Synageva BioPharma Announces Presentations At 11th Annual World Symposium

LEXINGTON, Mass., Feb. 11, 2015 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that data related to the company's programs will be the subject of three oral presentations and one poster presentation at the WORLDSymposium conference, February 9-13 in Orlando, Florida.

Oral presentations at this year's WORLD symposium include Kanuma (sebelipase alfa) data from the Phase 3 trial in children and adults with LAL Deficiency and the Phase 2/3 trial in infants with LAL Deficiency. 

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 DeficiencyThursday, February 12 at 3:30 p.m., presented by Barbara Burton, M.D., Professor of Pediatrics, Division of Genetics, Birth Defects and Metabolism, Northwestern University Feinberg School of Medicine, Chicago, IL.

Effect of Sebelipase Alfa on Survival and Liver Function in Infants with Rapidly Progressive Lysosomal Acid Lipase DeficiencyThursday, February 12 at 3:45 p.m., presented by Simon A. Jones, M.D., Consultant in Paediatric Inherited Metabolic Disease, Willink Unit, Manchester Centre for Genomic Medicine, Manchester Academic Health Sciences Centre, Genetic Medicine, St. Mary's Hospital, Manchester, UK.

In addition, the oral presentations include results from a study in individuals with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome).  The objectives of this study were to evaluate the integrity of the blood-brain barrier and structural brain abnormalities in MPS IIIB patients using multimodal magnetic resonance imaging (MRI), cerebrospinal fluid CSF-albumin index (CSF-AI), and measuring heparan sulfate (HS) levels in the cerebrospinal fluid (CSF), blood, and urine.   Five patients (between 6-8 years of age) with MPS IIIB enrolled in the study.  Clinical manifestations at baseline included cognitive delay, abnormal facial features, sleep disturbances and language delay.  Heparan sulfate analysis demonstrated elevated HS levels in the CSF, serum, and urine.  Results from the study revealed no leakage of the blood-brain barrier using dynamic contrast enhanced-MRI taken before, during and after intravenous injection of an MRI-visible contrast agent.  A small increase in CSF-AI was seen, suggestive of mild leakage of the blood-brain barrier.  Along with other preclinical data, the data from this study further support specific cellular transport, and not major leakage of the blood-brain barrier, as being important for movement of SBC-103 across the blood-brain barrier.

Evaluation of Blood-Brain Barrier (BBB) Integrity and Structural Abnormalities in MPS IIIB Patients Using Cerebrospinal Fluid/Serum Albumin Index (CSF-AI) and Multimodal MRIWednesday, February 11 at 10:15 a.m., presented by Sandra Rojas-Caro, M.D., Group Vice President, Clinical Research and Development, Synageva BioPharma, Lexington, MA.

A poster at the WORLD symposium included the following:

Effect of Sebelipase Alfa After Two Years in Adults with Lysosomal Acid Lipase Deficiency, Rojas-Caro et al.

Kanuma (sebelipase alfa) and LAL Deficiency

LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test.  LAL Deficiency causes progressive and multisystemic organ damage including cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications.  LAL Deficiency often manifests in childhood but can be diagnosed at all ages.  LAL Deficiency 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.

Kanuma is a recombinant form of the natural, human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency.  Kanuma 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, Kanuma received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.

SBC-103 and MPS IIIB

The mucopolysaccharidoses (MPS) consist of a group of rare lysosomal storage diseases 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.  MPS IIIB is caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of 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 natural human NAGLU enzyme that has favorable properties for enabling cellular uptake and has shown the ability to overcome the challenges previously encountered in producing recombinant human NAGLU.  The advancement of SBC-103 towards the clinic was supported by preclinical studies demonstrating that intravenously administered SBC-103 was able to cross the blood-brain barrier and reduce HS storage in the brain in an MPS IIIB animal model.  In addition, SBC-103 demonstrated transport across an in vitro model of the blood-brain barrier and distributed into the CSF in non-human primate studies.

The company is also conducting natural history studies in MPS IIIB.  These include a retrospective natural history study of deceased MPS IIIB patients that began in July 2013 and a prospective, longitudinal natural history study in living MPS IIIB patients that began in September 2014.  Natural history studies can help build an understanding of the manifestations and progression of disease and provide insights into biomarkers and other measures that may be useful as clinical outcomes.     

SBC-103 was granted orphan designation by the FDA in April 2013 and the European Medicines Agency (EMA) in June 2013 and received Fast Track designation by the FDA in January 2015.

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.

Medical information regarding Kanuma and LAL Deficiency is available by email at medinfo@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.   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, such as, the risk of delays in initiating or completing our clinical trials, unanticipated costs or delays in our research and development programs, unanticipated delays in our submission timelines, risk that the outcomes of our 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 the Company's prospectus supplement filed with the Securities and Exchange Commission (SEC) on January 7, 2015 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 these 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 BioPharma" and "Kanuma" are trademarks, and "Dedicated to Rare Diseases®" is a registered trademark, of Synageva BioPharma Corp. "WORLDSymposium" is a trademark of WORLDSymposia.

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SOURCE Synageva BioPharma Corp.