BioCryst Pharmaceuticals said Wednesday it began testing an experimental treatment on human volunteers for a rare and potentially fatal genetic disorder called hereditary angioedema.
The Durham-based drug developer said the safety test on about 80 healthy volunteers in the United Kingdom will run through the summer and the results will be reported in the third quarter. This will be the first human test of BCX7353, BioCryst’s second-generation treatment for hereditary angioedema that is based on taking one pill a day.
BioCryst’s earlier treatment, BCX4161, is based on three pills taken daily and is enrolling 96 people for its own clinical trial. In January, BCX4161 was granted fast track designation from the U.S. Food and Drug Administration, the agency that decides whether drugs are safe and effective for public use. The designation expedites development of promising drug candidates for treating serious or life-threatening conditions.
Hereditary angioedema, or HAE, afflicts about 1 in 50,000 people with painful swelling of hands, feet, face and airways. Patients experience excruciating pain, nausea, vomiting and, in some instances, asphyxiation. The primary treatment for HAE today is an intravenous injection rather than an oral pill.
In December, the FDA approved peramivir, BioCryst’s first drug, for treating serious cases of the flu. The federal government spent $235 million on developing the drug, now being sold under the brand name Rapivab.
The 53-employee company is also developing BCX4430, an antiviral for the treatment of Ebola and related viral diseases. BioCryst was founded in 1986 and went public in 1994.
The company’s stock closed Wednesday at $10.30, down 26 cents. The shares are down 15 percent this year.