“In a year with many milestones across our multiple advancing programs of oral medicines for rare diseases, BioCryst Pharmaceuticals, Inc. has achieved significant progress in the first quarter and we look forward to reporting data from our APeX-2 trial in the second quarter and filing a new drug application by the end of the year,” said Jon Stonehouse, president and CEO at the presentation of the company’s financial results for the first quarter ended 31 March 2019.
“We believe that oral BCX7353 could be transformative for many HAE patients and provide them with the opportunity for a normal life without the burden and discomfort of frequent injections and infusions,” Stonehouse added.
First Quarter 2019 Developments for HAE Program BCX7353:
- The company dosed the first patients in its APeX-J trial in Japan, designed to support potential Japanese approval of BCX7353 for the prevention of HAE attacks.
- In February 2019, the company announced data from the completed ZENITH-1 trial (including the 250 mg and 500 mg dose cohorts) of BCX7353 for the acute treatment of HAE attacks at the annual meeting of the American Academy of Allergy, Asthma & Immunology. The company plans to commence a Phase 3 trial, ZENITH-2, in the summer of 2019.
Upcoming Key Milestones for HAE Program BCX7353:
- Report 24-week safety and efficacy results from the Phase 3 APeX-2 clinical trial (Q2 2019)
- Begin ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the acute treatment of HAE (Summer 2019)
- File a new drug application for oral BCX7353 for the prevention of HAE attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)
- File a marketing authorization application for oral BCX7353 for the prevention of HAE attacks with the European Medicines Agency (EMA) (Q1 2020)