BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported the dosing of the first subject into its clinical study of ZENITH-1. This study is assessing up to 3 dosage strengths of BCX7353 administered as a single oral dose targeting acute treatment of angioedema attacks in people with hereditary angioedema.
Jon Stonehouse, the Chief Executive Officer of BioCryst, reported that they are thrilled to release the ZENITH-1 exploratory Phase II study. Based upon pharmacodynamic and pharmacokinetic properties of BCX7353, they consider it could be a convenient and efficacious oral option to parenteral therapies of acute angioedema attacks in people with HAE. A liquid formulation of BCX7353 can work as a strong complement to company’s prophylactic treatment program. The team consider this new formulation has the potential to fill an unmet need for people who are looking for easier and better ways to manage their ailments.
ZENITH-1 is a randomized, placebo-controlled, double-blind, adaptive dose-ranging study of the efficacy, tolerability and safety of BCX7353 for cure of acute angioedema attacks. The trial will enroll up to 60 people with HAE. Blinded trial drug will be administered as an oral liquid after beginning of symptoms, for up to three attacks in each person, with each subject getting both placebo (for one attack) and BCX7353 (for 2 attacks) in a randomized sequence.
The study is planned with up to 3 successive cohorts testing single doses of 250 mg (up to 12 subjects), 500 mg (up to 12 subjects) and 750 mg (from 12 to 36 subjects) starting with 750 mg. Efficacy evaluations comprise patient-reported composite VAS scores, change in symptoms, use of rescue medication and patient global assessment.
Treatment effect will be evaluated on accumulating results, starting after 12 people have completed trial in the first cohort, by assessing the proportion of placebo-treated and BCX7353-treated attacks which have an improved or stable composite VAS at four hours post dose. Once the therapy effect is shown, registration at the 500 mg dose level will start. If therapy effect at the 500 mg dose is same as that of 750 mg, the 250 mg dose cohort is on horizon.