Synlogic Presents New Preclinical Data at International Congress of Inborn Errors of Metabolism
September 07 2017 - 8:30AM
Business Wire
Data support the lead clinical program in urea
cycle disorders (UCD) and the further development of Synthetic
Biotic™ candidates as potential treatments for phenylketonuria
(PKU) and maple syrup urine disease (MSUD)
Synlogic (NASDAQ: SYBX) announced the presentation of new
preclinical data from programs investigating the company’s novel
Synthetic Biotic drug candidates at the 13th International Congress
of Inborn Errors of Metabolism (ICIEM), which is being held in Rio
de Janeiro, Brazil, September 5-8, 2017.
The data, generated in mouse and non-human primates, demonstrate
that Synlogic’s Synthetic Biotic medicines are active in the
gastrointestinal (GI) tract and are able to break down both dietary
and systemic sources of metabolites that build to toxic levels in
patients with UCD, PKU and MSUD.
“These data demonstrate that our Synthetic Biotic medicines can
act from the GI tract to potentially reduce the build-up of toxic
metabolites throughout the body in each of these disorders,” said
Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer.
“There is a huge need for improved treatment options for many
Inborn Errors of Metabolism (IEMs), and we believe that orally
administered living Synthetic Biotic medicines could provide a
promising option for patients.”
Presentation DetailsDevelopment of Genetically Engineered
E. coli Nissle Strains for the Treatment of Phenylketonuria and
Maple Syrup Urine Disease – Oral Presentation, Abstract number
248.
- PKU, caused by a defect in
phenylalanine hydroxylase (PAH) activity, is characterized by the
accumulation of systemic phenylalanine (Phe), which can lead to
severe neurological deficits unless patients are placed on a strict
low-Phe diet. SYN-PKU is an investigational Synthetic Biotic
medicine that can metabolize Phe into easily excreted compounds,
including hippurate, which is then excreted in the urine and serves
as a useful biomarker of SYN-PKU activity in mouse models and
non-human primates (NHP). In a mouse model of PKU challenged with
subcutaneous administration of Phe, oral dosing with SYN-PKU
resulted in significant reduction of Phe plasma levels compared to
controls. In studies in which NHPs were administered labeled Phe by
feeding, the data demonstrate that oral administration of SYN-PKU
enabled a significant decrease in blood Phe levels. Systemically
delivering labeled Phe to NHPs resulted in excretion of labeled
hippurate in the urine. These data suggest that systemic Phe
recirculates in the GI tract and is metabolized by SYN-PKU. These
data will be used to design the early phase clinical studies which
are planned to start in 2018.
- Other studies were performed using
SYN-MSUD, an investigational Synthetic Biotic candidate for the
treatment of MSUD, a rare genetic disorder associated with the
inability to break down certain branch chain amino acids (BCAAs).
In a mouse model of MSUD challenged with a high-protein diet, oral
delivery of SYN-MSUD resulted in significantly reduced plasma
levels of the BCAA leucine. In addition, a reduction in physical
symptoms of disease and reduced levels of leucine in the brains of
these animals was observed with SYN-MSUD treatment as compared with
controls.
A Genetically Engineered E. coli Nissle to Prevent
Hyperammonemia in Urea Cycle Disorder (UCD) – Poster
Presentation, Abstract number 377.
- Urea cycle disorders (UCDs) are a group
of inherited diseases in which the inability to efficiently convert
waste nitrogen into urea leads to the toxic accumulation of
systemic ammonia. An in vitro study demonstrated that SYNB1010 and
SYNB1020, investigational Synthetic Biotic candidates designed for
the treatment of UCD, were able to consume ammonia and produce
a beneficial metabolite, arginine.
- The systemic effects of SYNB1010 were
also studied in a UCD mouse model. The data demonstrated that
orally administered SYNB1010 functioned in the GI tract and
prevented systemic hyperammonemia caused by a high-protein diet,
significantly increasing survival of these animals.
About SYNB1020 and SYNB1618Earlier this year, Synlogic
initiated a Phase 1 clinical trial in healthy volunteers designed
to evaluate the safety and tolerability of its lead compound,
SYNB1020, as a potential treatment for UCD. Pending the success of
this first study, Synlogic plans to initiate two additional
clinical trials with the investigational candidate in symptomatic
patients with urea cycle disorders (UCD) or hepatic encephalopathy
(HE), both conditions in which patients experience elevated and
toxic blood ammonia levels. In the first half of 2018, the company
also plans to initiate a clinical trial with SYNB1618, a synthetic
biotic medicine designed to treat phenylketonuria (PKU), which is
caused by defective metabolism of the amino acid phenylalanine.
About Synthetic Biotic™ MedicinesSynlogic’s innovative
new class of Synthetic Biotic™ medicines leverages the tools and
principles of synthetic biology to genetically engineer probiotic
microbes to perform or deliver critical functions missing or
damaged due to disease. The company’s two lead programs target a
group of rare metabolic diseases – inborn errors of metabolism
(IEM). Patients with these diseases are born with a faulty gene,
inhibiting the body’s ability to break down commonly occurring
by-products of digestion that then accumulate to toxic levels and
cause serious health consequences. When delivered orally, these
medicines can act from the gut to compensate for the dysfunctional
metabolic pathway and have a systemic effect. Synthetic Biotic
medicines are designed to clear toxic metabolites associated with
specific metabolic diseases and have the potential to significantly
improve symptoms of disease for affected patients.
About Synlogic™Synlogic™ is pioneering the development of
a novel class of living Synthetic Biotic™ medicines based on its
proprietary drug discovery and development platform. Synlogic’s
initial pipeline includes Synthetic Biotic medicines for the
treatment of rare genetic diseases, such as Urea Cycle Disorder
(UCD) and Phenylketonuria (PKU). In addition, the company is
leveraging the broad potential of its platform to create Synthetic
Biotic medicines for the treatment of other diseases, such as liver
disease, inflammatory and immune disorders, and cancer. Synlogic is
collaborating with AbbVie to develop Synthetic Biotic-based
treatments for inflammatory bowel disease (IBD). For more
information, please visit synlogictx.com.
Forward-Looking StatementsThis press release contains
“forward-looking statements” that involve substantial risks and
uncertainties for purposes of the safe harbor provided by the
Private Securities Litigation Reform Act of 1995. All statements,
other than statements of historical facts, included in this press
release regarding strategy, future operations, future financial
position, future revenue, projected expenses, prospects, plans and
objectives of management are forward-looking statements. In
addition, when or if used in this press release, the words “may,”
“could,” “should,” “anticipate,” “believe,” “estimate,” “expect,”
“intend,” “plan,” “predict” and similar expressions and their
variants, as they relate to Synlogic may identify forward-looking
statements. Examples of forward-looking statements, include, but
are not limited to, statements regarding the potential of
Synlogic’s platform to develop therapeutics to address a wide range
of diseases including UCD, PKU and MSUD, the future clinical
development of SYNB1020 and its prospects as a potential treatment
for hyperammonemia, the future development of other product
candidates, such as SYN-PKU and SYN-MSUD and the approach Synlogic
is taking to discover and develop novel therapeutics using
synthetic biology. Actual results could differ materially from
those contained in any forward-looking statement as a result of
various factors, including, the uncertainties inherent in the
preclinical development process, the ability of Synlogic to protect
its intellectual property rights and legislative, regulatory,
political and economic developments.
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SynlogicMEDIA CONTACT:Courtney Heath,
617-872-2462courtney@scientpr.comorINVESTOR
CONTACT:Elizabeth Wolffe, Ph.D.,
617-207-5509liz@synlogictx.com
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