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

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

Business Wire

LEXINGTON, Mass. -- February 11, 2013

Synageva BioPharma Corp. (Synageva) (NASDAQ: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

“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®) (NYSE: 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

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 January3, 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
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