Confirmation of Fixed Quarterly Riliprubart Regimen in Patients with Cold Agglutinin Disease Using Population PK/PD and Exposure-Response Analyses.

ABSTRACT: Riliprubart is a second-generation, humanized immunoglobulin G4 that inhibits only the activated form of the C1s component of the proximal classical complement pathway. The clinical studies of riliprubart conducted thus far for the treatment of cold agglutinin disease (CAD), a rare autoimmune disease, include a Phase 1 first-in-human study in healthy participants and a Phase 1b single-dose study in 12 adult CAD patients. The objective of this study was to derive a riliprubart dosing regimen for CAD patients using model-informed drug development (MIDD) approaches utilizing available clinical data. A virtual population consisting of 1,000 CAD patients was created by sampling with replacement of the body weight distribution of CAD patients from a clinical database. The 3.5 g IV quarterly (q12

SUBMITTER: Chow T 

PROVIDER: S-EPMC12272316 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature