Plerixafor

Summary

Plerixafor (CHEMBL18442) is an approved small-molecule antineoplastic agent (ATC L03AX16) targeting CXCR4 and ACKR3; indicated across 39 conditions including plasma cell myeloma and lymphoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L03AX16
• Targets: 2 (CXCR4, ACKR3)
• Indications: 39 conditions
• Clinical trials: 134
• Chemistry: 502.8 Da · C28H54N8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL18442
Name	Plerixafor
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	65015
ChEBI	CHEBI:125354
ATC	L03AX16
Molecular formula	C28H54N8
Molecular weight	502.8
InChIKey	YIQPUIGJQJDJOS-UHFFFAOYSA-N

SMILES: C1CNCCNCCCN(CCNC1)CC2=CC=C(C=C2)CN3CCCNCCNCCCNCC3

IUPAC name: 1-[[4-(1,4,8,11-tetrazacyclotetradec-1-ylmethyl)phenyl]methyl]-1,4,8,11-tetrazacyclotetradecane

ChEBI definition: An azamacrocycle consisting of two cyclam rings connected by a 1,4-phenylenebis(methylene) linker. It is a CXCR4 chemokine receptor antagonist and a hematopoietic stem cell mobilizer. It is used in combination with grulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells to the perpheral blood for collection and subsequent autologous transplantation in patients with non-Hodgkin’s lymphoma and multiple myeloma.

Pharmacological roles (ChEBI): immunological adjuvant, antineoplastic agent, anti-HIV agent, C-X-C chemokine receptor type 4 antagonist.

Also known as: AMD 3100, AMD3100, JM 3100, Mozobil, Plerixafor, Plerixafor accord, Plerixafor hydrochloride, Plerixafor octahydrochloride, SID 791, AMD-3100, SID29217477, PLERIXAFOR

Parent form; salt/anhydrous children: CHEMBL1788331, CHEMBL2088494, CHEMBL2311028, CHEMBL2311089, CHEMBL2373140, CHEMBL5402188

Patent coverage: 3,076 distinct patent families (11,099 SureChEMBL compound mentions), from 1 matched compound structure(s). 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	6.19	0%	P61073
ACKR3	ACKR3	Agonist	6.85	0%	P25106

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, RecQ-like DNA helicase BLM, Alpha-2C adrenergic receptor, Menin/Histone-lysine N-methyltransferase MLL, C-X-C chemokine receptor type 4, Muscarinic acetylcholine receptor M2, Muscarinic acetylcholine receptor M1, Histamine H3 receptor, Stromal cell-derived factor 1, C-C chemokine receptor type 2.

Bioactivity

ChEMBL activities: 88 potent at pChembl ≥ 5 of 99 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CCR2	10.4	IC50	0.04	nM	CHEMBL_ACT_2186303
CCR2	10.05	IC50	0.09	nM	CHEMBL_ACT_2186299
CXCR4	9.09	IC50	0.81	nM	CHEMBL_ACT_2186297
CXCR4	9.09	IC50	0.81	nM	CHEMBL_ACT_25100052
HRH3	8.56	Ki	2.74	nM	CHEMBL_ACT_25741214
CXCR4	8.54	IC50	2.9	nM	CHEMBL_ACT_24992562
CXCR4	8.51	IC50	3.1	nM	CHEMBL_ACT_24891303
CXCL12	8.24	IC50	5.7	nM	CHEMBL_ACT_26133930
CXCR4	8.22	IC50	6	nM	CHEMBL_ACT_16670870
CXCR4	8.21	IC50	6.13	nM	CHEMBL_ACT_22781978
CXCR4	8.21	IC50	6.13	nM	CHEMBL_ACT_22983636
CXCR4	8.14	IC50	7.3	nM	CHEMBL_ACT_25886891
CXCR4	7.75	IC50	18	nM	CHEMBL_ACT_18542189
CXCR4	7.56	IC50	27.4	nM	CHEMBL_ACT_2610585
CXCR4	7.36	IC50	44	nM	CHEMBL_ACT_18314585
CXCR4	7.36	IC50	44	nM	CHEMBL_ACT_19218966
CXCR4	7.36	IC50	44	nM	CHEMBL_ACT_26133927
CXCR4	7.29	IC50	51	nM	CHEMBL_ACT_19218935
CXCR4	7.19	IC50	65	nM	CHEMBL_ACT_19438232
CXCR4	7.14	IC50	72.7	nM	CHEMBL_ACT_5163237
CXCR4	7.13	IC50	74	nM	CHEMBL_ACT_12149702
O08565	7	IC50	100	nM	CHEMBL_ACT_17723422
O08565	6.97	IC50	108	nM	CHEMBL_ACT_2578195
CXCR4	6.85	Ki	140	nM	CHEMBL_ACT_2183876
CXCR4	6.84	IC50	143.7	nM	CHEMBL_ACT_5163231
CXCR4	6.8	Ki	160	nM	CHEMBL_ACT_2183648
CXCR4	6.77	Ki	170	nM	CHEMBL_ACT_2185035
CXCR4	6.76	IC50	175.3	nM	CHEMBL_ACT_5163236
CXCR4	6.72	Ki	190	nM	CHEMBL_ACT_2183884
CXCR4	6.71	IC50	196.6	nM	CHEMBL_ACT_5163228

