Synageva BioPharma Corp.’s (GEVA) shares were up 0.2% immediately after the company announced the presentation of new data from a phase III double-blind placebo-controlled study (ARISE) on its lead candidate, sebelipase alfa, for the treatment of lysosomal acid lipase deficiency (LAL deficiency). Shares further gained 0.9% in the subsequent trading session. Overall, shares are up 1.1% since the announcement.
The data was presented at the 65th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD).
In this meeting, Synageva presented the impact of sebelipase alfa on a wide range of abnormalities related to LAL deficiency besides providing new insights into the severity of liver disease and dyslipidemia at baseline. Sebelipase alfa improved the markers of liver injury, dyslipidemia and liver fat content, which are the key parameters related to LAL deficiency. Moreover, the company said that sebelipase alfa sustained the reduction in markers of liver injury and led to further improvement in LDL cholesterol during the early part of an ongoing open-label extension study.
We remind investors that in Jun 2014, Synageva had announced that sebelipase alfa had met the primary endpoint in the ARISE study. Following 20 weeks of treatment, 31% of the patients (11/36) administered with sebelipase alfa reached normalization of alanine aminotransferase (ALT), a marker of liver injury, compared with 7% of the patients (2/30) receiving placebo.
Preliminary Results from Extension Study
In AASLD meeting, Synageva also announced the preliminary results from an ongoing open-label extension study in 65 out of the 66 patients enrolled in the phase III study on sebelipase alfa. Data showed that under administration of sebelipase alfa in the extension study, placebo-treated patients from the double-blind 20-week period experienced a similar response (in terms of ALT and LDL cholesterol) to the patients who had received sebelipase alfa during the 20-week treatment period. Moreover, sebelipase alfa-treated patients from the double-blind 20-week period showed sustained reduction in ALT and further reduction in LDL cholesterol under continued treatment with the candidate at week-36.
We note that Sebelipase alfa enjoys orphan drug status in both U.S. and Europe. It was also granted fast track designation and Breakthrough Therapy designation by the FDA for LAL deficiency in infants.
Meanwhile, in Oct 2014, Synageva initiated a rolling submission of a biologics license application for sebelipase alfa to the FDA, which is expected to be completed by the end of Jan 2015. The company also intends to submit a marketing authorization application to the European Medicines Agency by the end of Jan 2015.
Investors looking for well-placed stocks in the health care sector may consider Alcobra Ltd. (ADHD), Akorn, Inc. (AKRX) and Celgene Corporation (CELG). While Alcobra and Akorn carry a Zacks Rank #1 (Strong Buy), Celgene holds a Zacks Rank #2 (Buy).
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