The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: a randomized phase 1 study

P Kiessling, R Lledo-Garcia, S Watanabe… - Science translational …, 2017 - science.org
P Kiessling, R Lledo-Garcia, S Watanabe, G Langdon, D Tran, M Bari, L Christodoulou…
Science translational medicine, 2017science.org
Pathogenic immunoglobulin G (IgG) autoantibodies characterize some human autoimmune
diseases; their high concentration and long half-life are dependent on recycling by the
neonatal Fc receptor (FcRn). Inhibition of FcRn is an attractive new treatment concept for IgG-
mediated autoimmune diseases. Rozanolixizumab (UCB7665; CA170_01519. g57 IgG4P)
is an anti-human FcRn monoclonal antibody. In cynomolgus monkeys, rozanolixizumab
reduced IgG (maximum 75 to 90% by about day 10), was well tolerated, and did not increase …
Pathogenic immunoglobulin G (IgG) autoantibodies characterize some human autoimmune diseases; their high concentration and long half-life are dependent on recycling by the neonatal Fc receptor (FcRn). Inhibition of FcRn is an attractive new treatment concept for IgG-mediated autoimmune diseases. Rozanolixizumab (UCB7665; CA170_01519.g57 IgG4P) is an anti-human FcRn monoclonal antibody. In cynomolgus monkeys, rozanolixizumab reduced IgG (maximum 75 to 90% by about day 10), was well tolerated, and did not increase risk of infection. We also report a first-in-human, randomized, double-blind, placebo-controlled, dose-escalating study of intravenous (IV) or subcutaneous (SC) rozanolixizumab in healthy subjects (NCT02220153). The primary objective was to evaluate safety and tolerability. Secondary objectives were assessment of rozanolixizumab pharmacokinetics and pharmacodynamics, including effects on circulating IgG concentrations. Forty-nine subjects were randomized to receive rozanolixizumab (n = 36) or placebo (n = 13) across six cohorts. The first three cohorts received IV doses, and the subsequent three cohorts received SC doses, of rozanolixizumab 1, 4, or 7 mg/kg (n = 6 for each cohort; plus n = 7 or 6 for placebo, respectively). The most frequent treatment-emergent adverse event [TEAE; headache, 14 of 36 (38.9%) subjects] was dose-dependent and more prominent after IV administration. Severe TEAEs occurred in four subjects, all in the highest-dose IV group [headache (n = 3) and back pain (n = 1)]. Rozanolixizumab pharmacokinetics demonstrated nonlinear increases with dose. There were sustained dose-dependent reductions in serum IgG concentrations (IV and SC rozanolixizumab). These data provide clinical evidence for the therapeutic potential of rozanolixizumab.
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