Lusutrombopag

Summary

Lusutrombopag (CHEMBL2107831) is an approved small molecule (ATC B02BX07) targeting MPL; indicated across 3 conditions including thrombocytopenia and autoimmune thrombocytopenic purpura.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: B02BX07
• Targets: 1 (MPL)
• Indications: 3 conditions
• Clinical trials: 7
• Chemistry: 591.5 Da · C29H32Cl2N2O5S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2107831
Name	Lusutrombopag
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	49843517
ATC	B02BX07
Molecular formula	C29H32Cl2N2O5S
Molecular weight	591.5
InChIKey	NOZIJMHMKORZBA-KJCUYJGMSA-N

SMILES: CCCCCCO[C@@H](C)C1=CC=CC(=C1OC)C2=CSC(=N2)NC(=O)C3=CC(=C(C(=C3)Cl)/C=C(\C)/C(=O)O)Cl

IUPAC name: (E)-3-[2,6-dichloro-4-[[4-[3-[(1S)-1-hexoxyethyl]-2-methoxyphenyl]-1,3-thiazol-2-yl]carbamoyl]phenyl]-2-methylprop-2-enoic acid

Also known as: Lusutrombopag, Mulpleta, RSC888711, S-888711, LUSUTROMBOPAG, lusutrombopag

Patent coverage: 61 distinct patent families (122 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 101 (83%) 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
MPL	Thrombopoietin receptor	Agonist		0.2%	P40238

Broader ChEMBL bioactivity targets: 17 (assay-derived). Sample: Thrombopoietin receptor, Prothrombin, D(1A) dopamine receptor, Estrogen receptor, Thromboxane A2 receptor, Progesterone receptor, 5-hydroxytryptamine receptor 1A, Muscarinic acetylcholine receptor M1, Prostaglandin G/H synthase 1, Alpha-1A adrenergic receptor.

Bioactivity

ChEMBL activities: 13 potent at pChembl ≥ 5 of 19 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
MPL	8.96	ED50	1.09	nM	CHEMBL_ACT_16888437
ADORA3	6.48	AC50	333.8	nM	CHEMBL_ACT_25199055
DRD1	5.81	AC50	1540	nM	CHEMBL_ACT_25115637
PTGS1	5.67	AC50	2151	nM	CHEMBL_ACT_25205651
ESR1	5.54	AC50	2907	nM	CHEMBL_ACT_25139285
ABCB11	5.4	AC50	4000	nM	CHEMBL_ACT_25127171
Q01827	5.39	AC50	4100	nM	CHEMBL_ACT_25197401
KCNH2	5.31	AC50	4900	nM	CHEMBL_ACT_25118464
DRD1	5.26	AC50	5530	nM	CHEMBL_ACT_25234812
PTGS1	5.19	AC50	6420	nM	CHEMBL_ACT_25206581
HTR1A	5.16	AC50	6957	nM	CHEMBL_ACT_25165451
DRD3	5.08	AC50	8409	nM	CHEMBL_ACT_25194938
PGR	5.03	AC50	9338	nM	CHEMBL_ACT_25204718

Target pathways

Aggregated over 1 target gene(s): MPL.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Platelet Aggregation (Plug Formation)	1	MPL

Dominant GO biological processes

GO term	Targets
neutrophil homeostasis	1
platelet formation	1
monocyte homeostasis	1
thrombopoietin-mediated signaling pathway	1
positive regulation of lymphocyte proliferation	1
cellular response to hypoxia	1
positive regulation of platelet formation	1
eosinophil homeostasis	1
basophil homeostasis	1

Indications & clinical

Indications

3 indications (2 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
thrombocytopenia	4	MONDO:0002049	HP:0001873
autoimmune thrombocytopenic purpura	2	MONDO:0008558	EFO:0007160

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

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE2	3
PHASE4	1
PHASE3	1
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06426043	PHASE4	NOT_YET_RECRUITING	A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag
NCT02389621	PHASE3	COMPLETED	Safety and Efficacy Study of Lusutrombopag for Thrombocytopenia in Patients With Chronic Liver Disease Undergoing Elective Invasive Procedures
NCT01054443	PHASE2	TERMINATED	A Study to Investigate the Efficacy and Safety of Lusutrombopag (S-888711) Tablets Administered to Adults With Immune Thrombocytopenia (ITP)
NCT01129024	PHASE2	TERMINATED	An Open-label Safety Study of Lusutrombopag (S-888711) in Adults With Chronic Immune Thrombocytopenia (ITP)
NCT06287567	PHASE2	COMPLETED	Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP)
NCT03897413	PHASE1	COMPLETED	Effect of Food and Calcium on Pharmacokinetics of a Single Dose of S-888711 in Healthy Adult Volunteers
NCT07483385	Not specified	RECRUITING	Efficacy and Safety of Lusutrombopag After Allogeneic Hematopoietic Stem Cell Transplantation

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.

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

Molecule	Source	Status	Shared targets
ELTROMBOPAG	ChEMBL	Phase 4 (approved)	MPL