Pimicotinib Demonstrates Durable Response Among Patients With Tenosynovial Giant Cell Tumor

According to extended analysis from the phase 3 MANEUVER trial, there was continued improvement in tumor response with pimicotinib, with ongoing improvements to clinical outcome assessments, for patients with tenosynovial giant cell tumor.

These data were first presented by Xiaohui Niu, MD, Beijing Jishuitan Hospital, Beijing, China, at the 2025 European Society for Clinical Oncology (ESMO) Annual Congress in Berlin, Germany.

In the phase 3 MANEUVER trial, patients were randomized on a 2-to-1 basis to receive either pimicotinib or placebo. In part 1, patients received either 50 mg pimicotinib once daily or placebo for 24 weeks. In part 2, patients either continued or switched to pimicotinib for 24 weeks, followed by part 3, an extension phase. As previously reported, the primary end point was met, with a significant improvement to objective response rate with pimicotinib at week 25 compared to placebo (54.0% vs 3.2%; P < .0001). In this analysis, extended efficacy, including clinical outcome assessments evaluated at the end of part 2) and safety outcomes are reported.

With a median follow-up of 62 weeks, those patients who were randomized to the pimicotinib arm in part 1 (n = 63) had an objective response rate of 76.2% per RECIST v1.1 and 74.6% per tumor volume score. The median duration of response was not reached. After Part 2, there were ongoing improvements seen in all clinical outcome assessments (change from baseline to end of part 2), including active range of motion, worst stiffness numeric rating scale, brief pain inventory, and PROMIS-PF (Patient-Reported Outcomes Measurement Information System-Physical Function). 

There were no new safety signals observed, with no evidence of cholestatic hepatotoxicity or drug-induced liver injury, and no hair/skin hypopigmentation. Due to treatment-emergent adverse events, 6.3% of patients discontinued pimicotinib. It is noted that patients who switched from placebo to pimicotinib in part 2 also saw a benefit to objective response rate and clinical outcome assessments.

Dr Niu et al concluded that these data support “the timely initiation and long-term treatment of eligible patients with pimicotinib.”

Source:

Niu X, Gelderblom H, Ravi V, et al. Extended efficacy and safety from the phase III MANEUVER trial of pimicotinib in patients with tenosynovial giant cell tumor (TGCT). Presented at the 2025 ESMO Congress. October 17-21, 2025; Berlin, Germany. Abstract 2690MO.