FDA Grants Fast Track Designation to Concert Pharmaceuticals’ CTP-543 for the Treatment of Alopecia Areata

CTP-543 is a Novel JAK Inhibitor in Phase 2 for Alopecia Areata as
Potential First-in-Class Treatment

LEXINGTON, Mass.–(BUSINESS WIRE)–$CNCE #CNCEConcert
Pharmaceuticals, Inc.
(NASDAQ: CNCE) today announced that the U.S.
Food and Drug Administration (FDA) has granted Fast Track designation
for CTP-543, a novel, oral Janus kinase (JAK) inhibitor 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.

Fast Track designation is a process designed to facilitate the
development and expedite the review of new therapies to treat serious
conditions and address unmet medical needs. With Fast Track designation,
early and frequent communications between the FDA and the sponsor are
encouraged throughout the drug development and review process to help to
ensure that questions are resolved quickly.

“Fast Track designation recognizes that alopecia areata is a serious
disease in need of effective treatments. We look forward to
collaborating with the FDA on the development of CTP-543,” said James
Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals.
“There are no FDA-approved treatments for alopecia areata and CTP-543
would represent a significant medical advance as a potential
first-in-class treatment for moderate-to-severe disease.”

Concert is conducting a multi-center, double-blind, randomized,
placebo-controlled Phase 2a trial to evaluate the safety and efficacy of
CTP-543 in adults with moderate-to-severe alopecia areata. For
additional information on the ongoing CTP-543 Phase 2a trial, visit www.clinicaltrials.gov.

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. 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 FDA selected alopecia areata as one of eight new disease areas that
it focused on under its Patient-Focused Drug Development Initiative
(PFDDI) meetings in 2016-2017. The goal of the PFDDI is to bring patient
perspectives into an earlier stage of product development. The meeting
to discuss alopecia areata was held on Monday, September 11, 2017.
Additional information is available online at: https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm554443.htm

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 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
pipeline
 of innovative medicines targeting autoimmune and
inflammatory 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 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.

Contacts

Concert Pharmaceuticals, Inc.
Investors
Justine Koenigsberg,
781-674-5284
ir@concertpharma.com
or
Media
The
Yates Network
Kathryn Morris, 914-204-6412
kathryn@theyatesnetwork.com