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Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced six month results from an ongoing extension study of sebelipase alfa in adults with late onset Lysosomal Acid Lipase (LAL) Deficiency at the American Association for the Study of Liver Diseases (AASLD) annual meeting being held in Boston, MA, November 9-13, 2012.
“We are encouraged by the safety and efficacy profile of sebelipase alfa with longer term dosing in this trial of adults with late onset LAL Deficiency,” said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of Synageva BioPharma. “Sebelipase alfa replaces the deficient enzyme in these patients, and as anticipated, we observe normalization of serum transaminases, which are markers of liver damage, along with decreases in LDL-C and improvements in other lipid abnormalities associated with LAL Deficiency. We are also pleased to see that with sebelipase alfa treatment the improvements in blood disease activity markers are accompanied by decreases in liver fat content and liver volume as assessed by imaging studies. We look forward to providing additional updates from this ongoing trial as appropriate.”
Details from the extension study of sebelipase alfa in adults with late onset LAL Deficiency
Nine adults with LAL Deficiency were enrolled in the Phase I/II trial. After completing four weeks of treatment in the initial portion of the trial and at least four weeks of a post-treatment observation period, patients were allowed to continue treatment with sebelipase alfa as part of a long-term open-label extension study.
In the extension study, patients received four once-weekly infusions of sebelipase alfa (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg) and then transitioned to every other week infusions of sebelipase alfa (1.0 mg/kg or 3.0 mg/kg). Eight of nine patients have enrolled in the extension study. Data at the AASLD meeting were derived from the seven of the eight patients who completed the first six months of dosing in the extension study.