CTP-499: Phase II data

A double-blind, U.S. Phase II trial in 182 patients with Type II diabetic nephropathy showed that twice-daily 600 mg oral CTP-499 missed the primary endpoint of improving UACR from baseline to week 24 vs. placebo. At week 48, patients treated with CTP-499 had a smaller increase in UACR from baseline compared to placebo (24 vs. 223 mg/g, p=0.097), which Concert said may indicate stabilization of UACR in patients treated with CTP-499. On the secondary endpoint of mean serum creatinine levels, CTP-499 led to an increase of 0.13 mg/dL vs. an increase of 0.21 mg/dL for placebo at week 48 (p=0.057), which Concert said may indicate a slower decline in kidney function in patients treated with CTP-499. Additionally, 1 patient in the CTP-499 arm had a >=50% increase in serum creatinine levels after 48 weeks compared to 6 patients in the placebo arm (p=0.026).

At week 24, there were no significant differences between treatment groups in levels of the fibrotic biomarkers urinary fibronectin and plasma collagen IV. At week 48, CTP-499 led to a significant 52% reduction in urinary fibronectin and a significant 18% reduction in plasma collagen IV vs. placebo (p=0.0081 and p=0.022, respectively). ...