BioCryst (NASDAQ:BCRX) Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new data from the APeX-S clinical trial, which evaluated oral, once-daily ORLADEYO (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE), showing sustained reduction in disease burden for patients across multiple subgroups through 96 weeks of treatment. The data are being presented at the 13th C1-inhibitor Deficiency & Angioedema Workshop, which is being held in Budapest, Hungary, from May 4-7, 2023.
We continue to generate evidence that further strengthens confidence in ORLADEYO as a safe, effective and more convenient therapeutic option for people living with HAE. These data demonstrate how our oral, once-daily prophylactic treatment can consistently help patients spend more of their days without disruption from HAE attacks, regardless of their age, gender and experience with prior prophylactic treatment, including among pediatric patients aged 12-17. These additional analyses of long-term data reflect our continued commitment to define the potential benefits and bring ORLADEYO to as many HAE patients around the world as possible, said Dr. Ryan Arnold, chief medical officer of BioCryst.
BioCryst C1-inhibitor Deficiency & Angioedema Workshop Presentation Highlights
The oral presentation at the C1-inhbitor Deficiency & Angioedema Workshop will include post-hoc analyses from the APeX-S clinical study. APeX-S was a Phase 2, open label, international study evaluating the safety and effectiveness of ORLADEYO 110 mg and 150 mg once daily (QD) in patients with HAE Type I or Type II for up to 96 weeks in the United States and 240 weeks in all other countries. Overall, treatment-emergent adverse events (TEAEs) reported in APeX-S were mild and transient, indicating that ORLADEYO was generally well tolerated.
Attack-free status across subgroups of patients with hereditary angioedema after 96 weeks of berotralstat treatment: results from the APeX-S trial; Saturday, May 6, 8:00-8:15 am CEST
This analysis assessed the attack-free status of patients receiving ORLADEYO 150 mg through 96 weeks in APeX-S (n=287), stratified by baseline age, gender, and prior HAE prophylaxis treatment. The three subgroups included patients who were 12-17 (n=23), 18-64 (n=253) and 65 years of age (n=11), female (n=180) and male (n=107) and had prior experience with androgens (n=142) or C1-inhibitors (n=105).
Overall, a reduction in mean adjusted HAE attack rates was observed compared to Weeks 0-24 in patients treated with ORLADEYO 150 mg QD. Mean adjusted HAE attack rates decreased from 1.08 from Weeks 0-24 (n=287) to 0.69 and 0.59 from Weeks 25-48 (n=214) and Weeks 49-96 (n=158), respectively.
Attack-free status was consistently high through 96 weeks of treatment with ORLADEYO 150 mg regardless of patients age, gender and prior prophylactic treatment.
Patients aged 12-17, 18-64 and 65 remained attack free an average of 97, 94 and 98 percent of days, with a mean (SEM) of 196.8 (30.4), 99.3 (9.5) and 275.9 (104.4) days and a maximum duration of 461, 1,101 and 1,182 days between attacks, respectively.
Female patients remained attack free an average of 94 percent of days, with a mean (SEM) of 106 (11.8) days and a maximum duration of 1,182 days between attacks. Male patients remained attack free an average of 94 percent of days, with a mean (SEM) of 127.2 (17.4) days and a maximum duration of 1,101 days between attacks.
Patients who had prior treatment with androgens remained attack free an average of 93 percent of days, with a mean (SEM) of 87.1 (12.7) days and a maximum duration of 1,026 days between attacks. Patients who had prior treatment with C1-inhibitors remained attack free an average of 91 percent of days, with a mean (SEM) of 75.8 (9.8) days and a maximum duration of 584 days between attacks.
Long-term prophylaxis with ORLADEYO 150 mg led to a durable treatment effect and sustained reduction in disease burden through 96 weeks of treatment regardless of baseline characteristics including patients age, gender and prior prophylactic treatment.