Synageva BioPharma Reports Third Quarter 2012 Financial Results

  Synageva BioPharma Reports Third Quarter 2012 Financial Results

- Data at NSGC meeting highlights late onset LAL Deficiency disease severity -

Business Wire

LEXINGTON, Mass. -- November 06, 2012

Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage
biopharmaceutical company developing therapeutic products for rare disorders,
today reported third quarter 2012 financial results and announced data from
two posters on late onset LAL Deficiency presented at the recent National
Society for Genetic Counselors (NSGC) meeting held October 24-27, 2012, in
Boston, MA.

Late onset LAL Deficiency literature review data presented at NSGC meeting

During a poster session at the NSGC meeting, investigators presented their
analysis of 135 cases of late onset LAL Deficiency (or Cholesteryl Ester
Storage Disease, CESD) found in the published scientific literature for whom
clinical information, liver pathology, and serum lipid levels were reported.
The analysis revealed CESD-associated liver disease in all patients, including
hepatomegaly in 99% of patients and elevated transaminases in all 52 patients
with ALT & AST results. The investigators’ review of all 112 biopsy samples
demonstrated characteristic histopathology including massive lysosomal lipid
accumulation (microvesicular steatosis), cholesteryl ester crystals, fibrosis,
and/or micronodular cirrhosis. Liver failure resulted in esophageal varices in
12 patients, nine liver transplants, and 8 of 11 reported deaths.

An additional poster from the same group of cases provided further details of
the liver biopsy results. In CESD patients who had liver biopsies, fibrosis
and/or cirrhosis due to abnormal fat accumulation affected more than 50% of
patients, and importantly these abnormalities occurred in some patients within
the first year of life. “These analyses clearly demonstrate the serious,
chronic health consequences of late onset LAL Deficiency, and also remind us
again that these complications can strike very early in childhood,” said
Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical
Officer of Synageva BioPharma. “The insights from this extensive literature
review are consistent with findings from our ongoing review of the natural
history of late onset LAL Deficiency.”

Both posters entitled “Liver Disease and Chronic Liver Failure have Complete
Penetrance in Cholesteryl Ester Storage Disease, a Form of Lysosomal Acid
Lipase Deficiency” and “Cholesteryl Ester Storage Disease (CESD): An
Under-Recognized Disease of Lysosomal Acid Lipase Deficiency with Liver
Dysfunction/Failure and Hyperlipidemia” were submitted by Donna L. Bernstein,
MS, CGC et al, from the Division Of Medical Genetics, Steven and Alexandra
Cohen Medical Center of New York, North Shore-Long Island Jewish Health
System, New York.

Third Quarter 2012 Financial Results

For the quarter ended September 30, 2012, Synageva reported a net loss of
$10.2 million compared to a net loss of $6.8 million for the corresponding
quarter of the prior year. Results for the quarter ended September 30, 2011
refer to the results for Synageva BioPharma Corp., the private predecessor to
the company resulting from the combination of private Synageva and Trimeris.

Revenue for the quarter ended September 30, 2012 of $5.4 million consists of
$2.1 million of Fuzeon royalties from Roche, as well as revenue from
Synageva’s collaboration agreements of $3.3 million. Total operating expenses
for the quarter ended September 30, 2012, including R&D and G&A expenses,
totaled $15.6 million compared to $7.0 million for the corresponding quarter
of the prior year. Non-cash stock-based compensation expense totaled $1.6
million for the quarter ended September 30, 2012, compared to $0.1 million for
the corresponding quarter of the prior year. Non-cash amortization of
developed technology totaled $1.0 million for the quarter ended September 30,
2012. There was no amortization of developed technology in the corresponding
quarter of the prior year.

As of September 30, 2012, Synageva had a cash balance of $233.4 million that
includes net proceeds from a July 2012 public offering of the company’s common
stock. There is no outstanding debt.

2012 Financial Outlook

Synageva reiterates its previous net operating loss guidance of between $40
and $45 million for 2012. The net operating loss is primarily due to 
investments necessary to support the global clinical development of lead
program SBC-102 (sebelipase alfa), the expansion of a global commercial
infrastructure, and the advancement of pipeline programs.

About Synageva’s Lead Program

Sebelipase alfa is a recombinant form of the human Lysosomal Acid Lipase (LAL)
enzyme under development by Synageva as an enzyme replacement therapy for LAL
Deficiency, a lysosomal storage disorder (LSD). Synageva currently evaluates
sebelipase alfa in global clinical trials and 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

Lysosomal Acid Lipase Deficiency is a rare, autosomal recessive LSD that is
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, spleen and blood vessel walls leads to complications resulting in
significant morbidity and mortality. Early onset LAL Deficiency, sometimes
called Wolman disease, affects infants and is characterized by severe
malabsorption, growth failure, and hepatic failure and is usually fatal within
the first year of life.

About Synageva BioPharma Corp.

Synageva is a clinical stage biopharmaceutical company focused on the
discovery, development, and planned 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 assembled a team with a proven record of bringing therapies to
patients with rare diseases.

Further information regarding Synageva BioPharma Corp. is available at

Forward-Looking Statements

This news release contains “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 Quarterly Report
on Form 10-Q filed with the Securities and Exchange Commission (the “SEC”) on
August 3, 2012 and other filings Synageva periodically makes with the SEC, and
others of which are not. Synageva’s future financial results may differ from
those currently anticipated due to a number of factors, including
unanticipated costs in its research and development programs, fluctuations in
royalty revenues and unplanned costs associated with maintaining and enforcing
patents and other patent-related costs. 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.

Synageva BioPharma Corp.
Statement of Operations
(Unaudited and in thousands, except per share amounts)

                        Three Months Ended           Nine Months Ended
                        September 30,                September 30,
                          2012         2011          2012         2011
Royalty revenue         $ 2,132        $ -           $ 5,028        $ -
Collaboration and         3,296          88            4,990          193
license revenue
Other revenue            -             96           56            292
Total revenue             5,428          184           10,074         485
Operating expenses:
Research and              11,373         4,376         26,793         12,197
General and              4,244         2,627        11,051        5,906
Total operating          15,617        7,003        37,844        18,103
Loss from                 (10,189)       (6,819)       (27,770)       (17,618)
Other income              -              16            -              (183)
(expense), net
Interest income          7             (20)         12            (27)
(expense), net
Net loss                $ (10,182)     $ (6,823)     $ (27,758)     $ (17,828)
Basic and diluted
net loss per share      $ (0.43)       $ (92.21)     $ (1.26)       $ (143.77)
Weighted average
shares used in
basic and diluted        23,825        74           21,984        124
per share
computations (1)

(1) Per share computations for the three months ended September 30, 2011 are
based on Private Synageva’s historic common stock balance, which excludes the
impact of the conversion of preferred stock.

Synageva BioPharma Corp.
Consolidated Balance Sheet Data
(in thousands)

                        September 30,   December 31,
                                 2012             2011
Cash and cash equivalents      $  233,407        $  60,232
Working capital                   223,981           56,392
Total assets                      256,770           83,298
Accumulated deficit               (143,598)         (115,840)
Total stockholders' equity     $  243,022        $  74,048


Matthew Osborne, 781-357-9947
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