공고 • Jun 13
BioCryst Pharmaceuticals Presents New Clinical Data and Real-World Evidence for HAE Portfolio at the European Academy of Allergy and Clinical Immunology Annual Meeting
BioCryst Pharmaceuticals, Inc. announced new clinical data and real-world evidence for ORLADEYO (berotralstat), the first and only targeted oral prophylactic therapy for patients with hereditary angioedema (HAE) aged 2 and older, in addition to new data from the Phase 1b/2 multicenter, dose-ranging, open-label ALPHA-STAR study of navenibart, an investigational, long-acting, monoclonal antibody plasma kallikrein inhibitor for prophylaxis to prevent attacks of HAE. These data will be featured across multiple poster presentations during the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in Istanbul, Turkey, from June 12-15. Updated analysis of 48-week data from the ongoing APeX-P study, the largest trial of long-term prophylaxis (LTP) in pediatric patients with HAE, evaluating once-daily ORLADEYO in HAE patients aged 2 to. The number of HAE attacks requiring professional care decreased from 22 during the 12-week SOC period to 3 over 12 weeks of ORLADEYO treatment; this was sustained throughout the treatment period, with a trended decrease in level of professional care required from emergency department or urgent care treatment to physicians’ office, and further reduction to 0 attacks during Week 37-48. No significant safety concerns were identified over the 48-week treatment period. Additionally, a new post hoc analysis of the Phase 1b/2 ALPHA-STAR study of navenibart evaluating clinical outcomes across patient subgroups defined by baseline attack rate, body mass index (BMI), and age will be featured as a poster presentation (Poster D3.438). The analysis demonstrated that investigational navenibart consistently reduced HAE attacks, with results as follows: Reductions in overall HAE attack rate were observed across subgroups defined by baseline attack rate, BMI, and age. Following treatment, reductions in clinically relevant HAE outcomes were observed across analyzed subgroups, including reductions in moderate or severe attacks, and in baseline attack rate subgroup analyses, and reduced on-demand medication use. Reductions in the number of moderate or severe attacks were also observed with treatment across all BMI subgroups. Navenibart was previously shown to be well tolerated with no severe or serious treatment-emergent adverse events (TEAEs) reported and few injection site reactions. The most common TEAEs were headache, nasopharyngitis, and urinary tract infection. Together with the primary findings from Phase 1b/2 ALPHA-STAR, this analysis supports the ongoing Phase 3 evaluation of navenibart in the ALPHA-ORBIT trial as a potential long-acting therapeutic option for the broad HAE population. Through a comprehensive real-world evidence (RWE) generation program, BioCryst continues its commitment to generating meaningful, practice-informing research to demonstrate the value of long-term prophylaxis with ORLADEYO in routine clinical practice across the diverse HAE patient community. In addition to clinical data generated in clinical trials, RWE across multiple studies demonstrated ORLADEYO’s ability to achieve sustained reductions in HAE burden. Benefits were observed in adolescent and adult patients, including those switching to ORLADEYO from other LTP therapies, and translated into fewer HAE attacks, reduced healthcare resource utilization, and high patient satisfaction. This RWE will be presented in the following posters: Real-World Patient Characterization, Prior Long-Term Prophylactic Prescribing Patterns, and Treatment Outcomes for Adults on Berotralstat with Hereditary Angioedema in Japan; poster D1.407; Reductions in Hereditary Angioedema Attacks among Patients with C1 Esterase Inhibitor Deficiency Who Switched from Another Long-Term Prophylaxis to Berotralstat; poster D2.360; Hereditary Angioedema Attack Rates among Patients with Normal C1 Esterase Inhibitor Before and After Switching from Another Long-Term Prophylaxis to Berotralstat; poster D2.357; Reductions in Healthcare Resource Utilization in Adolescents with Hereditary Angioedema on Berotralstat; poster D2.361; Hereditary Angioedema Attack Frequency and Severity According to Individuals Taking Berotralstat for Long-Term Prophylaxis; poster D3.324.