和誉医药在《Future Oncology》杂志上全文发表了匹米替尼的全球III期临床研究设计

MANEUVER研究是一项由和誉医药发起的III期全球多中心临床试验，随机、安慰剂对照、双盲设计，分为三个阶段。

第一阶段，符合条件的受试者将按照2:1的比例随机分配，每日一次接受50mg的匹米替尼或安慰剂治疗，持续24周。第二阶段为开放标签治疗，所有受试者将接受24周的匹米替尼治疗，直至完成或退出研究。完成第二阶段的参与者可选择进入第三阶段，即开放标签的延长治疗期，以接受更长时间的治疗。此外，在第一阶段结束后，研究人员将邀请参与者进行离组访谈，以反映患者针对临床疗效的直观评价。

MANEUVER研究是首个实现亚洲和欧美TGCT患者均衡招募的国际多中心研究，覆盖中国、北美和欧洲的多个国家。这种广域代表性使得研究结果能够针对中国和中国以外地区分层进行详细比较，有助于深入理解疾病特征及不同人群中治疗反应的潜在差异。I期开放标签临床研究数据证明，匹米替尼可以有效缩小肿瘤体积，降低关节疼痛及僵硬程度，改善关节活动度，其有望成为TGCT患者更有效，更安全的治疗选择。

此前，匹米替尼于2022年10月获中国CDE临床III期批准，2023年3月获美国FDA临床III期批准，2023年9月获欧洲EMA临床III期批准。并于2024年3月29日，完成该项研究的全部患者入组。此项研究也是腱鞘巨细胞瘤疾病领域首个在中国、美国、加拿大和欧洲同步开展的全球III期研究。匹米替尼已获得中美欧三地突破性治疗药物和优先药物认定，以及美国快速通道认定，并于2024年1月获得欧洲孤儿药资格认定，用于治疗不可手术的腱鞘巨细胞瘤。

《Future Oncology》是一本混合型开放获取的学术期刊，收录于科学引文索引扩展版（SCIE）Q2区，影响因子3.0。该期刊经同行评议，专注于肿瘤治疗领域的最新进展，提供个性化医疗见解及其临床应用价值。期刊内容涵盖肿瘤创新疗法、进行中研究的方案、免疫治疗和放射治疗的最新动态，并且包括了医疗政策的讨论和真实世界研究的成果。

Abbisko Therapeutics has published Phase III study design of Pimicotinib in Future Oncology

MANEUVER is a Phase III, randomized, placebo-controlled, double-blind, multi-region study sponsored by Abbisko. 

This study consists of three parts. In Part 1, eligible participants were randomized in a 2:1 ratio to receive either pimicotinib 50 mg QD or matching placebo for 24 weeks (28-day cycles). Part 2 is an open-label treatment phase and all participants entering this phase will receive open-label pimicotinib until completion of 24 weeks of dosing or withdrawal from the study. Participants who complete Part 2 are given the option to enter the open-label extension treatment phase, namely Part 3, which facilitates prolonged treatment. In addition, upon the completion of Part 1, patients will be invited by the investigator to participate in an optional qualitative interview to reflect the patient's assessment of clinical efficacy. 

The MANEUVER study is the first global trial to enroll both Asian and Western patients with TGCT in balanced proportions across multiple regions including China, North America and Europe. This robust representation allows for detailed outcome comparisons by stratification factor – China sites versus non-China sites – thereby facilitating a deeper understanding of disease characteristics and potential variations in response across different populations. Data from Phase I open-label study has demonstrated Pimicotinib’s efficacy in effectively reducing tumor size, alleviating joint pain and stiffness, and improving joint mobility. If approved, it would provide an additional treatment option for patients with TGCT.

The approval to conduct this Phase III trial was received by the China NMPA in October 2022, the U.S. FDA in March 2023, and the EMA in September 2023. The completion of patient enrollment for this study by March 29 2024. It is the first global Phase III trial of TGCT simultaneously conducted in China, the U.S., Canada and Europe. Pimicotinib has obtained the Breakthrough Therapy Designation (BTD) from China NMPA and the U.S. FDA, as well as the PRIME designation from EMA. Furthermore, the U.S. FDA granted Fast Track Designation (FTD), and Orphan Drug Designation (ODD) granted by EMA in January 2024, for the treatment of TGCT patients who are not amenable to surgery.

Future Oncology is a hybrid open-access journal with a JCR Quartile ranking of Q2 and an impact factor of 3.0. It is a Science Citation Index Expanded (SCIE) -indexed, peer-reviewed publication that provides insights into cancer treatments, personalized strategies, and their clinical implications. The journal offers analyses on topics such as new anticancer agents, ongoing study protocols, immunotherapy, and advancements in radiotherapy, while also addressing policy debates and real-world outcomes research.