Concert Pharmaceuticals Presents Preclinical Results Demonstrating CTP-499, a Novel Treatment for Diabetic Kidney Disease, Ameliorated Renal Fibrosis and Inflammation In Vivo

Data Presented at Kidney Week 2012

SAN DIEGO--(BUSINESS WIRE)-- Concert Pharmaceuticals, Inc. today presented preclinical results which demonstrated CTP-499’s anti-fibrotic and anti-inflammatory properties in vivo. These findings were presented in a poster at the American Society of Nephrology’s (ASN) Kidney Week 2012. CTP-499 is currently in Phase 2 clinical development in patients with diabetic nephropathy, the leading cause of chronic kidney disease (CKD) worldwide.

“Kidney disease is a complex, multifactorial disease,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “Previous preclinical data showed that CTP-499 inhibits key pathological processes including pro-fibrotic gene expression and inflammatory cytokines. In the current studies, we demonstrated efficacy in an in vivo model of fibrosis. We look forward to the results from our ongoing Phase 2 clinical study to further understand CTP-499’s effectiveness as a treatment for diabetic kidney disease.”

The preclinical data showed CTP-499’s anti-fibrotic and anti-inflammatory properties in the Unilateral Ureteral Obstruction (UUO) rat model, a well-described in vivo model of renal fibrosis. In addition, it was found that the IL-13 pathway, a key mediator of tissue fibrosis, was modulated by CTP-499. Data highlights include:

  • CTP-499 significantly reduced kidney fibrosis, the key pathological mechanism in diabetic nephropathy that leads to end-stage renal disease.
  • CTP-499 protected against kidney tubule apoptosis, a characteristic pathophysiological process in kidney disease.
  • Gene expression analysis revealed that IL-13 and its downstream targets were significantly down-regulated in kidneys from rats dosed with CTP-499.
  • CTP-499 inhibited the expression of pro-inflammatory cytokines including TNF-α, MCP-1, and IL-6.
  • In vitro CTP-499 prevented the formation of matrix-forming fibroblasts from kidney proximal tubule cells, a process which may contribute to renal fibrosis in vivo.

About CTP-499

CTP-499, a novel, potentially first-in-class treatment for diabetic nephropathy, possesses multiple pharmacological properties of inhibiting inflammation, oxidation and fibrosis. Its unique qualities may enable it to intervene in the pathophysiology of diabetic nephropathy as well as 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.

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 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.

Concert Pharmaceuticals, Inc.
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Source: Concert Pharmaceuticals