Synageva BioPharma announced positive data on sebelipase alfa in a clinical study of patients with lysosomal acid lipase (LAL) deficiency at the American Association for the Study of Liver Diseases’ AASLD Liver Meeting, in Boston, MA.

The Phase 3, global, double-blind, placebo-controlled trial (n=66) randomized patients with LAL deficiency to every other week infusions of sebelipase alfa 1mg/kg or placebo for 20 weeks. Study results described the effect of sebelipase alfa on a range of disease-related abnormalities. The primary endpoint was met with 31% of the sebelipase alfa patients reaching normalization of ALT vs. 7% of placebo patients (P=0.027). All patients treated with sebelipase alfa showed a reduction in ALT with mean changes of -57.9U/L vs. -6.7U/L in the placebo group.

Further data shed insight into the severity of the participants’ baseline liver disease and dyslipidemia status. It was presented that of the patients who had biopsies at baseline, 47% had bridging fibrosis (defined as Ishak score 3 or 4) and another 31% (10/32) of patients had cirrhosis.

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Also, study authors presented data that showed additional improvements in LDL cholesterol at Week 36 from an ongoing open-label extension of the study. Fifty-eight percent of patients had LDL cholesterol levels at baseline in the very high category (>190mg/dL) although 24% of these patients were receiving lipid-lowering medications.

Sebelipase alfa is a recombinant form of the human LAL enzyme that is being investigated as an enzyme replacement therapy for LAL deficiency.

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