- BioCryst Pharmaceuticals (NASDAQ:BCRX -1%) announces that the first subject has been dosed in its two-part Phase 2 clinical trial, APeX-1, assessing next-generation killikrein inhibitor BCX7353 for the treatment of hereditary angioedema (HAE).
- APeX-1 is a randomized, double-blind, placebo-controlled dose-ranging study that will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of BCX7353 in ~50 HAE patients. Part 1 will randomize subjects 1:1 to receive either a once-daily oral dose of 350 mg of BCX7353 or placebo for four weeks. An interim analysis will be performed after the first 24 patients have completed 28 days of treatment. If a significant treatment effect is observed, Part 2 will be initiated. If the data aren't robust enough, 12 more patients will be recruited in Part 1. The sample size is flexible by design in order to cover a range of response options that will achieve statistical significance based on a reduction in HAE attack rate of at least 70% in the BCX7353 cohort, a placebo response rate of ~30% and a standard deviation of ~0.45 attacks/week.
- Part 2 will characterize the dose responses in 14 additional patients who will be randomized to receive either 250 mg or 125 mg of BCX7353 once daily or placebo.
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HAE is a rare inherited condition characterized by excessive swelling in the extremities, face, genitalia and airway, which can be life-threatening. A peptide called bradykinin is the mediator of acute swelling attacks in HAE sufferers. BCX7353 inhibits plasma kallikrein, an enzyme that plays a key role in bradykinin production.
- The company's first-generation killikrein inhibitor, avoralstat, failed to beat placebo in a Phase 2/3 HAE study.
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