LEXINGTON, Mass., June 4, 2015 /PRNewswire/ -- Synageva
BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company
developing therapeutic products for rare disorders, today announced
the completion of enrollment in a Phase 1/2 trial with SBC-103 in
patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as
Sanfilippo B syndrome).
About the trial
The trial reached its targeted enrollment of nine patients, who
are two years of age or greater but less than 12 years of age, with
a definitive diagnosis of MPS IIIB and developmental delay.
Patients are being treated in one of three different dosing cohorts
(0.3 mg/kg, 1.0 mg/kg or 3.0 mg/kg) with every other week
intravenous administrations of SBC-103 for 24 weeks. Patients
who meet qualifying criteria may continue dosing with SBC-103 for
an extended period.
The primary endpoint of the trial is safety and tolerability of
intravenous administration of SBC-103 in patients with MPS
IIIB. The study will also evaluate the effects of dosing with
SBC-103 on the change from baseline levels of total heparan sulfate
(HS) in urine, serum, and cerebral spinal fluid (CSF), as well as
measure the effects on neurocognitive and developmental function
and change in brain structures as assessed by magnetic resonance
imaging.
About SBC-103
MPS IIIB is a rare, devastating and life threatening disease
which typically presents in children during the first few years of
life. Genetic mutations result in decreased activity of the
alpha-N-acetyl-glucosaminidase (NAGLU) enzyme, which leads to a
buildup of abnormal amounts of HS in the brain and throughout the
body. Over time, this unrelenting systemic accumulation of HS
causes progressive and severe cognitive decline, behavioral
problems, speech loss, loss of mobility, and premature
death.
MPS IIIB patients have significant unmet medical need, as
current treatments are palliative for the behavioral problems,
sleep disturbances, seizures, and other complications, and do not
address the root cause of MPS IIIB and stop disease
progression.
SBC-103 is the recombinant form of natural human NAGLU designed
to replace the missing (or deficient) NAGLU enzyme. SBC-103
has favorable properties for enabling cellular uptake and has shown
the ability to overcome the challenges previously encountered in
producing recombinant human NAGLU. The advancement of SBC-103
towards the clinic was supported by preclinical studies
demonstrating that intravenously administered SBC-103 was able to
cross the blood-brain barrier and reduce HS storage in the brain in
an MPS IIIB animal model. In addition, SBC-103 demonstrated
transport across an in vitro model of the blood-brain barrier and
distributed into the CSF in non-human primate studies.
SBC-103 was granted orphan drug designation by the U.S. Food and
Drug Administration (FDA) in April
2013 and the European Medicines Agency (EMA) in June 2013 and received Fast Track designation by
the FDA in January 2015.
About Synageva
Synageva is a biopharmaceutical company focused on the
discovery, development, and commercialization of therapeutic
products for patients with rare diseases. The company's pipeline
consists of protein therapeutic programs for rare diseases with
unmet medical need which are currently at various stages of
development. The company is planning for a global launch of
Kanuma™ (sebelipase alfa) for the treatment of LAL Deficiency and
is dosing patients in a Phase 1/2 trial with its second,
first-mover program, SBC-103 for MPS IIIB. The company's
third, first-mover program, SBC-105, is an enzyme replacement
therapy in preclinical development for disorders of
calcification. In addition to these first-mover programs, the
pipeline also consists of opportunities that leverage the company's
manufacturing platform and other capabilities to create potentially
bio-superior treatments for patient populations where there is
still unmet medical need. The company has recently produced
enzymes targeting Hunter syndrome, Fabry disease and Pompe disease
with expression levels and activity that support further
preclinical development.
Forward-Looking Statements
This news release contains "forward-looking statements".
Such statements generally can be identified by the use of words
such as "anticipate," "expect," "plan," "could," "intend,"
"believe," "may," "will," "estimate," "forecast," "project," or
words of similar meaning. These forward-looking statements address,
among other matters, potential approval and plans for the potential
launch of Kanuma, the ability for the company's manufacturing
platform to create potentially bio-superior treatments, and plans
to further support preclinical development for a potentially
bio-superior program. 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, such as, unanticipated
costs or delays in the company's research and development programs,
risk of delays in completing the company's preclinical and clinical
trials, risk that the outcomes of the preclinical or clinical
trials may not support registration or further development of the
company's product candidates due to safety, efficacy or other
reasons, the timing for initiating and completing potential
preclinical and clinical studies, the timing for reporting data
from potential preclinical and clinical studies, the content and
timing of decisions by the FDA, EMA and other regulatory
authorities, our ability to prepare for potential commercial launch
of Kanuma, the ability to continue to produce favorable product
attributes and characteristics expressed using the company's
proprietary platform, and the risks identified under the heading
"Risk Factors" in the company's quarterly report on Form 10-Q for
the fiscal quarter ending March 31,
2015 filed with the Securities and Exchange Commission (SEC)
and other filings Synageva periodically makes with the SEC, and
others of which are not known. Preclinical and clinical trial
data are subject to differing interpretations, and regulatory
agencies, as well as medical and scientific experts, may not share
Synageva's views regarding these data or its implications.
Synageva may encounter problems or delays in preclinical and
clinical development and the regulatory process. 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. Our business is subject to
substantial risks and uncertainties, including those referenced
above. Investors, potential investors, and others should give
careful consideration to these risks and uncertainties.
"Synageva BioPharma™" and "Kanuma™" are trademarks, and
"Dedicated to Rare Diseases®" is a registered trademark, of
Synageva BioPharma Corp.
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SOURCE Synageva BioPharma Corp.