Date: Dec 04, 2013 Author: Don Seiffert Source: bizjournals (
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By Don Seiffert
Synageva BioPharma announced this week that it reached its goal of enrolling 50 patients in a late-stage trial of a drug aimed at a rare disease that causes liver damage in adults and kills infants, and expects to report the results of the trial in the second half of next year.
The Lexington biotech's Phase 3 trial of sebelipase alfa in children and adults with lysosomal acid lipase deficiency is the most advanced trial Synageva now has under way. The company reported positive results from an ongoing, earlier-stage trial of the same drug in September, showing a reduction in liver damage associated with the disease after one year.
Patients enrolled in the Phase 3 trial will receive infusions of sebelipase alfa or placebo every two weeks for a 20-week period, and then can enter into a long-term, open-label extension period.
LAL deficiency (historically called cholesteryl ester storage disease) causes cirrhosis and the accelerated buildup of plaque in the arteries. Infants with the disease, historically called Wolman disease, show rapid progression and usually die in the first six months of life.
Sebelipase alfa, an enzyme replacement therapy, has been granted orphan designation in the U.S., Europe and japan, and in the U.S. it has also received fast track designation, and Breakthrough Therapy designation for the treatment of infants.
Last month, Goldman Sachs analyst Navdeep Singh said in a research note that based on earlier trial data, he expects positive Phase 3 data, and estimates the market for the drug at more than $1 billion.
"We anticipate a strong launch, given management's experience with rare disease launches, the potential for a test to increase awareness/diagnosis, pricing power and the absence of a competitive threat," he wrote.
Goldman Sachs has a price target of $72 on the stock. As of 10:30 a.m. Wednesday, Synageva was trading at $62.67 a share, up 30 percent for the year to date.
