BioCryst (Nasdaq: BCRX) Pharmaceuticals CEO Jon Stonehouse came out of the Memorial Day holiday weekend especially rejuvenated on Wednesday.

BioCryst rolled out some of the best news it’s had since the company headquarters moved to Durham from Alabama four years ago. The publicly traded drug company reported positive results from a major clinical trial.

It’s for an oral medication that offers hope for people suffering a rare, debilitating and sometimes deadly genetic condition called hereditary angioedema (HAE).

The Phase 2a trial hit the sought-after targets for effectiveness in patients with HAE, indicating the drug developed by BioCryst scientists is on track to becoming a marketplace reality.

That doesn’t mean that BCX 4161 will be a blockbuster. HAE, fortunately, occurs in only about 1 in 10,000 to 1 in 50,000 people. So BioCryst spokesman Robert Bennett said the company plans to seek orphan drug status in the United States and Europe.

But it’s an important drug. If you’re among those “orphans” with HAE, your symptoms can include recurrent episodes of edema, meaning deposits of watery fluid, in various locations, including the hands, feet, face, genitalia and airway. In addition, HAE patients often have bouts of excruciating abdominal pain, nausea and vomiting that are caused by swelling in the intestinal wall. Airway swelling is particularly dangerous and can lead to death by asphyxiation. If that’s you, you’re plenty glad for BioCryst.

BioCryst expects to have approval by the U.S. Food & Drug Administration by Christmas for its first major drug, an intravenous flu treatment, peramivir. It’s also working on other drugs, including a promising antiviral currently dubbed BCX4430, to fight hemorrhagic fevers.

BioCryst has 24 employees in North Carolina and 14 in Birmingham, Ala. Its stock closed up nearly 11 percent at the end of trading Wednesday, then dropped about 3 percent after hours when the company announced a $100 million additional stock offering to pay for hauling its new drugs through the expensive clinical trials so they’ll all be tuned for the marketplace.

Editor’s note: Jim Shamp is director of media relations for the N.C. Biotechnology Center and is a frequent contributor to WRAL TechWire.

(C) N.C. Biotech Center