Vimseltinib

Summary

Vimseltinib (CHEMBL5095202) is an approved small molecule targeting PDGFRA, KIT, and CSF1R; indicated across 3 conditions including tenosynovial giant cell tumor and neoplasm.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Targets: 3 (PDGFRA, KIT, CSF1R)
• Indications: 3 conditions
• Clinical trials: 7
• Chemistry: 431.5 Da · C23H25N7O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095202
Name	Vimseltinib
Type	Small molecule
Max phase	3
FDA approved	yes
PubChem CID	86267612
Molecular formula	C23H25N7O2
Molecular weight	431.5
InChIKey	TVGAHWWPABTBCX-UHFFFAOYSA-N

SMILES: CC1=C(C=CC(=N1)C2=CN=C(N(C2=O)C)NC(C)C)OC3=CC(=NC=C3)C4=CN(N=C4)C

IUPAC name: 3-methyl-5-[6-methyl-5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-(propan-2-ylamino)pyrimidin-4-one

Also known as: Dcc-3014, DCC-3014, DP-6865, Vimseltinib, VIMSELTINIB

Patent coverage: 56 distinct patent families (138 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 125 (91%) 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
PDGFRA	platelet derived growth factor receptor alpha	Inhibition	6	6.2%	P16234
KIT	KIT proto-oncogene, receptor tyrosine kinase	Inhibition	7	0.5%	P10721
CSF1R	colony stimulating factor 1 receptor	Inhibition	8	0%	P07333

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Macrophage colony-stimulating factor 1 receptor, Platelet-derived growth factor receptor beta, Mast/stem cell growth factor receptor Kit, Receptor-type tyrosine-protein kinase FLT3, Platelet-derived growth factor receptor alpha, Tyrosine-protein kinase Lck, Cytochrome P450 2C9, Protein-tyrosine kinase 6.

Bioactivity

ChEMBL activities: 14 potent at pChembl ≥ 5 of 15 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CSF1R	8.7	IC50	2	nM	CHEMBL_ACT_26198618
CSF1R	8.66	IC50	2.2	nM	CHEMBL_ACT_29025725
CSF1R	8.52	IC50	3	nM	CHEMBL_ACT_24694156
CSF1R	8.43	IC50	3.7	nM	CHEMBL_ACT_24693960
CSF1R	7.57	IC50	27	nM	CHEMBL_ACT_24694088
PDGFRA	6.36	IC50	436	nM	CHEMBL_ACT_24694000
KIT	6.32	IC50	480	nM	CHEMBL_ACT_26198620
KIT	6.06	IC50	864	nM	CHEMBL_ACT_29025728
KIT	5.8	IC50	1600	nM	CHEMBL_ACT_24694157
PTK6	5.68	IC50	2100	nM	CHEMBL_ACT_24694158
PDGFRB	5.64	IC50	2300	nM	CHEMBL_ACT_24694020
PDGFRA	5.6	IC50	2500	nM	CHEMBL_ACT_29025731
FLT3	5.57	IC50	2700	nM	CHEMBL_ACT_29025734
LCK	5.55	IC50	2800	nM	CHEMBL_ACT_24694159

Target pathways

Aggregated over 3 target gene(s): PDGFRA, KIT, CSF1R.

Top Reactome pathways

51 total, by targets touching each:

Pathway	Targets	Genes
PIP3 activates AKT signaling	2	KIT, PDGFRA
Constitutive Signaling by Aberrant PI3K in Cancer	2	KIT, PDGFRA
RAF/MAP kinase cascade	2	KIT, PDGFRA
PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling	2	KIT, PDGFRA
Developmental Biology	1	KIT
Signaling by SCF-KIT	1	KIT
Regulation of KIT signaling	1	KIT
Signal Transduction	1	KIT
Disease	1	KIT
Downstream signal transduction	1	PDGFRA
Signaling by PDGF	1	PDGFRA
Negative regulation of the PI3K/AKT network	1	KIT
Generic Transcription Pathway	1	KIT
PI3K/AKT Signaling in Cancer	1	KIT
Other interleukin signaling	1	CSF1R
Diseases of signal transduction by growth factor receptors and second messengers	1	KIT
MAPK family signaling cascades	1	KIT
MAPK1/MAPK3 signaling	1	KIT
RNA Polymerase II Transcription	1	KIT
Gene expression (Transcription)	1	KIT
Transcriptional Regulation by VENTX	1	CSF1R
Transcriptional regulation by the AP-2 (TFAP2) family of transcription factors	1	KIT
TFAP2 (AP-2) family regulates transcription of growth factors and their receptors	1	KIT
Intracellular signaling by second messengers	1	KIT
Signaling by Receptor Tyrosine Kinases	1	KIT
Dasatinib-resistant KIT mutants	1	KIT
Imatinib-resistant KIT mutants	1	KIT
KIT mutants bind TKIs	1	KIT
Masitinib-resistant KIT mutants	1	KIT
Nilotinib-resistant KIT mutants	1	KIT

