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Concert Pharmaceuticals Initiates Phase 1 Trial Evaluating Deuterated Ivacaftor for Cystic Fibrosis

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Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today announced that is has initiated its Phase 1 clinical program for deuterium-modified ivacaftor, a novel, potentially disease-modifying treatment for cystic fibrosis. Ivacaftor is commercially available under the name Kalydeco(R). The first Phase 1 trial will be a crossover study to compare two proprietary deuterium-modified compounds in order to select one for further clinical evaluation. The Phase 1 program also encompasses single- and multiple-ascending doses to further evaluate the safety and pharmacokinetics of the selected candidate.

“This new program exemplifies Concert’s platform capability of advancing deuterium-modified versions of marketed or late-stage development compounds into Phase 1 testing in an expedited manner. Our goal with this Phase 1 evaluation is to efficiently evaluate the early clinical properties of two novel deuterium-modified analogs of ivacaftor in order to select the one with the best potential to benefit patients,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “Treatments for cystic fibrosis are evolving rapidly with multiple exploratory agents and mechanisms for CFTR modulation that we believe may be beneficial, and we are excited to develop our potentiator both as a single agent and to enable new combinations to expand patient options.”

The Phase 1 program is expected to enroll approximately 45 healthy volunteers. Dosing has been initiated in the first Phase 1 trial which is designed to assess single doses of two deuterium-modified compounds, each of which has demonstrated greater metabolic stability relative to Kalydeco in preclinical testing. Based on the results of this initial crossover study, Concert will select one compound for advancement into the single ascending dose portion of the Phase 1 program, which will assess single ascending doses of the selected compound compared to a single dose of Kalydeco. The final phase of the Phase 1 program will assess multiple ascending doses of the selected compound compared to placebo and is expected to begin in the second half of 2015. The Company expects to report top-line data upon completion of the multiple dose Phase 1 program. Additional information is available online at

“We are encouraged by our preclinical findings with deuterium-modified ivacaftor compounds which support the advancement of this program into clinical evaluation,” said Scott Harbeson, Ph.D., Vice President of Translational Research and Program Director of Cystic Fibrosis at Concert. “We look forward to executing our Phase 1 clinical development plan, as we believe that deuterium modification has the potential to offer an important new treatment option for cystic fibrosis patients.”

About Concert

Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform(R) (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 of deuterated-ivacaftor 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 Annual Report on Form 10-K 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(R) is a registered trademark of Vertex Pharmaceuticals, Inc.

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