Kiniksa Pharmaceuticals, Ltd.

Kiniksa Pharmaceuticals develops therapies targeting the innate immune system. Its lead candidate mavrilimumab is an anti-GM-CSF receptor alpha monoclonal antibody that achieved both primary and secondary efficacy endpoints in a Phase 2 trial for giant cell arteritis, significantly reducing flare risk and improving sustained remission rates versus placebo. The FDA granted Orphan Drug designation for mavrilimumab in GCA. Kiniksa also develops rilonacept-based therapies and other immunology programs. Preclinical animal testing is integral to the company's development: mavrilimumab was evaluated in in vivo models of human GCA where it reduced arterial inflammation and gamma interferon production. Ex vivo studies using cultured temporal arteries from GCA patients confirmed that blocking the GM-CSF receptor modulates inflammatory molecules and vascular remodelling pathways, with results presented at EULAR 2025.