Pimicotinib

Summary

Pimicotinib (CHEMBL5314535) is a phase-3 clinical-stage small molecule targeting CSF1R; indicated across 5 conditions including tenosynovial giant cell tumor and neoplasm.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (CSF1R)
• Indications: 5 conditions
• Clinical trials: 8
• Chemistry: 420.5 Da · C22H24N6O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5314535
Name	Pimicotinib
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	139549388
Molecular formula	C22H24N6O3
Molecular weight	420.5
InChIKey	NXFPMDWYDKHFMM-UHFFFAOYSA-N

SMILES: CC1=C(C=CC(=N1)NC(=O)N2CCC(C2=O)(C)C)OC3=CC(=NC=C3)C4=CN(N=C4)C

IUPAC name: 3,3-dimethyl-N-[6-methyl-5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyrrolidine-1-carboxamide

Also known as: ABSK-021 FREE BASE ANHYDROUS, ABSK021 FREE BASE ANHYDROUS, ASYM-133040, Pimicotinib, PIMICOTINIB

Parent form; salt/anhydrous children: CHEMBL6068433

Patent coverage: 6 distinct patent families (13 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 11 (85%) 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
CSF1R	colony stimulating factor 1 receptor	Inhibition	6.35	0%	P07333

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Macrophage colony-stimulating factor 1 receptor, Mast/stem cell growth factor receptor Kit, Platelet-derived growth factor receptor alpha.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CSF1R	7.71	IC50	19.48	nM	CHEMBL_ACT_26187730
KIT	7.11	IC50	76.98	nM	CHEMBL_ACT_26187725
PDGFRA	5.85	IC50	1399	nM	CHEMBL_ACT_26187728

Target pathways

Aggregated over 1 target gene(s): CSF1R.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Other interleukin signaling	1	CSF1R
Transcriptional Regulation by VENTX	1	CSF1R
Signaling by CSF1 (M-CSF) in myeloid cells	1	CSF1R

Dominant GO biological processes

GO term	Targets
positive regulation of protein phosphorylation	1
response to ischemia	1
inflammatory response	1
signal transduction	1
cell surface receptor protein tyrosine kinase signaling pathway	1
axon guidance	1
cell population proliferation	1
positive regulation of cell population proliferation	1
negative regulation of cell population proliferation	1
regulation of cell shape	1
positive regulation of macrophage chemotaxis	1
cell migration	1
peptidyl-tyrosine phosphorylation	1
cytokine-mediated signaling pathway	1
olfactory bulb development	1

Indications & clinical

Indications

5 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	2	MONDO:0005070	EFO:0000616
malignant pancreatic neoplasm	2	MONDO:0009831	EFO:1000359

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

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE1	6
PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06111274	PHASE2	RECRUITING	A Phase 2 Study of ABSK021 in Patients With Advanced Pancreatic Cancer
NCT07499362	PHASE2	RECRUITING	Study of Pimicotinib in Japanese Participants With Tenosynovial Giant Cell Tumor (TGCT) (J-MANEUVER)
NCT06884072	PHASE1	ACTIVE_NOT_RECRUITING	A Study to Evaluate the Effect of a High-fat Meal on the Exposure of Pimicotinib Capsule in Healthy Subjects
NCT06562946	PHASE1	COMPLETED	The Study to Assess the Pharmacokinetics of Pimicotinib in Subjects With Mild and Moderate Hepatic Impairment Relative to Subjects With Normal Hepatic Function
NCT06694948	PHASE1	COMPLETED	A Study to Evaluate the Relative Bioavailability of Two Different Pimicotinib Capsules in Healthy Subjects
NCT06779253	PHASE1	COMPLETED	A Study to Investigate The Effect of Pimicotinib on The Pharmacokinetics of Metformin, Fexofenadine and Rosuvastatin In Healthy Subjects
NCT07126249	PHASE1	COMPLETED	A Study to Evaluate the the Bioequivalence of Two Different Pimicotinib Capsules in Healthy Subjects
NCT07210996	PHASE1	COMPLETED	A Study to Evaluate the Relative Bioavailability of Pimicotinib Capsules Containing Free Base in Different Proportions in Healthy Subjects

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.

