Concert Pharmaceuticals Receives FDA Orphan Drug Designation for CTP-656 for the Treatment of Cystic Fibrosis
January 20 2017 - 8:30AM
Business Wire
Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced
that the U.S. Food and Drug Administration (FDA) has granted orphan
drug designation for CTP-656, Concert’s next generation CFTR
potentiator being developed for the treatment of cystic fibrosis.
In December 2016, Concert initiated a Phase 2 trial in the U.S.
evaluating CTP-656 in cystic fibrosis patients with gating
mutations. Topline results from the Phase 2 trial are expected by
year-end 2017.
“Receiving orphan drug designation is an important regulatory
milestone, and we are pleased that CTP-656 for cystic fibrosis has
been granted this status,” said Roger Tung, Ph.D., President and
Chief Executive Officer of Concert Pharmaceuticals. “We are
developing CTP-656 to potentially offer advantages over standard of
care, and our team is committed to advancing the clinical
development program to address the unmet needs of individuals with
cystic fibrosis.”
The Orphan Drug Act provides incentives for companies to develop
products for rare diseases affecting fewer than 200,000 people in
the United States. Incentives may include tax credits related to
clinical trial expenses, an exemption from the FDA user
fee, FDA assistance in clinical trial design and potential
market exclusivity for seven years following approval.
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.
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
clinical development 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:
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, whether orphan drug status will be
granted 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, Inc.
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version on businesswire.com: http://www.businesswire.com/news/home/20170120005277/en/
Concert Pharmaceuticals, Inc.Justine E. Koenigsberg (Investors),
781-674-5284ir@concertpharma.comorThe Yates NetworkKathryn Morris
(Media), 845-635-9828
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