling by WNT1AKT1
Metabolism of vitamins and cofactors1AKT1
AKT phosphorylates targets in the cytosol1AKT1
AKT phosphorylates targets in the nucleus1AKT1
Negative regulation of the PI3K/AKT network1AKT1
Membrane Trafficking1AKT1
TCF dependent signaling in response to WNT1AKT1
Metabolism of nitric oxide: NOS3 activation and regulation1AKT1
eNOS activation1AKT1
Regulation of gene expression in beta cells1AKT1
AKT-mediated inactivation of FOXO1A1AKT1
Generic Transcription Pathway1AKT1
PI3K/AKT Signaling in Cancer1AKT1
Cellular responses to stress1AKT1
Integrin signaling1AKT1
Transcriptional Regulation by TP531AKT1
Signaling by GPCR1AKT1
Deactivation of the beta-catenin transactivating complex1AKT1
GPCR downstream signalling1AKT1
Regulation of T cell activation by CD28 family1AKT1
Co-stimulation by CD281AKT1
CD28 dependent PI3K/Akt signaling1AKT1
Co-inhibition by CTLA41AKT1
G beta:gamma signalling through PI3Kgamma1AKT1
G-protein beta:gamma signalling1AKT1
VEGFA-VEGFR2 Pathway1AKT1
Signaling by Interleukins1AKT1

Dominant GO biological processes

GO termTargets
osteoblast differentiation1
maternal placenta development1
positive regulation of endothelial cell proliferation1
cell migration involved in sprouting angiogenesis1
complement receptor mediated signaling pathway1
sphingosine-1-phosphate receptor signaling pathway1
glycogen biosynthetic process1
regulation of glycogen biosynthetic process1
glucose metabolic process1
regulation of translation1
protein phosphorylation1
protein import into nucleus1
nitric oxide biosynthetic process1
activation-induced cell death of T cells1
inflammatory response1

Indications & clinical

Indications

5 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

IndicationPhaseMONDOEFO
leishmaniasis4MONDO:0011989EFO:0005044
visceral leishmaniasis4MONDO:0005445EFO:0005045
cutaneous leishmaniasis4MONDO:0005446EFO:0005046
mucocutaneous leishmaniasis4MONDO:0005859EFO:0007379
trypanosomiasis4MONDO:0000940DOID:10113

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)PhaseMONDOEFO
bipolar disorder3MONDO:0004985MONDO:0004985
urticaria2MONDO:0005492EFO:0005531

Clinical trials

Total trials: 36.

