Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, joins the National Organization for Rare Disorders (NORD), the European Organisation for Rare Diseases (EURORDIS), The Global Genes Project, and others around the world in observing Rare Disease Day. On the last day of February each year, patient organizations, industry, and other participants conduct special events to raise awareness and advance policies to improve conditions for people living with rare diseases. The theme, or slogan, this year for Rare Disease Day is “Join Together for Better Care” to help convey the global solidarity of the rare disease community which shares many of the same medical, social and economic challenges. "Rare Disease Day is a time to focus on the 30 million Americans living with rare diseases and issues such as the need for more research and greater access to care," said Peter L. Saltonstall, NORD President and CEO. "The theme for 2014 is Join Together for Better Care, and we are emphasizing the importance of collaboration in addressing the needs of those affected by these diseases, which tend to be serious and complex." Synageva is also proud to join efforts this year with The Global Genes Project, a leading non-profit organization for rare and genetic diseases. Along with displaying the signature Blue Denim Genes Ribbon™ associated with The Global Genes Project, Synageva employees will participate in a volunteer fund-raiser with proceeds to benefit the Support Organization for Lysosomal Acid Lipase Deficiency Advocacy, Care and Expertise (LAL Solace), a patient advocacy group for individuals affected by lysosomal acid lipase deficiency (LAL Deficiency). In the U.S., a rare disease is one that affects fewer than 200,000 Americans, and in Europe, less than one in 2,000 citizens. According to EURORDIS, approximately 80% of rare diseases have a genetic origin.
Approximately 7,000 rare diseases have been identified to date, affecting nearly 30 million Americans and 30 million Europeans.For more information about Rare Disease Day activities in the U.S., go to www.rarediseaseday.us. For information about Rare Disease Day activities globally, go to www.rarediseaseday.org. For more information about The Global Genes Project™, go to www.globalgenes.org. For more information about LAL Solace, go to www.lalsolace.org. Sebelipase alfa for LAL Deficiency LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency presenting in children and adults, historically called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis. These complications are due to the buildup of fatty material in the liver, blood vessel walls and other tissues as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression with death, usually in the first six months of life. Affected infants develop severe malabsorption, growth failure and liver complications. Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is evaluating sebelipase alfa in global Phase 3 clinical trials in infants, children and adults with LAL Deficiency. Sebelipase alfa has been granted orphan designation by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. SBC-103 for MPS IIIB 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 decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death. SBC-103 is a recombinant form of the human NAGLU enzyme being developed by Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing approaches, SBC-103 reduced HS substrate storage in the brain, liver and kidney in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA and the EMA. Synageva’s additional pipeline programs and manufacturing platform Synageva’s additional pipeline programs include other proteins targeting rare diseases at various stages of preclinical development. These diseases are characterized by significant morbidity and mortality and these programs are selected based on scientific rationale, high unmet medical need, potential to impact disease course and strategic alignment with the company’s corporate focus. In addition to these novel pipeline programs, Synageva is leveraging its manufacturing platform to develop improved biologic therapies for diseases with high unmet medical need.
Synageva’s proprietary manufacturing platform utilizes technology to produce drug product with consistent characteristics that enable robustness and flexibility during scale-up. In addition, the platform can provide favorable structural properties for bio-distribution and cell targeting in comparison to glycoproteins produced from other sources.Synageva routinely posts information that may be important to investors in the “Investor Relations” section of the company’s web site at www.synageva.com. Synageva encourages investors and potential investors to consult this web site regularly for important information about the company. Further information regarding Synageva BioPharma Corp. is available at www.synageva.com. 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, plans for leveraging our manufacturing platform to advance our pipeline programs and develop improved biologic therapies, and our belief that our platform can provide favorable structural properties for bio-distribution and cell targeting in comparison to glycoproteins produced from other sources. Many factors may cause actual results to differ materially from those expressed or implied by forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known, including risks that the results of our clinical trials may not support further development of our product candidates due to safety, efficacy or other reasons, the content and timing of decisions by the FDA and other regulatory authorities, the ability to maintain the current favorable protein structural properties for bio-distribution and cell targeting as compared to glycoproteins produced from other sources, and the risks identified under the heading “Risk Factors” in Synageva’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the “SEC”) on November 4, 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 of Synageva BioPharma Corp. “Synageva BioPharma™” is a trademark of Synageva BioPharma Corp.