Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context

Summary: Background: MET exon14 skipping mutations (METex14) is an established actionable driver oncogene of non-small-cell lung cancer (NSCLC). While ensartinib is a known second-generation tyrosine kinase inhibitor with primary activity against ALK translocation, it is also classified as a type I...

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Main Authors: Yang Xia, Panwen Tian, Mo Zhou, Jun Zhao, Yang Jin, Zhiyuan Guo, Xiuzhen Li, Weina Lu, Da Miao, Yuefei Lu, Wanting Xu, Yongchang Zhang, Xiuning Le, Wen Li
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:EClinicalMedicine
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Online Access:http://www.sciencedirect.com/science/article/pii/S2589537025000318
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author Yang Xia
Panwen Tian
Mo Zhou
Jun Zhao
Yang Jin
Zhiyuan Guo
Xiuzhen Li
Weina Lu
Da Miao
Yuefei Lu
Wanting Xu
Yongchang Zhang
Xiuning Le
Wen Li
author_facet Yang Xia
Panwen Tian
Mo Zhou
Jun Zhao
Yang Jin
Zhiyuan Guo
Xiuzhen Li
Weina Lu
Da Miao
Yuefei Lu
Wanting Xu
Yongchang Zhang
Xiuning Le
Wen Li
author_sort Yang Xia
collection DOAJ
description Summary: Background: MET exon14 skipping mutations (METex14) is an established actionable driver oncogene of non-small-cell lung cancer (NSCLC). While ensartinib is a known second-generation tyrosine kinase inhibitor with primary activity against ALK translocation, it is also classified as a type Ia MET inhibitor. We have previously shown anti-tumor activity against METex14 positive NSCLC both in vivo and in vitro. The EMBRACE trial aims to evaluate the clinical efficacy and safety of ensartinib for treatment of METex14 positive NSCLC. Methods: This is a multicenter single arm phase II investigator-initiated study that enrolled METex14 positive lung cancer after failing first line chemotherapy and/or immunotherapy. Eligible patients received ensartinib 225 mg orally once daily in a continuous 28-day treatment cycle until disease progression, unacceptable side effect, or death. Primary endpoint was investigator-assessed objective response rate (ORR), and the secondary end point included disease control rate (DCR), progression-free survival (PFS), duration of response (DoR) and safety profiles. The study was registered with the Chinese Clinical Trial Registry (ChiCTR2100048767). Findings: From July 2021 to February 2024, a total of 31 patients were enrolled and received ensartinib. Median follow-up time of the 30 evaluable patients was 9.2 months (95% Confidence Interval [CI], 6.3-not estimable). The ORR was 53.3% (16/30; 95% CI, 35.5–71.2) and DCR was 86.7% (26/30; 95% CI, 74.5–98.8). Median PFS was 6.0 months (95% CI, 3.0–8.8) and median DoR was 7.9 months (95% CI, 4.8–8.7). Adverse events (AEs) were reported in 24 patients (80%), with 7 (23.3%) of grade 3. The most common AEs were rash (14/30, 46.7%), followed by anemia (7/30, 23.3%), increased ALT (7/30, 23.3%), increased AST (7/30, 23.3%), and pruritus (6/30, 20%). No serious adverse events or treatment-related deaths occurred. Importantly, the exploratory ctDNA analysis indicates that clearance of circulating tumor DNA (ctDNA) at four weeks treatment was associated with more favorable treatment outcomes comparing with patients having positive ctDNA. Interpretation: Ensartinib has a promising anti-tumor activity and manageable safety in previously treated patients with METex14 positive lung cancer. Funding: This work was supported by the National Natural Science Foundation of China [82370028, 82422001] and the CSCO-MET Aberrant Solid Tumor Research Grant [Y-2022METAZMS-0066].
