Priority Review status expected to accelerate review period; PDUFA
date set for December 19, 2018
If approved, brexanolone IV would be the first and only medication
indicated for the treatment of postpartum depression
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 30, 2018--
Sage Therapeutics (NASDAQ: SAGE), a clinical-stage biopharmaceutical
company developing novel medicines to treat life-altering central
nervous system (CNS) disorders, today announced that the U.S. Food and
Drug Administration (FDA) has accepted the filing of a New Drug
Application (NDA) for Sage’s lead product candidate, an intravenous
formulation of brexanolone (SAGE-547) for the treatment of postpartum
depression (PPD). The NDA was granted Priority Review status and the FDA
assigned a Prescription Drug User Fee Act (PDUFA) target date of
December 19, 2018. As expected for a new molecular entity with a new
mechanism of action, the FDA is currently planning to hold an advisory
committee meeting to discuss the brexanolone IV application.
If approved, brexanolone IV would be the first medication indicated for
the treatment of PPD and would be Sage Therapeutics’ first commercial
product. The FDA grants Priority Review to investigational therapies
that, if approved, may offer significant improvements in the treatment,
prevention or diagnosis of a serious condition. Brexanolone IV received
Breakthrough Therapy Designation in September
2016, underscoring the significant unmet need in women with PPD.
PPD is the most common medical complication of childbirth affecting a
subset of women typically commencing in the third trimester of pregnancy
or in the months after giving birth. In the U.S., estimates of new
mothers identified with PPD each year vary by state from eight to 20
percent, with an overall average of 11.5 percent. There are no approved
therapies for PPD, and there is a clear unmet medical need for treatment.
The NDA is supported by data from the Hummingbird
Program, two Phase 3 multicenter, randomized, double-blind,
parallel-group, placebo-controlled trials in the U.S. (Study 202B and
Study 202C), designed to evaluate the safety and effectiveness of
brexanolone in women with moderate and severe PPD, aged between 18 and
45 years (inclusive) who were ≤6 months postpartum at screening and who
had onset of symptoms no earlier than the third trimester and no later
than the first four weeks following delivery. In both trials at all
doses, brexanolone achieved the primary endpoint, a significant mean
reduction from baseline in the Hamilton Rating Scale for Depression
(HAM-D) total score at 60 hours compared to placebo. Brexanolone was
generally well-tolerated in both studies with similar rates of adverse
events (AEs) across all treatment groups. The most common AEs in the
studies were headache, dizziness and somnolence.
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 the months 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 CNS disorders. Sage has a portfolio of novel
product candidates targeting critical CNS receptor systems, GABAA
and NMDA. Sage's lead program, a proprietary IV formulation of
brexanolone (SAGE-547), has completed Phase 3 clinical development for
postpartum depression and a New Drug Application is currently under
review with the U.S. Food and Drug Administration. 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: the potential timing of FDA review of our NDA
seeking approval of brexanolone IV in the treatment of PPD; the
potential for expedited review of the NDA as a result of Priority Review
status and Breakthrough Therapy Designation from the FDA; our
expectations regarding the possible approval of our NDA filing, and the
potential forbrexanolone IV to be the first medication indicated
for PPD; our estimates of the prevalence of PPD; and other statements
regarding our technology, business and 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 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 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 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 review of
brexanolone IV as a result of Priority Review status or Breakthrough
Therapy Designation from the FDA, and the FDA may not complete its
review of our filing within the target timelines; 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 of any of our product
candidates 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 Quarterly Report on Form 10-Q, 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20180530005482/en/
Source: Sage Therapeutics
Sage Therapeutics
Investors:
Paul Cox,
617-299-8377
paul.cox@sagerx.com
or
Media:
Maureen
L. Suda, 585-355-1134
maureen.suda@sagerx.com
