Mavorixafor

Summary

Mavorixafor (CHEMBL518924) is an approved small molecule (ATC L03AX24) targeting CXCR4; indicated across 7 conditions including neoplasm and neutropenia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L03AX24
• Targets: 1 (CXCR4)
• Indications: 7 conditions
• Clinical trials: 15
• Chemistry: 349.5 Da · C21H27N5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL518924
Name	Mavorixafor
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11256587
ATC	L03AX24
Molecular formula	C21H27N5
Molecular weight	349.5
InChIKey	WVLHHLRVNDMIAR-IBGZPJMESA-N

SMILES: C1C[C@@H](C2=C(C1)C=CC=N2)N(CCCCN)CC3=NC4=CC=CC=C4N3

IUPAC name: N’-(1H-benzimidazol-2-ylmethyl)-N’-[(8S)-5,6,7,8-tetrahydroquinolin-8-yl]butane-1,4-diamine

Also known as: Amd 070, AMD-070, AMD-11070, AMD070, AMD11070, Cxcr4 inhibitor x4p-001, Mavorixafor, X 4P-001, X4P-001, X4p001, Xolremdi, MAVORIXAFOR

Parent form; salt/anhydrous children: CHEMBL4203190

Patent coverage: 327 distinct patent families (791 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 528 (67%) of the total. 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).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
CXCR4	CXCR4	Antagonist	7.96	0%	P61073

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: C-X-C chemokine receptor type 4, Muscarinic acetylcholine receptor M2, D(2) dopamine receptor, Mu-type opioid receptor, Voltage-gated inwardly rectifying potassium channel KCNH2, Cytochrome P450 2D6.

Bioactivity

ChEMBL activities: 11 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CXCR4	8.3	IC50	5	nM	CHEMBL_ACT_18399360
CXCR4	8.29	IC50	5.18	nM	CHEMBL_ACT_23316731
CXCR4	7.89	IC50	13	nM	CHEMBL_ACT_3253208
CXCR4	7.64	Ki	23	nM	CHEMBL_ACT_25100049
CXCR4	7.52	IC50	30	nM	CHEMBL_ACT_13847048
CYP2D6	5.8	IC50	1600	nM	CHEMBL_ACT_18273664
CYP2D6	5.79	IC50	1640	nM	CHEMBL_ACT_18399470
CYP2D6	5.79	IC50	1640	nM	CHEMBL_ACT_23316806
DRD2	5.39	IC50	4100	nM	CHEMBL_ACT_24421263
KCNH2	5.28	IC50	5200	nM	CHEMBL_ACT_24421293
CHRM2	5.12	IC50	7500	nM	CHEMBL_ACT_24421273

Target pathways

Aggregated over 1 target gene(s): CXCR4.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Binding and entry of HIV virion	1	CXCR4
Signaling by ROBO receptors	1	CXCR4
Chemokine receptors bind chemokines	1	CXCR4
G alpha (i) signalling events	1	CXCR4
Formation of definitive endoderm	1	CXCR4
Specification of primordial germ cells	1	CXCR4
Developmental Lineage of Multipotent Pancreatic Progenitor Cells	1	CXCR4

Dominant GO biological processes

GO term	Targets
response to hypoxia	1
dendritic cell chemotaxis	1
apoptotic process	1
inflammatory response	1
immune response	1
G protein-coupled receptor signaling pathway	1
adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway	1
positive regulation of cytosolic calcium ion concentration	1
brain development	1
response to virus	1
calcium-mediated signaling	1
neurogenesis	1
regulation of cell adhesion	1
positive regulation of cell migration	1
CXCL12-activated CXCR4 signaling pathway	1

Indications & clinical

Indications

7 indications (1 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).

Indication	Trial phase	MONDO	EFO
neoplasm	4	MONDO:0005070	EFO:0000616
neutropenia	3	MONDO:0001475	MONDO:0001475
clear cell renal carcinoma	1	MONDO:0005005	EFO:0000349
melanoma	1	MONDO:0005105	EFO:0000756
HIV infectious disease	1	MONDO:0005109	EFO:0000764
Waldenstrom macroglobulinemia	1	MONDO:0100280	EFO:0009441
breast neoplasm	1	MONDO:0021100	MONDO:0007254

Clinical trials

Total trials: 15.

Phase distribution

Phase	Trials
PHASE1	7
PHASE1/PHASE2	5
PHASE3	2
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03995108	PHASE3	ACTIVE_NOT_RECRUITING	Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
NCT06056297	PHASE3	RECRUITING	A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
NCT00089466	PHASE1/PHASE2	COMPLETED	Safety and Activity of the Oral HIV Entry Inhibitor AMD11070 in HIV Infected Patients
NCT02667886	PHASE1/PHASE2	COMPLETED	Trial of X4P-001 in Participants With Advanced Renal Cell Carcinoma
NCT02923531	PHASE1/PHASE2	TERMINATED	Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma
NCT03005327	PHASE2	COMPLETED	A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
NCT04154488	PHASE1/PHASE2	COMPLETED	A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
NCT05103917	PHASE1/PHASE2	UNKNOWN	A Study to Assess the Safety, Tolerability and Antitumor Activity of X4P-001 in Combination With TNBC
NCT06858696	PHASE1	RECRUITING	A Study to Investigate Pharmacokinetics (PK) and Safety of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function
NCT00063804	PHASE1	COMPLETED	Safety of AMD070 When Administered Alone or Boosted With Low-Dose Ritonavir in HIV Uninfected Men
NCT00361101	PHASE1	COMPLETED	A Study of AMD11070 in HIV-infected Patients Carrying X4-tropic Virus
NCT02680782	PHASE1	TERMINATED	A Study Comparing Once-Daily vs. Twice-Daily Dosing of X4P-001 in Healthy Volunteers
NCT02823405	PHASE1	COMPLETED	X4P-001 and Pembrolizumab in Patients With Advanced Melanoma
NCT04274738	PHASE1	COMPLETED	A Study of Mavorixafor in Combination With Ibrutinib in Participants With Waldenstrom’s Macroglobulinemia (WM) Whose Tumors Express Mutations in MYD88 and CXCR4
NCT06914869	PHASE1	COMPLETED	Drug-Drug Interaction Potential of Mavorixafor

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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.

4 molecules share ≥1 primary target. Top 4 by shared-target count:

Molecule	Source	Status	Shared targets
CHLOROQUINE	ChEMBL	Phase 4 (approved)	CXCR4
PLERIXAFOR	ChEMBL	Phase 4 (approved)	CXCR4
ZALCITABINE	ChEMBL	Phase 4 (approved)	CXCR4
APLAVIROC	ChEMBL	Phase 3	CXCR4