Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial

Abstract Antibody-drug conjugates (ADCs) and tyrosine kinase inhibitors (TKIs) are widely used for HER2-positive metastatic breast cancer, but their efficacy in the neoadjuvant setting remains under investigation. The MUKDEN 06 trial (NCT05426486), a multicentre, randomised, phase 2b study, compared...

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Main Authors: Nan Niu, Jinqi Xue, Guanglei Chen, Fang Qiu, Qianshi Xu, Xinyu Zheng, Chao Liu, Yafei Zhao, Xi Gu, Yi Zhao, Hong Xu, Hao Zhang, Guijin He, Ke Li, Pengfei Li, Xiaoying Chen, Yong Li, Shuo Wang, Demiao Zhu, Tong Liu, Fei Xing, Yongqing Xu, Ye Han, Meiyue Tang, Mingxin Liu, Gege Jiao, Xiaofan Jiang, Tony Yuen, Zheng Pang, Caigang Liu
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-61213-2
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author Nan Niu
Jinqi Xue
Guanglei Chen
Fang Qiu
Qianshi Xu
Xinyu Zheng
Chao Liu
Yafei Zhao
Xi Gu
Yi Zhao
Hong Xu
Hao Zhang
Guijin He
Ke Li
Pengfei Li
Xiaoying Chen
Yong Li
Shuo Wang
Demiao Zhu
Tong Liu
Fei Xing
Yongqing Xu
Ye Han
Meiyue Tang
Mingxin Liu
Gege Jiao
Xiaofan Jiang
Tony Yuen
Zheng Pang
Caigang Liu
author_facet Nan Niu
Jinqi Xue
Guanglei Chen
Fang Qiu
Qianshi Xu
Xinyu Zheng
Chao Liu
Yafei Zhao
Xi Gu
Yi Zhao
Hong Xu
Hao Zhang
Guijin He
Ke Li
Pengfei Li
Xiaoying Chen
Yong Li
Shuo Wang
Demiao Zhu
Tong Liu
Fei Xing
Yongqing Xu
Ye Han
Meiyue Tang
Mingxin Liu
Gege Jiao
Xiaofan Jiang
Tony Yuen
Zheng Pang
Caigang Liu
author_sort Nan Niu
collection DOAJ
description Abstract Antibody-drug conjugates (ADCs) and tyrosine kinase inhibitors (TKIs) are widely used for HER2-positive metastatic breast cancer, but their efficacy in the neoadjuvant setting remains under investigation. The MUKDEN 06 trial (NCT05426486), a multicentre, randomised, phase 2b study, compared ARX788 (anti-HER2 ADC) plus pyrotinib (TKI) with the standard neoadjuvant regimen of docetaxel, carboplatin, trastuzumab, and pertuzumab (TCbHP) in female patients with early or locally advanced HER2-positive breast cancer. The primary endpoint was the pathological complete response (pCR, ypT0/is, ypN0) rate, analyzed in the intention-to-treat population. pCR was achieved in 70.6% (48/68) of patients receiving ARX788 plus pyrotinib, compared to 51.5% (35/68) in the TCbHP group, with a significant absolute difference of 19.1% (95% CI, 2.7–34.6; p = 0.023). No treatment-related deaths occurred. The most common grade 3–4 adverse events were diarrhea and hepatic dysfunction in the ARX788 plus pyrotinib group, and fatigue, nausea and anorexia in the TCbHP group. Interstitial lung disease (ILD)/pneumonitis and ocular events were observed with ARX788 plus pyrotinib, indicating a distinct safety profile. These findings offer clinical insights into the potential of dual HER2-targeted blockade with an ADC and TKI as an optional neoadjuvant strategy for patients with early or locally advanced HER2-positive breast cancer.
