Nile announces results of CD-NP Phase 2 study in renally compromised ADHF patients

Nile Therapeutics, Inc. (Nasdaq: NLTX), a biopharmaceutical company focused on the development of novel therapeutics for cardiovascular disease, today announced results of its Phase 2 study evaluating its lead compound CD-NP in patients with acute decompensated heart failure (ADHF) and mild to moderate renal insufficiency. Study results demonstrated that multiple doses were characterized as well tolerated with favorable drug activity in this acute patient population.

The open-label, single-blind, placebo-controlled Phase 2 study included 77 patients who were randomized into six cohorts at one of four doses of CD-NP (1.25, 2.5, 3.75 and 5 ng/kg/min) or placebo. Two cohorts were enrolled at each of the 1.25 and 2.5 ng/kg/min dose levels.  Patients received study drug for up to 72 hours and were followed for 30 days.  The primary objective of the study was to assess the safety and tolerability of CD-NP in a renally compromised ADHF population, the intended population of the therapy.  Secondary endpoints included several assessments of drug activity.  

CD-NP infusion at 1.25, 2.5 and 3.75 ng/kg/min appeared to be well tolerated.  A dose-dependent effect on blood pressure was observed, with minimal or mild blood pressure reduction at 1.25 and 2.5 ng/kg/min, and moderate blood pressure reduction at 3.75 ng/kg/min.  Dose escalation was limited by significant blood pressure reduction at 5 ng/kg/min.  

Secondary and exploratory analyses demonstrated favorable effects of CD-NP on renal function, particularly at the 1.25 and 2.5 ng/kg/min doses. At these doses, CD-NP appeared to preserve or enhance renal function compared to placebo, as evidenced by favorable trends in several biomarkers correlated with kidney function, including creatinine and cystatin-c. Data will be presented at an upcoming cardiology conference.

"The data from this trial appear to indicate that we have identified active doses of CD-NP suitable for evaluation in a larger double-blind, placebo controlled study in acute heart failure patients," said James Young, MD, Professor and Dean of Medicine of the Cleveland Clinic and member of Nile Therapeutics Scientific Advisory Board. "Particularly interesting is CD-NP's apparent effect on kidney function, which would be a unique and clinically important benefit over the current standard of care."

"We are excited and encouraged by this data, particularly by the demonstration of activity in the intended patient population, supporting our belief that CD-NP has the potential to be a valuable new therapy for patients with cardiovascular and renal disease," said Joshua Kazam, Chief Executive Officer of Nile Therapeutics. "We look forward to the continued advancement of the CD-NP program."