Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera

ABSTRACT Ropeginterferon alfa‐2b (ropeg) represents a new‐generation interferon‐based therapy approved for polycythaemia vera (PV) treatment. This study aimed to elucidate its population pharmacokinetics‐pharmacodynamics (PopPK‐PD) and exposure‐response (E‐R) relationships. A PopPK model was develop...

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Main Authors: Albert Qin, Kazuya Shimoda, Shanshan Suo, Rongfeng Fu, Keita Kirito, Daoxiang Wu, Jason Liao, Haoqi Chen, Lei Wu, Xia Su, Yucheng Gao, Toshiaki Sato, Yaning Li, Jingjing Zhang, Weihong Shen, Wei Wang, Lei Zhang, Jie Jin, Norio Komatsu
Format: Article
Language:English
Published: Wiley 2025-06-01
Series:Pharmacology Research & Perspectives
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Online Access:https://doi.org/10.1002/prp2.70109
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author Albert Qin
Kazuya Shimoda
Shanshan Suo
Rongfeng Fu
Keita Kirito
Daoxiang Wu
Jason Liao
Haoqi Chen
Lei Wu
Xia Su
Yucheng Gao
Toshiaki Sato
Yaning Li
Jingjing Zhang
Weihong Shen
Wei Wang
Lei Zhang
Jie Jin
Norio Komatsu
author_facet Albert Qin
Kazuya Shimoda
Shanshan Suo
Rongfeng Fu
Keita Kirito
Daoxiang Wu
Jason Liao
Haoqi Chen
Lei Wu
Xia Su
Yucheng Gao
Toshiaki Sato
Yaning Li
Jingjing Zhang
Weihong Shen
Wei Wang
Lei Zhang
Jie Jin
Norio Komatsu
author_sort Albert Qin
collection DOAJ
description ABSTRACT Ropeginterferon alfa‐2b (ropeg) represents a new‐generation interferon‐based therapy approved for polycythaemia vera (PV) treatment. This study aimed to elucidate its population pharmacokinetics‐pharmacodynamics (PopPK‐PD) and exposure‐response (E‐R) relationships. A PopPK model was developed using pooled data from four clinical studies, including two Phase I studies in healthy volunteers (n = 48) and two Phase II studies in Chinese or Japanese patients with PV (n = 78). Sequential modeling was used to analyze pharmacokinetics‐pharmacodynamics (PK‐PD) regarding hematological parameters, including hematocrit, platelet, and white blood cell counts. Hematological changes were simulated using fast‐ and slow‐dose titration regimens. Individual exposure values were used to analyze the E‐R relationships regarding complete hematologic response (CHR), driver mutation, JAK2V617F allele burden, and safety. In this study, we developed a target‐mediated drug disposition model. Sigmoid indirect effects elucidated the PK‐PD in terms of hematological changes. Simulations showed that the fast‐titration regimen significantly accelerated hematocrit reduction. Logistic regression models showed that the probability of achieving CHR increased with exposure at Week 24 but not at Week 52. In contrast, JAK2V617F allele reductions correlated with exposure at both Weeks 24 and 52. Exposure‐safety analysis revealed a manageable risk of adverse events associated with transaminase increases. This study established a robust framework for ropeg PK‐PD, providing insights into its E‐R relationships and disease‐modifying action. Trial Registration: A17‐102, A19‐201, and A20‐202 are registered at ClinicalTrials.gov. The registration numbers are as follows: A17‐102, NCT03546465; A19‐201, NCT04182100; and A20‐202, NCT05485948. A17‐101 is registered at www.chinadrugtrials.org.cn. The registration number is CTR20190451
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spelling doaj-art-398e9082a5cd4a8992ca32d917f31fae2025-08-20T02:39:32ZengWileyPharmacology Research & Perspectives2052-17072025-06-01133n/an/a10.1002/prp2.70109Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia VeraAlbert Qin0Kazuya Shimoda1Shanshan Suo2Rongfeng Fu3Keita Kirito4Daoxiang Wu5Jason Liao6Haoqi Chen7Lei Wu8Xia Su9Yucheng Gao10Toshiaki Sato11Yaning Li12Jingjing Zhang13Weihong Shen14Wei Wang15Lei Zhang16Jie Jin17Norio Komatsu18Medical Research & Clinical Operations, PharmaEssentia Corporation Taipei TaiwanDivision of Haematology, Diabetes and Endocrinology, Department of Internal Medicine, Faculty of Medicine University of Miyazaki Miyazaki JapanThe First Affiliated Hospital Zhejiang University School of Medicine Hangzhou Zhejiang ChinaState Key Laboratory of Experimental Haematology, National Clinical Research Centre for Blood Diseases, Institute of Haematology and Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin ChinaDepartment of Haematology and