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Synageva BioPharma™ Reports 2013 Full Year Financial Results

Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today reported 2013 full year financial results and provided 2014 goals, other key objectives, and financial guidance. Synageva’s management team will host a conference call today at 4:30 p.m. EST to review the financial results and provide a general business update. To participate in today’s call by telephone, please dial (877) 445-4603 for U.S. callers or (443) 295-9270 for international callers. In addition, the conference call will be webcast live from the “Webcasts & Presentations” section of the Investor Relations tab on the home page of Synageva’s website at

2013 Full Year Financial Results

For the year ended December 31, 2013, Synageva reported a net loss of $95.5 million compared to a net loss of $42.9 million for the corresponding period of the prior year.

Revenue for the full year ended December 31, 2013 of $13.4 million included $7.0 million of Fuzeon royalties from Roche, as well as revenue of $6.3 million from collaborations. Total costs and expenses for the full year 2013, including research and development (R&D) expenses, sales, general and administrative (SG&A) expenses, and amortization of developed technology, totaled $109.3 million. This compares to total costs and expenses for the full year 2012 of $58.0 million.

Synageva had cash, cash equivalents and short-term investments totaling $408.7 million on December 31, 2013, compared with cash, cash equivalents and short-term investments totaling $219.0 million on December 31, 2012.

2014 Financial Outlook

Synageva currently expects a net loss between $190 and $205 million for 2014. The net loss is primarily due to investments supporting the global clinical development program for sebelipase alfa, development of SBC-103, expansion of the global clinical, medical affairs and commercial infrastructure, expansion of manufacturing capabilities, as well as advancement of other preclinical pipeline programs.

Recent Company Highlights:
  • Initiated and completed enrollment of 66 children and adults with lysosomal acid lipase deficiency (LAL Deficiency) in the ARISE Phase 3 trial with sebelipase alfa
  • Completed the target enrollment in the Phase 2/3 trial in infants with LAL Deficiency and provided initial results
  • Presented 90-week data from the ongoing Phase 1/2 extension study of sebelipase alfa in adults with LAL Deficiency
  • Expansions to the global clinical, medical affairs and commercial infrastructure to further enhance disease awareness and identify more patients with LAL Deficiency
  • Completed construction on an additional commercial-scale manufacturing facility to further supply protein therapeutics for lead programs
  • Strengthened balance sheet as a result of completing follow-on offerings in January and September 2013, resulting in $281 million of net proceeds

Key Objectives:
  • Report top-line results from the Phase 3 ARISE trial with sebelipase alfa in children and adults with LAL Deficiency during the third quarter of 2014
  • Complete submission of a Biologic License Application (BLA) to the U.S. Food & Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for sebelipase alfa for the treatment of LAL Deficiency by the end of the first quarter of 2015
  • Continue global disease awareness programs to support identification of more infants, children and adults with LAL Deficiency
  • Initiate a clinical trial with SBC-103 in mucopolysaccharidosis IIIB (MPS IIIB) during mid-2014
  • Advance two additional programs into clinical trials by the end of 2016

Sebelipase alfa for LAL 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.

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. MPS IIIB, also known as Sanfilippo B syndrome, is caused by a 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.

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