BioCryst Pharmaceuticals, a pharmaceutical company focused on the development and commercialization of treatments for rare diseases, has announced plans to explore a new oral liquid formulation of BCX7353 for the treatment of acute attacks in patients with hereditary angioedema (HAE). The company has received initial regulatory approvals in Europe to initiate the ZENITH-1 exploratory clinical trial this summer.

"To complement the attractive profile of our prophylactic treatment programme, as evidenced by the interim results from our APeX-1 trial, we made a decision to explore an additional indication for the treatment of acute attacks with a new oral liquid formulation.  The rapid absorption and long half-life of BCX7353 observed after single oral doses in healthy volunteers, and the strong encouragement from disease experts and patient advocates have motivated us to explore ‘7353 as an acute treatment," said Jon Stonehouse, chief executive officer. "We believe this new formulation can fill an unmet need for patients with less frequent attacks who are looking for better ways to manage their illness. This initiative has the potential to provide patients with a more convenient option for the treatment of acute attacks, and if successful, the first oral acute therapy for HAE."

The purpose of ZENITH-1 is to explore whether single oral doses of a liquid formulation of BCX7353 could have utility in the treatment of acute angioedema attacks in patients with hereditary angioedema. It is designed as a randomized, double-blind, placebo controlled, dose-ranging trial with BCX7353 self-administered at home to treat attacks. Additional information and clinical trial design will be provided upon completion of the regulatory process and trial initiation.

Discovered by BioCryst, BCX7353 is a novel, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention of angioedema attacks in patients diagnosed with HAE. With the initiation of the ZENITH-1 clinical trial, the oral liquid formulation of BCX7353 will be evaluated for treating acute angioedema attacks. BCX7353 has been generally safe and well tolerated in the ongoing phase 2 ApeX-1 clinical trial and in clinical pharmacology studies in healthy volunteers.

BioCryst Pharmaceuticals designs, optimizes and develops novel small molecule drugs that block key enzymes involved in rare diseases. BioCryst has several ongoing development programs: BCX7353 and other second generation oral inhibitors of plasma kallikrein for hereditary angioedema, and galidesivir, a broad spectrum viral RNA polymerase inhibitor that is a potential treatment for filoviruses. RAPIVAB (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the US, Canada, Japan, Taiwan and Korea. Post-marketing commitment development activities for RAPIVAB are ongoing, as well as activities to support regulatory approvals in other territories.