Synageva BioPharma™ Initiates Dosing in Phase 3 Trial of Sebelipase Alfa in Children and Adults with


Synageva BioPharma™ Initiates Dosing in Phase 3 Trial of Sebelipase Alfa in Children and Adults with Late Onset 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 that the first patient initiated treatment in the ARISE trial (Acid Lipase Replacement Investigating Safety and Efficacy), a global, Phase 3, randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with late onset lysosomal acid lipase deficiency (LAL Deficiency).

"The buildup of abnormal fats in LAL Deficiency can cause progressive and severe liver damage including cirrhosis in children and adults, as well as accelerated atherosclerosis," said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer at Synageva. "By replacing the deficient enzyme that causes the accumulation of these abnormal fats, sebelipase alfa addresses the root cause of LAL Deficiency. Based on data from the previously conducted preclinical and clinical studies with sebelipase alfa, the Phase 3 ARISE trial was designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with LAL Deficiency."

About ARISE:A global Phase 3 trial of sebelipase alfa in children and adults with late onset LAL Deficiency

The ARISE trial will enroll 50 patients (children and adults) with late onset LAL Deficiency. Patients enrolled in the trial are randomized on a one-to-one basis to every other week infusions of sebelipase alfa (1 mg/kg), or placebo for the double-blind treatment period of 20 weeks. Results from the double-blind period will be used to demonstrate efficacy and safety in support of global submissions for product registration. Patients who participate in the trial will qualify to enter a long-term, open-label extension period.

The primary endpoint of the trial is the proportion of patients relative to placebo who achieve normalization of alanine aminotransferase (ALT), a marker of liver damage, at the completion of the double-blind treatment period (week 20). Key secondary endpoints include the relative reduction from baseline to week 20 in LDL-C, non-HDL-C, triglycerides, the proportion of patients who achieve aspartate aminotransaminase (AST) normalization, and the relative increase in HDL-C. Additional secondary endpoints, including reductions in liver fat content and liver volume and improvements in liver pathology, will be examined in a proportion of patients who undergo these assessments. Deficiency of LAL enzyme activity will be confirmed during patient screening with a dried blood spot biochemical enzyme activity assay performed by Laboratory Corporation of America® Holdings (LabCorp®) (NYS: LH) , the central diagnostic testing laboratory performing the tests.

About Synageva's Lead Program

Sebelipase alfa (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 a team with a proven record of bringing therapies to patients with rare diseases.

Further information regarding Synageva BioPharma Corp. is available at

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. Synageva cannot be sure if its Phase 3 trial design will allow it to sufficiently assess the effects of sebelipase alfa on a broad range of abnormalities associated with LAL Deficiency nor can it be sure that it will be able to enroll its target number of patients in the Phase 3 trial. In addition, the results of the Phase 3 trial may not support the Company's anticipated submissions for product registration. Earlier clinical results are not necessarily predictive of any results that may be achieved from the Phase 3 trial. Because of this, statements regarding expectations of clinical trial results, patient enrollment or expected timing of ultimate regulatory approval cannot be regarded as actual predictions of when Synageva will obtain regulatory approval for sebelipase alfa by a particular regulatory agency. 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.

Matthew Osborne, 781-357-9947

KEYWORDS: United States North America Massachusetts


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