Concert Pharmaceuticals, Inc. Presents Phase 1 Data for CTP-499 at American Society of Nephrology Kidney Week Meeting

Published: Nov 14, 2011

LEXINGTON, Mass.--(BUSINESS WIRE)-- Concert Pharmaceuticals, Inc. today announced data from a Phase 1 clinical trial of CTP-499, a novel agent for the potential treatment of diabetic nephropathy. The results demonstrated that a controlled-release formulation of CTP-499 was well-tolerated at single doses up to and including 1800 mg. The company presented these findings during a poster session at the American Society of Nephrology’s Kidney Week Meeting in Philadelphia, Pennsylvania. Concert expects to initiate a Phase 2 clinical trial of CTP-499 in patients with diabetic nephropathy during the first half of 2012.

“We believe that the properties of CTP-499 and its metabolites may result in favorable biologic activity as an anti-inflammatory, anti-oxidant and anti-fibrotic agent in chronic kidney disease,” said James Shipley, M.D., Chief Medical Officer of Concert Pharmaceuticals. “We look forward to advancing CTP-499 into a proof-of-concept study in patients with type 2 diabetes and renal impairment.”

The Phase 1 clinical trial was a randomized, double-blind, placebo-controlled, single ascending-dose study in 38 healthy volunteers. The primary objective of the study was to evaluate the safety, tolerability and pharmacokinetics of a controlled-release formulation of CTP-499 given as a single dose to four successive cohorts of 8 healthy subjects each (600 mg, 1200 mg, 1800 mg, and 2400 mg). For comparison purposes, six additional healthy subjects received a single dose of 400 mg of CTP-499 as an immediate release formulation. All adverse events were mild in nature and no serious adverse events were reported.

Previously, Concert conducted a Phase 1 study in 16 healthy volunteers to evaluate various formulations of CTP-499. The study supported the selection of a controlled-release formulation for the single ascending dose study and subsequent clinical development. CTP-499 will be dosed 600 mg twice daily in the Phase 2 proof-of-concept study.

The clinical studies were undertaken following promising results in the preclinical evaluation of CTP-499. In a series of in vitro experiments, CTP-499 exhibited anti-fibrotic, anti-inflammatory and anti-oxidative properties associated with inter-related pathological mechanisms that may underlie chronic kidney disease. In the streptozotocin (STZ) in vivo preclinical model of diabetic nephropathy, treatment with CTP-499 was protective against model-induced kidney pathologies and it reduced the expression of inflammatory cytokines and MMP-2. These results suggest CTP-499 can impact these key parameters of renal dysfunction, and support the potential of CTP-499 to treat diabetic nephropathy.

About CTP-499

CTP-499 is an analog of 1-((S)-5-hydroxyhexyl)-3,7-dimethylxanthine (HDX), an active metabolite of pentoxifylline. CTP-499, a potential first-in-class treatment for diabetic nephropathy, possesses a pleiotropic mechanism of action with anti-inflammatory, anti-oxidant and anti-fibrotic properties that are different from the current standard-of-care, including angiotensin converting enzyme inhibitors (ACEi) and angiotensin receptor blockers (ARBs). CTP-499 was developed using Concert’s DCE Platform™ to provide a new chemical entity with an improved pharmacokinetic profile. Concert expects to evaluate the potential of CTP-499 to protect kidney function and slow disease progression when added to existing standard-of-care therapy.

About Diabetic Nephropathy

Diabetic nephropathy is a frequent consequence of diabetes and is the leading cause of chronic kidney disease (CKD) and need for dialysis in the US. Current standard of care for CKD is treatment with blood pressure lowering agents that affect the renin-angiotensin system, including Angiotensin Converting Enzyme inhibitors (ACEi) and Angiotensin Receptor Blockers (ARBs). Despite the availability of these treatments, many patients progress to renal failure. According to the US Renal Data System, the number of end-stage renal failure patients in the US doubled between 1994 and 2008. There is a critical need for a new agent with a novel mechanism that delays or prevents the decline of kidney function and eventual need for dialysis.

About Deuterium Modification

Concert Pharmaceuticals specializes in the use of precision deuterium chemistry to create new chemical entities (NCEs) with unique properties based on known, pharmacologically active compounds. By selectively replacing one or more hydrogen atoms with deuterium, a stable and non-radioactive isotope of hydrogen, the company has been able to effect significant changes to the absorption, distribution, metabolism, or excretion (ADME) profile of a number of compounds with the potential for improvements in safety, tolerability, and/or efficacy.

About Concert

Concert Pharmaceuticals is a clinical stage biotechnology company focused on applying the company’s DCE Platform™ (deuterated chemical entity platform) to create novel and differentiated small molecule drugs. Concert’s approach leverages decades of pharmaceutical and clinical experience to reduce the time, risk and expense needed to create important new medicines. The company has a broad research pipeline encompassing many therapeutic areas including antiviral disease, renal disease, and CNS disorders, among others. Founded in 2006, Concert has raised more than $110 million of venture and institutional capital. For more information on Concert Pharmaceuticals, please visit

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

..Contact:. .Concert Pharmaceuticals, Inc. (Investors) Justine E. Koenigsberg, 781-674-5284 or The Yates Network (Media)

Kathryn Morris, 845-635-9828

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