Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ZY‐IL1 in Three Patients with Cryopyrin‐Associated Periodic Syndromes

Abstract We present the first results of the proof‐of‐concept phase 2a study of oral NLRP3 inflammasome inhibitor in subjects with cryopyrin‐associated periodic syndromes (CAPS). Three adult subjects with a confirmed diagnosis of CAPS were enrolled and administered 50 mg of ZYIL1 twice daily for 7 days. A total of 5 treatment‐emergent adverse events (TEAEs) were reported in 2 subjects. All 5 TEAEs were mild in severity and considered unrelated to the study drug. At steady state, the average plasma concentration and trough concentration ranged from 2.5 to 4.2 and 1.4 to 2.5 µg/mL, respectively. Inflammatory markers and disease activity (physician and patient global assessment score) decreased notably 12 hours post‐last dose.