Phase III trial of SBC 102 in LAL Deficiency meets primary endpoint-Synageva

Synageva has announced that the global, randomized, double-blind, placebo-controlled Phase III ARISE trial of SBC 102 (sebelipase alfa) in 66 children and adults with Lysosomal Acid Lipase Deficiency (LAL Deficiency) met the primary endpoint of normalization of alanine aminotransferase (ALT), a marker of liver injury. In addition, sebelipase alfa significantly improved multiple other disease-related abnormalities as measured by a number of secondary endpoints.

LAL Deficiency patients enrolled in the trial presented with multiple clinically important abnormalities at baseline. Fibrosis and/or cirrhosis was documented in 100% (32/32) of patients who had baseline biopsies even though the median age of patients enrolled in the trial was only 13 years. Dyslipidemia was common at baseline, with a median LDL cholesterol of 204 mg/dl, and an abnormally low median HDL cholesterol of 32.5 mg/dl. In addition, sebelipase alfa improved dyslipidemia with statistically significant reductions in LDL cholesterol, non-HDL cholesterol and triglycerides, as well as a statistically significant increase in HDL cholesterol, all compared with placebo from baseline to the completion of the double-blind treatment period of 20 weeks.

The sebelipase alfa and placebo arms had a similar number of patients with reported adverse events which were mild. The adverse events occurring in three or more sebelipase alfa treated patients, and which occurred more commonly in treated than placebo patients, were headache (28% versus 20%). Synageva plans to complete submission of a Biologic License Application to the FDA and a Marketing Authorization Application to the European Medicines Agency for sebelipase alfa for the treatment of LAL Deficiency by the end of the first quarter of 2015.