BioCryst Pharmaceuticals, Inc. has announced final results from its Phase 2 APeX-1 clinical trial in HAE. APeX-1 was a 3-part dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of orally administered once-daily (QD) BCX7353 for 28 days, as a preventative treatment to reduce the frequency of attacks in HAE patients. This final analysis evaluated data from all patients in Parts 1, 2 and 3 of the trial.
“We are delighted to see a robust treatment effect after completing the largest ever Phase 2 trial in HAE patients. We now have the information necessary to select doses for Phase 3,” said Jon Stonehouse, CEO & President. “The 125 mg once-daily oral dose of BCX7353 provided a high level of efficacy and excellent tolerability. This product profile will be an extremely attractive treatment option for physicians and patients.”
“An effective and tolerable prophylaxis of hereditary angioedema attacks is of paramount importance for many HAE patients and the benefit of an oral administration route for these chronically ill patients cannot be overestimated. In that respect, the results of this trial are extremely encouraging for the HAE patient community,” said Dr. Emel Aygören-Pürsün, MD, principal investigator for the APeX-1 trial and Head of Interdisciplinary Competence Center for Hereditary Angioedema, and Specialist in Internal Medicine and Hemostaseology Department of Child and Adolescent Medicine, Goethe University Hospital Frankfurt.
“With an active IND in the U.S. and the completion of APeX-1, we are now preparing for meetings in the fourth quarter of this year with the FDA and EMA to finalize the Phase 3 program,” said William Sheridan, Chief Medical Officer. “Our goal is to start the Phase 3 efficacy and long-term safety trials in the first quarter of 2018.”
Seventy-five subjects were randomized and included in the final analysis of pooled data from Parts 1, 2 and 3: 7 at 62.5 mg, 14 at 125 mg, 14 at 250 mg and 18 at 350 mg of BCX7353 QD; and 22 placebo. The qualifying attack rate was approximately 1/week. Baseline characteristics were generally well balanced across the treatment groups. Compliance with daily study drug dosing for 28 days was excellent (≥ 98% across all treatment groups).