LEXINGTON, Mass.–(BUSINESS WIRE)–$CNCE #CNCE—Concert
Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that it has
completed patient enrollment of an open label clinical trial to evaluate
once-daily compared to twice-daily oral dosing of CTP-543 in patients
with alopecia areata. The trial is designed to inform on the optimal
dosing regimen for CTP-543 for future clinical trials. Topline data from
the dose regimen trial is expected in the fourth quarter of 2019.
“This dose regimen trial helps us understand the safety and efficacy of
CTP-543 when dosed once daily and allows us to set our dosing strategy
for future late-stage trials. We are grateful to the patients who
participated in this and other trials, as we pursue a new treatment for
alopecia areata, a disease that currently has no FDA-approved
treatments,” said James V. Cassella, Ph.D., Chief Development Officer of
Concert Pharmaceuticals.
The open label trial (NCT03811912) is a randomized, multi-center study
to evaluate the efficacy and tolerability of once-daily versus
twice-daily dosing of CTP-543, in 57 adult patients with chronic,
moderate-to-severe alopecia areata. Patients in the trial were
randomized to receive CTP-543 either 8 mg twice-daily or 16 mg
once-daily over a 24 week treatment period. The trial will measure the
relative change in Severity of Alopecia Tool (SALT) score between Week
24 and baseline. All patients who complete 24 weeks of treatment will be
eligible to enroll into an extension study of CTP-543.
A second open label trial (NCT03941548) to evaluate once-daily compared
to twice-daily oral dosing was initiated in May 2019 and is currently
enrolling patients. Patients in the trial will be randomized to receive
either 12 mg twice-daily or 24 mg once-daily of CTP-543 over a 24 week
treatment period. All patients who complete 24 weeks of treatment will
be eligible to enroll into an extension study of CTP-543.
Additional information about CTP-543 clinical trials are available on www.clinicaltrials.gov.
About CTP-543 Interim Phase 2 Results
During the late-breaking clinical trials session at the American Academy
of Dermatology (AAD) Annual Meeting on March 2, 2019, the Company
presented interim results from an ongoing Phase 2 trial. At 24 weeks,
patients treated with an 8 mg twice-daily dose of CTP-543 met the
primary efficacy endpoint with a statistically significant difference
compared to placebo (p <0.001). The primary outcome measure was the
proportion of patients with a ≥ 50% relative reduction in their overall
SALT score between Week 24 and baseline. As reported, CTP-543 was
generally well tolerated and there were no serious adverse events
reported. Dosing in an additional cohort receiving a 12 mg twice-daily
dose of CTP-543 compared to placebo in the Phase 2 trial is currently
ongoing. Additional information about the CTP-543 Phase 2 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 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 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.
All other trademarks are those of their respective owners.
Contacts
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