Sebelipase Alfa Phase 3 Data In Children And Adults With LAL Deficiency Selected For Oral Presentation During The Late-Breaking Session At The Liver Meeting®

LEXINGTON, Mass., Oct. 8, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that data from its Phase 3, global clinical study evaluating sebelipase alfa in children and adults with LAL Deficiency has been selected for oral presentation during the late-breaking session at The Liver Meeting®, the 65^th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), being held November 7-11 in Boston, MA. 

The data will be presented during the late-breaking oral session on Monday, November 10, 4:00-4:15 p.m. EST:

• "Results of a Global Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Sebelipase Alfa as an Enzyme Replacement Therapy in Children and Adults with Lysosomal Acid Lipase Deficiency".  Publication number: LB-5
• Presenting Author: Manisha Balwani, M.D., Mount Sinai School of Medicine, New York, NY

The abstract supporting the late-breaking oral presentation is now available on the AASLD website at: http://aasld2014.abstractcentral.com/planner.jsp.

About Sebelipase Alfa for LAL Deficiency

Lysosomal Acid Lipase Deficiency (LAL D) is a life-threatening, underdiagnosed disease that manifests with significant morbidity and early mortality.  LAL D causes progressive and multisystemic organ damage including hepatic cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications.  LAL D often manifests in childhood but can be diagnosed at all ages with a simple blood test.  LAL D is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.  LAL D was historically called Cholesteryl Ester Storage Disease in children and adults, and Wolman disease in infants.

Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL D.  Synageva is evaluating sebelipase alfa in global Phase 3 clinical trials in infants, children and adults with LAL D.  Sebelipase alfa has been granted orphan designation 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, and Breakthrough Therapy designation by the FDA for LAL D presenting in infants.

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 planned global commercial launch of sebelipase alfa for LAL Deficiency.  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, 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 FDA 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 (SEC) on July 31, 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.

"Synageva" is a trademark, and "Dedicated to Rare Diseases" is a registered trademark of Synageva. 

"The Liver Meeting" is a registered trademark of the American Association for the Study of Liver Diseases. 

SOURCE Synageva BioPharma Corp.