Company to Host Conference Call and Webcast Today, January 17,
2017 at 8:30 A.M. ET
LEXINGTON, Mass.--(BUSINESS WIRE)--
Pharmaceuticals, Inc. (NASDAQ: CNCE) today provided further details
on the development plan in the U.S. and Europe for CTP-656, a next
generation CFTR potentiator being developed for the treatment of cystic
In the U.S., Concert initiated a placebo-controlled Phase 2 trial in
December 2016 to evaluate CTP-656 in cystic fibrosis patients with
gating mutations. Subsequent to the initiation of the study, the U.S.
Food and Drug Administration (FDA) informed Concert that, in order to
support dose selection for Phase 3, an adequate washout period, in which
Kalydeco® (ivacaftor) treatment is withheld, would be required in
addition to a placebo-control. The Company intends to further discuss
the additional feedback with FDA. The ongoing Phase 2 trial is being
conducted at multiple U.S. study sites within the Cystic Fibrosis
Foundation's Therapeutic Development Network with topline data expected
by year-end 2017.
In Europe, Concert intends to initiate an open-label Phase 2 trial in
the second quarter of 2017. The trial is expected to enroll 14 cystic
fibrosis patients with gating mutations stable on Kalydeco. Patients
will be switched to one dose of CTP-656 for two weeks followed by a
switch to a higher dose of CTP-656 for an additional two weeks.
Subsequently, patients will resume treatment with Kalydeco. The primary
analysis will be for non-inferiority for sweat chloride compared to the
Kalydeco baseline. Topline data from the European Phase 2 study of
CTP-656 is expected by year-end 2017.
"We are entering a new stage in which the next wave of drug candidates
for cystic fibrosis - beyond today's standard of care - are advancing
into later-stage clinical development. We look forward to further
discussions with FDA on the development of CTP-656 and believe that our
ongoing U.S. trial together with the planned European study will yield
data to support the progression of CTP-656," said Roger Tung, Ph.D.,
President and Chief Executive Officer of Concert Pharmaceuticals. "We
look forward to the continued advancement of CTP-656 for both
monotherapy and potential future combination therapy."
Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m.
EST to discuss CTP-656. To access the conference call, please dial (855)
354-1855 (U.S. and Canada) or (484) 365-2865 (International) five
minutes prior to the start time.
A live webcast of Concert's presentation may be accessed in the
Investors section of the Company's website at www.concertpharma.com.
Please log on to the Concert website approximately 15 minutes prior to
the scheduled webcast to ensure adequate time for any software downloads
that may be required. A replay of the webcast will be available on
Concert's website for two weeks.
About CTP-656 and Cystic Fibrosis
CTP-656 is a novel CFTR potentiator that may offer next generation,
once-daily dosing and was developed by Concert's novel application of
deuterium chemistry to modify ivacaftor. Ivacaftor is marketed by Vertex
Pharmaceuticals under the brand name Kalydeco. Concert is initially
developing CTP-656 as a potential monotherapy treatment for cystic
fibrosis due to gating mutations of the gene that encodes for cystic
fibrosis transmembrane conductance regulator (CFTR), a protein, which
regulates components of sweat, mucus clearance and digestion. The
Company also intends to enable potentially more effective combinations
to treat other mutations, including homozygous F508del, by partnering
with other potentially complementary CFTR modulators.
Cystic fibrosis is a life-threatening, hereditary genetic disease that
has systemic effects and can cause significantly reduced lung and
digestive system function. According to the Cystic Fibrosis Foundation,
an estimated 70,000 people worldwide have cystic fibrosis.
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 about the clinical development and outcomes of
CTP-656 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, 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, our ability to reach agreement with the FDA
regarding an adequate washout period to support dose selection for Phase
3 and our ability to recruit patients for any such trial, 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.
Concert Pharmaceuticals Inc., the Concert Pharmaceuticals Inc. logo and
DCE Platform are registered trademarks
of Concert Pharmaceuticals,
Kalydeco is a registered trademark of Vertex Pharmaceuticals,
View source version on businesswire.com: http://www.businesswire.com/news/home/20170117005339/en/
Concert Pharmaceuticals, Inc.
Justine E. Koenigsberg, 781-674-5284
Barbara Yates, 781-258-6153
Source: Concert Pharmaceuticals, Inc.
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