Concert Pharmaceuticals Announces CTP-543 Positive Top-Line Phase 1 Results
14 Diciembre 2016 - 6:30AM
Business Wire
CTP-543 to Advance into Phase 2 in First
Quarter of 2017
Concert 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 received CTP-543.
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 development.
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 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 press release.
References:1 Shi et al, J Clin Pharmacol, 2011.2 JCI Insight.
2016;1(15):e89790. doi:10.1172/jci.insight.89790.3 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.Jakafi® is a registered
trademark of Incyte Corporation.
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version on businesswire.com: http://www.businesswire.com/news/home/20161214005181/en/
Concert Pharmaceuticals, Inc.Justine Koenigsberg (investors),
781-674-5284ir@concertpharma.comorThe Yates NetworkKathryn Morris
(media), 845-635-9828
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