June 3, 2014
Synageva BioPharma Corp.
(Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced data presentations and a company-sponsored satellite symposium at the European Atherosclerosis Society (EAS) Congress being held
May 31-June 3, 2014
Sebelipase alfa continues to improve disease-related abnormalities at two years from an ongoing Phase 1/2 trial in adults with LAL Deficiency
As part of an oral presentation,
, M.D., M.P.H., Senior Medical Director at Synageva, provided an update to the study. With six of the eight patients enrolled in the ongoing Phase 1/2 extension study completing two years of treatment, sebelipase alfa continued to demonstrate sustained reductions in the biomarkers of liver damage (both ALT and AST), frequently into the normal range, from the pre-treatment baseline to two years of the extension study. Sebelipase alfa also maintained improvements in dyslipidemia associated with LAL Deficiency, with decreases in LDL and triglycerides and increases in HDL from the pre-treatment baseline to two years of the extension study.
Sebelipase alfa was generally well tolerated through two years of the extension study. Most adverse events were mild and unrelated to sebelipase alfa. Infusion associated reactions were uncommon, generally mild and gastrointestinal in nature (diarrhea, abdominal cramping). No anti-drug antibodies have been detected and no drug-related serious adverse events have been reported in this study to date. Two serious adverse events (cholecystitis/cholelithiasis) considered unlikely related to sebelipase alfa occurred in one patient and this patient continues treatment with sebelipase alfa in the study.
Satellite Symposium and Poster Presentation at EAS
Synageva sponsored a satellite symposium at the EAS on
Saturday, May 31
at 12:00 p.m.–1:30 p.m., CEST. The symposium entitled "Elevated LDL: Not Always Familial Hypercholesterolemia; Three Mystery Cases to Diagnose" was chaired by
, M.D., Lipid Clinic, Endocrinology & Nutrition Service, Institut d'Investigacions Biomediques August Pi Sunyer, Hospital Clinic,
In addition, Synageva announced the following as a poster presentation:
"Identification, Lipoprotein Profiling and Treatment of Patients with Lysosomal Acid Lipase Deficiency",
E.O. Stock, et al.
Sebelipase alfa for LAL Deficiency