Synageva BioPharma Corp. (GEVA) continues to progress well with the development of its lead candidate sebelipase alfa.
Synageva is developing sebelipase alfa for the treatment of lysosomal acid lipase deficiency (LAL deficiency).
The company recently announced that it has completed the enrolment target of 50 patients for its phase III trial ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) on candidate sebelipase alfa (formerly known as SBC-102).
Synageva expects to randomize the final patient for the trial by 2013 end. Synageva expects to report top-line results from the ARISE trial in the second half of 2014.
In Oct 2013, Synageva reported positive results from a 78-week extension study on sebelipase alfa in adults wherein it was observed that the candidate was generally well tolerated among patients.
We remind investors that sebelipase alfa was granted orphan drug designation by the U.S. Food and Drug Administration (:FDA), and the European Medicines Agency (:EMA). The FDA also granted “breakthrough therapy designation” to sebelipase alfa for LAL deficiency present in infants in May 2013.
Synageva primarily focuses on rare diseases. We note that sebelipase alfa is Synageva’s most advanced candidate in its pipeline. Hence, the successful development and commercialization of sebelipase alfa will be a major boost for Synageva.
Meanwhile, Synageva is also developing SBC-103, a replacement therapy for the treatment of mucopolysaccharidosis IIIB (MPS IIIB), also known as Sanfilippo B syndrome. The candidate is in late stages of preclinical testing and is anticipated to enter the clinic in mid-2014.
Synageva carries a Zacks Rank #3 (Hold). Currently, stocks like Actelion Ltd. (ALIOF), Shire (SHPG), and Isis Pharmacecuticals (ISIS) look attractive. While Actelion carries a Zacks Rank #1 (Strong Buy), both Shire and Isis Pharma are Zacks Rank #2 (Buy) stocks.