Results from the Phase 2, APeX-1 clinical trial of BCX7353 for the prevention of attacks in patients with HAE are published online in the July 26th issue of The New England Journal of Medicine.
The APeX-1 trial was a double blind, randomized, parallel group, placebo-controlled Phase 2 dose ranging trial comparing the safety and efficacy of 28 days of once-daily BCX7353 treatment, at doses of 62.5 mg, 125 mg, 250 mg, and 350 mg, with placebo. The trial demonstrated that oral administration of BCX7353 at a dose of 125 mg or more resulted in a significantly lower rate of attacks compared with placebo. Significant benefits with respect to quality of life were observed in the 125 mg and 250 mg dose groups. Mild gastrointestinal symptoms were the principal side effect, particularly in the 250 mg and 350 mg BCX7353 dose groups.
“For patients with HAE, the results of this trial suggest potential for the future to manage their disease with a prophylactic therapy that would combine efficacy and safety with the advantage of oral administration,” said Emel Aygören-Pürsün, M.D., principal investigator for the APeX-1 trial and Head of Interdisciplinary Competence Center for Hereditary Angioedema, and Specialist in Internal Medicine and Hemostaseology Department for Children and Adolescents, Goethe University Hospital Frankfurt.
“We are extremely pleased to have these important results published in The New England Journal of Medicine,” said Jon P Stonehouse, President and Chief Executive Officer of BioCryst Pharmaceuticals, Inc. “We look forward to confirming the results from APeX-1 in our pivotal Phase 3 trial, APeX-2, which we expect to report out in the first half of 2019.”