BioCryst Presents New Data Demonstrating Sustained Reductions in Attack Rates and Improvement in Quality of Life Among HAE Patients Following Long-Term Treatment with ORLADEYO® (berotralstat)

— Analysis from APeX-2 showed 94 percent attack-free days across all patients who completed 96 weeks of treatment —

RESEARCH TRIANGLE PARK, N.C., Feb. 24, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new long-term efficacy and safety data from the APeX-2 clinical trial evaluating oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) showing sustained reductions in attack rates and improvement in quality of life (QoL) among patients living with HAE, regardless of their baseline attack rates and initial responses to ORLADEYO.

The data are being presented at the 2022 American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting, which is being held live in Phoenix, Arizona, and virtually, from February 25-28, 2022.

“The long-term data from APeX-2 show that HAE patients in our clinical program had an experience consistent with what we are seeing commercially in the real world; that ORLADEYO provides noteworthy, sustained, consistent reductions in HAE attack rates that persist and maintain over time, resulting in meaningful quality of life improvements,” said Dr. William Sheridan, chief medical officer of BioCryst.

“The 96-week data from APeX-2 showcase the durable, long-term efficacy of ORLADEYO and build on the strong reductions in attack rates that have previously been reported, with all patients experiencing an average of 94 percent attack-free days at Week 96. ORLADEYO can be an effective prophylactic therapy for HAE patients regardless of their baseline attack rate,” said Dr. Emel Aygören-Pürsün, specialist in internal medicine at the division of oncology, hematology and hemostaseology at the department for children and adolescents of the University Hospital Frankfurt.

BioCryst AAAAI 2022 Presentation Highlights

APeX-2 included 121 HAE patients who were randomized 1:1:1 to ORLADEYO 110 mg or 150 mg, or placebo, once daily for 24 weeks (part 1 of the study). At Week 24, patients on ORLADEYO continued on the same dose and placebo patients were re-randomized to ORLADEYO 110 mg or 150 mg for another 24 weeks (part 2 of the study). At Week 48 and thereafter, all patients continued on ORLADEYO 150 mg (open-label phase).

The 96-week safety and efficacy data were previously reported in July 2021. These additional analyses from APeX-2, as reported in the posters at AAAAI, evaluated the long-term efficacy of ORLADEYO 150 mg in patients who completed 96 weeks of treatment (n=21). In APeX-2, ORLADEYO was safe and generally well tolerated, with no drug-related serious adverse events reported.

• Sustained Reductions in Hereditary Angioedema (HAE) Attack Rates Observed over 96 Weeks of Oral Berotralstat Treatment Regardless of Initial Response (Poster #490)
  • This analysis stratified all 21 patients by their initial reduction in HAE attack rate from baseline to Week 24 in three groups: Group A (