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spelling doaj-art-49f13b37da9d4ab6805afdf7c6ee48c32025-08-20T03:45:34ZengNature PortfolioNature Communications2041-17232025-07-011611910.1038/s41467-025-61213-2Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trialNan Niu0Jinqi Xue1Guanglei Chen2Fang Qiu3Qianshi Xu4Xinyu Zheng5Chao Liu6Yafei Zhao7Xi Gu8Yi Zhao9Hong Xu10Hao Zhang11Guijin He12Ke Li13Pengfei Li14Xiaoying Chen15Yong Li16Shuo Wang17Demiao Zhu18Tong Liu19Fei Xing20Yongqing Xu21Ye Han22Meiyue Tang23Mingxin Liu24Gege Jiao25Xiaofan Jiang26Tony Yuen27Zheng Pang28Caigang Liu29Department of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Breast Surgery, the First Affiliated Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Breast Surgery, Liaoning Cancer Hospital and InstituteDepartment of Breast Surgery, Liaoning Cancer Hospital and InstituteDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Breast Surgery, Anshan Cancer HospitalDepartment of Thoracic and Breast Surgery, Yanan People’s HospitalDepartment of Breast Surgery, Liaohe Oilfield General HospitalDepartment of Breast Surgery, Benxi Central HospitalDepartment of Breast Surgery, the First Affiliated Hospital of China Medical UniversityDepartment of Breast Surgery, the First Affiliated Hospital of Jinzhou Medical UniversityDepartment of Breast Surgery, Cancer Hospital of Harbin Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityDepartment of Oncology, Shengjing Hospital of China Medical UniversityCenter for Translational Medicine and Pharmacology, Icahn School of Medicine at Mount SinaiJiangsu Hengrui Pharmaceuticals Co., LtdDepartment of Oncology, Shengjing Hospital of China Medical UniversityAbstract Antibody-drug conjugates (ADCs) and tyrosine kinase inhibitors (TKIs) are widely used for HER2-positive metastatic breast cancer, but their efficacy in the neoadjuvant setting remains under investigation. The MUKDEN 06 trial (NCT05426486), a multicentre, randomised, phase 2b study, compared ARX788 (anti-HER2 ADC) plus pyrotinib (TKI) with the standard neoadjuvant regimen of docetaxel, carboplatin, trastuzumab, and pertuzumab (TCbHP) in female patients with early or locally advanced HER2-positive breast cancer. The primary endpoint was the pathological complete response (pCR, ypT0/is, ypN0) rate, analyzed in the intention-to-treat population. pCR was achieved in 70.6% (48/68) of patients receiving ARX788 plus pyrotinib, compared to 51.5% (35/68) in the TCbHP group, with a significant absolute difference of 19.1% (95% CI, 2.7–34.6; p = 0.023). No treatment-related deaths occurred. The most common grade 3–4 adverse events were diarrhea and hepatic dysfunction in the ARX788 plus pyrotinib group, and fatigue, nausea and anorexia in the TCbHP group. Interstitial lung disease (ILD)/pneumonitis and ocular events were observed with ARX788 plus pyrotinib, indicating a distinct safety profile. These findings offer clinical insights into the potential of dual HER2-targeted blockade with an ADC and TKI as an optional neoadjuvant strategy for patients with early or locally advanced HER2-positive breast cancer.https://doi.org/10.1038/s41467-025-61213-2
spellingShingle Nan Niu
Jinqi Xue
Guanglei Chen
Fang Qiu
Qianshi Xu
Xinyu Zheng
Chao Liu
Yafei Zhao
Xi Gu
Yi Zhao
Hong Xu
Hao Zhang
Guijin He
Ke Li
Pengfei Li
Xiaoying Chen
Yong Li
Shuo Wang
Demiao Zhu
Tong Liu
Fei Xing
Yongqing Xu
Ye Han
Meiyue Tang
Mingxin Liu
Gege Jiao
Xiaofan Jiang
Tony Yuen
Zheng Pang
Caigang Liu
Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
Nature Communications
title Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
title_full Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
title_fullStr Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
title_full_unstemmed Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
title_short Neoadjuvant ARX788 plus pyrotinib versus trastuzumab, pertuzumab, docetaxel and carboplatin for HER2-positive breast cancer: a randomised phase 2b trial
title_sort neoadjuvant arx788 plus pyrotinib versus trastuzumab pertuzumab docetaxel and carboplatin for her2 positive breast cancer a randomised phase 2b trial
url https://doi.org/10.1038/s41467-025-61213-2
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