Market Overview

Concert Pharmaceuticals Announces Completion of Enrollment in First Cohort of CTP-543 Phase 2a Trial


CTP-543 is Being Evaluated in Moderate-to-Severe Alopecia Areata

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

Pharmaceuticals, Inc.
(NASDAQ:CNCE) today announced that it has
completed enrollment of the first cohort of its Phase 2a 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 that is commercially available under the
brand name Jakafi® for the treatment of certain blood disorders.

In the Phase 2a trial, patients with alopecia areata in the first cohort
are administered 4 mg of CTP-543 or placebo twice daily for 24 weeks. An
independent Data Monitoring Committee (DMC) will conduct an interim
safety data review from the first cohort after patients have completed
three months of dosing. This review is expected to be completed in the
first quarter of 2018. Based on this review, the DMC will provide its
recommendation regarding initiating the second cohort in the trial,
which will be administered 8 mg of CTP-543 or placebo twice daily for 24
weeks. The Phase 2a trial is expected to be completed in the second half
of 2018.

"We are excited to see the enthusiastic participation by alopecia areata
patients in our study. Its sequential dosing design will allow us to
assess the safety and efficacy of CTP-543 in this disease in a
controlled and deliberate manner," said James Cassella, Ph.D., Chief
Development Officer of Concert Pharmaceuticals. "We look forward to the
DMC's assessment, and we are on track with our goal to complete the
trial in the second half of 2018."

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 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.

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) meeting in 2016-2017. The goal of the PFDDI is to bring patient
perspectives into an earlier stage of product development. The meeting
was held on Monday, 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

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,
availability of funding sufficient for our foreseeable and unforeseeable
operating expenses and capital expenditure requirements 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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