Synageva LAL ERT meets Phase III endpoints

Synageva BioPharma Corp. (NASDAQ:GEVA) said the Phase III ARISE trial in 66 patients ages >=4 with lysosomal acid lipase (LAL) deficiency showed that 1 mg/kg IV sebelipase alfa every other week met the primary endpoint of improving the proportion of patients with alanine aminotransferase (ALT) normalization at week 20 vs. placebo (p=0.027). Sebelipase alfa also met the secondary endpoints of reducing LDL-C (p<0.001), non-HDL-C (p<0.001) and triglyceride (p=0.038) levels and of increasing HDL-C levels (p<0.001) from baseline to week 20 vs. placebo. However,in 26 patients with evaluable paired biopsies, it missed the secondary endpoint of a greater proportion of patients with an improvement in hepatic steatosis vs. placebo (63% vs. 40%, p=not significant). Synageva said similar numbers of patients reported adverse events in each arm, but one patient receiving sebelipase alfa discontinued treatment after a severe, atypical infusion-related reaction. ...