P154 Safety and Tolerability of Once-Daily Oral Kallikrein Inhibitor Bcx7353 in Phase 3 Apex-2 Hae Study

Riedl, Marc A.; Lumry, William R.; Banerji, Aleena; Aygören‐Pürsün, Emel; Bernstein, J.; Maurer, Mari; Cicardi, Marco; Christiansen, S.; Zuraw, Bruce L.; Dobo, Sylvia; Cornpropst, Melanie; Iocca, Heather A.; Nagy, Emese; Murray, Sonya; Collis, P.; Sheridan, W.; Johnston, Dennis A.

doi:10.1016/j.anai.2019.08.258

Cited by 3 publications

(1 citation statement)

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“…The Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2) study, a phase 3, randomized, double-blind, placebo-controlled study showed a 44% reduction in attack rate compared with placebo [ 38 ]. Notably, gastrointestinal side effects occurred frequently across all three study arms (42% in the group that received 110 mg, 50% in the 150 mg arm, and up to 36% in the placebo group) [ 39 ]. An open-label long-term safety study assessing the dose of 125 mg once daily is currently being conducted and an application for EMA approval has been submitted [ 40 ].…”

Section: Introductionmentioning

confidence: 99%

Current and Prospective Targets of Pharmacologic Treatment of Hereditary Angioedema Types 1 and 2

Fijen

Bork

Cohn

2021

Clinic Rev Allerg Immunol

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Hereditary angioedema (HAE) is a rare disease that causes episodic attacks of subcutaneous and submucosal edema, which can be painful, incapacitating, and potentially fatal. These attacks are mediated by excessive bradykinin production, as a result of uncontrolled activation of the plasma kallikrein/kinin system, which is caused by a C1 esterase inhibitor deficiency or dysfunction in HAE types 1 and 2, respectively. For many years, treatment options were limited to therapies with substantial adverse effects, insufficient efficacy, or difficult routes of administration. Increased insights in the pathophysiology of HAE have paved the way for the development of new therapies with fewer side effects. In the last two decades, several targeted novel therapeutic strategies for HAE have been developed, for both long-term prophylaxis and on demand treatment of acute attacks. This article reviews the advances in the development of more effective and convenient treatment options for HAE and their anticipated effects on morbidity, mortality, and quality of life. The emergence of these improved treatment options will presumably change current HAE guidelines, but adherence to these recommendations may become restricted by high treatment costs. It will therefore be essential to determine the indications and identify the patients that will benefit most from these newest treatment generations. Ultimately, current preclinical research into gene therapies may eventually lead the way towards curative treatment options for HAE. In conclusion, an increasing shift towards the use of highly effective long-term prophylaxis is anticipated, which should drastically abate the burden on patients with hereditary angioedema.

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Section: Introductionmentioning

confidence: 99%

Current and Prospective Targets of Pharmacologic Treatment of Hereditary Angioedema Types 1 and 2

Fijen

Bork

Cohn

2021

Clinic Rev Allerg Immunol

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A review of berotralstat for the treatment of hereditary angioedema

Farkas

Balla

2022

Expert Review of Clinical Immunology

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Berotralstat (BCX7353) is a novel oral prophylactic treatment for hereditary angioedema: Review of phase II and III studies

Manning¹,

Kashkin²

2021

allergy asthma proc

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Background: Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling in various parts of the body. These attacks can be painful and debilitating, and affect apatient’s quality of life. Every patient who experiences an attack should be treated with on-demand medication to mitigateattack severity and duration. Many patients with HAE also receive long-term prophylaxis to reduce the frequency and severity of edema episodes. Although long-term prophylaxis reduces the disease burden for patients with HAE, available intravenous and subcutaneous treatments are accompanied by a significant treatment burden because of the logistical, emotional, and physical challenges posed by their long-term parenteral nature. Androgens are an effective oral prophylactic treatment; however, they are associated with significant adverse events and are not suitable for all patients. Thus, the HAE community has expressed interest in the development of alternative oral prophylactic therapies for preventing HAE attacks.Objective: Here, we review the phase II and III clinical data of berotralstat (BCX7353), which was approved by the U.S. Food and Drug Administration in December 2020.Results: Berotralstat is an oral, second-generation, synthetic, small-molecule plasma kallikrein inhibitor taken once dailyfor the prevention of HAE attacks in patients ages greater than or equal to 12 years. Results from the APeX studies (APeX-1 NCT02870972, APeX-2 NCT03485911, APeX-S NCT03472040, APex-J NCT03873116) demonstrated the efficacy of berotralstat as longterm prophylaxis for patients with HAE, which showed a reduction in the attack rate and on-demand medication usage.Berotralstat was well tolerated, and gastrointestinal treatment-emergent adverse events were generally mild and selflimited.Conclusion: Oral berotralstat is an effective and safe long-term prophylactic treatment for patients with HAE that will provide patients unable to tolerate parenteral therapies with the option of disease control. Berotralstat may be associated with reduced treatment burden compared with injectable therapies, highlighting the importance of patient preference with regard tothe administration route of their HAE prophylactic treatment.

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P154 Safety and Tolerability of Once-Daily Oral Kallikrein Inhibitor Bcx7353 in Phase 3 Apex-2 Hae Study

Cited by 3 publications

References 0 publications

Current and Prospective Targets of Pharmacologic Treatment of Hereditary Angioedema Types 1 and 2

Current and Prospective Targets of Pharmacologic Treatment of Hereditary Angioedema Types 1 and 2

A review of berotralstat for the treatment of hereditary angioedema

Berotralstat (BCX7353) is a novel oral prophylactic treatment for hereditary angioedema: Review of phase II and III studies

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