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spelling doaj-art-3aca6c0e7dea4c9ebc049bdac5856b132025-08-20T03:11:38ZengElsevierEClinicalMedicine2589-53702025-03-018110309910.1016/j.eclinm.2025.103099Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in contextYang Xia0Panwen Tian1Mo Zhou2Jun Zhao3Yang Jin4Zhiyuan Guo5Xiuzhen Li6Weina Lu7Da Miao8Yuefei Lu9Wanting Xu10Yongchang Zhang11Xiuning Le12Wen Li13Key Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China; Cancer Center, Zhejiang University, Hangzhou, Zhejiang, China; Corresponding author. Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, 310052, China.Department of Pulmonary and Critical Care Medicine, Lung Cancer Center/Lung Cancer Institute, West China Hospital of Sichuan University, Chengdu, Sichuan, ChinaKey Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China; Cancer Center, Zhejiang University, Hangzhou, Zhejiang, ChinaDepartment of Thoracic Medical Oncology, Peking University Cancer Hospital & Institute, Beijing, ChinaHubei Province Key Laboratory of Biological Targeted Therapy, Department of Respiratory and Critical Care Medicine, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, ChinaSecond Oncology Department, Handan Central Hospital, Handan, Hebei, ChinaDepartment of Pathology, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, ChinaKey Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, ChinaKey Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, ChinaKey Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, ChinaKey Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, ChinaDepartment of Medical Oncology, Lung Cancer and Gastrointestinal Unit, Hunan Cancer Hospital/The Affiliated Cancer Hospital of Xiangya School of Medicine, Central South University, Changsha, Hunan, ChinaDepartment of Thoracic/Head and Neck Medical Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, USA; Corresponding author. Department of Thoracic/Head and Neck Medical Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, 77030, USA.Key Laboratory of Respiratory Disease of Zhejiang Province, Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China; Cancer Center, Zhejiang University, Hangzhou, Zhejiang, China; Corresponding author. Department of Respiratory and Critical Care Medicine, Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, 310052, China.Summary: Background: MET exon14 skipping mutations (METex14) is an established actionable driver oncogene of non-small-cell lung cancer (NSCLC). While ensartinib is a known second-generation tyrosine kinase inhibitor with primary activity against ALK translocation, it is also classified as a type Ia MET inhibitor. We have previously shown anti-tumor activity against METex14 positive NSCLC both in vivo and in vitro. The EMBRACE trial aims to evaluate the clinical efficacy and safety of ensartinib for treatment of METex14 positive NSCLC. Methods: This is a multicenter single arm phase II investigator-initiated study that enrolled METex14 positive lung cancer after failing first line chemotherapy and/or immunotherapy. Eligible patients received ensartinib 225 mg orally once daily in a continuous 28-day treatment cycle until disease progression, unacceptable side effect, or death. Primary endpoint was investigator-assessed objective response rate (ORR), and the secondary end point included disease control rate (DCR), progression-free survival (PFS), duration of response (DoR) and safety profiles. The study was registered with the Chinese Clinical Trial Registry (ChiCTR2100048767). Findings: From July 2021 to February 2024, a total of 31 patients were enrolled and received ensartinib. Median follow-up time of the 30 evaluable patients was 9.2 months (95% Confidence Interval [CI], 6.3-not estimable). The ORR was 53.3% (16/30; 95% CI, 35.5–71.2) and DCR was 86.7% (26/30; 95% CI, 74.5–98.8). Median PFS was 6.0 months (95% CI, 3.0–8.8) and median DoR was 7.9 months (95% CI, 4.8–8.7). Adverse events (AEs) were reported in 24 patients (80%), with 7 (23.3%) of grade 3. The most common AEs were rash (14/30, 46.7%), followed by anemia (7/30, 23.3%), increased ALT (7/30, 23.3%), increased AST (7/30, 23.3%), and pruritus (6/30, 20%). No serious adverse events or treatment-related deaths occurred. Importantly, the exploratory ctDNA analysis indicates that clearance of circulating tumor DNA (ctDNA) at four weeks treatment was associated with more favorable treatment outcomes comparing with patients having positive ctDNA. Interpretation: Ensartinib has a promising anti-tumor activity and manageable safety in previously treated patients with METex14 positive lung cancer. Funding: This work was supported by the National Natural Science Foundation of China [82370028, 82422001] and the CSCO-MET Aberrant Solid Tumor Research Grant [Y-2022METAZMS-0066].http://www.sciencedirect.com/science/article/pii/S2589537025000318EnsartinibNSCLCMET exon 14 skipping mutation
spellingShingle Yang Xia
Panwen Tian
Mo Zhou
Jun Zhao
Yang Jin
Zhiyuan Guo
Xiuzhen Li
Weina Lu
Da Miao
Yuefei Lu
Wanting Xu
Yongchang Zhang
Xiuning Le
Wen Li
Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
EClinicalMedicine
Ensartinib
NSCLC
MET exon 14 skipping mutation
title Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
title_full Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
title_fullStr Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
title_full_unstemmed Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
title_short Ensartinib for advanced or metastatic non-small-cell lung cancer with MET exon 14 skipping mutations (EMBRACE): a multi-center, single-arm, phase 2 trialResearch in context
title_sort ensartinib for advanced or metastatic non small cell lung cancer with met exon 14 skipping mutations embrace a multi center single arm phase 2 trialresearch in context
topic Ensartinib
NSCLC
MET exon 14 skipping mutation
url http://www.sciencedirect.com/science/article/pii/S2589537025000318
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