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Article History

Received: 12 November 2024

Accepted: 23 March 2025

First Online: 1 July 2025

Declarations

: In the phase 2 dose-finding study, the ethical considerations were as follows: approval by the Ethics Review Board, adherence to the Declaration of Helsinki, provision of informed consent by all patients at enrollment, and clinical trial registration at ClinicalTrials.gov (NCT02094417). In the phase 2/3 study, the ethical considerations were as follows: approval by the Institutional Review Board at each center, adherence to the Declaration of Helsinki and International Conference on Harmonisation of Good Clinical Practice guidelines, provision of informed consent by all participants, and clinical trial registration at ClinicalTrials.gov (NCT02773290). Consent for secondary use of the data in this study was also obtained.

: K. Mitani received speakers bureau fees from Celgene K.K.; Otsuka Pharmaceutical Co., Ltd.; Bristol-Myers Squibb K.K.; Novartis Pharma K.K.; and Kyowa Kirin Co., Ltd.; and received research funding from Chugai Pharmaceutical Co., Ltd.; Kyowa Kirin Co., Ltd.; and Takeda Pharmaceutical Co., Ltd. Y.T. received speakers bureau fees from Kyowa Kirin Co., Ltd.; Novartis Pharma K.K.; Kissei Pharmaceutical Co., Ltd.; Sysmex Corporation; Chugai Pharmaceutical Co., Ltd.; and UCB Japan Co., Ltd.; received consultancy fees from Swedish Orphan Biovitrum Japan Co., Ltd.; Medical & Biological Laboratories Co., Ltd.; Kissei Pharmaceutical Co., Ltd.; Novartis Pharma K.K.; and Sysmex Corporation; and had membership on Board of Directors or advisory committees for Novartis Pharma K.K.; Sysmex Corporation; Takeda Pharmaceutical Co., Ltd.; and Argenx Japan K.K. K. Miyazaki received speakers bureau fees from Kyowa Kirin Co., Ltd.; Chugai Pharmaceutical Co., Ltd.; Pfizer Japan Inc.; Takeda Pharmaceutical Co., Ltd.; MSD K.K.; Novartis Pharma K.K.; Novo Nordisk Pharma Ltd.; KM Biologics Co., Ltd.; Nihon Pharmaceutical Co., Ltd.; and CSL Behring K.K.; and received research funding from Kyowa Kirin Co., Ltd.; Chugai Pharmaceutical Co., Ltd.; Pfizer Japan Inc.; Daiichi Sankyo Co., Ltd.; Astellas Pharma Inc.; and Otsuka Pharmaceutical Co., Ltd. K.N. received speakers bureau fees from Kyowa Kirin Co., Ltd. K.U. received speakers bureau fees from Novartis Pharma K.K.; AbbVie G.K.; Alexion Pharmaceuticals, Inc.; Amgen K.K.; Asahi Kasei Pharma Corporation; AstraZeneca K.K.; Bristol-Myers Squibb K.K.; Chugai Pharmaceutical Co., Ltd.; Eisai Co., Ltd.; Incyte Biosciences Japan G.K.; Kyowa Kirin Co., Ltd.; Meiji Seika Pharma Co., Ltd.; Nippon Shinyaku Co., Ltd.; Ono Pharmaceutical Co., Ltd.; Otsuka Pharmaceutical Co., Ltd.; PharmaEssentia Japan K.K.; Sanofi K.K.; Takeda Pharmaceutical Co., Ltd.; and Janssen Pharmaceutical K.K.; received research funding from Astellas Pharma Inc.; AbbVie G.K.; Alexion Pharmaceuticals, Inc.; Apellis Pharmaceuticals, Inc.; Bristol-Myers Squibb K.K.; Celgene K.K.; Chugai Pharmaceutical Co., Ltd.; Daiichi Sankyo Co., Ltd.; Eisai Co., Ltd.; Incyte Biosciences Japan G.K.; Kyowa Kirin Co., Ltd.; MSD K.K.; Nippon Shinyaku Co., Ltd.; Novartis Pharma K.K.; Ohara Pharmaceutical Co., Ltd.; Ono Pharmaceutical Co., Ltd.; Otsuka Pharmaceutical Co., Ltd.; Pfizer Japan Inc.; Sanofi K.K.; Takeda Pharmaceutical Co., Ltd.; and Yakult Honsha Co., Ltd.; and received consultancy fees from Alnylam Japan K.K.; Chugai Pharmaceutical Co., Ltd.; Ohara Pharmaceutical Co., Ltd.; and Swedish Orphan Biovitrum Japan Co., Ltd. N.U. received honoraria from Bristol-Myers Squibb K.K.; Janssen Pharmaceutical K.K.; Chugai Pharmaceutical Co., Ltd.; Nippon Shinyaku Co., Ltd.; and Otsuka Pharmaceutical Co., Ltd. T.F. received speakers bureau fees from Kyowa Kirin Co., Ltd.; Novartis Pharma K.K.; AbbVie G.K.; Janssen Pharmaceutical K.K.; Bristol-Myers Squibb K.K.; and Chugai Pharmaceutical Co., Ltd. H.K. received honoraria from Bristol-Myers Squibb K.K.; Celgene K.K.; Chugai Pharmaceutical