Xiaohui Niu, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, discusses a long-term analysis of the MANEUVER clinical trial of pimicotinib for the treatment of patients with tenosynovial giant cell tumor (TGCT).
TGCT is a rare condition characterized by the abnormal growth of tissue lining the joints and tendons throughout the body (synovium), resulting in a noncancerous tumor. There are two types of TGCT: the local or nodular form (where the tumor involves the tendons that support the joint, or in one area of the joint) and the diffuse form (where the entire lining of the joint is involved). Symptoms might include pain, limitation of movement, and locking of the joint. The knee is most commonly affected by this condition, though it can occur in other joints. The underlying cause of TGCT is unknown.
Pimicotinib is an orally administered, highly selective and potent small molecule inhibitor of CSF-1R. The MANEUVER clinical trial is a phase 3, three-part, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of pimicotinib in patients with TGCT who require systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. Longer-term results from the MANEUVER clinical trial were recently presented at European Society for Medical Oncology (ASMO).
The presented analysis showed that at a median follow-up of 14.3 months, the objective response rate for patients treated with pimicotinib from the beginning of the study increased to 76.2% from 54% at week 25. Additionally, objective response rate per BIRC based on tumor volume score increased to 74.6% from 61.9% at week 25. At the time of data cutoff, the median duration of response was not reached, with 93.7% of pimicotinib-treated patients experiencing a reduction in tumor size by BIRC per Response Evaluation Criteria in Solid Tumors v1.1 at longer-term follow-up.
Pimicotinib also demonstrated clinically meaningful improvements with longer-term follow-up up to week 73 in key patient-reported measures including range of motion, pain, stiffness, and physical function that significantly impacted patients living with TGCT. Additionally, patients who were initially randomized to placebo and then switched to pimicotinib in the open-label phase experienced an objective response rate of 64.5% with a median follow-up of 8.5 months after switching.
At longer-term follow-up there were no new safety concerns and no evidence of cholestatic hepatotoxicity, drug-induced liver injury, or hair/skin hypopigmentation. The majority of treatment-emergent adverse events were of mild severity and manageable.
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To learn more about TGCT and other rare cancers/tumors, visit https://checkrare.com/diseases/cancers/