Clinical stage biopharmaceutical company Synageva BioPharma has commenced dosing in its Phase III acid lipase replacement investigating safety and efficacy (ARISE) trial of sebelipase alfa in patients with late onset lysosomal acid lipase deficiency (LAL deficiency).

The ARISE trial is a double-blind, placebo-controlled study designed to enrol 50 patients including children and adults with late-onset LAL deficiency.

Synageva senior vice president and chief medical officer Dr Anthony Quinn said that progressive and severe liver damage, including cirrhosis in children and adults, in addition to accelerated atherosclerosis is caused by the accumulation of abnormal fats in LAL deficiency.

"By replacing the deficient enzyme that causes the accumulation of these abnormal fats, sebelipase alfa addresses the root cause of LAL deficiency," Quinn said.

"Based on data from the previously conducted preclinical and clinical studies with sebelipase alfa, the Phase III ARISE trial was designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with LAL deficiency."

"The ARISE trial is a double-blind, placebo-controlled study designed to enrol 50 patients including children and adults with late-onset LAL deficiency."

The patients will be randomised on a one-to-one basis to every other week infusions of sebelipase alfa (1mg/kg), or placebo for the double-blind treatment period of 20 weeks.

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The company expects to use results from the double-blind period to demonstrate efficacy and safety in global submissions for registration of the product.

The proportion of patients who achieve normalisation of alanine aminotransferase (ALT), a marker of liver damage, at the completion of the double-blind treatment period (week 20), compared to placebo, is the primary endpoint of the trial.

The study’s key secondary endpoints are the relative reduction from baseline to week 20 in LDL-C, non-HDL-C and triglycerides, the proportion of patients who achieve aspartate aminotransaminase normalisation and the relative increase in HDL-C.

The study will assess other secondary endpoints including reductions in liver fat content and liver volume, and improvements in liver pathology in a proportion of patients who undergo these assessments.