BioCryst Pharmaceuticals has dosed the first patient in the APeX-S trial that is designed to assess BCX7353 to treat patients with hereditary angioedema (HAE).
HAE is a rare and potentially fatal genetic condition that causes swelling under the skin in multiple locations throughout the body, including the throat or airway.
As part of the APeX-S trial, two dose levels of BCX7353 such as 110mg and 150mg once-daily will be provided over 48 weeks in patients with Type I and II HAE.
Around 160 patients will be enrolled in the trial, which is an open-label two-arm study.
The study will initially enrol patients who participated in a previous clinical trial of BCX7353, and will include patients who have not previously received the drug in due course of time.
Its endpoints include long-term safety, durability of response and quality of life measures.
BioCryst Pharmaceuticals CEO Jon Stonehouse said: “Initiation of the APeX-S trial is an important milestone to support filing and approval of BCX7353, and furthers our core strategy of bringing a once-daily, oral prophylactic treatment to HAE patients.
“Long-term safety results from this trial will supplement efficacy and safety data from the APeX-2 Phase III trial, which is also expected to commence in the first quarter of 2018.
“The ability to run these trials concurrently is beneficial, as it may allow us to more rapidly get this important medicine into the hands of patients who are seeking a better quality of life by eliminating their current injection-based treatment programmes.”
BioCryst’s BCX7353 is a new, oral, once-daily, selective inhibitor of plasma kallikrein currently being developed for the prevention and treatment of angioedema attacks in patients diagnosed with HAE.
The drug is reported to be safe and well tolerated in the Phase II APeX-1 trial.