Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology
company advancing novel, oral, non-systemically absorbed
biotherapeutics to transform the care of serious diseases, today
announced the initiation of Synpheny-3, a global, pivotal Phase 3
study evaluating the efficacy and safety of SYNB1934 as a potential
treatment for phenylketonuria (PKU).
“We have worked tirelessly to advance this program and today is
an important milestone for Synlogic and for the patient community
living with the extreme burden of PKU,” said Aoife Brennan,
M.B. Ch.B., Synlogic President and Chief Executive Officer.
“We have partnered on this pivotal trial design with key
stakeholders including clinician experts, global regulatory
agencies and people living with PKU and are very grateful for their
many insights. In particular, feedback from patients has heightened
our sense of urgency to bring forward a new oral therapeutic that
can be used either a monotherapy or adjunctive medical
treatment.”
“This is exciting news. The PKU
community is grateful to Synlogic for their commitment to bringing
a new treatment option forward for individuals with PKU, and for
seeking the input and partnership of our community in this
process,” said Lisa Milberg, Executive Director, National PKU
Alliance.
The Synpheny-3 Trial
Synpheny-3 is a randomized, placebo-controlled, global,
multi-center, pivotal Phase 3 clinical trial designed to evaluate
the efficacy and safety of SYNB1934 as a treatment for PKU. The
final trial design incorporates feedback from global regulatory
agencies including the U.S. Food and Drug Administration (FDA). The
global study will enroll approximately 150 patients with plasma
phenylalanine (Phe) levels at baseline of >360 μM. Synpheny-3
will include patients ages 18 years and older; an initial subset of
data from patients in Part 1 will be used to assess the opportunity
to lower the age of enrollment to 12 years of age. Study
participants may follow their usual diet while participating in the
trial.
Synpheny-3 is expected to be conducted at approximately 30
clinical sites across the United States, Canada, Germany, Denmark,
Israel, Turkey and Georgia. The study has been designed for patient
convenience, and offers opportunities to participate in person, or
in a virtual or hybrid format.
The study consists of three parts: Part 1 is a run-in period,
enabling individualized titration across three potential dose
levels (3x1011, 6x1011 and 1x1012), with patients spending a
minimum of three weeks at each dose. Part 2 of the trial is a
four-week, placebo-controlled, randomized withdrawal that will be
used to assess the primary endpoint: change in levels of plasma
Phe, with a primary analysis conducted among responders from Part
1. The definition for responders is a reduction in plasma Phe from
baseline of >20%. Part 3 is an open-label extension that may
extend for up to three years.
More information on the Synpheny-3 study is available at
www.clinicaltrials.gov, identifier NCT05764239 and also by visiting
pkuresearchstudy.com.
Business Update
In recognition of the important milestone of Synpheny-3
initiation and the progress of Synlogic’s clinical-stage programs,
the Company also announced efforts to direct resources towards its
late-stage portfolio of product candidates for rare metabolic
diseases. Changes include prioritizing activities including the
pivotal and pediatric studies of SYNB1934 for PKU, and advancing to
Phase 2 for SYNB1353 for HCU, as well as research activities that
support the current clinical pipeline and the ongoing collaboration
with Roche.
With that prioritization, the Company has reduced its workforce
by approximately 21%, which is expected to extend its cash runway
further into the second half of 2024. The Company estimates that it
will incur approximately $0.9 million of costs in connection with
the reduction in workforce related to severance pay and other
related termination benefits. The Company communicated the
workforce reduction on May 31, 2023 and expects the majority of the
costs associated with the reduction in force plan to be incurred
during the second quarter ending June 30, 2023 and the third
quarter ending September 30, 2023. The Company may also incur other
material charges not currently contemplated due to events that may
occur because of, or associated with, these actions.
About SYNB1934
SYNB1934 is an orally administered,
non-systemically absorbed, potential treatment for phenylketonuria
(PKU), a rare metabolic disease caused by inherited mutations that
impair the breakdown of phenylalanine (Phe), an amino acid found in
all protein-containing foods. The goal of PKU management is to
reduce plasma Phe below neurotoxic levels, reducing risk of
neurocognitive complications. Current treatment options for PKU are
limited due to safety and efficacy, leaving the majority of people
living with PKU without medical management and with uncontrolled
Phe. Synlogic designed SYNB1934 to target and consume Phe in
the GI tract, by applying precision genetic engineering to a
well-characterized probiotic. Results to date indicate the
potential for SYNB1934 to be the first therapeutic for PKU approved
as both a monotherapy and adjunctive medical treatment, and
following successful Phase 2 results, it has advanced to
Synpheny-3, a global, pivotal Phase 3 study. SYNB1934 has received
Orphan Drug Designation (ODD) and Rare Pediatric Disease
Designation (RPDD) from the FDA in addition to orphan
designation from the European Medicines Agency (EMA).
About Synlogic
Synlogic is a clinical-stage biotechnology company advancing
novel, oral, non-systemically absorbed biotherapeutics to transform
the care of serious diseases in need of new treatment options. The
Company’s late-stage pipeline is focused on rare metabolic
diseases, led by SYNB1934, currently being studied as a potential
treatment for phenylketonuria (PKU) in Synpheny-3, a global,
pivotal Phase 3 study. Additional product candidates address
diseases including homocystinuria (HCU), enteric hyperoxaluria,
gout, and cystinuria. This pipeline is fueled by the Synthetic
Biotic platform, which applies precision genetic engineering to
well-characterized probiotics. This enables Synlogic to create
GI-restricted, oral medicines designed to consume or modify
disease-specific metabolites – an approach well suited for PKU and
HCU, both inborn errors of metabolism, as well as other disorders
in which the disease–specific metabolites transit through the GI
tract, providing validated targets for these Synthetic Biotics.
Research activities include a partnership with Roche focused on
inflammatory bowel disease (IBD), and a collaboration with Ginkgo
Bioworks in synthetic biology, which has contributed to two
pipeline programs to date. For more information, please visit
www.synlogictx.com or follow us on Twitter or LinkedIn.
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, clinical development
plans, 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," “look forward,” "estimate," "expect," "intend," “on
track,” "plan," "predict," “prepare” 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 approach to Synthetic Biotics to develop therapeutics
to address a wide range of diseases including: inborn errors of
metabolism and inflammatory and immune disorders; our expectations
about sufficiency of our existing cash balance; the future clinical
development of Synthetic Biotics; the approach Synlogic is taking
to discover and develop novel therapeutics using synthetic biology;
and the expected timing of Synlogic's clinical trials of SYNB1934,
SYNB1353, SYNB8802 and SYNB2081 and availability of clinical trial
data. Actual results could differ materially from those contained
in any forward-looking statements as a result of various factors,
including: the uncertainties inherent in the clinical and
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 U.S. Securities and Exchange Commission. 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.
Media Contact: media@synlogictx.com
Investor Relations: investor@synlogictx.com
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