Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the
publication of safety and efficacy data from the randomized,
double-blind, placebo-controlled dose-ranging Phase 2 clinical
trial for CTP‑543 in the Journal of the American Academy of
Dermatology (JAAD). CTP-543 is an investigational oral Janus Kinase
(JAK) inhibitor being evaluated in ongoing clinical trials for the
treatment of adults with moderate to severe alopecia areata, an
autoimmune disease in which the immune system attacks hair
follicles and results in hair loss. The publication reports
clinically meaningful and statistically significant scalp hair
regrowth after 24 weeks of treatment with CTP-543 in both the 8 mg
twice-daily and 12 mg twice-daily dose groups in patients with
alopecia areata.
“We have made so much progress in alopecia areata in so short a
time, and it is utterly exciting to see JAK inhibitors advance as a
potential treatment for alopecia areata,” said Brett King, M.D.,
Department of Dermatology, Yale University School of Medicine and
lead author of the publication. “The therapeutic response in
patients as early as 12 weeks in this trial is significant, and we
hope that with continued development CTP-543 will become a
treatment option for the many people suffering from this serious
autoimmune disease.”
The publication highlights the establishment of two doses of
CTP-543 with statistically significant hair regrowth and a
generally well tolerated safety profile in patients with moderate
to severe alopecia areata. Additionally, patient-reported outcomes
of improvement following treatment with CTP-543 were statistically
significant.
The robust clinical results observed in the trial supported the
Breakthrough Therapy designation from the U.S. Food and Drug
Administration (FDA) and the progression of CTP-543 into the
THRIVE-AA pivotal Phase 3 clinical program. The Company recently
reported positive topline data from the THRIVE-AA1 trial, and
topline data from the THRIVE-AA2 trial is expected in the third
quarter of 2022.
“We are very pleased that the results from this trial are
published in this very prestigious journal and that its broad
readership across the field of dermatology will see the first
published results of CTP-543 in patients with alopecia areata,”
said James V. Cassella, Ph.D., Chief Development Officer of Concert
Pharmaceuticals and a co-author of the publication. “Today’s JAAD
publication further emphasizes the growing body of literature
showing the importance of the JAK mechanism in alopecia areata and
the potential therapeutic utility of CTP-543 as a new treatment
option for this important medical condition.”
CTP-543 Phase 2 Trial Results
The Phase 2 trial was a randomized, double-blind,
placebo-controlled, sequential dose trial to evaluate the safety
and efficacy of CTP-543 in adult patients with moderate to severe
alopecia areata. A total of 149 patients were randomized to receive
one of three doses of CTP-543 (4 mg, 8 mg or 12 mg) or placebo,
administered twice-daily. The primary outcome measure utilized the
Severity of Alopecia Tool (SALT) after 24 weeks of dosing.
Patients treated with either 8 mg twice-daily or 12 mg
twice-daily of CTP-543 met the primary efficacy endpoint with
statistically significant differences (p <0.001) relative to
placebo in the percentage of patients achieving a ≥ 50% relative
change from baseline at 24 weeks using the SALT score. The 8 mg
twice-daily and 12 mg twice-daily groups were also significantly
different from placebo in the number of patients achieving ≥ 75%
and ≥ 90% relative change in SALT from baseline at 24 weeks. At
Week 24, patients treated with 8 mg twice-daily and 12 mg
twice-daily compared to placebo also rated significantly greater
improvement in their alopecia areata on the Patient Global
Impression of Improvement Scale. For patients in the 4 mg cohort,
the relative reduction in their overall SALT score from baseline
was not significantly different from placebo.
In the Phase 2 study, CTP-543 was generally well tolerated and
safety results were consistent with the known safety profile of JAK
inhibitors. There was one serious adverse event reported in the 12
mg dose group where the patient was able to complete the trial
after a brief dose interruption.
A copy of the publication entitled, “Phase 2 randomized,
dose-ranging trial of CTP-543, a selective Janus Kinase inhibitor,
in moderate-to-severe alopecia areata” is available online on the
JAAD website and supplemental materials are available on
Mendeley.
About CTP-543 and Alopecia Areata
CTP-543 is an oral selective inhibitor of Janus kinases JAK1 and
JAK2.
Alopecia areata is an autoimmune disease in which the immune
system attacks hair follicles, resulting in partial or complete
loss of hair on the scalp and body. Alopecia areata may affect up
to approximately 1.5 million 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 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.
Following the FDA’s Patient-Focused Drug Development meeting held
in September 2017 on alopecia areata, the FDA summarized the input
shared by patients and patient representatives in a Voice of the
Patient report. Additional information on the PFDDI is available
online.
About Concert
Concert Pharmaceuticals is a clinical stage biopharmaceutical
company that is developing small molecule drugs that it discovered
through the application of its DCE Platform® (deuterated chemical
entity platform). Selective incorporation of deuterium into known
molecules has the potential, on a case-by-case basis, to provide
better pharmacokinetic or metabolic properties, thereby enhancing
their clinical safety, tolerability or efficacy. Concert’s lead
product candidate is in late-stage development for the treatment of
alopecia areata, a serious autoimmune dermatological condition.
Concert is also assessing a number of earlier-stage pipeline
candidates. 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, among others,
statements about our expectations regarding the development of
CTP-543 and the timing of availability of clinical trial data, and
any other statements containing the words “anticipate,” “believe,”
“continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “target,” “will,”
“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, timing and design of future clinical trials, the
availability and timing of data from ongoing and future clinical
trials and the results of such 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 Benigno M. Clinical, Cosmetic and
Investigational Dermatology 2020
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version on businesswire.com: https://www.businesswire.com/news/home/20220607005334/en/
For additional information: Justine E. Koenigsberg
(Investors) Concert Pharmaceuticals, Inc. (781) 674-5284
ir@concertpharma.com
Kathryn Morris (media) The Yates Network (914) 204-6412
kathryn@theyatesnetwork.com
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