Pimicotinib

Pimicotinib (ABSK021), an oral, highly potent and selective small molecule blocker of the colony-stimulating factor 1 receptor (CSF-1R) independently discovered by Abbisko Therapeutics. A number of studies have shown that blocking the CSF-1R signaling pathway could effectively modulate and change macrophage functions, and potentially treat many macrophage-dependent human diseases.^[1]

Pimicotinib

Clinical data
Other names	ABSK021
Routes of administration	Oral
Identifiers
IUPAC name 3,3-Dimethyl-N-[6-methyl-5-[2-(1-methylpyrazol-4-yl)pyridin-4-yl]oxypyridin-2-yl]-2-oxopyrrolidine-1-carboxamide
CAS Number	2253123-16-7
PubChem CID	139549388
ChemSpider	128942304
UNII	HV1XI8HST2
KEGG	D12938
Chemical and physical data
Formula	C22H24N6O3
Molar mass	420.473 g·mol−1
3D model (JSmol)	Interactive image
SMILES CC1=C(C=CC(=N1)NC(=O)N2CCC(C2=O)(C)C)OC3=CC(=NC=C3)C4=CN(N=C4)C
InChI InChI=1S/C22H24N6O3/c1-14-18(31-16-7-9-23-17(11-16)15-12-24-27(4)13-15)5-6-19(25-14)26-21(30)28-10-8-22(2,3)20(28)29/h5-7,9,11-13H,8,10H2,1-4H3,(H,25,26,30) Key:NXFPMDWYDKHFMM-UHFFFAOYSA-N

History

In December 2023, Abbisko Therapeutics entered into a licensing agreement for pimicotinib in all indications for China rights with Merck KGaA.^{[2] [3][4]}

In April 2023, a global phase III, randomized, double-blind, placebo-controlled, multicenter clinical trial designed to evaluate the safety and efficacy of pimicotinib in patients with tenosynovial giant cell tumor was started (NCT05804045).^[5]

Following with pimicotinib for tenosynovial giant cell tumor treatment in phase III, pimicotinib has also entered into a phase II trial in June 2023 for cGVHD treatment in China.^[6]

The U.S. Food and Drug Administration (FDA) and the Center for Drug Evaluation (CDE) of NMPA granted pimicotinib breakthrough therapy designation (BTD) for the treatment of tenosynovial giant cell tumor patients that are not amenable to surgery in January 2023 and July 2022, respectively.^[7]

Research

Pimicotinib is being investigated as a treatment for tenosynovial giant cell tumor,^[8][9] chronic graft-versus-host-disease (cGVHD), and pancreatic cancer.