Results from a phase 3 trial evaluating BCX7353 (BioCryst), an investigational therapy for hereditary angioedema (HAE), showed that the oral plasma kallikrein inhibitor significantly reduced the rate of HAE attacks, the primary endpoint of the study.  

In the APeX-2 trial, a total of 121 patients with Type I and II HAE were randomized 1:1:1 to receive either once-daily BCX7353 110mg (n=41), 150mg (n=40) or placebo (n=40) for 24 weeks. The primary efficacy endpoint of the study was the rate of investigator confirmed angioedema attacks over 24 weeks of study drug administration.

Results showed that the BCX7353 110mg group had a reduction in HAE attack rate of 30% (P =.024), while the 150mg dose group had a reduction in attack rate of 44% (P <.001), both compared with placebo; 50% of patients treated with the 150mg dose had a ≥70% reduction in their HAE attack rate compared to baseline vs 15% of placebo patients (P =.002).

Full, detailed results of APeX-2 will be submitted for peer review publication and presentation, according to the Company. BioCryst also plans to submit a New Drug Application based on the results from the trial.


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“The additional clinical information we now have from APeX-2 confirms that this is an oral kallikrein inhibitor that is effective at preventing HAE attacks in a large segment of the HAE patient population while having a very attractive tolerability profile,” said Bruce Zuraw, MD, professor of medicine and chief of the Division of Rheumatology, Allergy and Immunology at the University of California School of Medicine, and principal investigator of the APeX-2 trial.

Biocryst is also conducting an open label study (APeX-S) to evaluate the long term safety and effectiveness of BCX7353 in preventing acute angioedema attacks in patients with Type I and II HAE.

For more information visit Biocryst.com.