Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today reported 78-week results from an ongoing extension study with sebelipase alfa in adults with Lysosomal Acid Lipase (LAL) Deficiency. Chester B. Whitley, Ph.D., M.D., Professor, Director of the Gene Therapy Center, Advanced Therapies and PKU Clinic, Department of Pediatrics and Experimental and Clinical Pharmacology at the University of Minnesota, presented the results during an oral presentation at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting held in Chicago, IL. With six of the eight patients enrolled in the Phase 1/2 extension study completing 18 months of treatment, the results show sustained improvements in biochemical markers of liver damage and 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 NASPGHAN
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 statins, ezetimibe, and other medications.
Eight of nine patients continued treatment with sebelipase alfa as part of an ongoing, 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. One patient continues on treatment and has not yet reached the 78-week time point.