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Concert Pharmaceuticals Initiates Phase 1 Single-Ascending Dose Trial of CTP-692 as an Adjunctive Treatment for Schizophrenia

Thursday, January 24th 2019 at 12:00pm UTC

Pharmaceuticals, Inc.
(NASDAQ: CNCE) today announced that it has
initiated the second Phase 1 clinical trial with CTP-692, a novel
deuterium-modified form of D-serine being developed as an adjunctive
treatment for schizophrenia. The Phase 1 single-ascending dose trial
will evaluate the safety, tolerability, and pharmacokinetic profile of
CTP-692 in healthy volunteers.

“For decades all approved schizophrenia drugs have acted predominantly
by modulation of dopamine, or dopamine and serotonin receptors. By
activating glutamatergic pathways as an NMDA co-agonist, CTP-692 has the
potential to be a safe and effective new adjunctive treatment for
schizophrenia, and treat positive and negative symptoms and cognitive
dysfunction,” stated Roger Tung, Ph.D., President and Chief Executive
Officer of Concert Pharmaceuticals, Inc. “We are excited about
continuing our Phase 1 program and in the fourth quarter of 2019 are
preparing to advance CTP-692 into a Phase 2 trial that is intended to
support advancement into pivotal evaluation.”

The initial Phase 1 trial evaluated the safety, tolerability, and
pharmacokinetics of a single oral dose of CTP-692 versus D-serine in a
crossover study conducted in Australia. In individuals treated with both
compounds, CTP-692 was found to have increased plasma exposure compared
to D-serine. In addition, CTP-692 was found to be well tolerated in
healthy volunteers and no serious adverse events were reported. Under
its Investigational New Drug application in the United States, Concert
will conduct this second study in the Phase 1 program to assess the
safety, tolerability, and pharmacokinetics of single-ascending oral
doses of CTP-692 in a double-blind, placebo-controlled trial. The Phase
1 single-ascending dose trial will also evaluate the effect of food on
the pharmacokinetics of the compound. In addition, the Phase 1 program
will include a double-blind, placebo-controlled, multiple-ascending dose
trial assessing CTP-692 dosed orally over seven days. Topline data from
the Phase 1 program is expected in the first half of 2019.

The CTP-692 clinical program is supported by Concert’s preclinical
studies which have shown the potential of CTP-692 to improve upon the
safety profile of D-serine. D-Serine has been shown to cause
nephrotoxicity in published preclinical studies. Concert’s preclinical
studies have demonstrated that selective deuterium modification resulted
in increased exposure of CTP-692 relative to a similar dose of D-serine,
and administration of CTP-692 did not cause changes in serum creatinine
and blood urea nitrogen at doses where D-serine caused substantial
nephrotoxicity as assessed by these kidney function markers. These
preclinical results were presented by Concert at the American College of
Toxicology 2018 Annual Meeting in November 2018. A copy of the poster
may be accessed in the Scientific Presentations section of the Company’s
website at

About CTP-692

CTP-692 is a deuterium-modified analog of endogenous D-serine. Based on
documented effects of D-serine, the Company believes that CTP-692 has
the potential to restore NMDA receptor activity in key areas of the
brain and improve clinical outcomes in patients with schizophrenia.
CTP-692 has been shown to have similar ability to bind to and activate
human NMDA receptors relative to D-serine, with the potential for an
improved safety profile and improved clinical outcomes in the treatment
of schizophrenia. CTP-692 will be developed as an adjunctive therapy
administered in addition to standard antipsychotic medicines to improve
both positive and negative symptoms as well as cognitive function in
patients with schizophrenia.

An extensive body of evidence supports NMDA receptor hypofunction as a
key underlying mechanism of schizophrenia. The NMDA receptor comprises
two binding domains and, in addition to requiring glutamate binding,
activation with a co-agonist such as D-serine or glycine is necessary
for NMDA receptor activation. D-Serine is believed to be the most
important human NMDA synaptic co-agonist. It has been postulated for
some time that administration of NMDA co-agonists could benefit patients
with schizophrenia since there is evidence that plasma and cerebrospinal
fluid (CSF) levels of endogenous D-serine are reduced in patients with

About Schizophrenia

Schizophrenia is a chronic and devastating neuropsychiatric disorder
that is ranked as a leading cause of disability worldwide. The disease
afflicts nearly 1% of the world’s population, affecting both men and
women equally, and striking all ethnic and socioeconomic groups with a
similar level of prevalence. The illness is characterized by multiple
symptoms that are categorized into three main clusters known as positive
symptoms (hallucinations, delusional behaviors and thought disorder),
negative symptoms (social withdrawal, flattened affect and poverty of
speech), and cognitive dysfunction (diminished capacity for attention,
working memory and executive function). The underlying basis of the
current antipsychotic therapy is that excessive dopaminergic
neurotransmission and dysfunctional D2 receptor signaling play key
pathophysiological roles in the disease, and consequently all typical
and atypical antipsychotics in clinical practice possess some level of
D2 antagonist activity. Currently available antipsychotic drugs offer
some benefit for positive symptoms but many patients are not adequately
treated since currently available treatments are limited in their
capacity to treat negative symptoms and cognitive dysfunction which are
related to poor functional outcomes.

About Concert
is a clinical stage biopharmaceutical company
focused on applying its DCE
(deuterated chemical entity platform) to create novel
medicines designed to treat serious diseases and address unmet patient
needs. The Company’s approach starts with previously studied compounds,
including approved drugs, in which deuterium substitution has the
potential to enhance clinical safety, tolerability or efficacy.
Concert’s pipeline of
innovative medicines currently targets autoimmune diseases and central
nervous systems (CNS) disorders. For more information please visit
or follow us on Twitter at @ConcertPharma
or on LinkedIn.

Cautionary Note on Forward Looking Statements
Any statements
in this press release about our future expectations, plans and
prospects, including statements about the clinical development of
CTP-692 and other statements containing the words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,”
“plan,” “potential,” “predict,” “project,” “should,” “target,” “would,”
and similar expressions, constitute forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important factors,
including: the uncertainties inherent in the initiation of future
clinical trials, availability and timing of data from ongoing and future
clinical trials and the results of such trials, whether preliminary
results from a clinical trial will be predictive of the final results of
that trial or whether results of early clinical trials will be
indicative of the results of later clinical trials, expectations for
regulatory approvals and other factors discussed in the “Risk Factors”
section of our most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission and in other filings that we make
with the Securities and Exchange Commission. In addition, any
forward-looking statements included in this press release represent our
views only as of the date of this release and should not be relied upon
as representing our views as of any subsequent date. We specifically
disclaim any obligation to update any forward-looking statements
included in this press release.

Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and
DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.


Justine Koenigsberg (investors)
Concert Pharmaceuticals, Inc.

Kathryn Morris (media)
The Yates Network
(914) 204-6412

Source: Concert Pharmaceuticals, Inc.

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