Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that it has initiated a Phase 1 multiple ascending dose clinical trial with CTP-656. CTP-656 (deuterium-modified ivacaftor) is a novel, potentially disease-modifying treatment for cystic fibrosis for use as monotherapy as well as in combination with other CFTR modulators. Ivacaftor is commercially available under the name Kalydeco®.

"CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community," said James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals. "We believe the development of CTP-656 as a once-daily dose could help address adherence issues documented with the current standard of care by offering a simplified treatment option."

The multiple ascending dose Phase 1 study will be conducted in two parts and enroll 38 healthy volunteers to assess safety, tolerability and pharmacokinetics of CTP-656 in a tablet formulation. The first part will assess a single dose pharmacokinetic comparison of 150 mg of CTP-656 versus 150 mg of Kalydeco. The second part will assess three doses of CTP-656, starting at 75 mg and up to 300 mg daily for seven days compared to placebo. Additional information is available on

Concert expects to report top-line results from the Phase 1 multiple ascending dose study in the first half of 2016. The Company intends to initiate a single Phase 2 trial in patients with cystic fibrosis associated with gating or minimal function CFTR mutations in the second half of 2016.

About CTP-656 and Cystic Fibrosis

CTP-656 is a novel potentiator that may enable once-daily dosing that was developed by applying deuterium chemistry to modify ivacaftor. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis gating and minimal-function mutations of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein which regulates components of sweat, mucus clearance and digestion. 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 small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, genetic diseases, renal disease, inflammatory diseases and cancer. For more information, please visit

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 success of CTP-656, or potential dosing options for 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.

Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.

Kalydeco is a registered trademark of Vertex Pharmaceuticals, Inc.

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