Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan

Summary

This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan.

Timeline

Start

2018-12-27

Primary completion

2021-07-08

Completion

2021-07-08

Outcome

Sources disagree · no verdict is set; each source's reading is shown below

registry analysis (superiority test); Berotralstat 110mg Once Daily vs Placebo; p = 0.181 reads as mixed primary results NCT03873116 ↗

registry analysis (superiority test); Berotralstat 150mg Once Daily vs Placebo; p = 0.003; negative binomial regression model -49.1 (95% CI -67.5 to -20.4) reads as mixed primary results NCT03873116 ↗

release “APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks” reads as met primary endpoint biocryst.com ↗

release “The APeX-J trial met its primary endpoint” reads as met primary endpoint biocryst.com ↗

Publications

• Honda D, Hide M, Fukuda T, Koga K, Morita E, Moriwaki S, Sasaki Y, Suzuki Y, Collis P, Johnston DT, Tomita D, Desai B, Ohsawa I. Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial. World Allergy Organ J. 2024 Mar 2;17(3):100882. doi: 10.1016/j.waojou.2024.100882. eCollection 2024 Mar.
• Farkas H, Balla Z. A review of berotralstat for the treatment of hereditary angioedema. Expert Rev Clin Immunol. 2023 Feb;19(2):145-153. doi: 10.1080/1744666X.2023.2150611. Epub 2022 Nov 29.
• Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
• Ohsawa I, Honda D, Suzuki Y, Fukuda T, Kohga K, Morita E, Moriwaki S, Ishikawa O, Sasaki Y, Tago M, Chittick G, Cornpropst M, Murray SC, Dobo SM, Nagy E, Van Dyke S, Reese L, Best JM, Iocca H, Collis P, Sheridan WP, Hide M. Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial. Allergy. 2021 Jun;76(6):1789-1799. doi: 10.1111/all.14670. Epub 2020 Dec 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	berotralstat	Small molecule	110 mg	Oral
Subject	berotralstat	Small molecule	150 mg	Oral

Indications