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
Immunopharmacology Comments
X4 Pharmaceuticals announced (press release Jan 2017 ) a Phase 2/3 of X4P-001-LD, a low dose formulation of mavorixafor, as a chronic treatment for patients with WHIM syndrome. This is a genetic primary immunodeficiency disease caused by the aberrant trafficking of specific immune cells that are critical for proper immune system function which is caused by mutations in the CXCR4 receptor
Immunopharmacology Disease
Disease X-Refs Comment References
WHIM syndrome OMIM: 193670
Orphanet: ORPHA51636
Phase 3 clinical candidate for WHIM syndrome. FDA Breakthrough Therapy Designation for this indication was granted in November 2019.