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

Molecule	Source	Status	Shared targets
CRIZOTINIB	ChEMBL + PubChem	Phase 4 (approved)	CSF1R
PAZOPANIB	ChEMBL + PubChem	Phase 4 (approved)	CSF1R
AXITINIB	ChEMBL	Phase 4 (approved)	CSF1R
BOSUTINIB	ChEMBL	Phase 4 (approved)	CSF1R
BRIGATINIB	ChEMBL	Phase 4 (approved)	CSF1R
DASATINIB	ChEMBL	Phase 4 (approved)	CSF1R
ENTRECTINIB	ChEMBL	Phase 4 (approved)	CSF1R
FEDRATINIB	ChEMBL	Phase 4 (approved)	CSF1R
FILGOTINIB	ChEMBL	Phase 4 (approved)	CSF1R
IBRUTINIB	ChEMBL	Phase 4 (approved)	CSF1R
IMATINIB	ChEMBL	Phase 4 (approved)	CSF1R
MIDOSTAURIN	ChEMBL	Phase 4 (approved)	CSF1R
NERATINIB	ChEMBL	Phase 4 (approved)	CSF1R
NILOTINIB	ChEMBL	Phase 4 (approved)	CSF1R
NINTEDANIB	ChEMBL	Phase 4 (approved)	CSF1R
PACRITINIB	ChEMBL	Phase 4 (approved)	CSF1R
PEXIDARTINIB	ChEMBL	Phase 4 (approved)	CSF1R
PONATINIB	ChEMBL	Phase 4 (approved)	CSF1R
QUIZARTINIB	ChEMBL	Phase 4 (approved)	CSF1R
SORAFENIB	ChEMBL	Phase 4 (approved)	CSF1R
SUNITINIB	ChEMBL	Phase 4 (approved)	CSF1R
SUNITINIB MALATE	ChEMBL	Phase 4 (approved)	CSF1R
VANDETANIB	ChEMBL	Phase 4 (approved)	CSF1R
ALVOCIDIB	ChEMBL	Phase 3	CSF1R
BRIVANIB	ChEMBL	Phase 3	CSF1R
CEDIRANIB	ChEMBL	Phase 3	CSF1R
DACTOLISIB	ChEMBL	Phase 3	CSF1R
DOVITINIB	ChEMBL	Phase 3	CSF1R
LESTAURTINIB	ChEMBL	Phase 3	CSF1R
LINIFANIB	ChEMBL	Phase 3	CSF1R
MASITINIB	ChEMBL	Phase 3	CSF1R
MOTESANIB	ChEMBL	Phase 3	CSF1R
SEMAXANIB	ChEMBL	Phase 3	CSF1R
VATALANIB	ChEMBL	Phase 3	CSF1R
VIMSELTINIB	ChEMBL	Phase 3	CSF1R
ARRY-382	ChEMBL	Phase 2	CSF1R
BELVARAFENIB	ChEMBL	Phase 2	CSF1R
BMS-754807	ChEMBL	Phase 2	CSF1R
CC-115	ChEMBL	Phase 2	CSF1R
CENISERTIB	ChEMBL	Phase 2	CSF1R
CEP-11981	ChEMBL	Phase 2	CSF1R
CEP-32496	ChEMBL	Phase 2	CSF1R
CERDULATINIB	ChEMBL	Phase 2	CSF1R
DEFOSBARASERTIB	ChEMBL	Phase 2	CSF1R
DERAZANTINIB	ChEMBL	Phase 2	CSF1R
DORAMAPIMOD	ChEMBL	Phase 2	CSF1R
EDICOTINIB	ChEMBL	Phase 2	CSF1R
ELZOVANTINIB	ChEMBL	Phase 2	CSF1R
ENMD-2076	ChEMBL	Phase 2	CSF1R
ENRUPATINIB	ChEMBL	Phase 2	CSF1R
FORETINIB	ChEMBL	Phase 2	CSF1R
GZ-389988	ChEMBL	Phase 2	CSF1R
ILORASERTIB	ChEMBL	Phase 2	CSF1R
INIXACICLIB	ChEMBL	Phase 2	CSF1R
NARAZACICLIB	ChEMBL	Phase 2	CSF1R
NEFLAMAPIMOD	ChEMBL	Phase 2	CSF1R
ONATASERTIB	ChEMBL	Phase 2	CSF1R
PELITINIB	ChEMBL	Phase 2	CSF1R
R-406	ChEMBL	Phase 2	CSF1R
RAF-265	ChEMBL	Phase 2	CSF1R