Novartis today announced positive Phase IIb data showing remibrutinib (LOU064), a potentially best-in-class oral BTK inhibitor, demonstrated rapid and effective disease control in patients with inadequately controlled chronic spontaneous urticaria (CSU).
This randomized, double-blind, placebo-controlled study (NCT03926611) evaluated the efficacy and safety of remibrutinib over 12 weeks in patients inadequately controlled with antihistamines. Patients (n=311) were randomized to placebo or different doses of remibrutinib, taken orally. The primary endpoint was achieved with remibrutinib showing a statistically significant dose-response compared to placebo with respect to change from baseline in UAS7 score at Week 4, Novartis said.
All remibrutinib doses provided significant improvements with respect to change from baseline in UAS7 at Week 4 and at Week 12 (p<0.0001 for all doses vs placebo) and demonstrated a rapid improvement as of Week 1. Compared with placebo, more patients receiving any remibrutinib dose achieved a complete control with absence of hives and itch (UAS7=0) or well-controlled disease (UAS7≤6) until Week 12 (end of treatment). Remibrutinib showed a favorable safety profile and good tolerability across the entire dose range tested, with no dose-dependent pattern.
“Up to one percent of the world’s population is affected by CSU² and we are proud of our contribution to advancing treatment. Despite these advances, there continues to be a need for new CSU therapies and we are committed to challenge the boundaries of innovation,” said Angelika Jahreis, M.D., Ph.D., Novartis Global Head Development Unit Immunology, Hepatology & Dermatology. “The fast-onset of control achieved with this novel oral agent in patients with previously inadequately-controlled CSU is compelling, and we are excited to rapidly develop remibrutinib.”
CSU is a distressing and unpredictable disease, characterized by the occurrence of itchy wheals (hives), angioedema, or both for 6 weeks or more without specific external stimuli and can have a major negative impact on patients’ quality of life. It most commonly persists for 1-5 years, but in some cases even longer. Despite existing treatments, the disease remains inadequately controlled in a large portion of patients.
Remibrutinib is a highly selective, potent oral BTK inhibitor discovered within Novartis and being developed in a number of clinical and early settings. With an unmet need for new CSU therapies, highlighting the importance of targeting new modes of action, BTK inhibition may be an attractive therapeutic option for CSU, due to its pivotal role in FcεR1-mediated (high affinity receptor of IgE) signalling of mast cells and basophils and their relevance to CSU pathogenesis.