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Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of
“Children and adults with late onset LAL Deficiency can suffer from serious liver complications and accelerated atherosclerosis,” said Manisha Balwani, MD, MS, Assistant Professor of Genetics and Genomic Sciences and Assistant Professor of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, and principal investigator in the study. “Based on its mechanism of action, sebelipase alfa addresses the root cause of LAL Deficiency, and the Phase I/II extension study in adults with LAL Deficiency illustrates that sebelipase alfa improves patients’ abnormalities associated with the disease.”
About the Phase I/II 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 the initial portion of the Phase I/II trial, 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 enrolled in the extension study. Data published in
Hepatology were derived from the seven patients who completed the first 12 weeks of dosing in the extension study.
For these seven patients, sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to week 12 of the extension study of 52% and 36%, respectively (p<0.05 for both). In addition, sebelipase alfa resulted in mean percent decreases from the initial baseline to week 12 of the extension study for LDL-C of 27% (p=0.078), total cholesterol of 22% (p=0.047), triglycerides of 28% (p=0.016), as well as mean increases in HDL of 15% (p=0.016).