Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context

Summary: Background: Tazemetostat, the first enhancer of zeste homolog 2 (EZH2) inhibitor approved by the U.S. Food and Drug Administration, has shown efficacy in a global population with relapsed or refractory (R/R) follicular lymphoma (FL). This phase 2 study was primarily designed as a registrat...

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Main Authors: Junning Cao, Guangliang Chen, Lihua Qiu, Liling Zhang, Ming Jiang, Ying Cheng, Qiaohua Zhang, Lihong Liu, Ping Li, Yuerong Shuang, Huaqing Wang, Hongwei Xue, Huijing Wu, Meifang Zheng, Keshu Zhou, Zhiming Li, Hongmei Jing, Wei Yang, Zunmin Zhu, Wenyu Li, Jiaxuan Wangwu, Heyu Huang, Qiantao Jia, Dongmei Chen, Songhua Fan, M. Ming Shi, Weiguo Su
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:EClinicalMedicine
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Online Access:http://www.sciencedirect.com/science/article/pii/S2589537025003311
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author Junning Cao
Guangliang Chen
Lihua Qiu
Liling Zhang
Ming Jiang
Ying Cheng
Qiaohua Zhang
Lihong Liu
Ping Li
Yuerong Shuang
Huaqing Wang
Hongwei Xue
Huijing Wu
Meifang Zheng
Keshu Zhou
Zhiming Li
Hongmei Jing
Wei Yang
Zunmin Zhu
Wenyu Li
Jiaxuan Wangwu
Heyu Huang
Qiantao Jia
Dongmei Chen
Songhua Fan
M. Ming Shi
Weiguo Su
author_facet Junning Cao
Guangliang Chen
Lihua Qiu
Liling Zhang
Ming Jiang
Ying Cheng
Qiaohua Zhang
Lihong Liu
Ping Li
Yuerong Shuang
Huaqing Wang
Hongwei Xue
Huijing Wu
Meifang Zheng
Keshu Zhou
Zhiming Li
Hongmei Jing
Wei Yang
Zunmin Zhu
Wenyu Li
Jiaxuan Wangwu
Heyu Huang
Qiantao Jia
Dongmei Chen
Songhua Fan
M. Ming Shi
Weiguo Su
author_sort Junning Cao
collection DOAJ
description Summary: Background: Tazemetostat, the first enhancer of zeste homolog 2 (EZH2) inhibitor approved by the U.S. Food and Drug Administration, has shown efficacy in a global population with relapsed or refractory (R/R) follicular lymphoma (FL). This phase 2 study was primarily designed as a registration-intent bridging trial of tazemetostat in Chinese patients with EZH2-mutant (EZH2mut) R/R FL; the study also included evaluation in the EZH2 wild-type (EZH2wt) population. Methods: This multicentre, single-arm, phase 2 study was conducted at 19 sites in China. Eligible patients (aged ≥18 years) with histologically confirmed grade 1–3a FL were categorised by EZH2 status: mutant (EZH2mut, bridging cohort) and wild-type (EZH2wt), and received oral tazemetostat 800 mg twice daily in continuous 28-day cycles until investigator-assessed disease progression, intolerable toxicity, withdrawal of consent, or other protocol-specified criteria. The EZH2mut cohort primary endpoint was independent review committee (IRC)-assessed objective response rate (ORR). Efficacy in the EZH2mut cohort was assessed in patients who received at least one dose of tazemetostat and had centrally confirmed FL. Efficacy in the EZH2wt cohort and safety in both cohorts were assessed in all patients who received at least one dose of tazemetostat. This study is registered with ClinicalTrials.gov, NCT05467943. Findings: Between July 29, 2022, and Aug 31, 2023, 22 patients with EZH2mut R/R FL were enrolled (three lines [3L] of prior therapy, 31.8%; four lines [4L] of prior therapy or more, 27.3%). As of Aug 31, 2024 (median follow-up 14.4 months), IRC-assessed ORR was 63.6% (95% confidence interval [CI], 40.7%–82.8%), clinical benefit rate (CBR) was 90.9% (70.8%–98.9%), median duration of response (DoR) was not reached (18-month DoR rate, 51.6% [18.2%–77.3%]), and median progression-free survival was 15.4 (8.2–not estimable) months. All patients experienced treatment-related adverse events (TRAEs), and 13.6% had grade ≥3 TRAEs; most common TRAEs were haematological toxicities. For the EZH2wt cohort, 20 patients were enrolled (3L, 35.0%; ≥4L, 45.0%). Tazemetostat also demonstrated efficacy benefit and a manageable safety profile in the EZH2wt cohort, with an investigator-assessed ORR of 35.0% (95% CI, 15.4%–59.2%), CBR of 85.0% (62.1%–96.8%), and median DoR of 8.4 (1.9–15.7) months. Interpretation: These results suggest that tazemetostat has a clinically meaningful efficacy and was well tolerated in Chinese patients with R/R FL. Larger trials are warranted. A multicentre, randomised, phase 3 trial of tazemetostat combined with lenalidomide plus rituximab vs. lenalidomide plus rituximab in patients with R/R FL is ongoing (NCT04224493). Funding: HUTCHMED.
