First patient dosed in Phase 2 proof-of-concept
trial of SAGE-217 in Parkinson’s disease
Trial sites are open and screening patients for
the Phase 2a trial in essential tremor; patients expected to be
dosed imminently
Sage Therapeutics (NASDAQ:SAGE), a clinical-stage
biopharmaceutical company developing novel medicines to treat
life-altering central nervous system (CNS) disorders, today
announced the initiation of Phase 2 clinical development of
SAGE-217, a novel, internally-developed, next generation oral GABAA
receptor modulator that Sage is developing in both mood and
movement disorders. Dosing has now been initiated in the first of
two movement disorder indications. Patients are receiving SAGE-217
in a Phase 2 proof-of-concept trial in Parkinson’s disease. In
addition, trial sites are open and screening patients for a Phase
2a trial in essential tremor. Patients in this trial are expected
to be dosed imminently. Top-line results from the Part A open-label
study in Parkinson’s disease are expected in the first half of 2017
and the essential tremor study is anticipated to report results in
the second half of 2017. The Company also plans to initiate Phase 2
clinical trials of SAGE-217 in two mood disorders – major
depressive disorder (MDD) and postpartum depression (PPD).
“Sage continues to pioneer innovative approaches to neuroscience
drug development in CNS indications with high unmet need where we
can redefine treatment paradigms,” said Jeff Jonas, M.D., Chief
Executive Officer of Sage. “The SAGE-217 clinical program is an
excellent example of this approach. The initiation of mid-stage
trials of our novel, proprietary oral compound is a significant
corporate milestone and a credit to our talented team of
translational chemists, and clinical and regulatory leaders.”
“Administering the first dose of SAGE-217 in a proof-of-concept
study in Parkinson’s disease and the initiation of SAGE-217 in
essential tremor illustrate major progress in Sage’s effort to
address the serious need for additional effective treatments for
these movement disorders and in building our multi-product,
neuropsych portfolio,” said Steve Kanes, M.D., Ph.D., Chief Medical
Officer of Sage. “SAGE-217 is one of several product candidates
that Sage is developing to target the GABAA receptor system.
Dysfunction in this system is thought to be at the core of numerous
psychiatric and neurological disorders including essential tremor
and both the motor and non-motor symptoms in Parkinson’s
disease.”
The essential tremor study is a Phase 2a double-blind,
placebo-controlled, randomized withdrawal study that will evaluate
the efficacy, safety, tolerability and pharmacokinetics of SAGE-217
in approximately 60 patients with essential tremor. The primary
endpoint of the study is to compare the effect of one week of
SAGE-217 on overall kinetic tremor symptoms. Secondary endpoints
include additional accelerometer-derived and clinician-rated rating
scales.
The Parkinson’s disease program is a two-part Phase 2 clinical
trial evaluating the safety, tolerability, pharmacokinetics and
efficacy of SAGE-217 in moderate Parkinson’s disease patients. Part
A of the Phase 2 trial will be an open-label, proof-of-concept
study evaluating SAGE-217 in approximately 18 patients which, if
promising, may lead to a randomized, placebo-controlled Phase 2
trial. The primary endpoint for the Part A study will be to
evaluate the safety and tolerability of SAGE-217. The secondary
endpoint will be to evaluate improvement in motor symptoms as
assessed by the change from baseline after one week in the Movement
Disorder Society - Unified Parkinson’s Disease Rating Scale
(MDS-UPDRS) Part 3 (Motor Examination) total score.
About SAGE-217
SAGE-217 is a next generation positive allosteric modulator that
has been optimized for selectivity to synaptic and extrasynaptic
GABA receptors and a pharmacokinetic profile intended for daily
oral dosing. The GABA system is the major inhibitory signaling
pathway of the brain and CNS, and contributes significantly
to regulating CNS function. In a Phase 1 clinical
program, SAGE-217 was well-tolerated in single and multiple
ascending doses and the results were consistent with the predicted
pharmacokinetic and pharmacologic profile. SAGE-217 is
currently being developed for certain mood and movement disorders.
