Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced new results from Part 1 of its multiple ascending dose Phase 1 clinical trial in which a single-dose tablet formulation of CTP-656 was compared to the commercial tablet formulation of Kalydeco®, the current standard of care for cystic fibrosis patients with gating mutations. The results from this study confirmed CTP-656's superior pharmacokinetic profile to Kalydeco as was previously reported from Concert's single ascending dose trial, which demonstrated favorable pharmacokinetic properties with an aqueous suspension. Concert is using this tablet formulation of CTP-656 in its ongoing multiple ascending dose Phase 1 clinical trial and intends to use the same formulation in a Phase 2 clinical trial. The Company is evaluating CTP-656, deuterium-modified ivacaftor, as a novel, potentially disease-modifying treatment for cystic fibrosis as monotherapy as well as in combination with other CFTR modulators.

"We are pleased that the tablet dosage formulation of CTP-656 performed well and confirmed the superior pharmacokinetic profile compared to standard of care. We believe this formulation is suitable for late stage clinical development," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "These CTP-656 results are in line with our goal of providing superior benefits to patients including a more convenient, once-daily medicine that has the potential for improved efficacy."

Concert has completed Part 1 of a Phase 1 multiple ascending dose trial to assess CTP-656 in healthy volunteers. Part 1 of the trial, initiated in November 2015, compared a single dose of 150 mg of CTP-656 in a tablet formulation against a single dose 150 mg commercial tablet of Kalydeco in eight subjects, following a high-fat meal. The results confirmed CTP-656's superior pharmacokinetic profile compared to Kalydeco, including a longer half-life, reduced rate of clearance, substantially increased exposure and greater plasma levels at 24 hours. CTP-656's half-life of approximately 15 hours supports the potential for once-daily dosing.

With both the solid dose and suspension formulation, the overall metabolite exposure profile of CTP-656 differed from that of Kalydeco. For CTP-656, the greatest exposure in plasma was to parent drug, which is the most active species, whereas with Kalydeco the greatest plasma exposure was to a less-active metabolite.

Part 2 of the multiple ascending dose Phase 1 trial will assess three doses of CTP-656, ranging between 75 mg and 225 mg, to be administered daily for seven days, compared to placebo. Top-line results are expected in the second quarter of 2016.

Conference Call and Webcast

The Company will host a conference call and webcast tomorrow, March 1, 2016 at 8:30 a.m. ET to discuss full year 2015 financial results and the CTP-656 solid dose phase 1 results. 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 may be accessed in the Investors section of the company's website at 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 three months.

About CTP-656 and Cystic Fibrosis

CTP-656 is a novel CFTR potentiator that may enable once-daily dosing which was developed by applying deuterium chemistry to modify ivacaftor. Concert is initially developing CTP-656 as a potential treatment for cystic fibrosis as monotherapy in certain 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, impacting lungs, pancreas, intestines, testes, sweat ducts, and other organs. 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, and inflammatory diseases. 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 the clinical development and success 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.

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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