CTP-543 to Advance into Phase 2 in First Quarter of 2017
LEXINGTON, Mass.--(BUSINESS WIRE)--
Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the Phase 1
single and multiple ascending dose trial evaluating CTP-543 in healthy
volunteers has been completed. Concert is developing CTP-543 for the
treatment of alopecia areata, an autoimmune disorder in which the immune
system attacks hair follicles, resulting in patchy or complete hair
loss. CTP-543 is a deuterium-modified analog of ruxolitinib, a Janus
Kinase (JAK) inhibitor that is commercially available under the brand
name Jakafi® for the treatment of myelofibrosis and for polycythemia
vera. The Phase 1 program was designed to determine the safety,
tolerability and pharmacokinetics of CTP-543 as well as determine doses
for the planned Phase 2a clinical trial. Given the overall exposure and
safety parameters observed, the Company has selected four doses (4, 8,
12 and 16 mg BID) to explore in its Phase 2a trial. The Phase 2a
clinical trial is expected to begin in the first quarter of 2017.
"We are pleased with the pharmacokinetic profile emerging in our Phase 1
development with CTP-543," said James Cassella, Ph.D., Chief Development
Officer of Concert Pharmaceuticals. "There has been converging evidence
that JAK inhibition is important in promoting hair growth in individuals
with alopecia areata, representing a major advance in developing a new
treatment for this condition. We look forward to bringing CTP-543 into
its first efficacy study next year and believe we have the potential to
be the first to market with an FDA-approved oral treatment."
The single and multiple ascending dose trial enrolled a total of 77
healthy volunteers. The pharmacokinetic measurements showed increased
exposure with increasing doses. The half-life of CTP-543 was
approximately 3.3 hours, similar to that reported for non-deuterated
ruxolitinib1. CTP-543 was well-tolerated across all dose
groups and there were no serious adverse events reported in subjects who
The safety and exposure observed with 16 mg of CTP-543 twice daily
appeared comparable to the reported exposure of 20 mg ruxolitinib twice
daily. Published findings from an open-label clinical trial of 12
patients with alopecia areata conducted by investigators at Columbia
University demonstrated that 20 mg of ruxolitinib administered twice
daily resulted in significant hair regrowth in 75% of patients with
moderate to severe alopecia areata2.
The Company intends to present the Phase 1 findings at a medical
conference in 2017.
The Company's planned Phase 2a trial, which will enroll approximately
100 patients with moderate to severe alopecia areata, is a dose-ranging
trial with four active arms and a placebo comparator. The primary
outcome measure of the Phase 2a trial will utilize the severity
of alopecia tool (SALT) after 24 weeks of dosing. The trial will include
an extension arm where all patients enrolled in the study will receive
CTP-543 for an additional 28 weeks of dosing. The trial is expected to
commence in the first quarter of next year and topline primary outcome
data is expected by the end of 2017.
About CTP-543 and About Alopecia Areata
CTP-543 was discovered by applying Concert's deuterium chemistry
technology to modify ruxolitinib, which is commercially available under
the name Jakafi® in the United States for the treatment of myelofibrosis
and polycythemia vera. Ruxolitinib has been used to treat alopecia
areata in academic settings, including an investigator-sponsored
clinical trial, and has been shown 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 time3. 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.
In 2015, the FDA selected alopecia areata as one of eight new disease
areas that it will focus on under its Patient-Focused Drug Development
Initiative (PFDDI) meeting during fiscal year 2016-2017. The goal of the
PFDDI is to bring patient perspectives into an earlier stage of product
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 pulmonary diseases,
including cystic fibrosis, central nervous systems (CNS) disorders, as
well as autoimmune and inflammatory diseases. For more information
please visit www.concertpharma.com.
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
1 Shi et al, J Clin Pharmacol, 2011.
JCI Insight. 2016;1(15):e89790.
3 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.
is a registered trademark of Incyte Corporation.
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Concert Pharmaceuticals, Inc.
Justine Koenigsberg (investors),
Kathryn Morris (media), 845-635-9828
Source: Concert Pharmaceuticals, Inc.
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