A Phase 2 clinical trial in Parkinson’s disease is ongoing and
initiation of dosing in the essential tremor study is pending.
Phase 2 clinical trials in postpartum depression and major
depressive disorder are planned.
About Essential Tremor
Essential tremor is a common neurological condition that affects
an estimated 6 to 7 million in the U.S.1 Essential tremor causes a
rhythmic trembling of the hands, head, voice, legs or
trunk. Symptoms generally evolve over time and are both
visible and persistent following onset, which commonly occurs
either between 15-20 or 50-70 years of age. First-line
treatments for essential tremor include the anticonvulsant
primidone and the β-adrenergic blocker propranolol. Current
treatments for essential tremor are only moderately effective,
reducing, though not resolving, tremor amplitudes in about 50% of
patients. In addition, one out of three patients abandons
treatment due to side effects or poor efficacy.
About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disorder
that affects an estimated 700,000 patients in the U.S.2 and causes
impairment of motor function, including impaired movement, muscle
stiffness and tremors as well as non-motor symptoms including
anxiety, depression, sleep difficulties and gastrointestinal
disorders. Symptoms generally become more pronounced over time, and
the average age of Parkinson’s diagnosis is 60 years of age.
First-line treatments for Parkinson’s disease include combination
levodopa/carbidopa therapy. Current treatments for Parkinson’s
disease are only moderately effective in reducing symptoms in the
early stages of the disease, and patients become less responsive to
treatment as the disease progresses. Thus, there is a growing need
for innovative new treatments to prevent, delay onset or alleviate
symptoms of Parkinson’s disease.
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, SAGE-547,
is in Phase 3 clinical development for super-refractory status
epilepticus, a rare and severe seizure disorder, and for postpartum
depression. Sage is developing its next generation modulators,
including SAGE-217 and SAGE-718, with a focus on acute and chronic
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 expectations regarding development of SAGE-217 and our other
product candidates and their potential in the treatment of various
CNS disorders; the expected initiation and timing of clinical
trials and anticipated availability and announcement of data and
results; our estimates as to the number of patients with essential
tremor and Parkinson’s disease and our belief as to unmet need in
these populations. 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 experience slower than
expected clinical site initiation or enrollment in our clinical
trials, or the potential need for additional analysis or data or
the need to enroll additional patients, leading to possible delays
in completion of trials or in the availability of data; we may not
be able to generate supportive non-clinical data or to successfully
demonstrate the efficacy and safety of our product candidates at
each stage of development; success in our non-clinical studies
or in earlier stage clinical trials may not be repeated or observed
in ongoing or future studies involving the same compound or other
product candidates, and ongoing and future pre-clinical and
clinical results may not support further development of product
candidates or be sufficient to gain regulatory approval to market
any product; decisions or actions of regulatory agencies may affect
the initiation, timing, progress and cost of clinical trials, and
our ability to proceed with further clinical studies of a product
candidate or to obtain marketing approval; we may encounter adverse
events at any stage of development that negatively impact further
development; the actual size of the essential tremor and
Parkinson’s disease patient populations may be significantly lower
than our estimates; we may decide that a development pathway for
one of our product candidates in one or more indications is no
longer feasible or advisable or that the unmet need no longer
exists; and we may encounter technical and other unexpected
hurdles in the development and manufacture of our products which
may delay our timing or increase our expenses and use of cash, as
well as those risks more fully discussed in the section entitled
"Risk Factors" in our most recent Quarterly Report on Form 10-Q, 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.
1 Louis ED, Ottman R. How many people in the USA have essential
tremor? Deriving a population estimate based on epidemiological
data. Tremor Other Hyperkinet Mov. 2014; 4. doi:
10.7916/D8TT4P4B
2 Willis et al, Neuroepidemiology. 2010;34:143–151
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Investor Contact:Sage TherapeuticsPaul Cox,
617-299-8377paul.cox@sagerx.comorMedia Contact:Suda
Communications LLCMaureen L. Suda,
585-387-9248maureen.suda@sagerx.com
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