BioCryst Pharmaceuticals, Inc. announced new long-term efficacy and safety data from the APeX-2 clinical trial evaluating oral, once-daily ORLADEYO® for the prophylactic treatment of hereditary angioedema showing sustained reductions in attack rates and improvement in quality of life among patients living with HAE, regardless of their baseline attack rates and initial responses to ORLADEYO. 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 . 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.

At Week 48 and thereafter, all patients continued on ORLADEYO 150 mg. 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.

In APeX-2, ORLADEYO was safe and generally well tolerated, with no drug-related serious adverse events reported. This analysis stratified all 21 patients by their initial reduction in HAE attack rate from baseline to Week 24 in three groups: Group A, Group B and Group C. To note, Group C was a subset of Group B. A sustained reduction in HAE attack rates was observed from baseline to Week 96 across all three groups of patients. Group A had a mean decrease of 2.3 attacks/month, Group B had a mean decrease of 2.5 attacks/month and Group C had a mean decrease of 2.6 attacks/month.

The % age of attack-free days across all patients for the entire study duration was 94%, demonstrating ORLADEYO is an effective oral HAE prophylactic treatment even in patients who may have a lower initial response. This analysis stratified all 21 patients based on baseline attack rate: Group 1, Group 2 and Group 3. A >80 % reduction in mean attack rates was observed at Week 96 of treatment regardless of the patients' baseline attack rates. At Week 96, median attack rates were 0.0 regardless of baseline attack rate.

These data demonstrate ORLADEYO is an effective prophylactic therapy for patients with HAE regardless of baseline attack rate. important difference was defined as a change of six points in total score. A mean of 77% of patients reported clinically meaningful improvements using the AE-QoL total scores, including improvements observed as early as Week 4, and at each time point through 96 weeks of treatment.

The largest improvement was observed in the functioning domain with a mean improvement of 33.4 points at Week 96, suggesting patients reported less impairment in their day-to-day activities while on ORLADEYO 150 mg. The improvement in total AE-QoL scores and the % age with an MCID in these long-term results show that patients continue to experience QoL improvements compared to baseline over time with ORLADEYO. All posters are available to meeting registrants and will be on display in the poster hall in the Phoenix Convention Center during the meeting.