Axatilimab

drug
On this page

Also known as Axatilimab-csfrNiktimvoSndx-6352SNDX6352UCB-6352UCB6352

Summary

Axatilimab (CHEMBL4594608) is an approved antibody targeting CSF1R; indicated across 7 conditions including cholangiocarcinoma and hodgkins lymphoma.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Antibody
  • Targets: 1 (CSF1R)
  • Indications: 7 conditions
  • Clinical trials: 21

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL4594608
NameAxatilimab
TypeAntibody
Max phase4

Also known as: Axatilimab, Axatilimab-csfr, Niktimvo, Sndx-6352, SNDX-6352, SNDX6352, UCB-6352, UCB6352, AXATILIMAB

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
CSF1Rcolony stimulating factor 1 receptorBinding11.020%P07333

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): CSF1R.

Top Reactome pathways

3 total, by targets touching each:

PathwayTargetsGenes
Other interleukin signaling1CSF1R
Transcriptional Regulation by VENTX1CSF1R
Signaling by CSF1 (M-CSF) in myeloid cells1CSF1R

Dominant GO biological processes

GO termTargets
positive regulation of protein phosphorylation1
response to ischemia1
inflammatory response1
signal transduction1
cell surface receptor protein tyrosine kinase signaling pathway1
axon guidance1
cell population proliferation1
positive regulation of cell population proliferation1
negative regulation of cell population proliferation1
regulation of cell shape1
positive regulation of macrophage chemotaxis1
cell migration1
peptidyl-tyrosine phosphorylation1
cytokine-mediated signaling pathway1
olfactory bulb development1

Indications & clinical

Indications

7 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

IndicationTrial phaseMONDOEFO
cholangiocarcinoma2MONDO:0019087EFO:0005221
Hodgkins lymphoma2MONDO:0004952EFO:0000183
idiopathic pulmonary fibrosis2MONDO:0800504EFO:0000768
metastatic neoplasm1MONDO:0024883EFO:0009709
neoplasm1MONDO:0005070EFO:0000616
pulmonary fibrosis1MONDO:0002771EFO:0009448

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 21.

Phase distribution

PhaseTrials
PHASE211
PHASE15
PHASE1/PHASE24
Not specified1

Top trials by phase / activity

NCTPhaseStatusTitle
NCT04710576PHASE2ACTIVE_NOT_RECRUITINGA Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
NCT05491226PHASE2RECRUITINGReinvigorating TNBC Response to Immunotherapy With Combination Myeloid Inhibition and Radiation
NCT05723055PHASE2ACTIVE_NOT_RECRUITINGEvaluating Combination of Nivolumab and Axatilimab in Patients With Relapsed/Refractory Classical Hodgkin Lymphoma
NCT06132256PHASE2ACTIVE_NOT_RECRUITINGMAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)
NCT06320405PHASE1/PHASE2RECRUITINGAxatilimab in Combination With Retifanlimab and Paclitaxel for the Treatment of Patients With Advanced or Metastatic Solid Tumors
NCT06388564PHASE2ACTIVE_NOT_RECRUITINGA Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
NCT06523556PHASE1/PHASE2RECRUITINGAxatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia
NCT06663722PHASE2RECRUITINGAxatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
NCT07011810PHASE2RECRUITINGAxatilimab for Sclerotic Chronic Graft-versus-Host Disease
NCT07015853PHASE2NOT_YET_RECRUITINGASTRAEA: ReinvigorAting ReSponse To ImmunotheRApy in MEtAstatic TNBC With Combination Myeloid Inhibition and Radiation
NCT07128381PHASE1/PHASE2RECRUITINGPhase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML)
NCT07349771PHASE2NOT_YET_RECRUITINGAxatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Hematologic Cancer, ABRAXAS Trial
NCT03604692PHASE1/PHASE2COMPLETEDA Phase 1/2 Study to Evaluate Axatilimab in Participants With Active cGVHD
NCT04301778PHASE2COMPLETEDDurvalumab and SNDX-6532 Following Chemo or Radio-Embolization for Patients With Intrahepatic Cholangiocarcinoma
NCT04415073PHASE2TERMINATEDA Phase 2 Study to Evaluate Axatilimab for Hospitalized Participants With Respiratory Involvement Secondary to COVID-19
NCT06488378PHASE1RECRUITINGPhase Ib Study of Axatilimab in Combination With Olaparib in BRCA1/2 and PALB2- Associated Metastatic HER2-negative Breast Cancer
NCT06959537PHASE1RECRUITINGA Phase Ib Study of Novel Combination (New) of Low Dose Oral CyclophoSphamide (s) to Potentiate Axatilimab (A) + Retifanlimab (R) in Treating Metastatic Triple Negative Breast Cancer (TNBC)
NCT07511062PHASE1NOT_YET_RECRUITINGAxatilimab Combined With Decitabine/Venetoclax for the Treatment of TP53-mutated AML
NCT03238027PHASE1COMPLETEDA Phase 1 Study to Investigate Axatilimab Alone or in Combination With Durvalumab in Patients With Solid Tumors
NCT06713590PHASE1COMPLETEDA Study to Evaluate a Single-Dose Subcutaneous Axatilimab Compared With Intravenous Axatilimab in Healthy Participants
NCT05544032Not specifiedAVAILABLEExpanded Access of Axatilimab to Treat a Single Patient With Chronic Graft Versus Host Disease (GVHD)

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.

72 molecules share ≥1 primary target. Top 60 by shared-target count:

MoleculeSourceStatusShared targets
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)CSF1R
PAZOPANIBChEMBL + PubChemPhase 4 (approved)CSF1R
AXITINIBChEMBLPhase 4 (approved)CSF1R
BOSUTINIBChEMBLPhase 4 (approved)CSF1R
BRIGATINIBChEMBLPhase 4 (approved)CSF1R
DASATINIBChEMBLPhase 4 (approved)CSF1R
ENTRECTINIBChEMBLPhase 4 (approved)CSF1R
FEDRATINIBChEMBLPhase 4 (approved)CSF1R
FILGOTINIBChEMBLPhase 4 (approved)CSF1R
IBRUTINIBChEMBLPhase 4 (approved)CSF1R
IMATINIBChEMBLPhase 4 (approved)CSF1R
MIDOSTAURINChEMBLPhase 4 (approved)CSF1R
NERATINIBChEMBLPhase 4 (approved)CSF1R
NILOTINIBChEMBLPhase 4 (approved)CSF1R
NINTEDANIBChEMBLPhase 4 (approved)CSF1R
PACRITINIBChEMBLPhase 4 (approved)CSF1R
PEXIDARTINIBChEMBLPhase 4 (approved)CSF1R
PONATINIBChEMBLPhase 4 (approved)CSF1R
QUIZARTINIBChEMBLPhase 4 (approved)CSF1R
SORAFENIBChEMBLPhase 4 (approved)CSF1R
SUNITINIBChEMBLPhase 4 (approved)CSF1R
SUNITINIB MALATEChEMBLPhase 4 (approved)CSF1R
VANDETANIBChEMBLPhase 4 (approved)CSF1R
ALVOCIDIBChEMBLPhase 3CSF1R
BRIVANIBChEMBLPhase 3CSF1R
CEDIRANIBChEMBLPhase 3CSF1R
DACTOLISIBChEMBLPhase 3CSF1R
DOVITINIBChEMBLPhase 3CSF1R
LESTAURTINIBChEMBLPhase 3CSF1R
LINIFANIBChEMBLPhase 3CSF1R
MASITINIBChEMBLPhase 3CSF1R
MOTESANIBChEMBLPhase 3CSF1R
PIMICOTINIBChEMBLPhase 3CSF1R
SEMAXANIBChEMBLPhase 3CSF1R
VATALANIBChEMBLPhase 3CSF1R
VIMSELTINIBChEMBLPhase 3CSF1R
ARRY-382ChEMBLPhase 2CSF1R
BELVARAFENIBChEMBLPhase 2CSF1R
BMS-754807ChEMBLPhase 2CSF1R
CC-115ChEMBLPhase 2CSF1R
CENISERTIBChEMBLPhase 2CSF1R
CEP-11981ChEMBLPhase 2CSF1R
CEP-32496ChEMBLPhase 2CSF1R
CERDULATINIBChEMBLPhase 2CSF1R
DEFOSBARASERTIBChEMBLPhase 2CSF1R
DERAZANTINIBChEMBLPhase 2CSF1R
DORAMAPIMODChEMBLPhase 2CSF1R
EDICOTINIBChEMBLPhase 2CSF1R
ELZOVANTINIBChEMBLPhase 2CSF1R
ENMD-2076ChEMBLPhase 2CSF1R
ENRUPATINIBChEMBLPhase 2CSF1R
FORETINIBChEMBLPhase 2CSF1R
GZ-389988ChEMBLPhase 2CSF1R
ILORASERTIBChEMBLPhase 2CSF1R
INIXACICLIBChEMBLPhase 2CSF1R
NARAZACICLIBChEMBLPhase 2CSF1R
NEFLAMAPIMODChEMBLPhase 2CSF1R
ONATASERTIBChEMBLPhase 2CSF1R
PELITINIBChEMBLPhase 2CSF1R
R-406ChEMBLPhase 2CSF1R