BioCryst Presents Results on HAE Treatment and Patient Care at Global Symposium

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the presentation of six posters, including the first interim real-word evidence from the APeX-N trial, and new data highlighting the value of shared decision making (SDM) between healthcare providers (HCPs) and their hereditary angioedema (HAE) patients to provide optimal patient outcomes.

The company is presenting the posters at the 7th Bradykinin Symposium in Berlin from September 5-6, 2024.

• APeX-N interim results: Oral berotralstat for HAE prophylaxis in Europe
• HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making
• Adverse health outcomes and perspectives of androgen use in HAE
• Evaluation of adherence to berotralstat in patients with hereditary angioedema
• Tolerability and effectiveness of berotralstat for long-term prophylaxis in HAE
• Effectiveness and safety of berotralstat in HAE with normal C1-inhibitor

APeX-N interim results

APeX-N is a European multi-center observational study assessing the safety (primary objective), effectiveness and quality of life (secondary objectives) of berotralstat 150 mg in routine clinical use. This interim analysis included 56 patients from the United Kingdom, France, Germany and Sweden.

Non-serious gastrointestinal adverse events were reported in 12.5 percent of patients. Seven percent (n=4) of patients discontinued treatment (three due to unsatisfactory response, one to participate in a clinical trial). One patient had a severe HAE attack but continued treatment.

"These initial data from APeX-N in Europe reinforce and closely replicate the clinical trial and real-world evidence of berotralstat as the first oral prophylaxis for HAE," said Dr. Sorena Kiani, consultant immunologist at Royal Free London NHS Foundation.

HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making

This study, conducted in Germany, explored the dynamics between HCPs and patients in HAE management, identifying barriers to SDM and strategies to improve it. Ten HCPs participated in 60 minute interviews and simulated patient consultations. Eight HAE patients participated in 30 minute interviews. Participants then convened in structured focus groups to discuss their findings.

The participants identified a need for enhanced HCP awareness of patient perspectives, more comprehensive HCP-patient conversations and improved education about HAE treatment management.

"The findings from this study support the need for further insights to develop future guidance and HAE management strategies to facilitate successful shared decision making and improved patient quality of life," said Dr. Emel Aygören-Pürsün, department for children and adolescents, University Hospital Frankfurt, Germany.