Shares of BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) saw a handy gain on Tuesday after the company released the final results from its mid-stage APeX-1 clinical trial in hereditary angioedema (HAE). Overall, the Phase 2 results of BCX7353 strongly support its advancement into Phase 3 development.
Out of the four dose levels, the 125 mg dose level showed statistically significant and similar benefit for all attacks, and also when split into abdominal attacks and peripheral attacks. In contrast, at the 250 mg and 350 mg dose levels, there was no statistically significant effect for abdominal attacks, despite strong and statistically significant effects on peripheral attacks.
As expected, the lowest dose tested (62.5 mg) showed no statistically significant differences in attack rates (total, or when split into abdominal and peripheral) compared with placebo.
Looking ahead, BioCryst intends to meet with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) during the fourth quarter of 2017 to finalize the Phase 3 program required to support New Drug Application (NDA) and Marketing Authorization Application (MAA) submissions.
Jon Stonehouse, CEO and president, commented:
We are delighted to see a robust treatment effect after completing the largest ever Phase 2 trial in HAE patients. We now have the information necessary to select doses for Phase 3. The 125 mg once-daily oral dose of BCX7353 provided a high level of efficacy and excellent tolerability. This product profile will be an extremely attractive treatment option for physicians and patients.
William Sheridan, chief medical officer, added:
With an active IND in the U.S. and the completion of APeX-1, we are now preparing for meetings in the fourth quarter of this year with the FDA and EMA to finalize the Phase 3 program. Our goal is to start the Phase 3 efficacy and long-term safety trials in the first quarter of 2018.
Shares of BioCryst were up more than 13% at $5.66 just after Tuesday’s opening bell, but they had slipped into the red by mid-morning. The consensus analyst price target is $8.50 and a 52-week trading range of $3.75 to $9.25.