CSL behring presents results for garadacimab as preventive treatment in hereditary angioedema

CSL Behring, a global biotherapeutics leader, today announced results of a Phase 2 clinical trial for garadacimab (previously known as CSL312), an investigational novel Factor XIIa-inhibitory monoclonal antibody (FXIIa mAb) in development as a preventive treatment in hereditary angioedema (HAE). The data, presented at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress 2020, showed that the study met its primary endpoint, demonstrating reduced number of attacks compared to placebo in patients with HAE. Mean percentage reductions were 88.68%, 98.94%, and 90.50% in three garadacimab groups – 75, 200, and 600 mg subcutaneous (SC) – versus placebo. The study also showed garadacimab to be well-tolerated. 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. Garadacimab inhibits the plasma protein, FXIIa. FXIIa initiates the cascade of events that lead to edema formation. By targeting FXIIa, garadacimab can prevent the initiation of this cascade.

Additionally, last month, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to garadacimab as an investigational therapy for the prevention of bradykinin-mediated angioedema, which includes both hereditary and non-hereditary (acquired) angioedema. The FDA Office of Orphan Products Development (OOPD) grants orphan drug designation to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 U.S. patients. The designation qualifies companies with a range of incentives, including the potential for marketing exclusivity upon approval.

“The attacks that HAE patients experience can be very frightening, and clinicians want to do anything in their power to reduce the frequency of these attacks, lessen the need for rescue medicine and simplify treatment,” said lead study investigator Timothy Craig, D.O., Allergy, Asthma and Immunology, Department of Medicine and Pediatrics, Penn State Hershey, Hershey, PA. “The findings of this study are very encouraging and we look forward to further research assessing the safety and efficacy of garadacimab.”

Affecting about one in 40,000-50,000 people globally, attacks of HAE can happen suddenly and with little warning. Many people with HAE need both preventive treatment as well as on-demand (or “rescue”) therapies to treat an attack in progress, both of which are associated with frequent dosing.

“Consistent with our more than 40-year commitment to HAE therapeutic innovation, garadacimab represents a potentially first-in-class agent that utilizes a unique approach as a preventive treatment in HAE,” said Mittie Doyle, M.D., Vice President, Research and Development, Immunology Therapeutic Area at CSL Behring. “We are encouraged by the promising garadacimab data as well as the orphan drug designation milestone and look forward to advancing the clinical program to continue to deliver on our promise and improve the lives of people living with HAE.”  – PRNewswire