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Concert Pharmaceuticals Announces Initiation of Enrollment in Second Cohort of CTP-543 Phase 2a Trial for Alopecia Areata


Phase 2a Trial On Track to Complete in Second Half of 2018

Pharmaceuticals, Inc.
(NASDAQ:CNCE) today announced that it has
initiated enrollment of the second cohort of its Phase 2a clinical trial
evaluating CTP-543. Concert is developing CTP-543 for the treatment of
moderate-to-severe alopecia areata, an autoimmune disorder in which the
immune system attacks hair follicles, resulting in patchy or complete
hair loss. CTP-543 is a deuterium-modified analog of ruxolitinib, a
Janus Kinase (JAK) inhibitor.

An independent Data Monitoring Committee (DMC) conducted an interim
safety data review of the first cohort of the Phase 2a trial, following
12 weeks of dosing with 4 mg of CTP-543 or placebo twice daily. Based on
this review, the DMC provided its recommendation to continue with the
current cohort and to initiate dosing of the second cohort, whereby
patients will be administered 8 mg of CTP-543 or placebo twice daily for
24 weeks. The Company expects to report topline data from Phase 2a trial
in the fourth quarter of 2018.

"The alopecia areata community is eager for an effective and
well-characterized treatment for this important autoimmune disease and
we are extremely pleased that novel treatments, including CTP-543, are
advancing in clinical trials," said Dory Kranz, President and CEO of the
National Alopecia Areata Foundation.

"We are pleased that the CTP-543 trial is progressing as planned as we
continue to advance the evaluation of our innovative product candidate
for alopecia areata," said James Cassella, Ph.D., Chief Development
Officer of Concert Pharmaceuticals. "There is a significant unmet
medical need for alopecia areata and we intend to be at the forefront of
advancing a new oral treatment for patients."

The Phase 2a trial is a double-blind, randomized, placebo-controlled
trial to evaluate the safety and efficacy of CTP-543 in adults with
moderate-to-severe alopecia areata. Approximately 90 patients are being
enrolled in the study and sequentially randomized to receive one of two
doses of CTP-543 (4 mg or 8 mg) or placebo twice daily. The primary
outcome measure will utilize the severity of alopecia tool (SALT) after
24 weeks of dosing. Patient-reported outcome measures will be assessed
as secondary endpoints. If appropriate, the protocol may be amended to
explore higher doses of CTP-543. Additional information about the trial
is available on

CTP-543 has been well-tolerated in clinical evaluation to date and has
demonstrated a non-clinical safety profile consistent with therapeutic
JAK inhibition.

About CTP-543 and Alopecia Areata
CTP-543 was discovered by
applying Concert's deuterium chemistry technology to modify ruxolitinib,
a drug which selectively inhibits Janus kinases 1 and 2 (JAK1 and JAK2)
and is commercially available under the name Jakafi® in the United
States for the treatment of certain blood disorders. Ruxolitinib has
been used to treat alopecia areata in academic settings, including an
investigator-sponsored clinical trial, and has been reported to promote
hair growth in individuals with moderate-to-severe disease. Deuterium
modification of ruxolitinib was found to alter its human
pharmacokinetics in ways which may enhance its use as a treatment for
alopecia areata. The U.S. Food and Drug Administration (FDA) has granted
Fast Track designation for CTP-543.

Alopecia areata is an autoimmune disease that results in partial or
complete loss of hair on the scalp and body that may affect up to
650,000 Americans at any given time1. The scalp is the most
commonly affected area, but any hair-bearing site can be affected alone
or together with the scalp. Onset of the disease can occur throughout
life and affects both women and men. Alopecia areata can be associated
with serious psychological consequences, including anxiety and
depression. There are currently no drugs approved by the U.S. Food and
Drug Administration (FDA) for the treatment of alopecia areata.

The FDA selected alopecia areata as one of eight new disease areas that
it focused on under its Patient-Focused Drug Development Initiative
(PFDDI) in 2016-2017. The goal of the PFDDI is to bring patient
perspectives into an earlier stage of product development. The PFDDI
meeting on alopecia areata was convened by the FDA on September 11,
2017. Additional information is available online at:

About Concert
is a clinical stage biopharmaceutical company
focused on applying its DCE
(deuterated chemical entity platform) to create novel
medicines designed to address unmet patient needs. The Company's
approach starts with approved drugs in which deuterium substitution has
the potential to enhance clinical safety, tolerability or efficacy.
Concert has a broad
 of innovative medicines targeting autoimmune and
inflammatory 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 our expectations for clinical
development of CTP-543, 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, 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.

1 Fricke M. Epidemiology and Burden of alopecia areata: a
systemic review. Clinical, Cosmetic and Investigational Dermatology.
2015; Vol 8. 397-403.

Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and
DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.
Jakafi® is a registered trademark of Incyte Corporation.

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