Co., Ltd.; Janssen Pharmaceutical K.K.; Japan Blood Products Organization; Kyowa Kirin Co., Ltd.; MSD K.K.; Novartis Pharma K.K.; Ono Pharmaceutical Co., Ltd.; Sanofi K.K.; Takeda Pharmaceutical Co., Ltd.; Nippon Shinyaku Co. Ltd.; Novo Nordisk Pharma Ltd.; Kissei Pharmaceutical Co. Ltd.; GlaxoSmithKline K.K.; Daiichi Sankyo Co., Ltd.; and AstraZeneca K.K. I.M. received speakers bureau fees from Sanofi K.K.; Bristol-Myers Squibb K.K.; SymBio Pharmaceuticals Ltd.; AstraZeneca K.K.; Chugai Pharmaceutical Co., Ltd.; Takeda Pharmaceutical Co., Ltd.; Ono Pharmaceutical Co., Ltd.; AbbVie G.K.; Otsuka Pharmaceutical Co., Ltd.; Astellas Pharma Inc.; Pfizer Japan Inc.; Novartis Pharma K.K.; and Janssen Pharmaceutical K.K.; received research funding from Chugai Pharmaceutical Co., Ltd.; Kyowa Kirin Co., Ltd.; Sumitomo Pharma Co., Ltd.; Takeda Pharmaceutical Co., Ltd.; Mitsubishi Tanabe Pharma Corporation; Nippon Shinyaku Co., Ltd.; Eisai Co., Ltd.; Asahi Kasei Pharma Corporation; AbbVie G.K.; Taiho Pharmaceutical Co., Ltd.; Daiichi Sankyo Co., Ltd.; Nippon Kayaku Co., Ltd.; CSL Behring K.K.; Ayumi Pharmaceutical Corporation; and Eli Lily Japan K.K.; and received consultancy fees from Otsuka Pharmaceutical Co., Ltd. M.K. received speakers bureau fees from Bristol-Myers Squibb K.K.; Novartis Pharma K.K.; Janssen Pharmaceutical K.K.; Nippon Shinyaku Co., Ltd.; Takeda Pharmaceutical Co., Ltd.; and Ono Pharmaceutical Co., Ltd. M. Sawa received honoraria from GlaxoSmithKline K.K.; Daiichi Sankyo Co., Ltd.; AstraZeneca K.K.; Janssen Pharmaceutical K.K.; SymBio Pharmaceuticals Ltd.; CSL Behring K.K.; AbbVie G.K.; Mundipharma K.K.; Shire Japan K.K.; Mochida Pharmaceutical Co., Ltd.; Celgene K.K.; Sanofi K.K.; Takeda Pharmaceutical Co., Ltd.; Sumitomo Pharma Co., Ltd.; Otsuka Pharmaceutical Co., Ltd.; Eisai Co., Ltd.; Novartis Pharma K.K.; Asahi Kasei Pharma Corporation; Bristol-Myers Squibb K.K.; MSD K.K.; Ono Pharmaceutical Co., Ltd.; Nippon Shinyaku Co., Ltd.; Astellas Pharma Inc.; Pfizer Japan Inc.; Chugai Pharmaceutical Co., Ltd.; and Kyowa Kirin Co., Ltd. M.H. received honoraria from Chugai Pharmaceutical Co., Ltd. and JCR Pharmaceuticals Co., Ltd. S.I. received honoraria from Meiji Seika Pharma Co., Ltd.; and received research funding from AstraZeneca K.K. and Beigene Japan G.K. Y.Y. received honoraria from Alexion Pharmaceuticals Inc. and Novartis Pharma K.K.; and received research funding from Chugai Pharmaceutical Co., Ltd. K. Murotani received honoraria from Kyowa Kirin Co., Ltd., Chugai Pharmaceutical Co., Ltd., AstraZeneca K.K., Taiho Pharmaceutical Co., Ltd., MSD K.K., Nihon Shinyaku Co., Ltd. and Pfizer Japan Inc. M. Shimizu is currently employed at Kyowa Kirin Co., Ltd. A.M. received honoraria from Chugai Pharmaceutical Co., Ltd.; Alexion Pharmaceuticals Inc.; Nippon Shinyaku Co., Ltd.; Sumitomo Pharma Co., Ltd.; and Novartis Pharma K.K.; and received consultancy fees from Kyowa Kirin Co., Ltd. K.O. received honoraria from Human Life CORD Japan Inc.; Otsuka Pharmaceutical Co., Ltd.; Mitsubishi Tanabe Pharma Corporation; AbbVie G.K.; and Novartis Pharma K.K.; received research funding from Japan Agency for Medical Research and Development; received consultancy fees from Takara Bio Inc. and Human Life CORD Japan Inc.; is a current equity holder in Terumo Corporation; and has a membership on their Board of Directors or advisory committees. S.N. received honoraria from Novartis Pharma K.K. and Kyowa Kirin Co., Ltd.; SymBio Pharmaceuticals Ltd.; Sanofi K.K.; Asahi Kasei Pharma Corporation; Pfizer Japan Inc.; Swedish Orphan Biovitrum Japan Co., Ltd.; Alexion Pharmaceuticals, Inc.; received consultancy fees from SymBio Pharmaceuticals Ltd.; and received research funding from Alexion Pharmaceuticals Inc. J.W.L. received speakers bureau fees and grants from Alexion and AstraZeneca Rare Disease; and received consultancy fees from Kyowa Kirin Co., Ltd., Novartis Pharma K.K., and Sanofi K.K. The remaining authors declare no competing financial interests.