Synageva BioPharma™ Announces One Year of Treatment Data with Sebelipase Alfa in Late Onset LAL Defi

Synageva BioPharma™ Announces One Year of Treatment Data with Sebelipase Alfa in Late Onset LAL Deficiency

-Sebelipase alfa effects sustained and treatment generally well tolerated-

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (NAS: GEVA) , a biopharmaceutical company developing therapeutic products for rare diseases, today reported 12-month results from an ongoing extension study with sebelipase alfa in adults with late onset Lysosomal Acid Lipase (LAL) Deficiency. The data will be presented today by Reena Sharma, M.D., Consultant, Adult Inherited Metabolic Diseases, Salford Royal Hospital, U.K., during an oral presentation at the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) annual meeting being held in London, U.K., May 8-11, 2013.

Details from the extension study with sebelipase alfa in adults with late onset LAL Deficiency

Nine adults with LAL Deficiency enrolled in the Phase 1/2 trial. After completing the initial four-week portion of the trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study. Eight of nine patients enrolled in the extension study and six of these eight have now completed the first 12 months of the study.

At one year, the effects of sebelipase alfa in this study were sustained as compared to the effects at nine months. Sebelipase alfa continues to provide evidence of a reduction in liver damage with sustained reductions in both ALT and AST, frequently into the normal range. Sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to month 12 of the extension study of 56% and 40%, respectively (p=0.031 for both comparisons). In addition, sebelipase alfa maintained the improvements in the dyslipidemia associated with LAL Deficiency as measured by mean percent decreases from the initial baseline to month 12 of the extension study for LDL-C of 63% (p=0.031), total cholesterol of 42% (p=0.031), triglycerides of 47% (p=0.031), as well as a mean increase in HDL of 29% (p=0.031).

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 (diarrhea, abdominal cramping) events of mild severity. One patient with a moderate (Grade 2) allergic type infusion-related reaction, who paused treatment with sebelipase alfa at nine months of the extension study, remains off treatment with further tests pending. No anti-drug antibodies have been detected in any subjects 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.

Synageva-sponsored satellite symposium

During the ESPGHAN meeting, Synageva will sponsor a satellite symposium entitled, "LAL Deficiency: Why Missing the Diagnosis Matters" chaired by Patrick McKiernan, M.D., Consultant Paediatrician, The Liver Unit, Birmingham Children's Hospital, Birmingham, U.K. The symposium is being held May 9 at 5:45-7:15 p.m., GMT.

About Synageva's lead programs sebelipase alfa for LAL Deficiency and SBC-103 for MPS IIIB

LAL Deficiency is a rare autosomal recessive lysosomal storage disorder (LSD) caused by a marked decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis in children and adults. These complications are due to the buildup of fatty material in the liver and blood vessel walls as a result of decreased LAL enzyme activity. Early onset LAL Deficiency, sometimes called Wolman disease, is the most rapidly progressive form of LAL Deficiency and 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 under development by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is 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.

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's but represent a clinically distinct subset of MPS's 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 under development 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. Synageva plans to enter SBC-103 into human clinical trials for MPS IIIB during the first half of 2014.

About Synageva BioPharma Corp.

Synageva is a 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.

Further information regarding Synageva BioPharma Corp. is available at

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. 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 our ability to transition from preclinical programs to human clinical trials and those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on May 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 and "Synageva BioPharma™" is a trademark of Synageva BioPharma Corp.

Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947

KEYWORDS:   United Kingdom  United States  Europe  North America  Massachusetts


The article Synageva BioPharma™ Announces One Year of Treatment Data with Sebelipase Alfa in Late Onset LAL Deficiency originally appeared on

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