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Mol Ther Methods Clin Dev. 2017 Aug 18;6:207-215. doi: 10.1016/j.omtm.2017.08.001. eCollection 2017 Sep 15.

Safety and Efficacy of the Complement Inhibitor AMY-101 in a Natural Model of Periodontitis in Non-human Primates.

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University of Pennsylvania, School of Dental Medicine, Department of Microbiology, Philadelphia, PA 19104, USA.
Manila Central University, College of Dentistry, 1400 Caloocan City, Metro Manila, Philippines.
Simian Conservation Breeding and Research Center (SICONBREC), Makati City, Philippines.
University of Pennsylvania Perelman School of Medicine, Department of Pathology and Laboratory Medicine, Philadelphia, PA 19104, USA.
University of Pennsylvania, School of Dental Medicine, Department of Preventive and Restorative Sciences, Division of Pediatric Dentistry, Philadelphia, PA 19104, USA.
Amyndas Pharmaceuticals, 16675 Glyfada, Greece.


Periodontitis is a chronic inflammatory disease associated with overactivation of the complement system. Recent preclinical studies suggest that host-modulation therapies may contribute to effective treatment of human periodontitis, which may lead to loss of teeth and function if untreated. We previously showed that locally administered AMY-101 (Cp40), a peptidic inhibitor of the central complement component C3, can inhibit naturally occurring periodontitis in non-human primates (NHPs) when given once a week. This study was undertaken to determine the local safety of increasing doses of the drug as well as its efficacy when given at a reduced frequency or after systemic administration. Our findings have determined a local dose of AMY-101 (0.1 mg/site) that is free of local irritation and effective when given once every 3 weeks. Moreover, a daily subcutaneous dose of AMY-101 (4 mg/kg bodyweight) was protective against NHP periodontitis, suggesting that patients treated for systemic disorders (e.g., paroxysmal nocturnal hemoglobinuria) can additionally benefit in terms of improved periodontal condition. In summary, AMY-101 appears to be a promising candidate drug for the adjunctive treatment of human periodontitis, a notion that merits investigation in human clinical trials.


AMY-101; complement; compstatin; inflammation; non-human primates; periodontitis

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