Concert Pharmaceuticals Completes Enrollment in Phase 2a Trial of CTP-543 in Alopecia Areata
Topline Phase 2a Data from the 4 mg and 8 mg Cohorts Expected in Fourth Quarter of 2018
“The speed with which we enrolled the trial speaks to the significant
unmet need for alopecia areata and interest in this program. We are
grateful to the patients who volunteered to participate in our trial,”
The Phase 2a trial is a double-blind, randomized, placebo-controlled, sequential dose trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate-to-severe alopecia areata. Approximately 90 patients were randomized to receive one of two doses of CTP-543 (4 mg or 8 mg twice daily) or placebo. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. If appropriate, the protocol may be amended to explore 12 mg twice daily of CTP-543. Additional information about the trial is available on 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. Deuterium modification of ruxolitinib was
found to alter its human pharmacokinetics in ways which may enhance its
use as a treatment for alopecia areata.
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 (
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. Following the
U.S. Food and Drug Administration’s (
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.
1 Fricke M. Epidemiology and Burden of alopecia areata: a systemic review. Clinical, Cosmetic and Investigational Dermatology. 2015; Vol 8. 397-403.
Jakafi® is a registered trademark of
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Concert Pharmaceuticals, Inc.
Justine E. Koenigsberg, 781-674-5284
The Yates Network
Kathryn Morris, 914-204-6412