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The Company Expects to Initiate a Phase 2 Trial in Patients with
Schizophrenia in the Fourth Quarter of 2019

LEXINGTON, Mass.–(BUSINESS WIRE)–$CNCE #CNCEConcert
Pharmaceuticals, Inc.
(NASDAQ: CNCE) today reported positive results
from two studies in its Phase 1 program evaluating CTP-692, a novel
deuterium-modified form of D-serine being developed as an adjunctive
treatment for schizophrenia. The safety assessments in the single- and
multiple-ascending dose trials in healthy volunteers showed that the
drug was well tolerated over the dose ranges tested, which include the
doses expected to be evaluated in Phase 2 testing. Importantly, key
blood and urine markers of kidney function did not indicate any signs of
renal impairment. These data are consistent with preclinical
findings with CTP-692
indicating an improved renal safety profile
compared to non-deuterated D-serine which is known to produce renal
toxicity in rats. Despite evidence of benefit in multiple published
clinical studies of non-deuterated D-serine in the treatment of
schizophrenia, its use has been limited due to renal safety concerns.
Furthermore, in contrast to non-deuterated D-serine, which has been
reported to display highly variable pharmacokinetic behavior in humans,
CTP-692 was found to have low inter-individual pharmacokinetic
variability. The Company expects data from the single-and
multiple-ascending dose Phase 1 trials to be presented at a future
scientific meeting.

“We are pleased that CTP-692 demonstrated favorable clinical properties
including an excellent safety profile in its Phase 1 studies, and we
look forward to advancing it into Phase 2 evaluation later this year,”
said Roger Tung, Ph.D., President and Chief Executive Officer of Concert
Pharmaceuticals.

The Phase 1 program was designed to assess CTP-692’s safety,
tolerability and pharmacokinetic profile in healthy volunteers. The
Phase 1 program includes three studies: a crossover comparison of
CTP-692 versus D-serine, a single-ascending dose study that also
assessed the effect of food on the pharmacokinetics of CTP-692, and a
multiple-ascending dose trial assessing CTP-692 dosed orally over seven
days. In the single- and multiple-ascending dose trials, CTP-692 was
evaluated across doses ranging from 0.5 to 4 grams compared to placebo
in a total of 72 volunteers. CTP-692 demonstrated a favorable safety,
tolerability and pharmacokinetic profile with no serious adverse events
reported. In a separate study in 11 healthy volunteers treated in a
crossover design with both CTP-692 and D-serine, CTP-692 was found to
have increased plasma exposure compared to D-serine. These results were
presented as a poster
at the 2019 American Society of Clinical Psychopharmacology Annual
Meeting in Scottsdale, AZ.

About CTP-692
CTP-692 is a deuterium-modified analog of the
endogenous NMDA receptor co-agonist, 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 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 renal safety profile and improved clinical
outcomes in the treatment of schizophrenia. Preclinical studies have
demonstrated that CTP-692 provides preferentially higher concentrations
in the forebrain, compared to plasma and brainstem regions, as well as
overall higher exposure in the brain relative to non-deuterated
D-serine. CTP-692 will be initially developed as an adjunctive therapy
administered in addition to standard antipsychotic medicines with the
potential to improve 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 receptor co-agonist. It has been postulated for
some time that administration of NMDA co-agonists could benefit patients
with schizophrenia across multiple symptom domains since there is
evidence that plasma levels of endogenous D-serine are reduced in
patients with schizophrenia.

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 are frequently associated with neurologic and metabolic
adverse effects. 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
Concert
Pharmaceuticals
is a clinical stage biopharmaceutical company
focused on applying its DCE
Platform®
(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 www.concertpharma.com
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.

Contacts

Justine Koenigsberg (investors)
Concert Pharmaceuticals, Inc.
(781)
674-5284
[email protected]

Kathryn
Morris (media)
The Yates Network
(914) 204-6412
[email protected]