Efficacy and safety of vimseltinib in tenosynovial giant cell tumour (TGCT): Phase II expansion

TGCT is a rare, locally aggressive neoplasm caused by upregulation of the colony-stimulating factor 1 (CSF1) gene, resulting in aberrant CSF1 expression and recruitment of CSF1 receptor (CSF1R)-dependent inflammatory macrophages. Vimseltinib is an oral switch control tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R. We report safety, efficacy, and preliminary patient-reported outcome data for patients (pts) with TGCT treated with the recommended phase 2 dose (RP2D; 30 mg twice weekly).