Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies

Abstract Background Sacituzumab tirumotecan (sac-TMT) is an antibody–drug conjugate composed of an anti-TROP2 monoclonal antibody coupled to a cytotoxic belotecan-derived topoisomerase I inhibitor (KL610023) via a novel linker. We report results from the phase 1 dose-escalation cohorts in advanced s...

Full description

Saved in:
Bibliographic Details
Main Authors: Quchang Ouyang, Jordi Rodon, Yan Liang, Xinhong Wu, Qun Li, Lihua Song, Min Yan, Zhongsheng Tong, YunPeng Liu, Zev A. Wainberg, Ying Wang, Cuizhi Geng, Susanna V. Ulahannan, Guohua Yu, Manish R. Sharma, Xiang Wang, Judy S. Wang, Alexander Spira, Weihong Zhao, Rachel E. Sanborn, Ying Cheng, Xian Wang, Gesha Liu, Yaling Li, Junyou Ge, Elliot Chartash, Omobolaji O. Akala, Yongmei Yin
Format: Article
Language:English
Published: BMC 2025-06-01
Series:Journal of Hematology & Oncology
Subjects:
Online Access:https://doi.org/10.1186/s13045-025-01705-2
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849434263465754624
author Quchang Ouyang
Jordi Rodon
Yan Liang
Xinhong Wu
Qun Li
Lihua Song
Min Yan
Zhongsheng Tong
YunPeng Liu
Zev A. Wainberg
Ying Wang
Cuizhi Geng
Susanna V. Ulahannan
Guohua Yu
Manish R. Sharma
Xiang Wang
Judy S. Wang
Alexander Spira
Weihong Zhao
Rachel E. Sanborn
Ying Cheng
Xian Wang
Gesha Liu
Yaling Li
Junyou Ge
Elliot Chartash
Omobolaji O. Akala
Yongmei Yin
author_facet Quchang Ouyang
Jordi Rodon
Yan Liang
Xinhong Wu
Qun Li
Lihua Song
Min Yan
Zhongsheng Tong
YunPeng Liu
Zev A. Wainberg
Ying Wang
Cuizhi Geng
Susanna V. Ulahannan
Guohua Yu
Manish R. Sharma
Xiang Wang
Judy S. Wang
Alexander Spira
Weihong Zhao
Rachel E. Sanborn
Ying Cheng
Xian Wang
Gesha Liu
Yaling Li
Junyou Ge
Elliot Chartash
Omobolaji O. Akala
Yongmei Yin
author_sort Quchang Ouyang
collection DOAJ
description Abstract Background Sacituzumab tirumotecan (sac-TMT) is an antibody–drug conjugate composed of an anti-TROP2 monoclonal antibody coupled to a cytotoxic belotecan-derived topoisomerase I inhibitor (KL610023) via a novel linker. We report results from the phase 1 dose-escalation cohorts in advanced solid tumors and phase 2 expansion cohorts for metastatic triple-negative breast cancer (TNBC) from the first-in-human MK-2870-001 (KL264-01) study (NCT04152499). Methods Patients had unresectable locally advanced/metastatic solid tumors refractory to standard therapies. In the phase 1 dose-escalation cohorts, patients had unresectable locally advanced/metastatic solid tumors refractory to standard therapies. Sac-TMT was administered by intravenous administration every 2 weeks at 2 to 12 mg/kg. In phase 2, patients with TNBC and HR+/HER2− breast cancer received sac-TMT per recommended doses for expansion (RDEs) identified in phase 1. Primary objectives were determining maximum tolerated dose (MTD) of sac-TMT and establishing RDEs (phase 1) and determining ORR per RECIST v1.1 by investigator assessment (phase 2). Adverse events were assessed per NCI-CTCAE version 5.0. Results Thirty patients were enrolled in phase 1 and received sac-TMT 2 mg/kg (n = 4), 4 mg/kg (n = 7), 5 mg/kg (n = 7), 5.5 mg/kg (n = 5), and 6 mg/kg (n = 7). Five patients had dose-limiting toxicities: grade 3 stomatitis at 4, 5.5, and 6 mg/kg; grade 3 rash at 5 mg/kg; and grade 3 urticaria at 6 mg/kg. MTD was 5.5 mg/kg and RDEs were 4 and 5 mg/kg. In the phase 2 dose expansion, ORR (95% CI) was 34.8% (16.4%, 57.3%) in the 4-mg/kg group (n = 23) and 38.9% (23.1%, 56.5%) in the 5-mg/kg group (n = 36) for TNBC. ORR (95% CI) was 31.7% (18.1%, 48.1%) for HR+/HER2− breast cancer (n = 41). Conclusions Sac-TMT demonstrated manageable safety profile in patients with unresectable locally advanced/metastatic solid tumors and promising antitumor activity in metastatic TNBC and HR+/HER2 − breast cancer. Sac-TMT is being investigated in phase 3 studies. Trial registration ClinicalTrials.gov, NCT04152499.
