Concert Pharmaceuticals Completes Enrollment of 12 mg Cohort in Phase 2a Trial of CTP-543 in Alopecia Areata
January 22 2019 - 07:00AM
Business Wire
Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced
that it has completed patient enrollment of the final cohort
evaluating 12 mg twice daily of CTP-543 in its Phase 2a trial for
the treatment of moderate-to-severe alopecia areata. The trial
previously enrolled patients to receive 4 mg and 8 mg twice daily
compared to placebo. Data from the Phase 2a trial including the 12
mg cohort is expected in the third quarter of 2019.
“It is important early on in our development program to identify
the safest and most effective doses of CTP-543 for patients with a
chronic autoimmune disease like alopecia areata. The evaluation of
the twice daily 12 mg dose provides a comprehensive exploration of
the relevant dose range for CTP-543 and positions us well for later
stage development of the compound,” stated James V. Cassella,
Ph.D., Chief Development Officer of Concert Pharmaceuticals.
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. Patients were sequentially randomized to receive
one of three doses of CTP-543 (4 mg, 8 mg, or 12 mg) or placebo
twice daily. The primary outcome measure utilizes the severity
of alopecia tool (SALT) after 24 weeks of dosing.
Additional information about the trial is available on
www.clinicaltrials.gov.
In November 2018, Concert reported positive interim topline
results from the first two cohorts (4 mg and 8 mg twice daily) in
its Phase 2a trial. The 8 mg twice daily cohort achieved the
primary endpoint of ≥ 50% relative reduction in overall severity of
alopecia tool (SALT) score from baseline at 24 weeks in 47% of
patients compared to 8.6% of placebo patients (p <0.001). The 4
mg twice daily CTP-543 cohort had numerically higher responses than
placebo but was not statistically better than
placebo. Regrowth of hair did not appear to plateau at Week
24. Treatment with CTP-543 was generally well tolerated. No serious
adverse events were reported. Additional information about the
CTP-543 Phase 2a results is available in the Scientific
Presentations section of Company’s website.
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. 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 U.S. Food
and Drug Administration (FDA) has granted Fast Track designation
for CTP-543.
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 with the majority of patients initially having
symptoms by age 40. It is believed to equally affect 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) 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
(FDA) 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 ConcertConcert Pharmaceuticals is a clinical stage
biopharmaceutical company focused on applying its DCE Platform®
(deuterated chemical entity platform) to create novel medicines
designed to treat serious diseases and address unmet patient needs.
The Company’s approach starts with previously studied compounds,
including approved drugs, in which deuterium substitution has the
potential to enhance clinical safety, tolerability or efficacy.
Concert’s pipeline of innovative medicines currently
targets autoimmune 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 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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190122005193/en/
Justine E. Koenigsberg (Investors)Concert Pharmaceuticals,
Inc.(781) 674-5284ir@concertpharma.com
Kathryn Morris (media)The Yates Network(914)
204-6412kathryn@theyatesnetwork.com
Concert Pharmaceuticals (NASDAQ:CNCE)
Historical Stock Chart
From Feb 2024 to Mar 2024
Concert Pharmaceuticals (NASDAQ:CNCE)
Historical Stock Chart
From Mar 2023 to Mar 2024