SBC-102

SBC-102 is an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency, a lysosomal storage disorder (LSD).  The product is a recombinant form of the human LAL enzyme.  This enzyme is responsible for the metabolism of cholesteryl esters and triglycerides that are delivered to lysosomes by a variety of routes including low-density lipoprotein (LDL) receptor mediated endocytosis.  SBC-102 contains glycan structures that are specifically recognized and internalized by specific receptors into key target cells.  In November 2010, Synageva reported data from preclinical studies at the American Society of Human Genetics.  In addition to demonstrating uptake and localization to lysosomes in vitro in key cell types, including macrophages and fibroblasts, SBC-102 reduced lipid substrate levels in diseased tissues and corrected disease abnormalities associated with deficiency of the LAL enzyme, including growth failure and liver pathology in a disease model of LAL Deficiency.  Additional data including a more detailed analysis of the effects on the liver abnormalities and the dose response were reported at the European Society for the Study of Liver Disease in March 2011.

We received orphan drug designations in both the US and EU in 2010 for SBC-102 for LAL Deficiency, and human dosing in clinical trials is underway.  In addition to clinical studies, we have initiated Natural History Studies in approximately 20 countries.

We are pursuing a broad development strategy that includes clinical trials in patients with both early onset and late onset LAL Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease (CESD) respectively. Our program goals, which have been developed in collaboration with the global regulatory authorities, are to assess safety and tolerability in infants, children and adults to demonstrate clinical meaningful effects on the medical complications of LAL Deficiency.

For more information about our clinical programs for SBC-102, please visit clinicaltrials.gov or click here if you would like to contact Synageva regarding LAL Deficiency.

To watch an informational video about LAL Deficiency, please click here.  (If you have trouble viewing this video, please find further directions here.)