Berotralstat Reduces Use of On-demand Medication in Hereditary Angioedema (HAE) Patients Previously Treated with Prophylactic Therapies

The goal of HAE prophylaxis is to minimize the number attacks and associated disease burden, including treatment with on-demand medications. Berotralstat an oral once-daily selective plasma kallikrein inhibitor that has been shown reduce attack frequency in a Phase 3 study (NCT03485911). This analysis evaluates reduction medication patients prior prophylaxis. A double-blind, placebo-controlled (APeX-2) randomized berotralstat 110 mg (n=41):150 (n=40):placebo (n=40) daily for 24 weeks Part 1. For this post hoc analysis, were grouped according their prophylaxis: C1 esterase (C1-INH), androgen, or no prophylactic medication. Prior C1-INH androgen categories are not mutually exclusive. In (berotralstat 150 n=21; placebo n=16), rate use was significantly reduced vs (-59.2%, p=0.002). Similar reductions noted n=22; n=25) (-51.8%; p=0.004) without n=10; n=10) (-71.2%; p=0.019). corresponds approximately 2.2 fewer doses per month compared use, 1.6 those 1.4 Prophylactic resulted significant irrespective treatment.