Phase distribution

PhaseTrials
PHASE216
PHASE310
Not specified5
PHASE2/PHASE32
PHASE41
PHASE1/PHASE21
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT01462500PHASE4COMPLETEDPharmacokinetics of Miltefosine in Children and Adults
NCT06040489PHASE2/PHASE3RECRUITINGPilot Study: Oral Treatment of American Tegumentary Leishmaniasis (Cutaneous and Mucosal Forms) in the Elderly
NCT07463040PHASE3NOT_YET_RECRUITINGStudy Comparing Several Drugs to Understand Which Work Against Cutaneous Leishmaniasis (CL)
NCT00471705PHASE3COMPLETEDEfficacy and Safety of Miltefosine or Thermotherapy for Cutaneous Leishmaniasis in Colombia.
NCT00487253PHASE3UNKNOWNOral Miltefosine for the Treatment of Pediatric Cutaneous Leishmaniasis in Colombia
NCT00523965PHASE3COMPLETEDCombination Therapy in Indian Visceral Leishmaniasis
NCT00696969PHASE3COMPLETEDSafety and Efficacy Study to Evaluate Different Combination Treatment Regimens for Visceral Leishmaniasis
NCT01122771PHASE3COMPLETEDPhase III, Study of Three Short Course Combo (Ambisome®, Miltefosine, Paromomycin) Compared With AmBisome for the Treatment of VL in Bangladesh
NCT02011958PHASE3COMPLETEDEfficacy Trial of Ambisome Given Alone and Ambisome Given in Combination With Miltefosine for the Treatment of VL HIV Positive Ethiopian Patients.
NCT02193022PHASE3COMPLETEDMiltefosine for Children With PKDL
NCT03129646PHASE3COMPLETEDMiltefosine/Paromomycin Phase III Trial for Treatment of Primary Visceral Leishmaniasis (VL) Patients in Eastern Africa
NCT03829917PHASE2/PHASE3COMPLETEDOral Miltefosine Plus Topical Paromomycin In American Cutaneous Leishmaniasis
NCT04515186PHASE3COMPLETEDCombination, Miltefosine Monotherapy for Cutaneous Leishmaniasis in New World
NCT02530697PHASE2ACTIVE_NOT_RECRUITINGThe Association of Miltefosine and Pentoxifylline to Treat Mucosal and Cutaneous Leishmaniasis: A Clinical Trial in Brazil
NCT06550609PHASE2RECRUITINGTreatment of Bolivian L Braziliensis Mucosal Leishmaniasis With Inhaled Pentamidine Plus Oral Miltefosine
NCT00233545PHASE2COMPLETEDMiltefosine to Treat Cutaneous Leishmaniasis in Bolivia
NCT00370825PHASE2COMPLETEDCombination Chemotherapy for the Treatment of Indian Kala-Azar
NCT00371995PHASE2COMPLETEDShort Course of Miltefosine and Liposomal Amphotericin B for Kala-azar
NCT00373776PHASE1/PHASE2COMPLETEDMiltefosine for Mucosal Leishmaniasis
NCT00537953PHASE2UNKNOWNShort Course of Miltefosine and Antimony to Treat Cutaneous Leishmaniasis in Bolivia
NCT01050907PHASE2COMPLETEDMiltefosine to Treat Mucocutaneous Leishmaniasis
NCT01067443PHASE2COMPLETEDClinical Trial to Assess the Safety and Efficacy of Sodium Stibogluconate (SSG) and AmBisome® Combination, Miltefosine and AmBisome® and Miltefosine Alone for the Treatment Visceral Leishmaniasis in Eastern Africa
NCT01170949PHASE2TERMINATEDEfficacy and Safety of Miltefosine in Antihistamine Resistant Chronic Urticaria
NCT01377974PHASE2COMPLETEDClinical Trial of Miltefosine to Treat Mucosal Leishmaniasis
NCT01380301PHASE2TERMINATEDTreatment of Cutaneous Leishmaniasis With a Combination of Miltefosine and Antimony
NCT01380314PHASE2COMPLETEDOral Miltefosine Plus Topical Imiquimod to Treat Cutaneous Leishmaniasis
NCT01635777PHASE2COMPLETEDSafety and Efficacy of Oral Miltefosine in Patients With Post Kala Azar Dermal Leishmaniasis (PKDL)
NCT02431143PHASE2COMPLETEDPharmacokinetics/Safety of Miltefosine Allometric Dose for the Treatment of Visceral Leishmaniasis in Children in Eastern Africa
NCT02687971PHASE2COMPLETEDThermotherapy + a Short Course of Miltefosine for the Treatment of Uncomplicated Cutaneous Leishmaniasis in the New World¨
NCT03399955PHASE2UNKNOWNShort Course Regimens for Treatment of PKDL (Sudan)
NCT06251739EARLY_PHASE1COMPLETEDRepurposing Ivermectin for PKDL Treatment
NCT06514560Not specifiedRECRUITINGOPTImizing MIltefosine Treatment for Cutaneous LEISHmaniasis Patients
NCT02427308Not specifiedWITHDRAWNTreatment of Leishmaniasis With Impavido® (Miltefosine): Pregnancy Registry
NCT02429505Not specifiedWITHDRAWNTreatment of Leishmaniasis With Impavido® (Miltefosine): Higher-Weight Patient Registry
NCT02429518Not specifiedCOMPLETEDDedicated QT Study in Bolivian Patients Taking Impavido® (Miltefosine) for Mucocutaneous Leishmaniasis
NCT02431429Not specifiedCOMPLETEDSpermiogram Assessment in Bolivian Patients Taking Impavido® (Miltefosine) for Mucocutaneous Leishmaniasis

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

34 molecules share ≥1 primary target. Top 34 by shared-target count:

MoleculeSourceStatusShared targets
CAPIVASERTIBChEMBLPhase 4 (approved)AKT1
MIDOSTAURINChEMBLPhase 4 (approved)AKT1
NICLOSAMIDEChEMBLPhase 4 (approved)AKT1
AFURESERTIBChEMBLPhase 3AKT1
FASUDILChEMBLPhase 3AKT1
IPATASERTIBChEMBLPhase 3AKT1
LESTAURTINIBChEMBLPhase 3AKT1
LINIFANIBChEMBLPhase 3AKT1
PERIFOSINEChEMBLPhase 3AKT1
QUERCETINChEMBLPhase 3AKT1
EDELFOSINEChEMBLPhase 2AKT1
ELLAGIC ACIDChEMBLPhase 2AKT1
KALAFUNGINChEMBLPhase 2AKT1
LAUROGUADINEChEMBLPhase 2AKT1
MIRANSERTIBChEMBLPhase 2AKT1
MK-2206ChEMBLPhase 2AKT1
PF-04691502ChEMBLPhase 2AKT1
PICTILISIBChEMBLPhase 2AKT1
RUPITASERTIBChEMBLPhase 2AKT1
SOTRASTAURINChEMBLPhase 2AKT1
SULFAETHIDOLEChEMBLPhase 2AKT1
UPROSERTIBChEMBLPhase 2AKT1
AfatinibPubChemApprovedAKT1
belumosudilPubChemApprovedAKT1
BinimetinibPubChemApprovedAKT1
CrizotinibPubChemApprovedAKT1
dacomitinibPubChemApprovedAKT1
FostamatinibPubChemApprovedAKT1
IdelalisibPubChemApprovedAKT1
PazopanibPubChemApprovedAKT1
podofiloxPubChemApprovedAKT1
regorafenibPubChemApprovedAKT1
SelumetinibPubChemApprovedAKT1
TrametinibPubChemApprovedAKT1

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot