axatilimab   Click here for help

GtoPdb Ligand ID: 13497

Synonyms: Ab969 [US9908939] | axatilimab-csfr | INCA034176 | Niktimvo® | SNDX-6352 | UCB-6352
Approved drug
axatilimab is an approved drug (FDA (2024))
Compound class: Antibody
Comment: Axatilimab (SNDX-6352, formerly UCB-6352) is a humanized anti-CSF-1R antibody. It was initially developed as an alternative therapeutic modality for the treatment of chronic graft-versus-host disease (GvHD) [3]. Axatilimab targets macrophage-mediated inflammation and fibrosis that cause organ damage in GvHD, by blocking CSF-1R activation by either of its ligands (CSF-1 and IL-34).
Peptide sequences from the axatilimab INN entry align with sequences that are claimed in a UCB Biopharma patent originally filed in 2014 [1].
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
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Summary of Clinical Use Click here for help
The FDA approved axatilimab in August 2024, to treat (refractory) chronic graft-versus-host disease (GvHD) after failure of two prior systemic therapies [2]. Clinical trials are evaluating axatilimab in other diseases, including idiopathic pulmonary fibrosis and advanced cancers.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04710576 A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD) Phase 2 Interventional Syndax Pharmaceuticals FDA approval was based on positive safety and efficacy outcomes from this (AGAVE-201) study.
NCT05723055 Evaluating Combination of Nivolumab and Axatilimab in Patients With Relapsed/Refractory Classical Hodgkin Lymphoma Phase 2 Interventional University of Utah
NCT06132256 Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF) Phase 2 Interventional Syndax Pharmaceuticals
NCT06388564 A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease Phase 2 Interventional Incyte Corporation