BioCryst Pharmaceuticals, Inc. has announced financial results for the fourth quarter and full year ended December 31, 2015.
“In 2015, we gained clarity on both of our HAE drug candidates. Our efforts are now focused on completing the bioavailability study of a solid dosage form of avoralstat and completing the APeX-1 clinical trial of BCX7353,” said Jon P. Stonehouse, President & CEO. “We ended 2015 with a balance sheet that enables us to achieve these two near-term data events without the need to raise capital. These two programs give us two shots at achieving our goal of developing a highly effective, conveniently dosed, oral drug candidate for the prophylactic treatment of HAE patients.”
On February 8, 2016, we announced results from OPuS-2 (Oral P rophylaxiS-2), a clinical trial of avoralstat administered three times daily in a liquid-filled soft gel formulation for the prophylactic treatment of HAE attacks. The primary efficacy endpoint was angioedema attack frequency. Treatment with 500 mg and 300 mg of avoralstat three times daily failed to demonstrate a statistically significantly lower mean attack rate versus placebo. Statistically significant improvements in duration of attacks and in the Angioedema Quality of Life total score were observed comparing the 500 mg three times a day avoralstat arm to placebo. Following the analysis of OPuS-2 results, the decision was made to discontinue further development of softgel avoralstat formulation in order to focus development efforts on a novel solid dosage form of avoralstat.
BioCryst expects to report results from a relative bioavailability study testing the novel solid dosage form of avoralstat by mid-year 2016. The primary goal of this study is to achieve meaningfully better drug exposure in a twice daily dosing regimen.
BioCryst expects to report results from the BCX7353 APeX-1 dose ranging study in HAE patients by year end. The design of APeX-1 trial will be described once it is initiated.