Hepatology Publishes 12-Week Phase I/II Extension Study Data of Sebelipase Alfa in Adults with Late

Hepatology Publishes 12-Week Phase I/II Extension Study Data of Sebelipase Alfa in Adults with Late Onset LAL Deficiency

- Sebelipase alfa improves markers of liver damage and dyslipidemia associated with LAL Deficiency-

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (NAS: GEVA) , a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced publication of the 12-week Phase I/II extension study data of sebelipase alfa in adults with lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Hepatology.

"Children and adults with late onset LAL Deficiency can suffer from serious liver complications and accelerated atherosclerosis," said Manisha Balwani, MD, MS, Assistant Professor of Genetics and Genomic Sciences and Assistant Professor of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, and principal investigator in the study. "Based on its mechanism of action, sebelipase alfa addresses the root cause of LAL Deficiency, and the Phase I/II extension study in adults with LAL Deficiency illustrates that sebelipase alfa improves patients' abnormalities associated with the disease."

About the Phase I/II extension study of sebelipase alfa in adults with late onset LAL Deficiency

Nine adults with LAL Deficiency were enrolled in the Phase I/II trial. After completing the initial portion of the Phase I/II trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study.

In the extension study, patients received four once-weekly infusions of sebelipase alfa (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg) and then transitioned to every other week infusions of sebelipase alfa (1.0 mg/kg or 3.0 mg/kg). Eight of nine patients enrolled in the extension study. Data published in Hepatology were derived from the seven patients who completed the first 12 weeks of dosing in the extension study.

For these seven patients, sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to week 12 of the extension study of 52% and 36%, respectively (p<0.05 for both). In addition, sebelipase alfa resulted in mean percent decreases from the initial baseline to week 12 of the extension study for LDL-C of 27% (p=0.078), total cholesterol of 22% (p=0.047), triglycerides of 28% (p=0.016), as well as mean increases in HDL of 15% (p=0.016).

Patients in the Phase I/II trial had a mean age of 32 years (range 19-45); two-thirds were male. Eight of the nine patients had evidence of hepatomegaly on clinical exam, and two of the nine patients had evidence of more advanced liver disease, including cirrhosis and portal hypertension. Seven patients had a history of other cardiovascular conditions. Seven of the nine patients were receiving lipid modifying therapies including ezetimibe, statins, and other medications.

Sebelipase alfa was well tolerated throughout the initial 12 weeks 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 (diarrhea, abdominal cramping) events of mild severity. No anti-drug antibodies were detected in any subjects in either the initial portion or the 12-week extension portion of the Phase I/II 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 has continued treatment with sebelipase alfa without changes in dosing and administration.

"The publication of these data in Hepatology underscores the ability of sebelipase alfa to reduce markers of liver damage and improve dyslipidemia in these adults with LAL Deficiency," said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of Synageva BioPharma. "Along with the Phase III trial in children and adults with late onset LAL Deficiency, this publication will also help support efforts to raise awareness for a disease that remains an underappreciated cause of cirrhosis in children and adults."

About Synageva's Lead Program

Sebelipase alfa (formerly referred to as SBC-102) is a recombinant form of the human LAL enzyme under development by Synageva as an enzyme replacement therapy for LAL Deficiency, a lysosomal storage disorder (LSD). Synageva is currently 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.

About LAL Deficiency

LAL Deficiency is a rare autosomal recessive LSD caused by a marked decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In these patients, the buildup of fatty material in the liver and blood vessel walls may lead to liver cirrhosis, liver failure and accelerated atherosclerotic events. Early onset LAL Deficiency, sometimes called Wolman disease, affects infants and is characterized by severe malabsorption, growth failure and liver failure, and is usually fatal within the first six months of life. There are no approved pharmacological therapies for LAL Deficiency. Success with stem cell and liver transplant appears to be limited by procedure-related morbidity and mortality.

About Synageva BioPharma Corp.

Synageva is a clinical stage 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. The company has assembled a team with a proven record of bringing therapies to patients with rare diseases.

Further information regarding Synageva BioPharma Corp. is available at www.synageva.com.

Forward-Looking Statements

This news release and oral statements made from time to time by Synageva representatives in respect of the same subject matter may contain "forward-looking statements" under the provisions of the Private Securities Litigation Reform Act of 1995. Such statements can be identified by introductory words such as "expects," "plans," "intends," "believes," "will," "estimates," "forecasts," "projects," or words of similar meaning and by the fact that they do not relate strictly to historical or current facts. 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 those identified under the heading "Risk Factors" in the Company's prospectus supplement filed with the Securities and Exchange Commission (the "SEC") on January 3, 2013, and other filings Synageva periodically makes with the SEC and others of which are not. 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.

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

For Synageva:
Matthew Osborne, 781-357-9947
matthew.osborne@synageva.com

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