Nile Therapeutics, Inc. (Nasdaq: NLTX), a biopharmaceutical company focused on the development of novel therapeutics for cardiovascular disease, 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."
About Heart Failure
Heart failure is the fastest-growing clinical cardiac disease in the U.S. according to the American Heart Association, affecting over 5 million Americans. Over 1 million patients in the U.S. each year are hospitalized with ADHF, an acute exacerbation of heart failure. This hospitalization rate is almost double the rate seen 15 years ago, and is the most frequent cause of hospital admission in the U.S. for patients older than 65 years, generating annual inpatient costs of more than $33 billion.
Safe Harbor Paragraph for Forward-Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding the timing, progress and anticipated results of the clinical development, regulatory processes, clinical trial and data analysis timelines, anticipated benefits of CD-NP, Nile's strategy, future operations, outlook, milestones, the timing and success of Nile's product development, future financial position, future financial results, plans and objectives of management are forward-looking statements. Nile may not actually achieve these plans, intentions or expectations and Nile cautions investors not to place undue reliance on Nile's forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements Nile makes. Various important factors that could cause actual results or events to differ materially from the forward-looking statements that Nile makes include Nile's need to raise additional capital to fund its product development programs to completion, Nile's reliance on third-party researchers to develop its product candidates, and its lack of experience in developing and commercializing pharmaceutical products. Additional risks are described in greater detail in the reports Nile files with Securities and Exchange Commission, including those described under the caption "Risk Factors" in Item 1A of its Annual Report on Form 10-K for the year ended December 31, 2009 filed with the Securities and Exchange Commission on March 3, 2010. Nile is providing this information as of the date of this press release and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise.
Source: Nile Therapeutics, Inc