format Article
id doaj-art-542c4c40c63a4772b52cf3c4d31ce9aa
institution Kabale University
issn 1756-8722
language English
publishDate 2025-06-01
publisher BMC
record_format Article
series Journal of Hematology & Oncology
spelling doaj-art-542c4c40c63a4772b52cf3c4d31ce9aa2025-08-20T03:26:43ZengBMCJournal of Hematology & Oncology1756-87222025-06-0118111310.1186/s13045-025-01705-2Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapiesQuchang Ouyang0Jordi Rodon1Yan Liang2Xinhong Wu3Qun Li4Lihua Song5Min Yan6Zhongsheng Tong7YunPeng Liu8Zev A. Wainberg9Ying Wang10Cuizhi Geng11Susanna V. Ulahannan12Guohua Yu13Manish R. Sharma14Xiang Wang15Judy S. Wang16Alexander Spira17Weihong Zhao18Rachel E. Sanborn19Ying Cheng20Xian Wang21Gesha Liu22Yaling Li23Junyou Ge24Elliot Chartash25Omobolaji O. Akala26Yongmei Yin27Hunan Cancer HospitalUniversity of Texas MD Anderson Cancer CenterJiangsu Province HospitalHubei Cancer HospitalShanghai East HospitalShandong Cancer HospitalThe Affiliated Cancer Hospital of Zhengzhou University & Henan Cancer HospitalTianjin Cancer HospitalThe First Hospital of China Medical UniversityUCLA University of California Los AngelesSun Yai-Sen Memorial HospitalThe Fourth Hospital of Hebei Medical UniversityStephenson Cancer Center, University of Oklahoma/SCRIWeifang People’s HospitalSTART Center for Cancer Research - MidwestXuzhou Central HospitalFlorida Cancer Specialists/Sarah Cannon Research InstituteVirginia Cancer Specialists (Fairfax)Chinese PLA General HospitalEarle A. Chiles Research Institute, Providence Cancer InstituteJilin Cancer HospitalSir Run Run Shaw Hospital Zhejiang University School of MedicineSichuan Kelun-Biotech Biopharmaceutical Co., Ltd.Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.Merck & Co., Inc.Merck & Co., Inc.Jiangsu Province HospitalAbstract Background Sacituzumab tirumotecan (sac-TMT) is an antibody–drug conjugate composed of an anti-TROP2 monoclonal antibody coupled to a cytotoxic belotecan-derived topoisomerase I inhibitor (KL610023) via a novel linker. We report results from the phase 1 dose-escalation cohorts in advanced solid tumors and phase 2 expansion cohorts for metastatic triple-negative breast cancer (TNBC) from the first-in-human MK-2870-001 (KL264-01) study (NCT04152499). Methods Patients had unresectable locally advanced/metastatic solid tumors refractory to standard therapies. In the phase 1 dose-escalation cohorts, patients had unresectable locally advanced/metastatic solid tumors refractory to standard therapies. Sac-TMT was administered by intravenous administration every 2 weeks at 2 to 12 mg/kg. In phase 2, patients with TNBC and HR+/HER2− breast cancer received sac-TMT per recommended doses for expansion (RDEs) identified in phase 1. Primary objectives were determining maximum tolerated dose (MTD) of sac-TMT and establishing RDEs (phase 1) and determining ORR per RECIST v1.1 by investigator assessment (phase 2). Adverse events were assessed per NCI-CTCAE version 5.0. Results Thirty patients were enrolled in phase 1 and received sac-TMT 2 mg/kg (n = 4), 4 mg/kg (n = 7), 5 mg/kg (n = 7), 5.5 mg/kg (n = 5), and 6 mg/kg (n = 7). Five patients had dose-limiting toxicities: grade 3 stomatitis at 4, 5.5, and 6 mg/kg; grade 3 rash at 5 mg/kg; and grade 3 urticaria at 6 mg/kg. MTD was 5.5 mg/kg and RDEs were 4 and 5 mg/kg. In the phase 2 dose expansion, ORR (95% CI) was 34.8% (16.4%, 57.3%) in the 4-mg/kg group (n = 23) and 38.9% (23.1%, 56.5%) in the 5-mg/kg group (n = 36) for TNBC. ORR (95% CI) was 31.7% (18.1%, 48.1%) for HR+/HER2− breast cancer (n = 41). Conclusions Sac-TMT demonstrated manageable safety profile in patients with unresectable locally advanced/metastatic solid tumors and promising antitumor activity in metastatic TNBC and HR+/HER2 − breast cancer. Sac-TMT is being investigated in phase 3 studies. Trial registration ClinicalTrials.gov, NCT04152499.https://doi.org/10.1186/s13045-025-01705-2Sac-TMTAntibody–drug conjugateTriple-negative breast cancerHR+/HER2− breast cancer
