Synlogic Granted Fast Track Designation from FDA for labafenogene marselecobac (SYNB1934) for Treatment of Phenylketonuria
11 Julho 2023 - 8:00AM
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 that the U.S. Food and Drug Administration (FDA) has
granted Fast Track designation to labafenogene marselecobac
(previously known as SYNB1934) for the treatment of phenylketonuria
(PKU). Labafenogene marselecobac has also received Rare Disease
Designation (RPDD) and Orphan Drug Designation (ODD) by the FDA and
orphan designation from the European Medicines Agency (EMA).
“We are pleased that this potentially transformative therapy has
now received three important regulatory designations from the FDA,
and orphan designation from the EMA, reflecting a shared
understanding of the urgent need for new medical treatment options
that can effectively and safely lower Phe levels in patients with
PKU,” said Aoife Brennan, M.B. Ch.B., Synlogic President
and Chief Executive Officer. “This milestone re-enforces our own
urgency as we execute our pivotal study, Synpheny-3.”
The FDA’s Fast Track process is designed to facilitate the
development and expedite the review of drugs to treat serious
conditions and fill an unmet medical need. To qualify, available
clinical and non-clinical data need to demonstrate meaningful
therapeutic potential. The benefits of Fast Track designation
include opportunities for frequent meetings with the FDA to discuss
trial design, development plans and data needed to support drug
approval, as well as the ability to submit a registrational filing
for approval on a rolling basis, and eligibility for priority
review, if relevant criteria are met.
About labafenogene marselecobac
Labafenogene marselecobac (previously known as 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
labafenogene marselecobac 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 labafenogene marselecobac as 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. Labafenogene marselecobac has received Orphan Drug
Designation (ODD), Fast Track designation 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 labafenogene marselecobac (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" 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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