CSL has presented results from the pivotal placebo-controlled Phase 3 VANGUARD clinical trial of garadacimab (CSL312).
Garadacimab is the company's investigational first-in-class monoclonal antibody being developed as a long-term prophylactic treatment for patients with hereditary angioedema (HAE).
HAE is a rare, genetic and potentially life-threatening condition that causes painful, debilitating and unpredictable episodes of swelling of the abdomen, larynx, face and extremities, among other areas of the body.
CSL said that 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.
It said that Patients taking once monthly garadacimab experienced a statistically lower monthly attack rate versus placebo, resulting in a mean attack rate reduction of 86.5 per cent and a median attack rate reduction of 100 per cent.
"Overall, during the six-month trial, a majority (61.5 per cent) of patients taking garadacimab were attack-free whereas no patients on the placebo arm were attack-free; 74.4 per cent of patients taking garadacimab achieved ≥90 per cent attack reduction versus the run-in period," said CSL.
“Targeting FXIIa and the HAE cascade from the start, as opposed to intervening downstream, is an innovative treatment approach that could help stop the process in its tracks,” said Dr Timothy Craig, Tenured Professor of Medicine, Pediatrics and Biomedical Sciences at Penn State University and Principal Investigator of the study.
“The Phase 3 data we are presenting support the potential use of garadacimab as a prophylactic therapy for HAE.”
"The data being shared at AAAAI showcase the efficacy and safety profile of garadacimab administered as a convenient monthly subcutaneous injection. The clinical trial results support garadacimab as a novel, first-in-class potential treatment that could offer a significant benefit to patients with HAE,” said Catherine Milch, Vice President R&D Immunology and CSL. “Garadacimab represents the next chapter in delivering on our promise to bring disruptive innovation and treatment options to patients living with rare diseases.”
CSL said it plans regulatory submissions to global health authorities later this calendar year seeking the approval of garadacimab.