Abstract oral presentations: Session 1

The Scientific Track then turned to the first of two oral presentation sessions on HAE-related research.

1. Clinical Validation of a Novel Kinin Biomarker Assay to Characterize Bradykinin-Mediated Angioedema. Professor Henriette Farkas, Hungary

Prof Farkas presented on the validation of a novel biomarker assay to characterize bradykinin-mediated angioedema, which she described as probably a game-changer in the diagnosis of bradykinin.

The need for further diagnostic tools is most pressing, according to Prof Farkas, in HAE with normal C1 inhibitor. However, detecting biomarkers for any disease related to bradykinin is a major challenge, as its half-life is less than 30 seconds.

Prof Farkas said the study examined the feasibility of tracking bradykinin using a new method. They recruited 13 patients with HAE with C1-inhibitor deficiency and another with a plasminogen genetic variant (HAE with normal C1-inhibitor deficiency). The new procedure used a cold process to effectively measure bradykinin levels, which were elevated in both types of HAE patients compared with healthy controls.

2. Results From the ALPHA-STAR Trial, A Phase 1b/2 Single and Multiple Dose Study to Assess the Safety, Tolerability, Clinical Activity, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Navenibart in Participants with Hereditary Angioedema (HAE). Dr William Lumry, United States of America

The following oral presentation was on data on a potential new treatment for HAE, called navenibart. Dr Lumry gave a brief history of HAE treatment, noting that on-demand intravenous (IV) and subcutaneous (sc) treatment has been augmented by long-term IV and sc injections to prevent attacks, as well as daily oral treatment. This study, he said, examined the potential to further reduce patients’ treatment burden by using a medicine that lasts longer in the body.

The trial was, he said, a small one. Still, it showed that navenibart was well tolerated. Out of 16 patients across 3 different cohorts receiving treatment, the most common side effect was a blocked nose or headache, and treatment-related side effects were primarily injection-site reactions. The therapy reduced attack frequency effectively.

Dr Lumry concluded that navenibart demonstrated a favorable safety profile and no dose-related treatment-emergent side effects. Navenibart showed a 94 to 100% median reduction in monthly attack rates compared to the patient’s baseline, and has the potential to be an effective and safe prophylactic medication for HAE with administration every 4 or 6 months.