Target pathways

Aggregated over 2 target gene(s): CXCR4, ACKR3.

Top Reactome pathways

13 total, by targets touching each:

Pathway	Targets	Genes
Chemokine receptors bind chemokines	2	ACKR3, CXCR4
G alpha (i) signalling events	2	ACKR3, CXCR4
Signal Transduction	1	ACKR3
Binding and entry of HIV virion	1	CXCR4
Signaling by GPCR	1	ACKR3
Class A/1 (Rhodopsin-like receptors)	1	ACKR3
Peptide ligand-binding receptors	1	ACKR3
Signaling by ROBO receptors	1	CXCR4
GPCR downstream signalling	1	ACKR3
GPCR ligand binding	1	ACKR3
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
immune response	2
G protein-coupled receptor signaling pathway	2
positive regulation of cytosolic calcium ion concentration	2
calcium-mediated signaling	2
cell chemotaxis	2
chemotaxis	2
signal transduction	2
chemokine-mediated signaling pathway	2
response to hypoxia	1
dendritic cell chemotaxis	1
apoptotic process	1
inflammatory response	1
adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway	1
brain development	1
response to virus	1

Indications & clinical

Indications

39 indications (7 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
plasma cell myeloma	4	MONDO:0009693	EFO:0001378
lymphoma	4	MONDO:0005062	EFO:0000574
acute lymphoblastic leukemia	4	MONDO:0004967	EFO:0000220
non-Hodgkin lymphoma	4	MONDO:0018908	EFO:0005952
childhood malignant neoplasm	4	MONDO:0006517	EFO:1000654
neoplasm	4	MONDO:0005070	EFO:0000616
Hodgkins lymphoma	2	MONDO:0004952	EFO:0000183
diffuse large B-cell lymphoma	2	MONDO:0018905	EFO:0000403
systemic sclerosis	2	MONDO:0005100	EFO:0000717
exocrine pancreatic carcinoma	2	MONDO:0005192	EFO:0002618
gliosarcoma	2	MONDO:0016681	EFO:1001465
head and neck cancer	2	MONDO:0005627	EFO:0006859
T-cell non-Hodgkin lymphoma	2	MONDO:0015760	MONDO:0015760
hematopoietic and lymphoid system neoplasm	2	MONDO:0002334	MONDO:0044881
lymphopenia	2	MONDO:0003783	MONDO:0003783
sickle cell disease	2	MONDO:0011382	MONDO:0011382
chronic granulomatous disease	2	MONDO:0018305	MONDO:0018305
Fanconi anemia	2	MONDO:0019391	MONDO:0019391
graft versus host disease	1	MONDO:0013730	EFO:0004599
acute myeloid leukemia	1	MONDO:0018874	EFO:0000222
B-cell chronic lymphocytic leukemia	1	MONDO:0004948	EFO:0000095
myelodysplastic syndrome	1	MONDO:0018881	EFO:0000198
leukemia	1	MONDO:0005059	EFO:0000565
mantle cell lymphoma	1	MONDO:0018876	EFO:1001469
thalassemia	1	MONDO:0000984	EFO:1001996
sarcoma	1	MONDO:0005089	EFO:0000691
kidney disorder	1	MONDO:0005240	EFO:0003086
neutropenia	1	MONDO:0001475	MONDO:0001475
follicular lymphoma	1	MONDO:0018906	MONDO:0018906
type 1 diabetes mellitus	1	MONDO:0005147	MONDO:0005147
acute biphenotypic leukemia	1	MONDO:0020322	MONDO:0019460
beta thalassemia	1	MONDO:0019402	Orphanet:848
glioblastoma	1	MONDO:0018177	EFO:0000519
syndromic disease	0	MONDO:0002254	MONDO:0002254
blood platelet disease	0	MONDO:0002245	MONDO:0002245

