AMY-101 as complement C3 inhibitor for periodontitis therapy: Mechanisms, efficacy, and clinical translation

Li, Jialun, Xu, Zhi, Nishio Ayre, Wayne ORCID: https://orcid.org/0000-0003-2405-1876 and Liu, Xiaohan 2025. AMY-101 as complement C3 inhibitor for periodontitis therapy: Mechanisms, efficacy, and clinical translation. Frontiers in Immunology 16 , 1587126. 10.3389/fimmu.2025.1587126

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Abstract

Periodontitis is a chronic inflammatory disease characterized by gingival inflammation, alveolar bone resorption, and periodontal tissue destruction. Complement activation, particularly through the C3 component, plays a critical role in the inflammatory processes underlying periodontitis. AMY-101, a selective inhibitor of complement C3, has demonstrated significant potential in modulating complement activity and mitigating periodontal inflammation. This study comprehensively evaluates AMY-101’s effects through in vitro, preclinical, and clinical studies. Mechanistic investigations revealed that AMY-101 effectively suppresses pro-inflammatory cytokines and matrix metalloproteinases (MMPs), reducing tissue destruction. Preclinical models confirmed AMY-101’s ability to improve clinical parameters such as probing pocket depth, attachment loss, and bone preservation. Moreover, clinical trials demonstrated AMY-101’s safety and efficacy in reducing gingival inflammation and bleeding without serious adverse events. These findings highlight AMY-101’s therapeutic potential for periodontitis and broader applicability in other complement-driven inflammatory diseases.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Schools > Dentistry
Funders:	National Natural Science Foundation of China, Innovation and Entrepreneurship Training Program for Chinese Medical University Students
Date of First Compliant Deposit:	10 April 2025
Date of Acceptance:	8 April 2025
Last Modified:	16 May 2025 09:47
URI:	https://orca.cardiff.ac.uk/id/eprint/177575

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