New data from the APeX-2 trial shows that 150 mg of oral, once-daily berotralstat (BCX7353) for prophylaxis of HAE attacks reduced patients’ monthly use of standard of care (SoC) on-demand medicine by 53.6 percent compared to placebo, and reduced the number of HAE attacks requiring acute SoC treatment by 49.2 percent compared to placebo. Additional data show that the percentage of HAE attacks requiring re-treatment with multiple doses of on-demand therapy was lower for patients receiving berotralstat (150 mg) than placebo.
“Patients receiving berotralstat had fewer attacks, treated fewer attacks, experienced less severe attacks and used less on-demand medication compared to placebo. These data from APeX-2 provide further evidence that HAE patients are seeing significant benefits from oral, once-daily berotralstat,” says Dr. William Sheridan, chief medical officer of BioCryst Pharmaceuticals, Inc.
In APeX-2, patients experienced a rapid and sustained decrease in their attack frequency over 48 weeks. Thirty patients who were randomized to 150 mg of berotralstat at the beginning of the trial and completed 48 weeks of therapy had a baseline mean attack rate of 2.9 attacks per month, which declined to 1.4 attacks per month after one month and to 1.0 attacks per month at month 12. APeX-2 patients who switched from placebo to 150 mg of berotralstat after week 24 saw dramatic and sustained reductions in their HAE attack rate. Their mean attack rate dropped to 0.5 attacks per 28 days at month seven and to 0.4 attacks per 28 days at month 12.
An integrated 48-week analysis across both the APeX-2 and APeX-S trials showed berotralstat was safe and generally well tolerated in a total of 342 patients with a total of 232 patient-years of daily oral dosing. The most frequent adverse drug reactions were mild-to-moderate gastrointestinal events that were brief in duration and self-limited.
APeX-2 is a randomized, double-blind, placebo-controlled, three-arm trial testing two dose levels of orally administered once-daily berotralstat (110 mg and 150 mg) for prevention of angioedema attacks. The trial enrolled 121 patients with Type I and II HAE in the United States, Canada and Europe. Following completion of the 24-week analysis period, patients continued on study drug in an ongoing extension phase of APeX-2 through 48 weeks. Patients randomized to placebo for 24 weeks were re-randomized to receive one of the two doses of study drug in the extension phase of the trial. Patients who complete 48 weeks may continue in the trial on open-label berotralstat.