CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sage Therapeutics (NASDAQ: SAGE), a clinical-stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders, today announced the Company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its lead product candidate, an intravenous formulation of brexanolone (SAGE-547), for the treatment of postpartum depression (PPD).
Brexanolone IV received Breakthrough Therapy Designation in September 2016, underscoring the significant unmet need in women with PPD. Breakthrough Therapy Designation is intended to offer a potentially expedited development path and review for promising drug candidates intended to treat serious conditions, including increased interaction and guidance from the FDA.
The NDA submission is supported by data from the Hummingbird Program. This clinical program included three multicenter, randomized, double-blind, parallel-group, placebo-controlled trials (Study 202A, Study 202B and Study 202C), each designed to evaluate the safety and effectiveness of brexanolone in women with moderate or severe PPD, aged between 18 and 45 years who were ≤6 months postpartum at screening in the United States.
About FDA Breakthrough Therapy Designation
The FDA's
Breakthrough Therapy Designation is intended to expedite the development
and review of a drug candidate that is planned for use, alone or in
combination with one or more other drugs, to treat a serious or
life-threatening disease or condition when preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement over
existing therapies on one or more clinically significant endpoints. The
benefits of Breakthrough Therapy Designation include the same benefits
as Fast Track Designation, plus an organizational commitment involving
the FDA's senior managers with more intensive guidance from the FDA.
Breakthrough Therapy Designation does not change the standards for
approval.
About Postpartum Depression
Postpartum depression (PPD) is a
distinct and readily identified major depressive disorder that is the
most common medical complication of childbirth, affecting a subset of
women typically commencing in the third trimester of pregnancy or within
four weeks after giving birth. PPD may have devastating consequences for
a woman and for her family, which may include significant functional
impairment, depressed mood and/or loss of interest in her newborn, and
associated symptoms of depression such as loss of appetite, difficulty
sleeping, motor challenges, lack of concentration, loss of energy and
poor self-esteem. Suicide is the leading cause of maternal death
following childbirth. In the U.S., estimates of new mothers identified
with PPD each year vary by state from 8 to 20 percent, with an overall
average of 11.5 percent. More than half of these cases may go
undiagnosed without proper screening. There are no FDA approved
therapies for PPD and there is a high unmet medical need for improved
pharmacological therapy in PPD.
About Brexanolone (SAGE-547)
Brexanolone (SAGE-547) is an
allosteric modulator of both synaptic and extrasynaptic GABAA receptors.
Allosteric modulation of neurotransmitter receptor activity results in
varying degrees of desired activity rather than complete activation or
inhibition of the receptor. Sage’s proprietary intravenous (IV)
formulation of brexanolone is being developed for the treatment of
postpartum depression (PPD) and has been granted Breakthrough Therapy
Designation by the FDA and PRIority MEdicines (PRIME) designation from
the European Medicines Agency (EMA) in PPD.
About Sage Therapeutics
Sage Therapeutics is a
clinical-stage biopharmaceutical company committed to developing novel
medicines to transform the lives of patients with life-altering central
nervous system (CNS) disorders. Sage has a portfolio of novel product
candidates targeting critical CNS receptor systems, GABA and NMDA.
Sage's lead program, a proprietary IV formulation of brexanolone
(SAGE-547), has completed Phase 3 clinical development for postpartum
depression. Sage is developing its next generation modulators, including
SAGE-217 and SAGE-718, in various CNS disorders. For more information,
please visit 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 or implying: the
potential for approval of brexanolone IV in the treatment of PPD; the
potential for expedited development and review of brexanolone IV in PPD
as a result of the Breakthrough Therapy Designation from the FDA; our
expectations regarding the possible acceptance of our NDA filing; our
estimates of the prevalence of PPD; and our view as to the potential for
brexanolone IV and the other product candidates in our portfolio. 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: the FDA may decide not to accept
for filing our NDA for brexanolone IV in PPD; the clinical and
non-clinical data we have generated with our proprietary formulation of
brexanolone to date may be determined by the FDA, despite prior advice,
to be insufficient to file for or gain regulatory approval to launch and
commercialize our product in PPD and the FDA may determine that
additional trials or data are necessary in order to file for or obtain
approval; the FDA may find fault with the data generated at particular
clinical site or sites or with the activities of our trial monitor or
may disagree with our analyses of the results of our trials or identify
issues with our manufacturing or quality systems, and any such findings
or issues could require additional data or analyses or changes to our
systems that could delay or prevent us from gaining approval of
brexanolone IV; we may not achieve expedited development or review of
brexanolone IV as a result of the Breakthrough Therapy Designation from
the FDA; the actual size of the PPD patient population may be
significantly lower than our estimates and, even if brexanolone IV is
successfully approved for PPD, it may only be approved or used to treat
a subset of the PPD population; we may encounter unexpected safety or
tolerability issues with brexanolone IV in ongoing clinical trials; we
may not be able to successfully demonstrate the efficacy and safety of
any of our other product candidates at each stage of development;
success in early stage clinical trials may not be repeated or observed
in ongoing or future studies of our product candidates; ongoing
and future clinical results may not support further development or be
sufficient to gain regulatory approval to market our product candidates;
and we may encounter technical and other unexpected hurdles in the
development and manufacture of our product candidates; as well as those
risks more fully discussed in the section entitled "Risk Factors" in our
most recent Annual Report on Form 10-K, and 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.