Multinational phase 3 trial of vimseltinib versus placebo for tenosynovial giant cell tumor

Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm for which few systemic treatment options exist. The multinational phase 3 MOTION study randomized patients with symptomatic TGCT not amenable to surgery 2:1 to vimseltinib (an oral switch control CSF1R inhibitor) or placebo for 24 weeks. The primary end point was objective response rate. The objective response rate was 40% in the vimseltinib group versus 0% in the placebo group (p<0.0001). Most treatment-emergent adverse events were grade 1 or 2. Vimseltinib resulted in clinically meaningful functional and symptomatic improvements. The authors conclude that vimseltinib could be an effective treatment option for TGCT patients.