Pexidartinib hydrochloride (PLX-3397 HCl) is a selective, orally active, ATP-competitive CSF1R/M-CSFR inhibitor and c-Kit inhibitor with IC50 values of 20 and 10 nM, respectively, capable of inducing cancer cell apoptosis.

c-Kit:10 nM, FLT1:880 nM, NTRK3:890 nM, KDR:350 nM, cFMS:20 nM, FLT3:160 nM, LCK:860 nM

Pexidartinib hydrochloride is a highly potent, selective, ATP-competitive inhibitor of CSF1R (cFMS) and c-Kit. It exhibits 10–100 times greater inhibitory selectivity for c-Kit and CSF1R compared to other related kinases (such as FLT3, KDR (VEGFR2), LCK, FLT1 (VEGFR1), and NTRK3 (TRKC)), with IC50 values of 160 nM, 350 nM, 860 nM, 880 nM, and 890 nM, respectively [1].

In the neonatal mouse model, Pexidartinib hydrochloride (0.25 and 1 mg/kg, intraperitoneal injection, twice daily for 8 days) inhibited the proliferation of microglia and BrdU-positive cells [2].
Pexidartinib hydrochloride (1 mg/kg, twice daily for 8 days) did not significantly affect the number of cleaved caspase-3-positive cells in mice [2].
Pexidartinib hydrochloride (50 mg/kg, oral administration, once every two days for 3 weeks) reduced macrophage levels in mouse tissues without interfering with Vitis vinifera glucose homeostasis [4].