Motixafortide

Summary

Motixafortide (CHEMBL5095030) is a phase-3 clinical-stage protein (ATC L03AX23) targeting CXCR4; indicated across 14 conditions including neoplasm and chronic myeloid leukemia.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Protein
• ATC class: L03AX23
• Targets: 1 (CXCR4)
• Indications: 14 conditions
• Clinical trials: 21
• Chemistry: 2159.5 Da · C97H144FN33O19S2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095030
Name	Motixafortide
Type	Protein
Max phase	3
FDA approved	no
PubChem CID	166625217
ATC	L03AX23
Molecular formula	C97H144FN33O19S2
Molecular weight	2159.5
InChIKey	JJVZSYKFCOBILL-DPNRMINZSA-N

SMILES: C1C[C@H]2C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@H](C(=O)N[C@@H](C(=O)N[C@H](C(=O)N[C@@H](C(=O)N2C1)CCCCN)CCCCN)CCCNC(=O)N)CC3=CC=C(C=C3)O)NC(=O)[C@H](CC4=CC5=CC=CC=C5C=C4)NC(=O)[C@H](CCCNC(=N)N)NC(=O)[C@H](CCCNC(=N)N)NC(=O)C6=CC=C(C=C6)F)C(=O)N[C@@H](CCCNC(=N)N)C(=O)N)CCCNC(=O)N)CCCNC(=N)N)CC7=CC=C(C=C7)O

IUPAC name: (3R,6S,9R,12S,15R,20R,23S,26S,29S,32S)-3,6-bis(4-aminobutyl)-N-[(2S)-1-amino-5-carbamimidamido-1-oxopentan-2-yl]-15-[[(2S)-2-[[(2S)-5-carbamimidamido-2-[[(2S)-5-carbamimidamido-2-[(4-fluorobenzoyl)amino]pentanoyl]amino]pentanoyl]amino]-3-naphthalen-2-ylpropanoyl]amino]-26-(3-carbamimidamidopropyl)-9,23-bis[3-(carbamoylamino)propyl]-12,29-bis[(4-hydroxyphenyl)methyl]-2,5,8,11,14,22,25,28,31-nonaoxo-17,18-dithia-1,4,7,10,13,21,24,27,30-nonazabicyclo[30.3.0]pentatriacontane-20-carboxamide

Also known as: 4F-BENZOYL-TN-14003, 4F-benzoyl-TN14003, BKT-140, Bkt140, BKT140, Bl 8040, BL-8040, Motixafortida, Motixafortide, T140, TF 14016, TF-14016

Patent coverage: 438 distinct patent families (1,170 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	9.04	0%	P61073

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

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

13 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
neoplasm	3	MONDO:0005070	EFO:0000616
chronic myeloid leukemia	2	MONDO:0011996	EFO:0000339
Hodgkins lymphoma	2	MONDO:0004952	EFO:0000183
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
acute lymphoblastic leukemia	2	MONDO:0004967	EFO:0000220
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
plasma cell myeloma	2	MONDO:0009693	EFO:0001378
aplastic anemia	2	MONDO:0015909	HP:0001915
non-Hodgkin lymphoma	2	MONDO:0018908	EFO:0005952
gastric adenocarcinoma	1	MONDO:0005036	EFO:0000503
carcinoma of esophagus	1	MONDO:0019086	EFO:0002916
sickle cell disease	1	MONDO:0011382	MONDO:0011382
adenocarcinoma	1	MONDO:0004970	MONDO:0003219

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

Clinical trials

Total trials: 21.

Phase distribution

Phase	Trials
PHASE2	8
PHASE1/PHASE2	5
PHASE1	5
PHASE3	2
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07101445	PHASE4	RECRUITING	Evaluating Premedication Regimens (Methylprednisolone vs Dexamethasone-based) for the Prevention of Systemic and Injection Site Reactions to Motixafortide in Patients With Multiple Myeloma Undergoing Stem Cell Mobilization, PARADE Trial
NCT03246529	PHASE3	ACTIVE_NOT_RECRUITING	A Phase III, Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and Granulocyte Colony Stimulating Factor (G-CSF) as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects With Multiple Myeloma (MM)
NCT06514508	PHASE3	RECRUITING	Mobilization of Stem Cells With Motixafortide (BL-8040) in Combination With G-CSF in Multiple Myeloma Patients
NCT04543071	PHASE2	RECRUITING	Chemo4METPANC Combination Chemokine Inhibitor, Immunotherapy, and Chemotherapy in Pancreatic Adenocarcinoma
NCT07392970	PHASE2	NOT_YET_RECRUITING	Motixafortide for MRD Sensitization in AML
NCT01010880	PHASE1/PHASE2	COMPLETED	Safety Study of a Chemokine Receptor (CXCR4) Antagonist in Multiple Myeloma Patients
NCT01838395	PHASE2	COMPLETED	Phase IIa Study Evaluating Safety and Efficacy of BL-8040 in Relapsed/Refractory AML Patients
NCT02115672	PHASE1/PHASE2	WITHDRAWN	Imatinib and BL-8040 (Novel Anti CXCR4 Antagonist) for Improving Molecular Response in Chronic Myelogenous Leukemia
NCT02462252	PHASE2	COMPLETED	Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
NCT02502968	PHASE2	UNKNOWN	BL-8040 Addition to Consolidation Therapy in AML Patients
NCT02639559	PHASE2	COMPLETED	Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies
NCT02763384	PHASE2	TERMINATED	BL-8040 and Nelarabine for Relapsed or Refractory T-Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma
NCT02826486	PHASE2	COMPLETED	Study Assessing Safety and Efficacy of Combination of BL-8040 and Pembrolizumab in Metastatic Pancreatic Cancer Patients
NCT03154827	PHASE1/PHASE2	TERMINATED	Safety, Tolerability and Efficacy of the BL-8040 and Atezolizumab for Maintenance Treatment in Subjects With Acute Myeloid Leukemia (AML)
NCT03193190	PHASE1/PHASE2	COMPLETED	A Study of Multiple Immunotherapy-Based Treatment Combinations in Participants With Metastatic Pancreatic Ductal Adenocarcinoma (Morpheus-Pancreatic Cancer)
NCT03281369	PHASE1/PHASE2	COMPLETED	A Study of Multiple Immunotherapy-Based Treatment Combinations in Patients With Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction Cancer (G/GEJ) or Esophageal Cancer (Morpheus-Gastric and Esophageal Cancer)
NCT06442761	PHASE1	RECRUITING	SCD Stem Cell Mobilization and Apheresis Using Motixafortide
NCT06506461	PHASE1	RECRUITING	Gene Editing For Sickle Cell Disease
NCT02073019	PHASE1	COMPLETED	A Phase I Study Evaluating Safety, Tolerability, PK and PD of BL-8040 for Stem Cell Mobilization in Healthy Volunteers
NCT05618301	PHASE1	COMPLETED	Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)
NCT06547112	PHASE1	COMPLETED	A Pharmacodynamic Study of the Apheresis Product of Multiple Myeloma Patients Undergoing Quad-induction Followed by Motixafortide + G-CSF Mobilization

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

5 molecules share ≥1 primary target. Top 5 by shared-target count:

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