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

Clinical trials

Total trials: 134.

Phase distribution

Phase	Trials
PHASE2	48
PHASE1	34
PHASE1/PHASE2	28
Not specified	14
PHASE4	5
PHASE3	3
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01164475	PHASE4	COMPLETED	Evaluation of Approved Weight-Based Dose Compared to Fixed Dose of Plerixafor in Patients With Non-Hodgkin’s Lymphoma (NHL) Weighing Less Than 70 Kilograms
NCT02006225	PHASE4	UNKNOWN	Stem Cell Harvesting Using GCSF Plus Plerxiafor, in First -Line, for Heavily Pre- Treated Pediatric Oncology Patients.
NCT02056210	PHASE4	COMPLETED	Stem Cell Mobilization With Plerixafor in Diabetic vs Control Subjects
NCT05087212	PHASE4	COMPLETED	Mobilization of Stem Cells With AMD3100 (Plerixafor) in Combination With G-CSF in Multiple Myeloma Patients
NCT05510544	PHASE4	UNKNOWN	Plerixafor for Poorly Mobilized Lymphoma
NCT01146834	PHASE3	COMPLETED	Trial of Three Stem Cell Mobilization Regimens for Multiple Myeloma
NCT01301963	PHASE3	TERMINATED	Filgrastim With or Without Plerixafor in Treating Patients With Multiple Myeloma Previously Treated With Lenalidomide
NCT01767714	PHASE3	COMPLETED	Evaluation of Plerixafor Plus G-CSF to Mobilize and Collect 5×10^6CD34+ Cells/kg in Non-Hodgkin’s Lymphoma (NHL) Patients for Autologous Transplantation
NCT02231879	PHASE2/PHASE3	COMPLETED	Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
NCT00967785	PHASE1/PHASE2	RECRUITING	A Phase I Study of Mozobil in the Treatment of Patients With WHIMS
NCT01318317	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Genetically Engineered Lymphocyte Therapy After Peripheral Blood Stem Cell Transplant in Treating Patients With High-Risk, Intermediate-Grade, B-cell Non-Hodgkin Lymphoma
NCT02015013	PHASE2	RECRUITING	Hematopoietic Stem Cell Mobilization in Idiopathic CD4 Lymphocytopenia Patients and Healthy Controls for the Study of T Cell Maturation and Trafficking in Murine Models
NCT02570542	PHASE2	ACTIVE_NOT_RECRUITING	Study of the Impact of CD34+ Cell Dose on Absolute Lymphocyte Count Following High-Dose Therapy and Autologous Stem Cell Transplantation for Relapsed and Refractory Diffuse Large B-cell Lymphoma (DLBCL)
NCT03055247	PHASE2	RECRUITING	Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD
NCT05357482	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
NCT05470491	PHASE1/PHASE2	RECRUITING	Trial of Allogeneic Reduced-Intensity, HLA-Haploidentical Allogeneic Hematopoietic Cell Bone Marrow Transplantation Followed by Graft-versus-Host-Disease (GVHD) Prophylaxis With Cyclophosphamide, Bortezomib and Maraviroc for Hematologic Malignancies …
NCT06158828	PHASE1/PHASE2	RECRUITING	Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML
NCT06207799	PHASE2	RECRUITING	Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma
NCT06325709	PHASE1/PHASE2	RECRUITING	Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCT06851767	PHASE1/PHASE2	ENROLLING_BY_INVITATION	Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCT07188090	PHASE2	RECRUITING	Mozobil for Autologous Hematopoietic Stem Cell Transplantation
