Synageva BioPharma Corp. (GEVA) announced that it has finished enrolling patients for its phase III study (Acid Lipase Replacement Investigating Safety and Efficacy - ARISE) on sebelipase alfa.
The study is being conducted in children and adults suffering from lysosomal acid lipase deficiency (LAL Deficiency). The company said that 66 patients were randomized in the study – this was above Synageva’s original target of 50 patients.
Synageva remains on track to present top-line results from the study in the second half of this year.
Meanwhile, Synageva also said that it has met the enrollment target in a phase II/III study being conducted in infants with LAL Deficiency. Preliminary results will be out at the 10th Annual Lysosomal Disease Network (:LDN) WORLD Symposium being held between Feb 11-13.
Sebelipase alfa is Synageva’s lead pipeline candidate. It enjoys orphan drug status in the U.S., EU as well as Japan. Moreover, it has fast track designation in the U.S. as well as Breakthrough Therapy designation (LAL Deficiency presenting in infants).
Other pipeline candidates at Synageva include SBC-103, which is being developed for mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo B syndrome. SBC-103 has orphan drug designation in the U.S. as well as in the EU. The candidate is slated to move into clinical studies in mid-2014.
With sebelipase alfa being the lead candidate at Synageva, its successful development and approval would be a major boost for the company. We expect investor focus to remain on pipeline updates.
Synageva carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the same sector include Horizon Pharma, Inc. (HZNP), Achillion Pharmaceuticals, Inc. (ACHN) and Alimera Sciences, Inc. (ALIM). While Horizon Pharma is a Zacks Rank #1 (Strong Buy) stock, Achillon and Alimera are Zacks Rank #2 (Buy) stocks.