mavorixafor   Click here for help

GtoPdb Ligand ID: 8580

Synonyms: AMD 070 | AMD-070 | AMD-11070 | AMD11070 | compound 2 [PMID: 20297846] | X4P-001 | X4P-001-IO | X4P-001-LD
Immunopharmacology Ligand
Compound class: Synthetic organic
Comment: Mavorixafor (AMD070) is a potent, selective and bioavailable CXCR4 chemokine receptor allosteric antagonist [1]. Originally developed for HIV treatment, it is now being repurposed by X4 Pharmaceuticals as X4P-001 for the treatment of WHIM syndrome, a sub-type of a primary immunodeficiency disease caused by CXCR4 mutations. The compound is exemplified in a process patent US7332605 and as compound 89 from a series of 169 analogues in WO2003055876 but neither filing includes activity data.
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 4
Hydrogen bond donors 2
Rotatable bonds 7
Topological polar surface area 70.83
Molecular weight 349.23
XLogP 2.23
No. Lipinski's rules broken 0
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Canonical SMILES NCCCCN(C1CCCc2c1nccc2)Cc1nc2c([nH]1)cccc2
Isomeric SMILES NCCCCN([C@H]1CCCc2c1nccc2)Cc1nc2c([nH]1)cccc2
InChI InChI=1S/C21H27N5/c22-12-3-4-14-26(15-20-24-17-9-1-2-10-18(17)25-20)19-11-5-7-16-8-6-13-23-21(16)19/h1-2,6,8-10,13,19H,3-5,7,11-12,14-15,22H2,(H,24,25)/t19-/m0/s1
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Summary of Clinical Use Click here for help
Mavorixafor is being evaluated in clinical trials in patients with WHIM syndrome and in neutropenia. Click here to link to's full list of mavorixafor studies. A Phase 3 trial was recruiting WHIM syndrome patients as of November 2019. This drug has Orphan Designation in the US (October 2018) and EU (July 2019) as a treament for WHIM syndrome. In November 2019 the US FDA granted mavorixafor Breakthrough Therapy Designation for this rare indication.