Erianne Leatherman | Apr 14, 2017

BioCryst reports encouraging results in Phase 2 APeX-1 trial of hereditary angioedema treatment

BioCryst Pharmaceuticals Inc. reported positive results for its interim analysis of its Phase 2 APeX-1 trial to evaluate BCX7353 in reducing attacks in patients with hereditary angioedema (HAE).

The APeX-1 trial centered on evaluating the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered once daily BCX7353 for 28 days, which is used as a preventive treatment to cut down on the frequency of attacks in HAE patients, BioCryst said in a news release.

"The results of this interim analysis are extraordinarily encouraging," Dr. Emel Aygören-Pursun, principal investigator for the APeX-1 trial and head of Interdisciplinary Competence Center for Hereditary Angioedema and a specialist in internal medicine and hemostaseology in the Department of Child and Adolescent Medicine, Goethe University Hospital Frankfurt, said in the release. "Confirmation of the results would lead to a huge step forward in the treatment of hereditary angioedema, toward an effective, safe and easy to administer prophylaxis for the debilitating attacks connected with this condition."

The interim trial included 28 patients randomized equally to receive BCX7353 or a placebo for 28 days.

“No clinically significant changes in hematology parameters, renal function tests, electrolytes, or urinalysis were observed,” BioCryst said.

In addition, BCX7353’s efficacy, safety and tolerability profile in the interim analysis “strongly supports its continued investigation as a prophylactic treatment for HAE,” BioCryst said.

"We are extremely excited to have such a strong treatment effect in reducing HAE attacks with our once daily oral therapy," BioCryst CEO nad President Jon Stonehouse said in the release. "What is even more encouraging is the dramatic benefit seen in the reduction of peripheral attacks and mixed peripheral and abdominal attacks. A once daily oral therapy with an 88 percent reduction in these attacks has the potential to make a huge difference in HAE patients' lives."

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