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Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced a poster presentation at the 12
th International Congress of Inborn Errors of Metabolism (ICIEM) conference being held in Barcelona, Spain, September 3-6, 2013.
The poster, entitled “Long Term Effect of Sebelipase Alfa After One Year in Patients with Lysosomal Acid Lipase (LAL) Deficiency,” V. Valayannopoulos et al, describes the results from the ongoing Phase 1/2 extension study in adults with LAL Deficiency treated with sebelipase alfa at one year. With seven of the eight patients enrolled in the study completing one full year of treatment, the results continue to show reduction in liver damage and improvement in the dyslipidemia associated with LAL Deficiency.
Details from the Phase 1/2 extension study with sebelipase alfa in adults with LAL Deficiency presented at ICIEM
Nine adults with LAL Deficiency with a median age of 29 years (range 19-45) enrolled in the Phase 1/2 trial. Seven of nine patients had a history of hepatomegaly and/or splenomegaly, and two of nine patients had evidence of more advanced liver disease, including cirrhosis and portal hypertension. All nine patients had a history of dyslipidemia, and seven of nine patients also had a history of other cardiovascular conditions. Seven of nine patients received treatment with lipid modifying therapies including ezetimibe, statins, and other medications.
Eight of nine patients continued treatment with sebelipase alfa as part of a long term open-label extension study. The ninth patient delayed entering the extension study and, while off treatment, experienced progression of liver disease requiring an urgent liver transplant. As previously reported, one patient paused treatment but has recently been deemed eligible to resume treatment by an independent safety committee.
At one year of treatment with sebelipase alfa, patients had sustained reductions in both ALT and AST, frequently into the normal range, with mean percentage decreases in ALT and AST from the pre-treatment baseline to month 12 of the extension study of 58% and 40%, respectively (p=0.016 for both comparisons).