BioCryst Announces New Clinical Data and Therapy for HAE
BioCryst announced new clinical data and real-world evidence for c, the first and only targeted oral prophylactic therapy for patients with hereditary angioedema, or 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, or 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 in pediatric patients with HAE, evaluating once-daily Orladeyo in HAE patients aged 2 to less than12 years will be featured in a poster presentation. Analysis of 48-week trial data showed that treatment with Orladeyo was associated with early and sustained reductions in rate and number of HAE attacks requiring on-demand treatment and professional care, with results as follows. The median adjusted HAE attack rate requiring on-demand treatment decreased from 0.691 attacks/month during the 12-week standard of care period to 0.169 attacks/month during the 48-week Orladeyo treatment period. 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, and age will be featured as a poster presentation. 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 reported and few injection site reactions. The most common TEAEs were headache, nasopharyngitis, and urinary tract infection.
