Concert Pharmaceuticals Announces Issuance of U.S. Patent for its Lead Drug Candidate CTP-499

Concert Pharmaceuticals Announces Issuance of U.S. Patent for its Lead Drug Candidate CTP-499

InvestorsConcert Pharmaceuticals, Inc.Justine E. Koenigsberg, 781-674-5284orMediaThe Yates NetworkKathryn Morris, 845-635-9828

today announced that it has been granted U.S. Patent No. 8,263,601 which covers CTP-499, the company’s lead drug candidate in development for the treatment of diabetic nephropathy, a form of chronic kidney disease (CKD). This patent claims the composition of matter and pharmaceutical compositions of CTP-499, which is currently in Phase 2 clinical testing.

“We are very pleased to obtain this patent for our lead clinical candidate. This follows patent protection we recently received for several of our priority programs,” said Robert Silverman, Senior Vice President and General Counsel. “We have now used our DCE Platform to make a wide range of substantially differentiated agents which are covered by a worldwide patent estate that includes 26 issued U.S. patents.”

CTP-499 is a novel, potentially first-in-class treatment for diabetic nephropathy, possesses a pleiotropic mechanism of action with anti-inflammatory, anti-oxidant and anti-fibrotic properties. Its unique qualities may enable it to intervene in the pathophysiology of diabetic nephropathy, other forms of kidney disease and certain fibrotic indications.

CTP-499 was developed using Concert’s DCE Platform in which deuterium was incorporated at select positions of 1-((S)-5-hydroxyhexyl)-3,7-dimethylxanthine (HDX), an active metabolite of pentoxifylline. Deuterium incorporation enhances the metabolism profile of HDX, resulting in high and consistent plasma levels of both CTP-499 and active metabolites. CTP-499 is intended to be additive to the current standard of care, angiotensin converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) therapy, to prevent or slow progression of kidney damage in diabetic nephropathy and other types of chronic kidney disease.

A Phase 2 clinical trial is underway to evaluate the safety and efficacy of CTP-499 in patients with type 2 diabetes and chronic kidney disease.

The current pipeline for novel CKD treatments is limited and there is a need for more effective treatments to slow disease progression and prevent dialysis. Diabetic nephropathy is a frequent consequence of diabetes and is the leading cause of CKD and need for dialysis in the US. The current standard of care for CKD is treatment with blood pressure lowering agents that affect the renin-angiotensin system, including ACEi and 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.

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 renal disease, hematologic disorders, 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 Inc., the CoNCERT Pharmaceuticals Inc. Logo and DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.