Synageva BioPharma™ Highlights SBC-103 Data At The Lysosomal Disease Network (LDN) WORLD Symposium™

Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced SBC-103 data from an oral and poster presentation at the LDN WORLD Symposium being held February 11-13 in San Diego, California. Synageva is investigating SBC-103 as an enzyme replacement for the alpha-N-acetyl-glucosaminidase (NAGLU) enzyme for mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). MPS IIIB is caused by a decrease in NAGLU enzyme activity which leads to the buildup of abnormal amounts of heparan sulfate in the brain and other organs. The SBC-103 preclinical data presented at the meeting confirms results from previous studies, describes a potential mechanism for central nervous system uptake, and supports further clinical investigation of SBC-103 with intravenous administration.

As part of an oral presentation, Sandra Rojas-Caro, MD, Head of Clinical Research and Development at Synageva presented data from a preclinical study with SBC-103 in a mouse model of MPS IIIB. Data from this study confirmed that SBC-103 delivered by intravenous and intrathecal administration reduced abnormal heparan sulfate levels in the brain of NAGLU-deficient mice. In addition, intravenously administered SBC-103 increased NAGLU enzyme activity levels in the brain of a MPS IIIB mouse model and increased cerebral spinal fluid NAGLU enzyme activity in non-human primates in preclinical studies. These findings suggest that SBC-103 may have properties that allow it to cross the normal blood-brain barrier.

Additional data supporting this observation was provided as a poster from a study that investigated SBC-103 in an in vitro model of the blood-brain barrier. In this study, SBC-103 was effectively transported from the apical side (representing the blood) to the opposite basolateral side (representing the brain tissue). The addition of mannose-6-phospate inhibited directional transport by more than 90%, suggesting that the observed transport of SBC-103 was mediated by the mannose-6-phosphate receptor. These data suggest that the previously reported effects of intravenous SBC-103 on central nervous system substrate accumulation in an MPS IIIB disease model may be mediated by specific cellular transport across the blood-brain barrier.

If you liked this article you might like

The Nominees for Best Biopharma CEO of 2015 Are...

Bluebird, Pacira Pharmaceuticals Excite Life Science Investors

Bluebird, Pacira Pharmaceuticals Gain Steam in Burgeoning Life Science Space

New Lifetime High Today: Synageva BioPharma (GEVA)

Where the Money Was Made the Last 5 Years

Where the Money Was Made the Last 5 Years