Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema

A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Parallel-group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BCX7353 as a Preventative Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema

This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 compared to placebo.

Key Inclusion Criteria:

• A clinical diagnosis of HAE type I or II
• Documented HAE attacks within a defined calendar period
• Access to acute attack medications
• Sexually active women of child-bearing potential and sexually active men must utilize effective contraception

Key Exclusion Criteria:

• Women who are pregnant or breast-feeding
• Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
• Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks
• History of or current alcohol or drug abuse
• Infection with hepatitis B, hepatitis C or HIV
• Participation in any other investigational drug study currently or within the last 30 days