"We are excited to initiate our Phase 2 trial with CTP-656 in
individuals with cystic fibrosis," said
The Phase 2 clinical trial is a randomized, parallel-group,
double-blind, placebo-controlled, clinical trial to evaluate the safety
and efficacy of CTP-656 in cystic fibrosis patients with gating
mutations. Patients enrolled in the 28-day study will receive one of
three doses (20 mg, 100 mg and 150 mg) of CTP-656 once-daily or placebo.
There will also be an open-label Kalydeco comparator arm in the trial.
Approximately 30-40 patients will be enrolled in the Phase 2 trial which
will be conducted in multiple centers in the
In Phase 1 clinical evaluations, CTP-656 provided superior key exposure parameters compared to Kalydeco. CTP-656 was well-tolerated and its safety profile was comparable to that of Kalydeco. No serious adverse events were reported in Phase 1 studies.
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
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including statements about 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 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.
Kalydeco is a registered trademark of Vertex Pharmaceuticals, Inc.
Justine E. Koenigsberg, 781-674-5284
The Yates Network
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