|[February 04, 2013]
Hepatology Publishes 12-Week Phase I/II Extension Study Data of Sebelipase Alfa in Adults with Late Onset LAL Deficiency
LEXINGTON, Mass. --(Business Wire)--
BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage
biopharmaceutical company developing therapeutic products for rare
diseases, today announced publication of the 12-week Phase I/II
extension study data of sebelipase alfa in adults with lysosomal acid
lipase deficiency (LAL Deficiency) in the online version and an upcoming
print edition of Hepatology.
"Children and adults with late onset LAL Deficiency can suffer from
serious liver complications and accelerated atherosclerosis," said
Manisha Balwani, MD, MS, Assistant Professor of Genetics and Genomic
Sciences and Assistant Professor of Medicine, Icahn School of Medicine
at Mount Sinai, New York, NY, and principal investigator in the study.
"Based on its mechanism of action, sebelipase alfa addresses the root
cause of LAL Deficiency, and the Phase I/II extension study in adults
with LAL Deficiency illustrates that sebelipase alfa improves patients'
abnormalities associated with the disease."
About the Phase I/II extension study of sebelipase alfa in adults
with late onset LAL Deficiency
Nine adults with LAL Deficiency were enrolled in the Phase I/II trial.
After completing the initial portion of the Phase I/II trial, patients
were allowed to continue treatment with sebelipase alfa as part of a
long-term, open-label extension study.
In the extension study, patients received four once-weekly infusions of
sebelipase alfa (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg) and then
transitioned to every other week infusions of sebelipase alfa (1.0 mg/kg
or 3.0 mg/kg). Eight of nine patients enrolled in the extension study.
Data published in Hepatology were derived from the seven patients
who completed the first 12 weeks of dosing in the extension study.
For these seven patients, sebelipase alfa produced mean percent
decreases for ALT and AST from the initial baseline to week 12 of the
extension study of 52% and 36%, respectively (p<0.05 for both). In
addition, sebelipase alfa resulted in mean percent decreases from the
initial baseline to week 12 of the extension study for LDL-C of 27%
(p=0.078), total cholesterol of 22% (p=0.047), triglycerides of 28%
(p=0.016), as well as mean increases in HDL of 15% (p=0.016).
Patients in the Phase I/II trial had a mean age of 32 years (range
19-45); two-thirds were male. Eight of the nine patients had evidence of
hepatomegaly on clinical exam, and two of the nine patients had evidence
of more advanced liver disease, including cirrhosis and portal
hypertension. Seven patients had a history of other cardiovascular
conditions. Seven of the nine patents were receiving lipid modifying
therapies including ezetimibe, statins, and other medications.
Sebelipase alfa was well tolerated throughout the initial 12 weeks of
the extension study. The majority of adverse events were mild and
unrelated to sebelipase alfa. Infusion-related reactions were uncommon
and the majority were gastrointestinal (diarrhea, abdominal cramping)
events of mild severity. No anti-drug antibodies were detected in any
subjects in either the initial portion or the 12-week extension portion
of the Phase I/II study. A single patient during the extension study
developed acute cholecystitis and cholelithiasis (two serious adverse
events) which were later treated with elective cholecystectomy. These
two serious adverse events were considered unlikely related to
sebelipase alfa. This patient has continued treatment with sebelipase
alfa without changes in dosing and administration.
"The publication of these data in Hepatology underscores the
ability of sebelipase alfa to reduce markers of liver damage and improve
dyslipidemia in these adults with LAL Deficiency," said Anthony Quinn,
MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of
Synageva BioPharma. "Along with the Phase III trial in children and
adults with late onset LAL Deficiency, this publication will also help
support efforts to raise awareness for a disease that remains an
underappreciated cause of cirrhosis in children and adults."
About Synageva's Lead Program
alfa (formerly referred to as 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
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
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 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.
This news release and oral statements made from time to time by Synageva
representatives in respect of the same subject matter may contain
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Securities Litigation Reform Act of 1995. Such statements can be
identified by introductory words such as "expects," "plans," "intends,"
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similar meaning and by the fact that they do not relate strictly to
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differ materially from forward-looking statements, including inaccurate
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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 (News - Alert)") on January 3, 2013, and
other filings Synageva periodically makes with the SEC and others of
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