China's Abbisko Therapeutics has reported extended follow-up data from the Phase III MANEUVER trial, showing 76.2% objective response rate (ORR) at median 14.3 months for CSF-1R inhibitor pimicotinib in tenosynovial giant cell tumour (TGCT), with median duration of response not reached. The oral small molecule demonstrated sustained clinical outcome assessment improvements including 23.9% joint range of motion enhancement at 73 weeks, with a safety profile consistent with previous analyses and supporting long-term treatment feasibility. Cross-over patients achieving 64.5% response rate after switching from placebo further validated the therapeutic benefit in this rare proliferative disorder affecting synovial joints and tendon sheaths.

Pimicotinib holds breakthrough therapy designation (BTD) in the US and priority medicines status from the European Medicines Agency, with China National Medical Products Administration (NMPA) priority review underway for systemic treatment of tenosynovial giant cell tumour. Abbisko partnered global commercialisation rights with Merck in December 2023 following a positive top-line results announcement. The CSF-1R inhibitor addresses pathological CSF-1 overexpression driving inflammatory cell accumulation, representing a first-in-class mechanism for non-malignant tumour management with potential applications in oncology and inflammatory diseases.

According to PharmCube's NextBiopharm® database, pimicotinib ranks second globally in terms of development among CSF-1R inhibitors. Click here to request a free trial for NextBiopharm®.