BioCryst Reports 96-week Data from APeX-2 Showing ORLADEYO®(berotralstat) Reduced HAE Attack Rate by 80 Percent from Baseline

—Multiple data presentations at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2021 highlight durability of effect and long-term safety of ORLADEYO—

RESEARCH TRIANGLE PARK, N.C., July 10, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that hereditary angioedema (HAE) patients who were randomized to receive 150 mg of oral, once-daily ORLADEYO® (berotralstat) at the start of the APeX-2 trial had an 80 percent average reduction in their mean attack rate per month during weeks 25-96 of the trial, compared to baseline. Median attack rates also decreased from 2.7 attacks/month at baseline to 0.0 attacks per month in 16 of 17 months through the same period.

ORLADEYO was generally well-tolerated during the treatment period with fewer drug-related adverse events reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24) and part 2 (weeks 25-48). Eighty-one percent of the patients who entered part 3 completed the trial.

“The long-term data we now see from two years of therapy in the clinical program reinforces the substantial, sustained reduction in HAE attacks patients experienced with ORLADEYO. These results are consistent with the experience many patients are having in the real world since we launched ORLADEYO, which is a key driver for the strong patient demand we are seeing as patients switch from injectable prophylactic agents and injectable acute-only therapies to oral, once-daily ORLADEYO for control of their HAE attacks,” said Dr. William Sheridan, chief medical officer of BioCryst.

The data are scheduled to be presented in an oral presentation on Monday, July 12, 2021 at 2:15pm CET at the EAACI Hybrid Congress 2021.

Additional Presentations of New ORLADEYO Data at EAACI

On-demand medication use was reduced in HAE patients treated with ORLADEYO (150 mg) in APeX-2

• In HAE patients taking oral, once-daily ORLADEYO 150 mg who had a ≥50 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was a 78 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.1 fewer doses of on-demand medication per month. In patients who had a ≥70 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was an 85 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.2 fewer doses of on-demand medication per month.
• The data were presented in poster #250 at the EAACI Hybrid Congress 2021.

ORLADEYO demonstrated consistently low attack rates in adolescent patients in APeX-S

• In an analysis of adolescent patients (ages 12-17) treated with oral, once-daily ORLADEYO 150 mg in the open-label safety study, APeX-S, the mean (SEM) attack rate at week 4 was 0.4 attacks/month, which was generally sustained through week 48.
• Median attack rates in these adolescents were 0.0 attacks/month throughout the 48 weeks of treatment.
• Greater than 70 percent of patients were attack-free in weeks 4 to 48.
• ORLADEYO was generally well-tolerated in APeX-S.
• The data were presented in poster #336 at the EAACI Hybrid Congress 2021.

ORLADEYO demonstrated consistently low HAE attack rates during COVID-19

• Stress is a documented trigger for HAE attacks and recently published physician and patient survey data shows an increase in patient-reported HAE attack rates due to an increase in stress related to the COVID-19 pandemic (pre-COVID: 1.5 attacks/3-months vs during COVID: 4.4 attacks/3-months).1
• In an analysis of HAE patients taking oral, once-daily ORLADEYO 150 mg in the APeX-S trial, monthly HAE attack rates, pre-COVID and during COVID, remained consistently low,