BioCryst Pharmaceuticals, Inc. announced new data from the APeX-S and APeX-2 clinical trials which evaluated oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) demonstrating sustained reductions in attack rates and improvement in quality of life (QoL) among patients living with HAE, highlighting its profile as a well-tolerated, effective and convenient prophylactic HAE therapeutic option. The company also announced additional analyses from new real-world data that further demonstrate a meaningful reduction in attack rates experienced by patients on ORLADEYO, in addition to findings from a survey that underscore a significant disease and treatment burden among pediatric HAE patients, as reported by their caregivers. The data are being presented at the 2023 American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting, which is being held in San Antonio, Texas, from February 24-27, 2023.

BioCryst AAAAI 2023 Presentation Highlights: The posters being presented at AAAAI include analyses from the APeX-2 and APeX-S clinical studies, as well as real-world data from patients taking ORLADEYO in the United States. APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group, three-part study evaluating ORLADEYO versus placebo for the prevention of HAE attacks in patients with HAE Type I or Type II. APeX-S was a Phase 2, open label, international study evaluating the safety and effectiveness of ORLADEYO 110 mg once daily (QD) and 150 mg QD in patients with HAE Type I or Type II for up to 96 weeks in the US and 240 weeks in all other countries.

Overall, treatment-emergent adverse events (TEAEs) reported in APeX-2 and APeX-S were mild and transient, indicating that ORLADEYO was generally well tolerated.