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June 03, 2014

Synageva BioPharma™ Highlights Data Presentations And Satellite Symposium At The European Atherosclerosis Society Congress

Synageva BioPharma Corp. (Synageva) (GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced data presentations and a company-sponsored satellite symposium at the European Atherosclerosis Society (EAS) Congress being held May 31-June 3, 2014 in Madrid, Spain. 

Sebelipase alfa continues to improve disease-related abnormalities at two years from an ongoing Phase 1/2 trial in adults with LAL Deficiency

As part of an oral presentation, Radhika Tripuraneni, M.D., M.P.H., Senior Medical Director at Synageva, provided an update to the study.  With six of the eight patients enrolled in the ongoing Phase 1/2 extension study completing two years of treatment, sebelipase alfa continued to demonstrate sustained reductions in the biomarkers of liver damage (both ALT and AST), frequently into the normal range, from the pre-treatment baseline to two years of the extension study.  Sebelipase alfa also maintained improvements in dyslipidemia associated with LAL Deficiency, with decreases in LDL and triglycerides and increases in HDL from the pre-treatment baseline to two years of the extension study. 

Sebelipase alfa was generally well tolerated through two years of the extension study.  Most adverse events were mild and unrelated to sebelipase alfa.  Infusion associated reactions were uncommon, generally mild and gastrointestinal in nature (diarrhea, abdominal cramping).  No anti-drug antibodies have been detected and no drug-related serious adverse events have been reported in this study to date.  Two serious adverse events (cholecystitis/cholelithiasis) considered unlikely related to sebelipase alfa occurred in one patient and this patient continues treatment with sebelipase alfa in the study.

Satellite Symposium and Poster Presentation at EAS

Synageva sponsored a satellite symposium at the EAS on Saturday, May 31 at 12:00 p.m.–1:30 p.m., CEST.  The symposium entitled "Elevated LDL: Not Always Familial Hypercholesterolemia; Three Mystery Cases to Diagnose" was chaired by Emilio Ros, M.D., Lipid Clinic, Endocrinology & Nutrition Service, Institut d'Investigacions Biomediques August Pi Sunyer, Hospital Clinic, Barcelona, Spain.

In addition, Synageva announced the following as a poster presentation: "Identification, Lipoprotein Profiling and Treatment of Patients with Lysosomal Acid Lipase Deficiency", E.O. Stock, et al.

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 liver complications, malabsorption, and growth failure.

Sebelipase alfa 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 and Synageva's additional pipeline programs

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.  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 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.

Synageva routinely posts information that may be important to investors in the "Investor Relations" section of the company's website at www.synageva.com.  Synageva encourages investors and potential investors to consult this website regularly for important information about the company.

Further information regarding Synageva 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, our plans to develop our product candidates and pipeline programs, and their potential to address unmet medical need and impact disease course.  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, unanticipated costs or delays in our research and development programs, risk of delays in completing our clinical trials, risk that the outcomes of our preclinical or clinical trials may not support registration or further development of our product candidates due to safety, efficacy or other reasons, the content and timing of decisions by the U.S. Food and Drug Administration and other regulatory authorities, 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 May 1, 2014 and other filings Synageva periodically makes with the SEC, and others of which are not known.  Preclinical and clinical trial data are subject to differing interpretations, and regulatory agencies, as well as medical and scientific experts, may not share Synageva's views regarding this data or its implications.  Synageva may encounter problems or delays in preclinical and clinical development and the regulatory process. 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.  "Synageva BioPharma™" is a trademark of Synageva BioPharma Corp.