Market Overview

Concert Pharmaceuticals Initiates CTP-543 Phase 2 Trial in Alopecia Areata


Pharmaceuticals, Inc.
(NASDAQ:CNCE) today announced the initiation
of a Phase 2a clinical trial evaluating 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 an orally-administered selective inhibitor of
Janus kinases 1 and 2, known as JAK1 and JAK2, which are enzymes
believed to be involved in this autoimmune disorder. The Phase 2a trial
is designed to evaluate the safety and efficacy of CTP-543 after 12
months of dosing with the primary efficacy analysis at week 24. The
Company expects to report topline data in the first quarter 2018.

"Alopecia areata is a devastating autoimmune disease that impacts half a
million or more patients in the U.S. at any given time. There is a
significant unmet medical need with no FDA-approved therapy," said Roger
Tung, Ph.D., President and Chief Executive Officer of Concert
Pharmaceuticals. "We intend to be at the forefront of advancing a new
oral treatment for alopecia areata patients and look forward to
assessing CTP-543's therapeutic efficacy in the Phase 2 trial."

"The treatment community, and patients suffering from this disease, have
been eager for new treatments for alopecia areata. Though more work
needs to be done to explore safety and efficacy, there is a growing and
encouraging body of evidence supporting the Janus kinase mechanism in
this indication," stated Julian Mackay-Wiggan, MD, MS., Associate
Professor of Dermatology and Director of Clinical Research in
Dermatology at Columbia University. "I believe this is a new era in
which we are advancing treatments into clinical evaluation that target
the underlying biology of the disease."

The Phase 2a trial is a double-blind, randomized, placebo-controlled,
parallel dose trial to evaluate the safety and efficacy of CTP-543 in
adult patients with moderate-to-severe alopecia areata. Approximately
100 patients will be randomized to receive one of four doses of CTP-543
(4, 8, 12 and 16 mg twice daily) or placebo. The primary outcome measure
will utilize the severity of alopecia tool (SALT) after 24 weeks of
dosing. The trial will include an additional 28 weeks of dosing where
all patients enrolled in the study will receive CTP-543. Additional
information about the trial will be available on

Clinical Studies with CTP-543 and JAK Inhibitors in Alopecia Areata

In Phase 1 clinical studies to date with CTP-543 in healthy volunteers,
the pharmacokinetic profile showed increased exposure with increasing
doses of CTP-543. CTP-543 was well tolerated across all dose groups and
there were no serious adverse events reported. The most commonly
reported adverse event was headache. There were no withdrawals or dose
modifications related to CTP-543. The average exposure observed with 16
mg of CTP-543 twice daily appeared to be comparable to published
findings for the reported average exposure of 20 mg ruxolitinib twice
daily. In an open-label clinical trial of 12 patients with alopecia
areata, investigators at Columbia University reported that 20 mg of
ruxolitinib administered orally twice-daily resulted in significant hair
regrowth in 75% of individuals with moderate to severe alopecia areata1.

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 time2. 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.

In 2015, the FDA selected alopecia areata as one of eight new disease
areas that it will focus on in its Patient-Focused Drug Development
Initiative (PFDDI) meetings during fiscal year 2016-2017. The goal of
the PFDDI is to bring patient perspectives into an earlier stage of
product development.

As part of Concert's activities with the treatment community, the
Company is furthering the development of educational tools about
alopecia areata. The Company has recently launched a website designed to
provide educational content about research for new treatment options for
those impacted with alopecia areata. To learn more, please visit,

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 pulmonary diseases,
including cystic fibrosis, autoimmune and inflammatory diseases and
central nervous systems (CNS) disorders. For more information please

Cautionary Note on Forward Looking Statements
Any statements
in this press release about our future expectations, plans and
prospects, including statements about our expectations on the 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 Annual Report on Form 10-K 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.

1JCI Insight. 2016;1(15):e89790.
2 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.
is a registered trademark of Incyte Corporation.

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