During the meeting, the FDA indicated general agreement with Concert’s proposed Phase 3 development plan providing flexibility to conduct either two clinical trials with CTP-499 or a single trial evaluating two doses of CTP-499 compared to placebo. A reduction in the rate of progression of renal disease, measured by a time-to-event analysis of the composite of increases in serum creatinine (greater than or equal to 50%) or end stage renal disease, versus placebo-treated patients, was agreed to be an acceptable Phase 3 endpoint to support the filing of a potential New Drug Application (NDA). The Company currently plans to submit a request for a Special Protocol Assessment (SPA) to the FDA later this year.
“We are very pleased with the outcome of the End-of-Phase 2 meeting and the feedback from our discussion with the FDA,” stated Roger Tung, Ph.D., President and Chief Executive Officer. “We believe the results from the Phase 2 clinical trial of CTP-499 in diabetic nephropathy are encouraging and appreciate the valuable guidance that the FDA has provided us regarding the design and conduct of a Phase 3 registration program.”
About Diabetic Nephropathy
Diabetic nephropathy, or type 2 diabetic kidney disease, a condition in which kidney function is progressively lost, can result in the need for dialysis or kidney transplantation. It is associated with increased morbidity and mortality, and is the leading cause of end-stage renal disease. The current standard of care for diabetic nephropathy is treatment with ACEis or ARBs, which are blood pressure lowering agents that affect the renin-angiotensin system. Despite the availability of these treatments, many patients progress to end stage renal failure and require dialysis or kidney transplants. Approximately 40% of patients with a history of type 2 diabetes are believed to have diabetic nephropathy.
CTP-499 is a novel, deuterium-containing, oral anti-fibrotic agent. It is a multi-subtype selective PDE inhibitor that is being developed to slow the progression of diabetic nephropathy in patients with macroalbuminuria. CTP-499 is a deuterated analog of 1-(S)-5-hydroxyhexyl-3,7-dimethylxanthine, or HDX, an active metabolite of pentoxifylline. It is being developed as an additive treatment to angiotensin modulation with an ACEi or ARB. In April 2014, Concert announced 48-week results of its Phase 2 clinical trial of CTP-499 in patients with diabetic nephropathy which were presented in a late-breaking session at the National Kidney Foundation 2014 Spring Clinical Meeting.
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to create novel small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, renal disease, inflammation and cancer.
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including statements about the potential effectiveness of CTP-499 in treating diabetic nephropathy, our plans and timelines for the clinical development and regulatory approval of CTP-499 and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the "Risk Factors" section of our Annual Report on Form 10-Q filed with the Securities and Exchange Commission on May 14, 2014 and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.
SOURCE: Concert Pharmaceuticals, Inc.