NCT07605416	PHASE2	NOT_YET_RECRUITING	Testing an Experimental Approach to Treat Patients With Plasma Cell Leukemia, The QUANTUM Trial
NCT00241358	PHASE1/PHASE2	COMPLETED	Study Evaluating AMD3100 for Transplantation of Sibling Donor Stem Cells in Patients With Hematological Malignancies
NCT00322387	PHASE2	COMPLETED	Mobilization of Stem Cells With Plerixafor, Chemotherapy and G-CSF in Multiple Myeloma or Non-Hodgkin’s Lymphoma Patients
NCT00396383	PHASE2	TERMINATED	Treatment With AMD3100 (Plerixafor) in MM Patients to Mobilize PBCs For Collection and for Transplantation
NCT00396968	PHASE1/PHASE2	WITHDRAWN	AMD3100 With Busulfan, Fludarabine and Thymoglobulin for Allogeneic Stem Cell Transplant for AML and MDS
NCT00479115	PHASE1/PHASE2	COMPLETED	Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
NCT00512252	PHASE1/PHASE2	COMPLETED	AMD3100 Plus Mitoxantrone, Etoposide and Cytarabine in Acute Myeloid Leukemia
NCT00665314	PHASE2	COMPLETED	Evaluation of the Safety and Efficacy of the Addition of AMD3100 to a G-CSF Mobilization Regimen in Patients With Lymphoma (NHL and HD) and Multiple Myeloma (MM).
NCT00669669	PHASE1/PHASE2	TERMINATED	O6-Benzylguanine-Mediated Tumor Sensitization With Chemoprotected Autologous Stem Cell in Treating Patients With Malignant Gliomas
NCT00733824	PHASE1/PHASE2	COMPLETED	Intravenous AMD3100 for Collection of Autologous Peripheral Blood Stem Cells in Patients With Lymphoma
NCT00822770	PHASE1/PHASE2	COMPLETED	Plerixafor and Granulocyte Colony-stimulating Factor (G-CSF) With Busulfan, Fludarabine and Thymoglobulin
NCT00903968	PHASE1/PHASE2	COMPLETED	Combination Plerixafor (AMD3100)and Bortezomib in Relapsed or Relapsed/Refractory Multiple Myeloma
NCT00906945	PHASE1/PHASE2	COMPLETED	Chemosensitization With Plerixafor Plus G-CSF in Acute Myeloid Leukemia
NCT00914849	PHASE2	COMPLETED	Intravenous (IV) AMD3100 for Mobilization and Matched Related Transplant for Advanced Hematological Malignancies
NCT00998049	PHASE2	COMPLETED	Plerixafor in Treating Patients With Multiple Myeloma Previously Treated With Lenalidomide and Planning to Undergo Autologous Stem Cell Transplant
NCT01037517	PHASE2	COMPLETED	Trial of an Augmented Mobilization Strategy With Plerixafor (Mozobil®) in a Population at Risk for Poor Stem Cell Mobilization
NCT01095757	PHASE2	COMPLETED	Evaluation of the Drug Plerixafor in Combination With Chemotherapy and G-CSF for Stem Cell Collection
NCT01097057	PHASE2	COMPLETED	Rituximab, Combination Chemotherapy, Filgrastim (G-CSF), and Plerixafor in Treating Patients With Non-Hodgkin Lymphoma Undergoing Mobilization of Autologous Peripheral Blood Stem Cells
NCT01149863	PHASE2	COMPLETED	Alteration in Timing of Plerixafor Administration

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
MAVORIXAFOR	ChEMBL + PubChem	Phase 4 (approved)	CXCR4
CHLOROQUINE	ChEMBL	Phase 4 (approved)	CXCR4
ZALCITABINE	ChEMBL	Phase 4 (approved)	CXCR4
APLAVIROC	ChEMBL	Phase 3	CXCR4