spellingShingle Quchang Ouyang
Jordi Rodon
Yan Liang
Xinhong Wu
Qun Li
Lihua Song
Min Yan
Zhongsheng Tong
YunPeng Liu
Zev A. Wainberg
Ying Wang
Cuizhi Geng
Susanna V. Ulahannan
Guohua Yu
Manish R. Sharma
Xiang Wang
Judy S. Wang
Alexander Spira
Weihong Zhao
Rachel E. Sanborn
Ying Cheng
Xian Wang
Gesha Liu
Yaling Li
Junyou Ge
Elliot Chartash
Omobolaji O. Akala
Yongmei Yin
Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
Journal of Hematology & Oncology
Sac-TMT
Antibody–drug conjugate
Triple-negative breast cancer
HR+/HER2− breast cancer
title Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
title_full Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
title_fullStr Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
title_full_unstemmed Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
title_short Results of a phase 1/2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
title_sort results of a phase 1 2 study of sacituzumab tirumotecan in patients with unresectable locally advanced or metastatic solid tumors refractory to standard therapies
topic Sac-TMT
Antibody–drug conjugate
Triple-negative breast cancer
HR+/HER2− breast cancer
url https://doi.org/10.1186/s13045-025-01705-2
work_keys_str_mv AT quchangouyang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT jordirodon resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yanliang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT xinhongwu resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT qunli resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT lihuasong resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT minyan resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT zhongshengtong resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yunpengliu resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT zevawainberg resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yingwang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT cuizhigeng resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT susannavulahannan resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT guohuayu resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT manishrsharma resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT xiangwang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT judyswang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT alexanderspira resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT weihongzhao resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT rachelesanborn resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yingcheng resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT xianwang resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT geshaliu resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yalingli resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT junyouge resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT elliotchartash resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT omobolajioakala resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies
AT yongmeiyin resultsofaphase12studyofsacituzumabtirumotecaninpatientswithunresectablelocallyadvancedormetastaticsolidtumorsrefractorytostandardtherapies