RESEARCH TRIANGLE PARK, N.C., June 25, 2018 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc.(NASDAQ:BCRX), a pharmaceutical company focused on the development and commercialization of treatments for rare diseases, today announced that the Company has reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for Hereditary Angioedema (“HAE”) with the Pharmaceuticals and Medical Devices Agency (“PMDA”) in Japan.
The Phase 3 trial design agreed upon for Japan, APeX-J, is a randomized, placebo-controlled double-blind trial of 24 weeks duration with a 28-week safety extension. Approximately 24 Japanese subjects with HAE will be enrolled. The APeX-J trial design closely follows the design of APeX-2, a Phase 3 clinical trial being conducted in the U.S., Canada and European countries. APeX-J tests the same dose levels of BCX7353 as in APeX-2, 110 mg daily and 150 mg daily, and the endpoints are identical to those in APeX-2. Data from the APeX-J and APeX-2 trials will be combined for regulatory submission in Japan.
“Japanese HAE patients would welcome an oral prophylactic drug as another treatment alternative, which provides HAE patients ease in daily life as well as relief from travelling outside their home town for treatment to receive on-demand therapy of HAE attacks in a hospital,” said Professor Michihiro Hide, M.D., Ph.D, Dean of the School of Medicine and Professor and Chairman, Department of Dermatology, Hiroshima University, and a leading expert in HAE in Japan.
BCX7353 was one of the first products granted Sakigake designation by the Japanese PMDA in October 2015. Sakigake designation is awarded to innovative products addressing an unmet medical need in Japan or providing improved efficacy compared to available treatment. Sakigake designation is associated with accelerated review of a Japanese NDA as well as pricing and other corporate benefits for the holder.
"Now that we have agreement on the regulatory requirements for marketing authorization of BCX7353 in Japan, we can move forward with executing APeX-J and selecting a commercialization partner for this region," said Jon P. Stonehouse, President & Chief Executive Officer. "A once-a-day oral treatment for this rare disease represents a high unmet medical need in Japan and an excellent partnering opportunity."
About BCX7353
Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 has been generally safe and well tolerated in the Phase 2 APeX-1 clinical trial. BioCryst is currently conducting the Phase 3 APeX-2 clinical trial and the long-term safety APeX-S clinical trial, both evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. BioCryst is also conducting the ZENITH-1 clinical trial. ZENITH-1 is a proof-of-concept Phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals designs, optimizes and develops novel small-molecule medicines that address both common and rare conditions. BioCryst has several ongoing development programs including BCX7353, an oral treatment for hereditary angioedema, galidesivir, a potential treatment for filoviruses, and a preclinical program to develop oral Alk-2 inhibitors for the treatment of fibrodysplasia ossificans progressive (“FOP”). 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 U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the Company's website at www.BioCryst.com.
