Data from phase III VANGUARD clinical trial of CSL 312 showing reduced attack rate in hereditary angioedema presented at AAAAI meeting

Results from the trial, the first to investigate targeting activated Factor XII (FXIIa) to prevent HAE attacks, showed that once-monthly subcutaneous injections of garadacimab significantly reduced the attack rate compared to placebo.

During the double-blind, randomized, placebo-controlled, multicenter, parallel-group study, patients taking once monthly garadacimab (n=39) experienced a statistically lower monthly attack rate versus placebo (n=24) (p< 0.001), resulting in a mean attack rate reduction of 86.5% versus placebo, and a median attack rate reduction of 100% versus placebo. The mean attack rate reduction compared with placebo after adjusting for baseline attack rate was 89.2%. Overall, during the six-month trial, a majority (61.5%) of patients taking garadacimab were attack-free whereas no patients on the placebo arm were attack-free; 74.4% of patients taking garadacimab achieved a minimum 90% attack reduction versus the run-in period.

The study also showed that garadacimab demonstrated a favorable safety and tolerability profile. There were no adverse events that led to treatment discontinuation. Five injection site reactions, all mild, were reported in two (5.1%) patients treated with garadacimab and three patients (12%) on placebo. Based on the full study data, which are consistent with the positive top line results announced in August 2022, CSL plans regulatory submissions to global health authorities later this calendar year for approval of garadacimab. The data were presented during the 2023 American Academy of Allergy Asthma & Immunology (AAAAI) Annual Meeting.