Dominant GO biological processes

GO term	Targets
cell surface receptor protein tyrosine kinase signaling pathway	3
positive regulation of cell population proliferation	3
cell migration	3
positive regulation of cell migration	3
protein autophosphorylation	3
positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction	3
protein phosphorylation	3
hematopoietic progenitor cell differentiation	2
peptidyl-tyrosine phosphorylation	2
regulation of actin cytoskeleton organization	2
cell chemotaxis	2
positive regulation of ERK1 and ERK2 cascade	2
chemotaxis	2
anatomical structure morphogenesis	2
inflammatory response	2

Indications & clinical

Indications

3 indications (0 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
tenosynovial giant cell tumor	3	MONDO:0002522	EFO:1000562
tenosynovial giant cell tumor, diffuse type	3	MONDO:0024686	MONDO:0024686
neoplasm	1	MONDO:0005070	EFO:0000616

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE1	4
PHASE3	1
PHASE1/PHASE2	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05059262	PHASE3	ACTIVE_NOT_RECRUITING	Study of Vimseltinib for Tenosynovial Giant Cell Tumor
NCT03069469	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Study of Vimseltinib (DCC-3014) in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
NCT06619561	PHASE2	RECRUITING	A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
NCT07539090	PHASE1	RECRUITING	Evaluate the Effect of Vimseltinib on Pharmacokinetics of Combined Oral Contraceptive (Ethinyl Estradiol/Levonorgestrel)
NCT04242238	PHASE1	COMPLETED	Study of DCC-3014 in Combination With Avelumab in Patients With Advanced or Metastatic Sarcomas
NCT07158398	PHASE1	COMPLETED	Evaluate the Effect of Vimseltinib on the Pharmacokinetics of a BCRP and OATP1B1 Substrate
NCT07158411	PHASE1	COMPLETED	Evaluate the Effect of Vimseltinib on Organic Cation Transporter 2 (OCT2)

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.

112 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
Crizotinib	ChEMBL + PubChem	Phase 4 (approved)	CSF1R, KIT, PDGFRA
Gefitinib	ChEMBL + PubChem	Phase 4 (approved)	CSF1R, KIT, PDGFRA
IMATINIB	ChEMBL + PubChem	Phase 4 (approved)	CSF1R, KIT, PDGFRA
PAZOPANIB	ChEMBL + PubChem	Phase 4 (approved)	CSF1R, KIT, PDGFRA
REGORAFENIB	ChEMBL + PubChem	Phase 4 (approved)	CSF1R, KIT, PDGFRA
AXITINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
BOSUTINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
DASATINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
FEDRATINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
MIDOSTAURIN	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
NILOTINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
NINTEDANIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
PEXIDARTINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
PONATINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
QUIZARTINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
SORAFENIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
SUNITINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
VANDETANIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT, PDGFRA
ALVOCIDIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
BRIVANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
CEDIRANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
DOVITINIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
LESTAURTINIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
LINIFANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
MASITINIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
MOTESANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
PIMICOTINIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
SEMAXANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
VATALANIB	ChEMBL	Phase 3	CSF1R, KIT, PDGFRA
CENISERTIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
DEFOSBARASERTIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
DORAMAPIMOD	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
ENMD-2076	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
FORETINIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
ILORASERTIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
R-406	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
RAF-265	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
REBASTINIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
SOTULETINIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
SU-014813	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
TANDUTINIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
TOZASERTIB	ChEMBL	Phase 2	CSF1R, KIT, PDGFRA
Afatinib	PubChem	Approved	CSF1R, KIT, PDGFRA
Idelalisib	PubChem	Approved	CSF1R, KIT, PDGFRA
Selumetinib	PubChem	Approved	CSF1R, KIT, PDGFRA
AVAPRITINIB	ChEMBL + PubChem	Phase 4 (approved)	KIT, PDGFRA
BRIGATINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT
CERITINIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
ENTRECTINIB	ChEMBL	Phase 4 (approved)	CSF1R, KIT
ERLOTINIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
INFIGRATINIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
LENVATINIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
RIPRETINIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
TIVOZANIB	ChEMBL	Phase 4 (approved)	KIT, PDGFRA
BARASERTIB	ChEMBL	Phase 3	KIT, PDGFRA
CANERTINIB	ChEMBL	Phase 3	KIT, PDGFRA
SARACATINIB	ChEMBL	Phase 3	KIT, PDGFRA
AMUVATINIB	ChEMBL	Phase 2	KIT, PDGFRA
BEMCENTINIB	ChEMBL	Phase 2	KIT, PDGFRA
BMS-777607	ChEMBL	Phase 2	KIT, PDGFRA