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What: Shares of BioCryst Pharmaceuticals (NASDAQ:BCRX), a biotechnology company focused on developing small-molecule drugs designed to block enzymes which are vital to the disease development process, spiked higher by as much as 22% after reporting positive data from its OPuS-1 mid-stage study involving BCX4161 as a treatment for hereditary angioedema, or HAE, a rare immune disorder that can cause swelling of the face, breathing airways, and also causes abdominal cramping.
So what: According to the preliminary results from BioCryst's proof-of-concept study, BCX4161 met both the primary and secondary endpoints. The primary endpoint – the reduction of angioedema attacks – showed that BCX4161 "demonstrated a statistically significant mean attack rate reduction of 0.45 attacks per week versus placebo" over the four-week study. The secondary endpoint of safety and tolerability was also met with none of the 24 patients dropping out of the study and only one serious adverse events (an abdominal HAE attack) occurring during the study. The number of attack-free days also climbed to 22 for BCX4161 compared to 19 for the placebo, with three BCX4161 intent-to-treat patients completing the study without an attack compared to zero for the placebo. Finally, the Angioedema Quality of Life Questionnaire and Angioedema Activity Score also notably favored BioCryst's investigational drug. Following this news, shares of Dyax (UNKNOWN:DYAX.DL) also spiked higher by double-digits, though this appears to be as a reassurance to current investors that BioCryst's investigational therapy isn't a direct threat in that it's designed to prevent HAE attacks before they start whereas Dyax's HAE therapy is given during an attack.
Now what: The next step for BioCryst appears pretty clear: the OPuS-2 trial which will begin later this year and run for 12 weeks. This data would certainly suggest in the early going that BCX4161 could give the lone HAE preventative therapy currently approved by the Food and Drug Administration, Shire's Cinryze, a run for its money, eventually. Of course, the best part about this is rare disease sufferers get a potentially new therapy to choose from, while BioCryst gets to place a high, but protected, price tag on its therapy if it's eventually approved. BioCryst certainly just got more exciting and I would suggest biotech-savvy investors get this name on their watchlist.
Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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