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- Data at NSGC meeting highlights late onset LAL Deficiency disease severity -
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
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
Third Quarter 2012 Financial Results
For the quarter ended
Revenue for the quarter ended
As of
2012 Financial Outlook
Synageva reiterates its previous net operating loss guidance of between
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
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 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
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
"Dedicated to Rare Diseases®"is a registered trademark and "Synageva
BioPharma™" is a trademark of
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| Statement of Operations | ||||||||||||||||||||||
| (Unaudited and in thousands, except per share amounts) | ||||||||||||||||||||||
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Three Months Ended
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Nine Months Ended
September 30, |
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| 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
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| Consolidated Balance Sheet Data | |||||||||||||
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| 2012 | 2011 | ||||||||||||
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(Unaudited) |
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| 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 | |||||||||
Synageva
matthew.osborne@synageva.com
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