CAMBRIDGE, Mass.--(BUSINESS WIRE)--Synlogic (Nasdaq: SYBX), a clinical stage company applying synthetic biology to probiotics to develop novel living medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SYNB1618, Synlogic’s preclinical-stage drug candidate for the treatment of phenylketonuria (PKU) , an inborn error of metabolism (IEM) caused by a mutation in the gene that breaks down the amino acid phenylalanine (Phe). Phe accumulation in the blood and brain can lead to neurocognitive abnormalities and treatment currently requires severe dietary protein restriction.
SYNB1618, an orally administered medicine, is designed to complement the missing function in patients with PKU by providing alternative metabolic pathways to consume Phe. Synlogic plans to file an investigational new drug application (IND) with the FDA for SYNB1618 for the potential treatment of PKU in early 2018.
“We believe our Synthetic Biotic medicines could transform the treatment of PKU,” said Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer. “Consequently, we were pleased to receive the FDA’s orphan drug designation which validates our approach and represents an important step toward achieving our goal of bringing novel treatments to the patients and families affected by this challenging disease.”
The FDA’s Orphan Drug Program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
About PKU
Phe is an essential amino acid that enters the
body as a component of dietary protein and can be toxic if its levels
accumulate in the blood and brain. PKU is caused by a defect in the gene
encoding phenylalanine hydroxylase (PAH), a liver enzyme that
metabolizes Phe. Current disease management of PKU involves strict
dietary protein restriction with the consumption of Phe-free protein
supplements. The only currently approved medication, Kuvan®,
is indicated for a subgroup of patients and does not eliminate the need
for ongoing dietary management. Life-long Phe control is challenging due
to the highly restrictive nature of the diet and patients typically
experience worsening neurological function depending on the severity of
their genetic mutation and their treatment compliance. PKU is diagnosed
at birth, and the National PKU Alliance estimates that there are
currently 16,500 people living with the disorder in the U.S.
About Synthetic Biotic Medicines
Synlogic’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 medicines, Synthetic Biotic medicines, based on
its proprietary drug development platform. Synlogic’s initial pipeline
includes Synthetic Biotic medicines for the treatment of rare genetic
diseases, such as urea cycle disorders (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 more
common diseases, including 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 www.synlogictx.com.
Forward-Looking Statements
This 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: inborn errors of metabolism, liver disease,
inflammatory and immune disorders, and cancer; the future clinical
development of Synthetic Biotic medicines; the approach Synlogic is
taking to discover and develop novel therapeutics using synthetic
biology; the potential of Synlogic’s technology to treat
phenylketonuria; the expected timing of Synlogic’s anticipated IND
application and clinical trial; and the benefit of orphan drug status.
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, as well as
those risks identified under the heading “Risk Factors” in Synlogic’s
filings with the SEC. The forward-looking statements contained in this
press release reflect Synlogic’s current views with respect to future
events. Synlogic anticipates that subsequent events and developments
will cause its views to change. However, while Synlogic may elect to
update these forward-looking statements in the future, Synlogic
specifically disclaims any obligation to do so. These forward-looking
statements should not be relied upon as representing Synlogic’s view as
of any date subsequent to the date hereof.
