KING OF PRUSSIA, PA — Global biotechnology leader CSL (ASX: CSL; USOTC: CSLLY) yesterday presented results from the pivotal placebo-controlled Phase 3 VANGUARD clinical trial of garadacimab (CSL312). Garadacimab is CSL’s investigational first-in-class monoclonal antibody being developed as a long-term prophylactic treatment for patients with hereditary angioedema (HAE). 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. The data were presented during the 2023 American Academy of Allergy Asthma & Immunology (AAAAI) Annual Meeting, February 24-27, in San Antonio, Texas.
“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.”
Key Data from the Phase 3 VANGUARD Trial
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 ≥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.
“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, CSL. “Garadacimab represents the next chapter in delivering on our promise to bring disruptive innovation and treatment options to patients living with rare diseases.”
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.
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