Synageva BioPharma™ Highlights Data at the International Congress of Inborn Errors of Metabolism

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Synageva BioPharma™ Highlights Data at the International Congress of Inborn Errors of Metabolism

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (NAS: GEVA) , a biopharmaceutical company developing therapeutic products for rare diseases, today announced a poster presentation at the 12th International Congress of Inborn Errors of Metabolism (ICIEM) conference being held in Barcelona, Spain, September 3-6, 2013.

The poster, entitled "Long Term Effect of Sebelipase Alfa After One Year in Patients with Lysosomal Acid Lipase (LAL) Deficiency," V. Valayannopoulos et al, describes the results from the ongoing Phase 1/2 extension study in adults with LAL Deficiency treated with sebelipase alfa at one year. With seven of the eight patients enrolled in the study completing one full year of treatment, the results continue to show reduction in liver damage and improvement in the dyslipidemia associated with LAL Deficiency.


Details from the Phase 1/2 extension study with sebelipase alfa in adults with LAL Deficiency presented at ICIEM

Nine adults with LAL Deficiency with a median age of 29 years (range 19-45) enrolled in the Phase 1/2 trial. Seven of nine patients had a history of hepatomegaly and/or splenomegaly, and two of nine patients had evidence of more advanced liver disease, including cirrhosis and portal hypertension. All nine patients had a history of dyslipidemia, and seven of nine patients also had a history of other cardiovascular conditions. Seven of nine patients received treatment with lipid modifying therapies including ezetimibe, statins, and other medications.

Eight of nine patients continued treatment with sebelipase alfa as part of a long term open-label extension study. The ninth patient delayed entering the extension study and, while off treatment, experienced progression of liver disease requiring an urgent liver transplant. As previously reported, one patient paused treatment but has recently been deemed eligible to resume treatment by an independent safety committee.

At one year of treatment with sebelipase alfa, patients had sustained reductions in both ALT and AST, frequently into the normal range, with mean percentage decreases in ALT and AST from the pre-treatment baseline to month 12 of the extension study of 58% and 40%, respectively (p=0.016 for both comparisons).

In addition, sebelipase alfa maintained improvement in dyslipidemia associated with LAL Deficiency by reducing the mean levels of LDL from the pre-treatment baseline of 144 mg/dL (range 70-300) to 50 mg/dL (range 17-79) at month 12 of the extension study and by reducing the mean levels of triglycerides from the pre-treatment baseline of 152 mg/dL (range 80-277) to 91 mg/dL (range 43-166) at month 12 of the extension study. Sebelipase alfa increased mean levels of HDL from the pre-treatment baseline of 35 mg/dL (range 22-49) to 45 mg/dL (range 32-63) at month 12 of the extension study. These results translated into mean percentage decreases from the pre-treatment baseline to month 12 of the extension study in LDL of 60% (p=0.016) and triglycerides of 36% (p=0.047), as well as a mean increase in HDL of 29% (p=0.016).

Sebelipase alfa was generally well tolerated throughout the initial 12 months of the extension study. The majority of adverse events were mild and unrelated to sebelipase alfa. Infusion-related reactions were uncommon and the majority were gastrointestinal events (diarrhea, abdominal cramping) of mild severity. No anti-drug antibodies have been detected in any patients tested to date in either the initial portion or extension portion of the Phase 1/2 study. A single patient during the extension study developed acute cholecystitis and cholelithiasis (two serious adverse events) which were later treated with elective cholecystectomy. These two serious adverse events were considered unlikely related to sebelipase alfa. This patient continues treatment with sebelipase alfa without a change in dosing and administration.

About sebelipase alfa for LAL Deficiency

Lysosomal acid lipase deficiency (LAL Deficiency) is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency in children and adults, sometimes 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 and organs as a result of decreased LAL enzyme activity. Infants with LAL Deficiency, sometimes called Wolman disease, suffer from the most rapidly progressive form of LAL Deficiency that 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 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 early onset LAL Deficiency.

About 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 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 being developed 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 and the EMA. Synageva plans to be in clinical trials with SBC-103 during the first half of 2014.

About 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 our 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 our web site at www.synageva.com. Synageva encourages investors and potential investors to consult our web site regularly for important information about us.

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, our plans to enter into human clinical trials for MPS IIIB during the first half of 2014, our plans for leveraging the platform to 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 forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known, including, the possibility that preclinical data or regulatory delays cause our MPS IIIB program to not enter human clinical trials in the first half of 2014, the ability to maintain the current favorable protein structural properties for bio-distribution and cell targeting as compared to glycoproteins produced from other sources, the ability to rely on Breakthrough Therapy designation the implications of which cannot be determined at this time, and those additional risks identified under the heading "Risk Factors" in the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on August 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 of Synageva BioPharma Corp. "Synageva BioPharma™" is a trademark of Synageva BioPharma Corp.



Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947
matthew.osborne@synageva.com

KEYWORDS:   United States  North America  Massachusetts

INDUSTRY KEYWORDS:

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