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spelling doaj-art-ecd3bf49b60e4438906ec2a25636e6d62025-08-20T03:07:12ZengElsevierEClinicalMedicine2589-53702025-09-018710339910.1016/j.eclinm.2025.103399Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in contextJunning Cao0Guangliang Chen1Lihua Qiu2Liling Zhang3Ming Jiang4Ying Cheng5Qiaohua Zhang6Lihong Liu7Ping Li8Yuerong Shuang9Huaqing Wang10Hongwei Xue11Huijing Wu12Meifang Zheng13Keshu Zhou14Zhiming Li15Hongmei Jing16Wei Yang17Zunmin Zhu18Wenyu Li19Jiaxuan Wangwu20Heyu Huang21Qiantao Jia22Dongmei Chen23Songhua Fan24M. Ming Shi25Weiguo Su26Department of Medical Oncology, Fudan University Shanghai Cancer Center, Shanghai Medical College, Fudan University, Shanghai, China; Corresponding author. Department of Medical Oncology, Fudan University Shanghai Cancer Center, Shanghai Medical College, Fudan University, Shanghai, 200032, China.Department of Medical Oncology, Fudan University Shanghai Cancer Center, Shanghai Medical College, Fudan University, Shanghai, ChinaDepartment of Lymphoma, Tianjin Medical University Cancer Hospital, National Clinical Research Center for Cancer, Tianjin Key Laboratory of Cancer Prevention and Therapy, Sino-US Center for Lymphoma Diagnosis and Treatment, Tianjin, ChinaCancer Center, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, ChinaDepartment of Oncology, West China Hospital of Sichuan University, Chengdu, ChinaDepartment of Oncology, Jilin Cancer Hospital, Changchun, ChinaDepartment of Oncology, Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital, Shanxi, ChinaDepartment of Hematology, The Fourth Hospital of Hebei Medical University, Shijiazhuang, ChinaDepartment of Hematology, Tongji Hospital of Tongji University, Tongji University School of Medicine, Shanghai, ChinaDepartment of Lymphoma Oncology, Jiangxi Cancer Hospital, Nanchang, ChinaDepartment of Oncology, Tianjin Union Medical Center, Nankai University, Tianjin, China; The Institute of Translational Medicine, Tianjin Union Medical Center, Nankai University, Tianjin, China; Tianjin Cancer Institute of Integrative Traditional Chinese and Western Medicine, Tianjin, ChinaDepartment of Oncology, The Affiliated Hospital of Qingdao University, Qingdao, ChinaDepartment of Lymphoma Medicine, Hubei Cancer Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, ChinaDepartment of Hematology, Linyi Cancer Hospital, Linyi, ChinaDepartment of Hematology, The Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, ChinaDepartment of Medical Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaDepartment of Hematology, Lymphoma Research Center, Peking University Third Hospital, Beijing, ChinaDepartment of Hematology, Shengjing Hospital of China Medical University, Shenyang, ChinaInstitute of Hematology, Henan Provincial People's Hospital, Zhengzhou, ChinaDepartment of Lymphoma, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Southern Medical University, Guangzhou, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaHUTCHMED, Shanghai, ChinaSummary: Background: Tazemetostat, the first enhancer of zeste homolog 2 (EZH2) inhibitor approved by the U.S. Food and Drug Administration, has shown efficacy in a global population with relapsed or refractory (R/R) follicular lymphoma (FL). This phase 2 study was primarily designed as a registration-intent bridging trial of tazemetostat in Chinese patients with EZH2-mutant (EZH2mut) R/R FL; the study also included evaluation in the EZH2 wild-type (EZH2wt) population. Methods: This multicentre, single-arm, phase 2 study was conducted at 19 sites in China. Eligible patients (aged ≥18 