Sage Therapeutics Announces U.S. Food and Drug Administration Granted SAGE-718 Orphan Drug Designation for the Treatment of Huntington’s Disease
October 18 2023 - 5:30AM
Business Wire
Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical
company leading the way to create a world with better brain health,
today announced the U.S. Food and Drug Administration (FDA) granted
Orphan Drug Designation (ODD) 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 potential lead indication of HD-related cognitive
impairment, as well as 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 devastating condition that often
affects patients in their prime years, and it can significantly
impact a patient’s ability to live independently. Cognitive
impairment is one of the most underrecognized aspects of this
disease,” said Laura Gault, M.D., Ph.D., Chief Medical Officer,
Sage Therapeutics. “There are currently no approved treatments to
address cognitive impairment for people with HD and a growing sense
of urgency among researchers and people living with HD to address
cognitive impairment early so that patients can maintain
independence longer. The ODD designation from the FDA provides
continued momentum in our efforts to help patients and their
families impacted by this aspect of HD.”
Orphan drug designation is granted by the FDA Office of Orphan
Products Development to assist and encourage companies to develop
safe and effective therapies for the treatment of rare diseases and
disorders. Under the Orphan Drug Act, the FDA may provide grant
funding towards clinical trial costs, tax advantages, FDA user-fee
benefits, and the potential for seven years of market exclusivity
in the United States for the drug in the orphan indication
following drug approval by the FDA. The approval of an orphan
designation request does not alter the standard regulatory
requirements and process for obtaining marketing approval. For more
information about orphan designation, please visit the FDA website
at www.fda.gov.
SAGE-718 previously received Fast Track Designation from the FDA
for HD, and orphan drug designation for HD by the European
Medicines Agency.
About SAGE-718 SAGE-718, a first-in-class investigational
NMDA receptor positive allosteric modulator (PAM), is in
development as a potential oral therapy for cognitive disorders
associated with NMDA receptor dysfunction, including HD, PD and AD.
Sage is advancing a 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 open-label
safety study in HD cognitive impairment.
About Huntington’s disease and cognition HD is a rare,
inherited neurodegenerative disease that progresses over time and
affects up to an estimated 40,000 adults in the U.S. each year.
Cognitive impairment can severely affect people with HD. There are
currently no treatment options available to improve the cognitive
effects of the disease.
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 FDA; the
estimated number of people with HD in the U.S.; 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 FDA; 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 or cause us to
terminate further work; 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, lengthen the regulatory
pathway for approval or negatively impact our ability to proceed
with further development or may in other ways impair the potential
for successful development; even if we are successful in our
development efforts and receive FDA approval of SAGE-718 in HD with
orphan exclusivity that prevents others from gaining approval of
the same drug in this indication during the exclusivity period,
competitors may receive approval of different products for HD; our
estimates of the number of people with HD and the unmet need for
new options to improve treatment may prove not to be correct; 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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version on businesswire.com: https://www.businesswire.com/news/home/20231018464957/en/
Investor Ashley Kaplowitz 786-252-1419
ashley.kaplowitz@sagerx.com
Media Matthew Henson 917-930-7147
Matthew.Henson@sagerx.com
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