LEXINGTON, Mass., Jan. 26, 2015 /PRNewswire/ -- Synageva
BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company
developing therapeutic products for rare disorders, announced today
dosing with SBC-103 in patients with mucopolysaccharidosis IIIB
(MPS IIIB, also known as Sanfilippo B syndrome) has begun as part
of a Phase 1/2 study. In addition, the U.S. Food and Drug
Administration (FDA) recently granted SBC-103 Fast Track
designation. Fast Track is a process designed to facilitate
development and expedite review of drugs to treat serious and
life-threatening conditions and fill an unmet medical need to get
important new drugs to patients expeditiously.
MPS IIIB is a rare disease caused by a decrease in
alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads
to the buildup of abnormal amounts of heparan sulfate (HS) in the
brain and other organs. The accumulation of abnormal HS in
multiple cells and tissues leads to severe cognitive decline,
behavioral problems, speech loss, increasing loss of mobility, and
premature death. SBC-103 is a recombinant form of natural
human NAGLU designed to replace the missing (or deficient) NAGLU
enzyme.
"This is a very exciting time for individuals with MPS IIIB and
their families and we are delighted to help support the advancement
of this important program," said Christine
Lavery, Chief Executive of the Society for
Mucopolysaccharide Diseases (the MPS Society U.K.). "MPS IIIB
is a progressive, life-limiting disease and it is encouraging to
see this program progress from preclinical to clinical
studies."
Chester B. Whitley, Ph.D., M.D.,
Professor, Advanced Therapies Program, Department of Pediatrics and
Experimental and Clinical Pharmacology at the University of Minnesota, Minneapolis, MN, and
investigator in the trial said, "Administering the very first dose
of this recombinant enzyme is an important milestone for patients
afflicted with this otherwise untreatable, lethal disease.
This approach challenges the century-long dogma that the
blood-brain barrier is impenetrable. The potential impact for
these children cannot be overestimated. Sanfilippo syndrome
was discovered at the University of
Minnesota, and our team is uniquely prepared and excited to
help assess treatment for these patients."
"The start of dosing in patients with Synageva's second,
first-mover program and the first clinical trial of an enzyme
replacement therapy for MPS IIIB is an important step forward for
this community and the company," said Sanj
K. Patel, President and Chief Executive Officer of
Synageva. "The advancement of this program to clinical trials
was made possible through the dedication and commitment from
patients and their families affected by MPS IIIB and the diligent
efforts by our research and development team. Lysosomal
disease enzyme replacement therapies with the recombinant form of
the natural human enzymes are the gold standard of treatment for
patients with these devastating diseases, and the results from this
clinical trial will help further our understanding of the role of
SBC-103 in MPS IIIB."
About the Phase 1/2 study
The trial will enroll approximately nine patients, two years of
age or greater but less than 12 years of age, with definitive
diagnosis of MPS IIIB and developmental delay. Patients will
be 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 therapy with SBC-103 for an
extended treatment period that will last up to 128 weeks.
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 determine the effects of dosing
with SBC-103 on the onset, magnitude, and reversibility of changes
in levels of total HS in cerebral spinal fluid (CSF), serum, and
urine as well as measure the effects of neurocognitive and
developmental function and change in brain structures as assessed
by magnetic resonance imaging. Exploratory biomarkers,
SBC-103 concentration in CSF, MPS IIIB disease characteristics,
symptoms, and quality of life will also be measured. The
company plans to report preliminary data from this study during the
second half of this year.
About SBC-103
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.
The company is also conducting natural history studies in MPS
IIIB. These include a retrospective natural history study of
deceased MPS IIIB patients that began in July 2013 and a prospective, longitudinal natural
history study in living MPS IIIB patients that began in September
2014. Natural history studies can help build an understanding
of the manifestations and progression of disease and provide
insights into biomarkers and other measures that may be useful as
clinical outcomes.
SBC-103 was granted orphan designation by the FDA in
April 2013 and the European Medicines
Agency (EMA) in June 2013 and
received Fast Track designation by the FDA in January 2015.
Synageva routinely posts information that may be important to
investors in the "Investor Relations" section of the company's
website at www.synageva.com. Synageva encourages investors
and potential investors to consult this website regularly for
important information about the company.
Further information regarding Synageva is available at
www.synageva.com.
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, the potential impact of
Fast Track designation, the number of patients we intend to enroll
and the length of treatment, the timing to report preliminary data
from the Phase 1/2 study, and the potential impact of natural
history studies. 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, the risk of delays
in initiating or completing our clinical trials, unanticipated
costs or delays in our research and development programs,
unanticipated delays in our submission timelines, risk that the
outcomes of our clinical trials may not support registration or
further development of our product candidates due to safety,
efficacy or other reasons, the content and timing of decisions by
the FDA and other regulatory authorities, and the risks identified
under the heading "Risk Factors" in the Company's prospectus
supplement filed with the Securities and Exchange Commission (SEC)
on January 7, 2015 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" is a trademark, and "Dedicated to Rare
Diseases" is a registered trademark, of Synageva.
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