years) with histologically confirmed grade 1–3a FL were categorised by EZH2 status: mutant (EZH2mut, bridging cohort) and wild-type (EZH2wt), and received oral tazemetostat 800 mg twice daily in continuous 28-day cycles until investigator-assessed disease progression, intolerable toxicity, withdrawal of consent, or other protocol-specified criteria. The EZH2mut cohort primary endpoint was independent review committee (IRC)-assessed objective response rate (ORR). Efficacy in the EZH2mut cohort was assessed in patients who received at least one dose of tazemetostat and had centrally confirmed FL. Efficacy in the EZH2wt cohort and safety in both cohorts were assessed in all patients who received at least one dose of tazemetostat. This study is registered with ClinicalTrials.gov, NCT05467943. Findings: Between July 29, 2022, and Aug 31, 2023, 22 patients with EZH2mut R/R FL were enrolled (three lines [3L] of prior therapy, 31.8%; four lines [4L] of prior therapy or more, 27.3%). As of Aug 31, 2024 (median follow-up 14.4 months), IRC-assessed ORR was 63.6% (95% confidence interval [CI], 40.7%–82.8%), clinical benefit rate (CBR) was 90.9% (70.8%–98.9%), median duration of response (DoR) was not reached (18-month DoR rate, 51.6% [18.2%–77.3%]), and median progression-free survival was 15.4 (8.2–not estimable) months. All patients experienced treatment-related adverse events (TRAEs), and 13.6% had grade ≥3 TRAEs; most common TRAEs were haematological toxicities. For the EZH2wt cohort, 20 patients were enrolled (3L, 35.0%; ≥4L, 45.0%). Tazemetostat also demonstrated efficacy benefit and a manageable safety profile in the EZH2wt cohort, with an investigator-assessed ORR of 35.0% (95% CI, 15.4%–59.2%), CBR of 85.0% (62.1%–96.8%), and median DoR of 8.4 (1.9–15.7) months. Interpretation: These results suggest that tazemetostat has a clinically meaningful efficacy and was well tolerated in Chinese patients with R/R FL. Larger trials are warranted. A multicentre, randomised, phase 3 trial of tazemetostat combined with lenalidomide plus rituximab vs. lenalidomide plus rituximab in patients with R/R FL is ongoing (NCT04224493). Funding: HUTCHMED.http://www.sciencedirect.com/science/article/pii/S2589537025003311Follicular lymphomaEZH2 inhibitorBridging studyTazemetostatChinese
spellingShingle Junning Cao
Guangliang Chen
Lihua Qiu
Liling Zhang
Ming Jiang
Ying Cheng
Qiaohua Zhang
Lihong Liu
Ping Li
Yuerong Shuang
Huaqing Wang
Hongwei Xue
Huijing Wu
Meifang Zheng
Keshu Zhou
Zhiming Li
Hongmei Jing
Wei Yang
Zunmin Zhu
Wenyu Li
Jiaxuan Wangwu
Heyu Huang
Qiantao Jia
Dongmei Chen
Songhua Fan
M. Ming Shi
Weiguo Su
Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
EClinicalMedicine
Follicular lymphoma
EZH2 inhibitor
Bridging study
Tazemetostat
Chinese
title Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
title_full Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
title_fullStr Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
title_full_unstemmed Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
title_short Efficacy and safety of tazemetostat, an EZH2 inhibitor, in Chinese patients with relapsed/refractory follicular lymphoma: a multicentre, single-arm, phase 2 studyResearch in context
title_sort efficacy and safety of tazemetostat an ezh2 inhibitor in chinese patients with relapsed refractory follicular lymphoma a multicentre single arm phase 2 studyresearch in context
topic Follicular lymphoma
EZH2 inhibitor
Bridging study
Tazemetostat
Chinese
url http://www.sciencedirect.com/science/article/pii/S2589537025003311
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