Concert Pharmaceuticals, Inc. announced it has made significant progress advancing CTP-499, its novel anti-inflammatory, anti-oxidant, and anti-fibrotic agent for diabetic nephropathy and other forms of Chronic Kidney Disease, (CKD) into clinical development. Based on encouraging preclinical results and successful formulation assessment in healthy volunteers, Concert has initiated a phase I single ascending dose study of CTP-499 in healthy volunteers. CTP-499, invented using Concert’s DCE platform (Deuterated Chemical Entity platform), is a potential first-in-class treatment for Chronic Kidney Disease, a major and growing health problem.

“We are excited about the progress of CTP-499 and its potential to treat diabetic nephropathy and, more generally, chronic kidney disease,” said Roger Tung, PhD, president and chief executive officer of Concert Pharmaceuticals. “CKD is a progressive disease that has become a major medical concern associated with substantial health care costs running to tens of billions of dollars per year in the US. Preclinical evidence that CTP-499 may protect kidney function and slow disease progression is encouraging. We look forward to advancing CTP-499 in clinical testing to evaluate its benefits in the treatment of diabetic nephropathy and other forms of Chronic Kidney Disease.”

Diabetic nephropathy is associated with diabetes and is the leading cause of chronic kidney disease in the US. Current standard of care for kidney disease is treatment with blood pressure lowering agents that affect the renin-angiotensin cascade, including Angiotensin Converting Enzyme inhibitors (ACEi) and Angiotensin Receptor Blockers (ARBs). Despite the use of current treatments, many patients progress to renal failure. There is a critical need for agents with novel mechanisms that further delay or prevent the decline of kidney function.

CTP-499 is a deuterium containing new chemical entity which was derived from an active metabolite of a drug approved for a different indication. It possesses a pleiotropic mechanism of action with anti-inflammatory, anti-oxidant, and anti-fibrotic properties that are different from the current standard of care. CTP-499 is initially being developed for the treatment of type 2 diabetic nephropathy. Formulation studies, including an assessment of CTP-499 controlled release formulations in healthy subjects, have been completed. Based on its effectiveness in preclinical cellular and in vivo models of renal injury, including a diabetic nephropathy model, CTP-499 has the potential to protect kidney function and slow disease progression when added to existing therapy.

Deuterium is a safe, non-radioactive relative of hydrogen that can be isolated from sea water and has been used extensively in human metabolic and clinical studies. Since deuterium is an isotope of hydrogen, deuterium-containing compounds are expected to have similar pharmacological activity as their hydrogen analogues. However, because deuterium is heavier than hydrogen, it forms a stronger chemical bond to carbon. The stronger chemical bond obtained by selective deuterium modification may in certain cases substantially improve the drug’s metabolic properties, potentially resulting in better safety, tolerability and/or efficacy.

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 infectious disease, renal disease and CNS disorders, among others.