Synageva BioPharma™ Joins the International MPS Network in Support of MPS Awareness Day 2013

Synageva BioPharma™ Joins the International MPS Network in Support of MPS Awareness Day 2013

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (NAS: GEVA) , a biopharmaceutical company developing therapeutic products for rare diseases, joins the International MPS Network and others around the world in observing MPS Awareness Day 2013. During MPS Awareness day, patient organizations, industry, and other participants conduct special events to raise awareness of Mucopolysaccharidoses (MPS) disease. Today, Synageva employees will host a breakfast with family members of individuals affected by MPS IIIB, also known as Sanfilippo B syndrome, to discuss their various experiences and challenges when faced with this devastating disease.

"We are excited to be a part of MPS Awareness Day," said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of Synageva. "As a company focused on rare diseases, we understand the impact MPS diseases have on the lives of patients and their families. We are pleased to be able to support the various organizations, patients and families, in their efforts to raise disease awareness around the world."

"We are very happy to have Synageva as a partner to increase awareness about MPS diseases today," said representatives of the International MPS Network in a joint statement. "There remains significant unmet need for many of the MPS diseases, and we look forward to the progress of Synageva's SBC-103 program for MPS IIIB."

MPS diseases are genetic lysosomal storage diseases (LSD) caused by the body's inability to produce specific enzymes. These missing or insufficient enzymes prevent the proper degradation of certain types of sugar molecules, or mucopolysaccharides, in cells, resulting in the storage of these sugar molecules in various tissues and organs throughout the body. There are 11 known enzyme deficiencies that give rise to seven distinct MPS diseases. Clinical features shared by the MPS diseases include a chronic and progressive disease course and multi-system involvement, but each MPS disease may present with unique clinical features. The International MPS Network estimates the occurrence of MPS diseases in the general population to be one in 25,000 births.

Synageva is developing SBC-103 as an enzyme replacement therapy for MPS IIIB. Most recently at the Lysosomal Disease Network meeting in February 2013, investigators presented preclinical data with SBC-103 demonstrating reductions in accumulated substrate levels in the brain of a MPS IIIB mouse model using various dosing approaches. Synageva plans to enter SBC-103 into human clinical trials for MPS IIIB during the first half of 2014.

For more information about MPS Awareness Day activities visit the websites of the International MPS Network ( and the National MPS Society (

About Synageva's lead programs sebelipase alfa for LAL Deficiency and SBC-103 for MPS IIIB

Lysosomal acid lipase deficiency (LAL Deficiency) is a rare autosomal recessive lysosomal storage disorder (LSD) caused by a marked decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis in children and adults. These complications are due to the buildup of fatty material in the liver and blood vessel walls as a result of decreased LAL enzyme activity. Early onset LAL Deficiency, sometimes called Wolman disease, is the most rapidly progressive form of LAL Deficiency and is usually fatal within the first six months of life. Affected infants develop severe malabsorption, growth failure and liver failure. There are no approved therapies for LAL Deficiency.

Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme under development by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is evaluating sebelipase alfa in global clinical trials for both early and late onset LAL Deficiency. Sebelipase alfa has been granted orphan designations by the U.S. Food and Drug Administration (FDA), the European Medicines Agency, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA.

The Mucopolysaccharidoses (MPS) consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. Mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is caused by a marked decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal sugars called heparan sulfate disaccharides (HSD) in the brain and other organs. The accumulation of abnormal HSD, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death. There are no approved therapies for MPS IIIB.

SBC-103 is a recombinant form of the human NAGLU enzyme under development by Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing approaches, SBC-103 reduced HSD substrate storage in the brains, liver and kidney tissues in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA.

About Synageva BioPharma Corp.

Synageva is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with life-threatening rare diseases and unmet medical need. Synageva has several protein therapeutics in its drug development pipeline.

Further information regarding Synageva BioPharma Corp. is available at

Forward-Looking Statements

This news release contains "forward-looking statements". Such statements generally can be identified by the use of words such as "anticipate," "expect," "plan," "could," "intend," "believe," "may," "will," "estimate," "forecast," "project," or words of similar meaning. These forward-looking statements address, among other matters, our expectations for entering into human clinical trials for MPS IIIB. Many factors may cause actual results to differ materially from forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known, including the risk that the results from our preclinical activities in MPS IIIB may not be sufficient to enter into human clinical trials in the timeframe we expect and those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on May 7, 2013, and other filings Synageva periodically makes with the SEC, and others of which are not known. No forward-looking statement is a guarantee of future results or events, and investors should avoid placing undue reliance on such statements. Synageva undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

"Dedicated to Rare Diseases®" is a registered trademark and "Synageva BioPharma™" is a trademark of Synageva BioPharma Corp.

Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947

KEYWORDS:   United States  North America  Massachusetts


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