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Soft Tissue Tumors

(P 407) PIMICOTINIB IN TENOSYNOVIAL GIANT CELL TUMOR (TGCT): LONG-TERM EFFICACY, SAFETY, AND PATIENT-REPORTED OUTCOMES FROM THE PHASE 3 MANEUVER STUDY

    Author(s)

  • Vinod Ravi, MD

    Professor, Department of Sarcoma Medical Oncology, Division of Cancer Medicine
    The University of Texas MD Anderson Cancer Center
    Houston, Texas, United States

    • Co-Author(s)

  • Hans Gelderblom, MD, PhD

    Professor medical oncology
    Leiden University Medical Center
    Leiden, Zuid-Holland, Netherlands

  • Javier Martín-Broto, MD, PhD

    Medical Oncologist
    Fundación Jiménez Díaz University Hospital
    Madrid, Madrid, Spain

  • YZ

    Yong Zhou, N/A, MD

    West China Hospital, Sichuan University
    Chengdu, Sichuan, China (People's Republic)

  • AR

    Albiruni R A Razak, MBBCh, MRCP

    Princess Margaret Cancer Center
    Toronto, Ontario, Canada

  • RS

    Ramy Saleh, MD

    Medical Director
    Cedars Cancer Center, McGill University Health Center
    Montreal, Quebec, Canada

  • Jingnan Shen, PhD

    leader
    Musculoskeletal Oncology, 1st Affiliated Hospital of Sun Yat-sen University
    Guangzhou, Guangdong, China (People's Republic)

  • TL

    Tang Liu, MD, PhD

    The Second Xiangya Hospital of Central South University
    Changsha, Hunan, China (People's Republic)

  • Silvia Stacchiotti, MD

    Medical Oncologist
    Fondazione IRCCS Istituto Nazionale dei Tumori
    Milan, Lombardia, Italy

  • KS

    Kamlesh K. Sankhala, MD

    Medical Oncologist, Cedars Sinai Medical Center Director, Precision NextGen Oncology and Research Center
    Precision NextGen Oncology and Research Center
    Los Angeles, California, United States

  • César Serrano, MD, PhD

    Head
    Sarcoma Translational Research Program, Hospital Universitari Vall d'Hebron – Vall d'Hebron Institut d'Oncologia
    Barcelona, Catalonia, Spain

  • JW

    Jing Wang, MD, PhD

    Hunan Provincial People's Hospital
    Changsha, Hunan, China (People's Republic)

  • Yingqi Hua, MD, PhD

    Professor
    Department of Orthopedic Oncology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
    Shanghai, Shanghai, China (People's Republic)

  • Piotr Rutkowski, MD, PhD

    prof. dr hab. n. med.
    Maria Sklodowska-Curie National Research Institute of Oncology
    warsaw, Mazowieckie, Poland

  • XZ

    Xiaojing Zhang, PhD

    Liaoning Cancer Hospital & Institute
    Shenyang, Liaoning, China (People's Republic)

  • YF

    Yi Feng, MD, PhD

    The Second Hospital of Shanxi Medical University
    Taiyuan, Shaanxi, China (People's Republic)

  • TL

    Tao Li, MD, PhD

    Zhejiang Cancer Hospital
    Hangzhou, Zhejiang, China (People's Republic)

  • Giacomo Giulio G. Baldi, MD

    Medical Oncologist
    Department of Oncology, Hospital of Prato, Azienda USL Toscana Centro
    Prato, Toscana, Italy

  • Hairong Xu, MD

    Associate Chief Physician/Associate professor
    Department of Orthopaedic Oncology Surgery, Beijing Jishuitan Hospital, Peking University
    Beijing, Beijing, China (People's Republic)

  • Xiaohui Niu, MD

    Chief physician
    Department of Orthopaedic Oncology Surgery, Beijing Jishuitan Hospital, Peking University
    Beijing, Beijing, China (People's Republic)

Objective: TGCT is a rare, locally aggressive and often debilitating synovial tumor driven by overproduction of colony-stimulating factor-1 (CSF-1), highlighting the unmet need for effective systemic therapies. Pimicotinib (pimi) is an oral, highly selective, potent CSF-1 receptor inhibitor. MANEUVER (NCT05804045) is the first global Phase 3, randomized, placebo (pbo)-controlled trial evaluating efficacy and safety of pimi in patients (pts) with TGCT from Asia, Europe, and North America. The primary endpoint (objective response rate [ORR] by blinded independent review committee [BIRC] per RECIST v1.1) and all key secondary endpoints were met in Part 1 at Week 25 (Niu et al. ASCO 2025).

Methods: In Part 1 (double-blind), pts received pimi 50 mg once daily or pbo (2:1) for 24 weeks; at Week 25, pts continued or switched to pimi open-label for a further 24 weeks (Part 2), followed by an open-label extension phase. This analysis reports the extended efficacy (including clinical outcome assessments [COAs]) and safety outcomes at the end of Part 2.

Results: There were 94 pts randomized (pimi = 63; pbo = 31). Median age was 40 years (range: 18–69); 68% were female; 50% had disease in the knee. At the final analysis data cutoff (Mar 12, 2025; median follow-up: 62 weeks), when all pts had completed Part 2, ORR in pts randomized to pimi was 76.2% (95% confidence interval [CI] 63.8, 86.0) per RECIST v1.1 and 74.6% (95% CI 62.1, 84.7) per tumor volume score; all COAs, including EuroQol visual analog scale, had ongoing improvement (Table). Pts switching from pbo to pimi in Part 2 derived ORR and COA benefits. No new safety signals, or evidence of cholestatic hepatotoxicity or drug-induced liver injury, emerged. Treatment-emergent adverse events led to discontinuations in 4 (6.3%) pts randomized to pimi and 2 (6.5%) pts randomized to pbo who switched to pimi.

Conclusion: Sustained treatment with pimi in pts with TGCT demonstrated continued improvements in efficacy, enhanced patient outcomes, and an acceptable safety profile, reinforcing its potential for long-term use in eligible pts.

Clinical Outcome Assessments
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