Abstract
TGCT is a rare, locally aggressive neoplasm caused by upregulation of the colony-stimulating factor 1 (CSF1) gene, resulting in aberrant CSF1 expression and the 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 long-term safety and efficacy for patients (pts) with TGCT from the phase I arm (dose escalation) of the phase I/II study.
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