Berotralstat Consistently Demonstrates Reductions in Attack Frequency in Hereditary Angioedema (HAE) Irrespective of Baseline Attack Rate: Subgroup Analysis from the APeX-2 Trial

Berotralstat is an oral plasma kallikrein inhibitor in development for HAE attack prophylaxis. characterized by unpredictable, episodic attacks; some patients experience frequent attacks without treatment. This analysis sought to understand whether baseline frequency correlates with responder rates berotralstat. 121 were randomized berotralstat 110 mg:150 mg:placebo daily 24 weeks a phase 3 double-blind, placebo-controlled study (NCT03485911). post hoc examined the reduction of rate Cohort 1: < 2 attacks/month; 2: ≥2 4 3: ≥4 attacks/month. In 1, median per month 1.3 (berotralstat 150 mg; n= 10) and 1.7 (placebo; 12) which declined 0.41 1.3, respectively. 2, 2.7 n=20) 3.1 n=21) 1.2 2.7, For 3, 5.2 mg (n=10) 4.5 placebo (n=6) 1.9 2.5, Cohorts treatment resulted ≥50% relative 70%, 55%, 50% patients, addition, 60%, 45%, respectively, had ≥70% rate. These results demonstrate consistent berotralstat, adding potential prophylactic option armamentarium physicians.