Synageva BioPharma™ Highlights Data at the NASPGHAN Meeting


Synageva BioPharma™ Highlights Data at the NASPGHAN Meeting

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (NAS: GEVA) , a biopharmaceutical company developing therapeutic products for rare diseases, today reported 78-week results from an ongoing extension study with sebelipase alfa in adults with Lysosomal Acid Lipase (LAL) Deficiency. Chester B. Whitley, Ph.D., M.D., Professor, Director of the Gene Therapy Center, Advanced Therapies and PKU Clinic, Department of Pediatrics and Experimental and Clinical Pharmacology at the University of Minnesota, presented the results during an oral presentation at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting held in Chicago, IL. With six of the eight patients enrolled in the Phase 1/2 extension study completing 18 months of treatment, the results show sustained improvements in biochemical markers of liver damage and 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 NASPGHAN

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 statins, ezetimibe, and other medications.

Eight of nine patients continued treatment with sebelipase alfa as part of an ongoing, 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. One patient continues on treatment and has not yet reached the 78-week time point.

At 78 weeks of treatment with sebelipase alfa, patients continued to have sustained reductions in both ALT and AST, frequently into the normal range, from the pre-treatment baseline. In addition, sebelipase alfa maintained improvement in dyslipidemia associated with LAL Deficiency with decreases in LDL and triglycerides and increases in HDL from the pre-treatment baseline to week 78 of the extension study. Sebelipase alfa also reduced patients' liver fat fraction and liver volume from the beginning of the extension study to week 52, the latest time point for these assessments. Liver fat fraction and liver volume were measured by multi-echo magnetic resonance imaging (MRI).

Sebelipase alfa was generally well tolerated through 78 weeks of the extension study. Most adverse events were mild and unrelated to sebelipase alfa. Infusion-related reactions were uncommon and the majority were mild and gastrointestinal in nature (diarrhea, abdominal cramping). No anti-drug antibodies have been detected in the patients tested to date in either the initial portion or extension portion of the Phase 1/2 study. Two serious adverse events (cholecystitis/cholelithiasis) considered unlikely related to sebelipase alfa occurred in one patient and this patient continues treatment with sebelipase alfa in the study. No drug-related serious adverse events have been reported in this study to date.

Natural history study data from infants with LAL Deficiency

Data from a natural history study of infants diagnosed with Wolman disease (LAL Deficiency presenting in infants) were also presented as a poster at this year's NASPGHAN meeting. The natural history study in infants represents a retrospective, observational study based on the compilation of patient records from more than 30 infants diagnosed with this condition. The median age of death in these patients was less than four months of age.

Synageva-sponsored satellite symposium

During the NASPGHAN meeting, Synageva sponsored a satellite symposium entitled "Fatty Liver Disease: It's Not All Related to Obesity." The symposium was chaired by Maureen Jonas, M.D., Clinical Director, Hepatology Medical Director, Liver Transplant Program, Professor of Pediatrics, Harvard Medical School, Boston, MA.

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 presenting in children and adults, historically 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 as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression with death, usually in the first six months of life. Affected infants develop severe malabsorption, growth failure and liver complications.

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 LAL Deficiency presenting in infants.

About Synageva's Next Program: 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 amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.

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 HS substrate storage in the brain, liver and kidney in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA and the EMA.

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 the company's web site at Synageva encourages investors and potential investors to consult this web site regularly for important information about the company.

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 for additional pipeline programs that meet certain criteria, our plans for leveraging our manufacturing platform to develop improved biologic therapies and our belief that our platform can provide consistent characteristics in drug products and 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 our additional pipeline programs do not meet all of the criteria that we use for assessment, these pipeline programs are not able to advance beyond an early stage, our efforts to develop improved biologic therapies may not be successful, we are unable to maintain the current favorable protein structural properties for bio-distribution and cell targeting as compared to glycoproteins produced from other sources, we are unable to rely on Breakthrough Therapy designation, the implications of which cannot be determined at this time, and the risks identified under the heading "Risk Factors" in the Company's prospectus supplement filed with the Securities and Exchange Commission (the "SEC") on September 25, 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

KEYWORDS: United States North America Massachusetts


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