Oncology University of Yamanashi Yamanashi JapanPharmaEssentia Biotech (Beijing) Limited Beijing ChinaBiostatistics and Data Sciences, PharmaEssentia Corporation Taipei TaiwanPharmaron Clinical Services Co., Ltd Chengdu ChinaPharmaron Clinical Services Co., Ltd Chengdu ChinaPharmaron Clinical Services Co., Ltd Chengdu ChinaPharmaron Clinical Services Co., Ltd Chengdu ChinaPharmaEssentia Japan KK Tokyo JapanPharmaEssentia Biotech (Beijing) Limited Beijing ChinaPharmaEssentia Biotech (Beijing) Limited Beijing ChinaPharmaEssentia Biotech (Beijing) Limited Beijing ChinaPharmaEssentia Biotech (Beijing) Limited Beijing ChinaState Key Laboratory of Experimental Haematology, National Clinical Research Centre for Blood Diseases, Institute of Haematology and Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin ChinaThe First Affiliated Hospital Zhejiang University School of Medicine Hangzhou Zhejiang ChinaPharmaEssentia Japan KK Tokyo JapanABSTRACT Ropeginterferon alfa‐2b (ropeg) represents a new‐generation interferon‐based therapy approved for polycythaemia vera (PV) treatment. This study aimed to elucidate its population pharmacokinetics‐pharmacodynamics (PopPK‐PD) and exposure‐response (E‐R) relationships. A PopPK model was developed using pooled data from four clinical studies, including two Phase I studies in healthy volunteers (n = 48) and two Phase II studies in Chinese or Japanese patients with PV (n = 78). Sequential modeling was used to analyze pharmacokinetics‐pharmacodynamics (PK‐PD) regarding hematological parameters, including hematocrit, platelet, and white blood cell counts. Hematological changes were simulated using fast‐ and slow‐dose titration regimens. Individual exposure values were used to analyze the E‐R relationships regarding complete hematologic response (CHR), driver mutation, JAK2V617F allele burden, and safety. In this study, we developed a target‐mediated drug disposition model. Sigmoid indirect effects elucidated the PK‐PD in terms of hematological changes. Simulations showed that the fast‐titration regimen significantly accelerated hematocrit reduction. Logistic regression models showed that the probability of achieving CHR increased with exposure at Week 24 but not at Week 52. In contrast, JAK2V617F allele reductions correlated with exposure at both Weeks 24 and 52. Exposure‐safety analysis revealed a manageable risk of adverse events associated with transaminase increases. This study established a robust framework for ropeg PK‐PD, providing insights into its E‐R relationships and disease‐modifying action. Trial Registration: A17‐102, A19‐201, and A20‐202 are registered at ClinicalTrials.gov. The registration numbers are as follows: A17‐102, NCT03546465; A19‐201, NCT04182100; and A20‐202, NCT05485948. A17‐101 is registered at www.chinadrugtrials.org.cn. The registration number is CTR20190451https://doi.org/10.1002/prp2.70109complete hematologic response (CHR)exposure‐response (E‐R)JAK2V617F allele burdenpolycythemia vera (PV)population pharmacokinetics‐pharmacodynamics (PopPK‐PD)Ropeginterferon alfa‐2b (ropeg)
spellingShingle Albert Qin
Kazuya Shimoda
Shanshan Suo
Rongfeng Fu
Keita Kirito
Daoxiang Wu
Jason Liao
Haoqi Chen
Lei Wu
Xia Su
Yucheng Gao
Toshiaki Sato
Yaning Li
Jingjing Zhang
Weihong Shen
Wei Wang
Lei Zhang
Jie Jin
Norio Komatsu
Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
Pharmacology Research & Perspectives
complete hematologic response (CHR)
exposure‐response (E‐R)
JAK2V617F allele burden
polycythemia vera (PV)
population pharmacokinetics‐pharmacodynamics (PopPK‐PD)
Ropeginterferon alfa‐2b (ropeg)
title Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
title_full Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
title_fullStr Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
title_full_unstemmed Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
title_short Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera
title_sort population pharmacokinetics pharmacodynamics and exposure response of ropeginterferon alfa 2b in chinese and japanese patients with polycythemia vera
topic complete hematologic response (CHR)
exposure‐response (E‐R)
JAK2V617F allele burden
polycythemia vera (PV)
population pharmacokinetics‐pharmacodynamics (PopPK‐PD)
Ropeginterferon alfa‐2b (ropeg)
url https://doi.org/10.1002/prp2.70109
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