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Concert Pharmaceuticals Presents Clinical Results Demonstrating
CTP-499 is Well-Tolerated in Patients with Stage 3 Chronic Kidney Disease
Results Presented at National Kidney Foundation 2012 Spring Clinical Meetings
May 11, 2012
Lexington, MA – Concert Pharmaceuticals, Inc. today announced results from its first clinical trial evaluating the safety and tolerability of CTP-499 in patients with chronic kidney disease. The Phase 1b clinical trial demonstrated that CTP-499 was well-tolerated in patients with stage 3 chronic kidney disease. The results of this study were presented in a poster this week at the National Kidney Foundation’s 2012 Spring Clinical Meeting in Washington, DC. CTP-499 is currently in Phase 2 clinical development in patients with diabetic nephropathy, the leading cause of chronic kidney disease (CKD) worldwide. The Phase 2 study was initiated in February 2012.
“We are encouraged by these Phase 1b results in patients with CKD which showed that CTP-499 was well-tolerated at doses that provide plasma levels of drug and active metabolites that we believe are desirable based on preclinical models,”said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “We look forward to completing our Phase 2 study in 2013 to evaluate the benefits of CTP-499 as a potential treatment for diabetic nephropathy to delay or prevent the need for dialysis and kidney transplantation.”
The Phase 1b clinical trial was a randomized, double-blind, placebo-controlled, multi-center study in patients with stage 3 chronic kidney disease (eGFR 30-59 mL/min/1.73m2). The primary objective of the study was to evaluate the safety and tolerability of CTP-499 controlled release tablets at a dose of 600 mg once-a-day for 2 weeks followed by a dose of 600 mg twice-a-day for 2 weeks. In addition, the pharmacokinetic profile on the first and last day of dosing was characterized.
A total of 25 patients with stage 3 chronic kidney disease were randomized to CTP-499 and 8 patients were randomized to placebo, with 23 and 8 patients completing the 4-week treatment period, respectively. All patients succeeded in titrating to 600 mg twice-a-day after 2 weeks of once-a-day dosing. All adverse events were mild in nature. No serious adverse events and no clinically significant laboratory abnormalities were reported, and there no discontinuations associated with CTP-499 treatment.
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. Its unique qualities may enable it to intervene in the pathophysiology of diabetic nephropathy, other forms of kidney disease and fibrotic indications.
CTP-499 was developed using Concert’s DCE Platform™ in which deuterium was incorporated at select positions in order to enhance the metabolic profile of the compound. In a clinical study reported last year, it was found that CTP-499 provides high levels of active species with the desired pleiotropic activities, in particular forming a substantial quantity of a metabolite which Concert scientists have identified as being pharmacologically relevant to chronic kidney disease. 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.
About Diabetic Nephropathy
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 with the need for dialysis. 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 antiviral disease, 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 www.concertpharma.com.
Concert Pharmaceuticals, the CoNCERT logo and the DCE Platform are trademarks of Concert Pharmaceuticals, Inc.
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