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Randomized Controlled Trial
. 2021 Jun;9(6):2305-2314.e4.
doi: 10.1016/j.jaip.2021.03.057. Epub 2021 Apr 15.

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)

H James Wedner  1 , Emel Aygören-Pürsün  2 , Jonathan Bernstein  3 , Timothy Craig  4 , Richard Gower  5 , Joshua S Jacobs  6 , Douglas T Johnston  7 , William R Lumry  8 , Bruce L Zuraw  9 , Jessica M Best  10 , Heather A Iocca  10 , Sharon C Murray  10 , Bhavisha Desai  10 , Eniko Nagy  10 , William P Sheridan  11 , Sorena Kiani-Alikhan  12
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Free article
Randomized Controlled Trial

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)

H James Wedner et al. J Allergy Clin Immunol Pract. 2021 Jun.
Free article

Abstract

Background: Berotralstat (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 a favorable safety and tolerability profile.

Objective: Evaluate berotralstat safety, tolerability, and effectiveness over 48 weeks.

Methods: APeX-2 is a phase 3, parallel-group, multicenter trial (NCT03485911) in patients with HAE due to C1 esterase inhibitor deficiency. Part 1 was double-blind and placebo-controlled, with patients randomized to 24 weeks of berotralstat 150 mg, 110 mg, or placebo. In part 2, patients continued berotralstat the same dose or, if initially randomized to placebo, were rerandomized to berotralstat 150 mg or 110 mg through weeks 24 to 48. The primary end point was safety and tolerability.

Results: One hundred eight patients received 1 or more doses of berotralstat in part 2. Treatment-emergent adverse events (TEAEs) occurred in 30 of 39 patients (77%) in the placebo group during part 1, and 25 of 34 patients (74%) re-randomized from placebo to berotralstat 110 mg or 150 mg in part 2, with drug-related TEAEs in 13 of 39 (33%), and 11 of 34 (32%) in the same groups. Most TEAEs were mild or moderate, with no serious drug-related TEAEs. The most common TEAEs were upper respiratory tract infections, abdominal pain, diarrhea, and vomiting. Mean (±standard error of the mean) monthly attack rates at baseline and week 48 were 3.06 (±0.25) and 1.06 (±0.25) in the berotralstat 150mg 48-week group and 2.97 (±0.21) and 1.35 (±0.33) in the berotralstat 110mg 48-week group.

Conclusions: The safety, tolerability, and effectiveness of berotralstat were maintained over 48 weeks of treatment.

Keywords: BCX7353; Berotralstat; C1 inhibitor; HAE; Hereditary angioedema; Kallikrein inhibitor; Oral therapy; Phase 3 trial; Prophylaxis.

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