Pexidartinib, sold under the brand name Turalio, is a kinase inhibitor drug for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.

Pexidartinib (PLX-3397) is a potent, orally active, selective, and ATP-competitive colony stimulating factor 1 receptor (CSF1R or M-CSFR) and c-Kit inhibitor, with IC50 s of 20 and 10 nM, respectively. Pexidartinib (PLX-3397) exhibits 10- to 100-fold selectivity for c-Kit and CSF1R over other related kinases.

Pexidartinib (PLX3397) is an orally available, CNS permeable, selective tyrosine kinase inhibitor that preferentially targets the macrophage colony stimulating factor-1 receptor (CSF-1R), but also inhibits closely related family members c-Kit and FLT3 [1].

Here, we investigated the effect of PLX3397 (pexidartinib) as a potent inhibitor of the CSF1 receptor (CSF1R). PLX3397 was recently approved by the Food and Drug Administration (FDA) to treat tenosynovial giant cell tumor and reprogram TAMs whose infiltration correlates with unfavorable prognosis of sarcomas.

It is a potent multi-targeted receptor tyrosine kinase inhibitor of CSF-1R, KIT, and FLT3 (IC50 of 20 nM, 10 nM and 160 nM, respectively). Approved by the FDA for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT).

A small-molecule CSF-1R inhibitor, pexidartinib (PLX3397), is the highlighted CSF-1R inhibitor in this review and casts the broadest net of clinical studies using monotherapy CSF-1R inhibition in the setting of c-KIT-mutated melanoma, prostate cancer, glioblastoma, classical Hodgkin lymphoma, neurofibroma, sarcoma, and leukemia.33 In addition ...

2019年8月21日 · We designed and synthesized a highly selective brain-penetrant CSF1R inhibitor (PLX5622) allowing for extended and specific microglial elimination, preceding and during pathology development.

PLX3397 is an oral, small molecule that selectively inhibits CSF1R and KIT, penetrates the blood-brain barrier in model systems, and represents a novel approach for clinical development. Methods: We conducted a phase II study in patients with recurrent GB.

2019年3月29日 · Colony-stimulating factor 1 (CSF1) controls macrophage differentiation, and here we sought to determine the effect of a CSF1 receptor inhibitor, PLX3397, on adipose tissue macrophage levels and...