Randomized Trial of the Efficacy and Safety of Berotralstat (BCX7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: Results of APeX-2 Through 48 Weeks (Part 2)

BackgroundBerotralstat (BCX7353) is a recently approved, oral, once-daily kallikrein inhibitor for hereditary angioedema (HAE) prophylaxis. In the APeX-2 trial, berotralstat reduced HAE attack rates over 24 weeks, with favorable safety and tolerability profile.ObjectiveEvaluate safety, tolerability, effectiveness 48 weeks.MethodsAPeX-2 phase 3, parallel-group, multicenter trial (NCT03485911) in patients due to C1 esterase deficiency. Part 1 was double-blind placebo-controlled, randomized weeks of 150 mg, 110 or placebo. part 2, continued same dose or, if initially placebo, were rerandomized mg through 48. The primary end point tolerability.ResultsOne hundred eight received more doses 2. Treatment-emergent adverse events (TEAEs) occurred 30 39 (77%) placebo group during 1, 25 34 (74%) re-randomized from drug-related TEAEs 13 (33%), 11 (32%) groups. Most mild moderate, no serious TEAEs. most common upper respiratory tract infections, abdominal pain, diarrhea, vomiting. Mean (±standard error mean) monthly at baseline week 3.06 (±0.25) 1.06 150mg 48-week 2.97 (±0.21) 1.35 (±0.33) 110mg group.ConclusionsThe maintained treatment. Berotralstat profile. Evaluate weeks. tolerability. One group.