Sage Therapeutics Announces European Medicines Agency Granted SAGE-718 Orphan Drug Designation for the Treatment of Huntington’s Disease
February 22 2023 - 6:30AM
Business Wire
Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical
company leading the way to create a world with better brain health,
today announced the European Medicines Agency (EMA) granted Orphan
Drug Designation to SAGE-718 for the treatment of Huntington’s
disease (HD). SAGE-718 is in development as a potential oral
therapy for cognitive disorders associated with NMDA receptor
dysfunction. Multiple clinical studies are ongoing with SAGE-718
across several disease areas, including two placebo-controlled
Phase 2 studies and a Phase 3 open-label safety study in the lead
indication of HD-related cognitive impairment, and additionally
Phase 2 placebo-controlled studies in mild cognitive impairment
(MCI) associated with Parkinson’s disease (PD) and MCI and mild
dementia due to Alzheimer’s disease (AD).
“Huntington’s disease is a serious, debilitating condition that
interferes with daily functioning in the prime years of life and is
associated with significant morbidity and early mortality, and yet
there are currently no approved therapies for the treatment of
HD-related cognitive impairment,” said Laura Gault, M.D., Ph.D.,
Chief Medical Officer, Sage Therapeutics. “With this exciting
development of Orphan Drug Designation from the EMA, we have
further momentum in our efforts to address this unmet need. Our
goal with SAGE-718 is to provide rapid, meaningful, and sustained
improvement in cognitive functioning early in the disease so that
patients can maintain independence longer.”
Orphan drug designation is reserved for medicines treating rare,
life-threatening, or chronically debilitating diseases that meet
certain specified criteria. Potential benefits for Sage as a result
of this designation include Protocol Assistance or scientific
advice specifically designed for orphan medicines, European
centralized authorization procedure, if development is successful
and a marketing authorization application is filed, and if
approved, ten years of market exclusivity and protection from
similar medicines with the same indication being approved.
About SAGE-718
SAGE-718, Sage’s first-in-class NMDA receptor positive
allosteric modulator (PAM) and lead neuropsychiatric drug
candidate, is in development as a potential oral therapy for
cognitive disorders associated with NMDA receptor dysfunction,
potentially including HD, PD and AD. Sage is advancing a robust
clinical program for SAGE-718 with multiple ongoing
placebo-controlled Phase 2 studies across multiple disease areas,
including its potential lead indication, cognitive impairment
associated with HD, as well as cognitive impairment due to AD and
PD. The Company is also conducting a Phase 3 safety extension study
in HD cognitive impairment. SAGE-718 has received Fast Track
Designation from the FDA for the potential treatment of HD.
About Sage Therapeutics
Sage Therapeutics is a biopharmaceutical company fearlessly
leading the way to create a world with better brain health. Our
mission is to pioneer solutions to deliver life-changing brain
health medicines, so every person can thrive. For more information,
please visit http://www.sagerx.com.
Forward-Looking Statements
Various statements in this release concern Sage's future
expectations, plans and prospects, including without limitation our
statements regarding: the goals, potential benefit, and potential
indications for SAGE-718; the potential benefit of ODD granted to
SAGE-718 by the EMA; the potential for success of this programs,
and the opportunity to help patients; and the mission and goals for
our business. These statements constitute forward-looking
statements as that term is defined in the Private Securities
Litigation Reform Act of 1995. These forward-looking statements are
neither promises nor guarantees of future performance, and are
subject to a variety of risks and uncertainties, many of which are
beyond our control, which could cause actual results to differ
materially from those contemplated in these forward-looking
statements, including the risks that: we may not seek or achieve
some or any of the benefits associated with orphan drug designation
of SAGE-718 by the EMA; we may not be successful in the development
of SAGE-718 or of any of our other product candidates; success in
earlier clinical trials may not be repeated or observed in ongoing
or future studies, and ongoing and future clinical trials may not
meet their primary or key secondary endpoints which may
substantially impair development; unexpected concerns may arise
from additional data, analysis or results from any of our completed
studies; we may encounter adverse events at any stage that
negatively impact further development or the potential for approval
or the potential for successful commercialization, if approved, or
that require additional nonclinical and clinical work which may not
yield positive results; we may encounter delays in initiation,
conduct, completion of enrollment or completion of our ongoing and
planned clinical trials, including as a result of slower than
expected site initiation, slower than expected enrollment, the need
or decision to expand the trials or other changes, that may impact
our ability to meet our expected timelines and increase our costs;
decisions or actions of the FDA or other regulatory agencies may
affect the initiation, timing, design, size, progress and cost of
clinical trials and our ability to proceed with further development
or may impair the potential for successful development; and we may
encounter technical and other unexpected hurdles in the development
and manufacture of SAGE-718 or our other product candidates or the
commercialization of any current or future marketed product which
may delay our timing or change our plans, increase our costs or
otherwise negatively impact our business; as well as those risks
more fully discussed in the section entitled "Risk Factors" in our
most recent quarterly report, as well as discussions of potential
risks, uncertainties, and other important factors in our subsequent
filings with the Securities and Exchange Commission. In addition,
any forward-looking statements represent our views only as of
today, and should not be relied upon as representing our views as
of any subsequent date. We explicitly disclaim any obligation to
update any forward-looking statements.
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Investor Contact Helen Rubinstein 315-382-3979
helen.rubinstein@sagerx.com
Media Contact Matthew Henson 917-930-7147
matthew.henson@sagerx.com
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