Synageva BioPharma (GEVA) announced that the first patient initiated treatment in the ARISE trial, a global, Phase 3, randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with late onset lysosomal acid lipase deficiency. The ARISE trial will enroll 50 patients with late onset LAL Deficiency. Patients enrolled in the trial are randomized on a one-to-one basis to every other week infusions of sebelipase alfa, or placebo for the double-blind treatment period of 20 weeks. The primary endpoint of the trial is the proportion of patients relative to placebo who achieve normalization of alanine aminotransferase, a marker of liver damage, at the completion of the double-blind treatment period. Key secondary endpoints include the relative reduction from baseline to week 20 in LDL-C, non-HDL-C, triglycerides, the proportion of patients who achieve aspartate aminotransaminase normalization, and the relative increase in HDL-C. Additional secondary endpoints, including reductions in liver fat content and liver volume and improvements in liver pathology, will be examined in a proportion of patients who undergo these assessments. Deficiency of LAL enzyme activity will be confirmed during patient screening with a dried blood spot biochemical enzyme activity assay performed by Laboratory Corporation of America Holdings (LH), the central diagnostic testing laboratory performing the tests.