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