Merck KGaA's Phase III MANEUVER trial of pimicotinib, an investigational drug developed by Abbisko Therapeutics Co., Ltd., has met its primary endpoint, demonstrating a significant improvement in objective response rate (ORR) in patients with tenosynovial giant cell tumor (TGCT). The study's findings offer a potential new treatment option for this rare tumor.
The MANEUVER trial revealed that pimicotinib achieved an ORR of 54.0% at week 25, compared to a mere 3.2% in the placebo group. This statistically significant difference underscores the potential of pimicotinib as a targeted therapy for TGCT.
Secondary Endpoint Improvements
Beyond the primary endpoint, the trial also assessed several secondary endpoints related to patient-reported outcomes. Results indicated that pimicotinib treatment led to statistically significant and clinically meaningful improvements in stiffness, as measured by the Numeric Rating Scale, and pain, assessed using the Brief Pain Inventory. These improvements suggest a comprehensive benefit of pimicotinib in managing TGCT-related symptoms.
Safety and Tolerability
In terms of safety, pimicotinib was well-tolerated by patients in the MANEUVER trial. The observed safety profile was consistent with previously reported data, and notably, there was no evidence of cholestatic hepatotoxicity, a potential liver-related adverse effect.
About Tenosynovial Giant Cell Tumor (TGCT)
Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm that affects the synovium of joints, tendon sheaths, and bursae. Current treatment options are limited, highlighting the need for more effective therapies. The positive results from the MANEUVER trial suggest that pimicotinib could potentially fill this unmet need.
