Concert Pharmaceuticals Presents Positive Data from Multiple Dose Phase 1 Clinical Trial of CTP-354, Lead Candidate for the Treatment of Spasticity

– No sedation or ataxia observed and treatment was well tolerated
– Company plans to initiate a Phase 2 trial this year
– Data presented at the American Neurological Association Annual Meeting

LEXINGTON, Mass.–(Oct 13, 2014)– Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) announced today Phase 1 data of CTP-354, a novel, potentially first-in-class, non-sedating, once-daily oral treatment for spasticity. In this multiple ascending dose trial, no sedation or ataxia was observed with CTP-354 and the drug was generally well tolerated across all dose cohorts. The Company will be presenting these findings today during a poster session at the American Neurological Association’s Annual Meeting in Baltimore, Maryland. Concert expects to initiate a Phase 2 clinical trial evaluating CTP-354 in patients with spasticity associated with spinal cord injury by the end of 2014.

“We are very pleased to have completed our clinical evaluation of CTP-354 in this Phase 1 trial. We remain on track to advance the program into Phase 2 testing later this year, initially targeting spasticity in patients with spinal cord injury followed by the start of a Phase 2 trial in multiple sclerosis patients in early 2015,” stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals.

Dr. Tung added, “CTP-354 was designed to avoid dose-limiting sedation and ataxia, which currently limit the broader use of other GABAA receptor modulators such as benzodiazepines. This trial provides evidence that CTP-354 can achieve high plasma levels without causing those side effects. In addition to being generally well tolerated in our Phase 1 clinical trials, CTP-354 also demonstrated highly favorable pharmacokinetics with low variability, dose-proportional exposure, a long half-life in the body and high levels of GABAA receptor occupancy. Taken together, we believe these results support once-daily dosing, which would provide a substantial improvement over the three-times-daily dosing required by current standard-of-care oral spasticity medicines.”

The Phase 1 multiple ascending dose clinical trial was a randomized, double-blind, placebo-controlled study in 30 healthy volunteers. The primary objective of the trial was to evaluate the safety, tolerability and pharmacokinetics of 10-day repeat dosing of 2 mg, 6 mg and 12 mg of CTP-354. Clinical highlights include:

• Safety. CTP-354 was generally well tolerated. There were no serious adverse events and no treatment discontinuations. The most common adverse events were dose-related mild and moderate dizziness and somnolence (drowsiness). No sedation or ataxia was observed at the doses evaluated.

• Pharmacokinetics. Across all doses, plasma half-life was approximately 20 hours at steady state. CTP-354 exposure was generally dose-proportional across daily doses ranging from 2 mg to 12 mg.

• Food Effect. Administration of CTP-354 under both fed and fasted conditions provided similar exposure, indicating that it can be dosed without regard to meals.

The Company previously reported positive results from both a Phase 1 single ascending dose trial and a Phase 1 brain imaging trial as measured by positron emission tomography, or PET imaging. The long half-life and pharmacokinetic profile observed in the single ascending dose support once-daily dosing of CTP-354. In addition, CTP-354 provided high and sustained brain GABAA receptor occupancy levels in the brain imaging trial in both single and repeat doses. A copy of the poster is available online at:

Based on the results of the Phase 1 trials, Concert intends to advance CTP-354 into Phase 2 clinical evaluation later this year. The Phase 2 program is projected to include two trials: one evaluating spasticity associated with spinal cord injury and one evaluating spasticity associated with multiple sclerosis.

About CTP-354
CTP-354 is a novel, potentially first-in-class, non-sedating, once-daily oral treatment for spasticity. Concert is initially developing CTP-354 for use in patients with spinal cord injury and in patients with multiple sclerosis to address significant unmet medical needs. CTP-354 is a subtype-selective GABAA receptor modulator. GABAA receptors are found in the nervous system and, when activated, reduce the transmission of certain nerve signals. Several classes of widely used drugs target GABAA receptors, including benzodiazepines, but do not have the receptor subtype selectivity of CTP-354.

About Spasticity
Spasticity is a chronic condition characterized by involuntary tightness, stiffness or contraction of muscles that occurs in patients who have damage to the brain and/or spinal cord. Spasticity can result from a wide range of disorders, including multiple sclerosis, spinal cord injury, cerebral palsy, amyotrophic lateral sclerosis, stroke and hereditary spastic paraplegia. Symptoms can range from mild muscle tightness to more severe symptoms, including crippling and painful inability to move limbs that can result in disability and diminished quality of life. The American Association of Neurologic Surgeons estimated in 2006 that there were 12 million patients suffering from spasticity worldwide.

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 or efficacy. The company is developing a broad pipeline targeting CNS disorders, renal disease, inflammation 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 the safety or efficacy of CTP-354 or our plans and timelines for the clinical development of CTP-354 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, 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.

Concert Pharmaceuticals, Inc.
Justine Koenigsberg, 781-674-5284
The Yates Network
Kathryn Morris, 845-635-9828