BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage
biopharmaceutical company developing therapeutic products for rare
diseases, today announced presentations at the upcoming LDN WORLD
Symposium being held February 13-15 in Orlando, Florida. During an oral
presentation on Friday, February 15, at 1:00 PM EST, Dr. Manisha Balwani
will present 38-week data from the Phase I/II extension study of
sebelipase alfa in adults with late onset lysosomal acid lipase
deficiency (LAL Deficiency). An additional poster at the LDN WORLD
Symposium will highlight the ability of SBC-103 to reduce the
accumulation of substrate in the brain of a Mucopolysaccharidosis IIIB
(also known as MPS IIIB, or Sanfilippo B) animal model.
Data Presentations and Synageva Satellite Symposium at the LDN WORLD
On Friday, February 15, at 1:00 PM EST, Manisha Balwani, MD, MS,
Assistant Professor of Genetics and Genomic Sciences and Assistant
Professor of Medicine, Mount Sinai School of Medicine, New York, NY,
will present 38-week data from the Phase I/II extension study of
sebelipase alfa. 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. Eight of nine
patients have enrolled in the extension study and seven of these eight
have now completed the first 38 weeks of the study.
Sebelipase alfa effects observed in this study at 24 weeks were
sustained through 38 weeks. Sebelipase alfa continues to reduce liver
damage with sustained reductions in both ALT and AST, frequently into
the normal range. In addition, maintenance of the improvements in the
dyslipidemia associated with LAL Deficiency was also observed.
Sebelipase alfa produced mean percent decreases for ALT and AST from the
initial baseline to week 38 of the extension study of 54% and 40%,
respectively (p=0.016 for both comparisons). In addition, sebelipase
alfa resulted in mean percent decreases from the initial baseline to
week 38 of the extension study for LDL-C of 47% (p=0.016), total
cholesterol of 33% (p=0.016), triglycerides of 29% (p=0.047), as well as
a mean increase in HDL of 15% (p=0.313).
Sebelipase alfa was generally well tolerated throughout the initial 38
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. One patient with a moderate (Grade 2)
allergic type infusion-related reaction has paused treatment with
sebelipase alfa pending further testing. No anti-drug antibodies have
been detected in any subjects in either the initial portion or 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 treatedwith elective cholecystectomy.
This patient has continued treatment with sebelipase alfa without
changes in dosing and administration. These two serious adverse events
were considered unlikely related to sebelipase alfa.
SBC-103 reduces accumulated substrate levels in the brain of a MPS
IIIB mouse model
A poster entitled "Biochemical Evidence of the Effects of SBC-103, A
Recombinant Human Alpha-N-Acetylglucosaminidase in a
Mucopolysaccharidosis IIIB Mouse Model Using an Improved Analytical
Method for Substrate Quantification," describes the preclinical activity
Measurement of abnormal substrate in the brain has historically been
challenging in this disease. This poster describes an improved method
for quantifying heparan sulfate disaccharides (or HSD, the elevated
substrate in MPS IIIB patients). This improved method showed clear
increases in substrate in the MPS IIIB mouse disease model and that
treatment with SBC-103 using various dosing approaches produced
dose-dependent HSD level reductions in the brain, liver and kidney
tissues of the mouse disease model.
An additional poster at this year's LDN WORLD Symposium is:
"Co-Localization of Macrophage Aggregation and Fibrosis in a Rat Model
of Lysosomal Acid Lipase (LAL) Deficiency and the Effects of Enzyme
Replacement with SBC-102," J. Rutkowski, et al.
Synageva-sponsored satellite symposium
On Friday, February 15th at 6:30-7:30 AM EST, Synageva will
sponsor a breakfast symposium entitled, "LAL Deficiency: Are We Missing
the Diagnosis and Does it Matter " chaired by Gregory A. Grabowski, MD,
The A. Graeme Mitchell Chair of Human Genetics, Professor and Director,
Cincinnati Children's Hospital Medical Center.
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 lysosomal storage disease
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 the LDN WORLD Symposium
The LDN WORLD Symposium is an ACCME-accredited annual symposium which
includes lectures and poster presentations on basic, translational and
clinical research for lysosomal storage disorders. The goal of the
meeting is to provide an interdisciplinary forum to explore and discuss
specific areas of interest, research and clinical applicability related
to lysosomal diseases.
About Synageva BioPharma Corp.
Synageva is a clinical stage biopharmaceutical company focused on
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
"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 January 3, 2013, and
other filings Synageva periodically makes with the SEC and others of
which are not. 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.
[ Back To NFVZone's Homepage ]