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Dear Shareholders,
As we enter the new year, I am very pleased to look back at 2008 and the significant progress in the development of our lead drug, CD-NP, for the treatment of heart failure. I am also very aware of the macroeconomic turmoil which has had a negative affect on everyone. At Nile Therapeutics, we focus internally on development activities while maintaining a pragmatic understanding of the broader financial conditions which inform our strategy.
During 2008, we built on the foundation of our 2007 achievements. Our primary focus was on the clinical development of CD-NP. We are executing a clearly designed clinical development plan for CD-NP, which will allow us to initiate our Phase 2b proof-of-concept study in 2009. In 2008, as planned, CD-NP entered and completed its Phase 1b open label dose escalating safety and pharmacodynamic study in stable heart failure patients. The objectives of the Phase 1b study were to establish the maximum tolerated dose (MTD) and to assess the pharmacologic effects of CD-NP on blood pressure and renal function in heart failure patients. The results of this study showed:
- CD-NP was well tolerated at doses of up to 20 ng/kg/min
- Blood pressure effects were dose dependent and well characterized
- CD-NP demonstrated diuretic effects comparable to furosemide
- CD-NP produced statistically significant changes on biomarkers consistent with enhanced renal function
In parallel to the Phase 1b study, we initiated a second Phase 2a multi-center, open label, dose escalating hemodynamic study in stabilized acute heart failure patients. The objective of the Phase 2a study is to assess hemodynamic and renal effects of CD-NP. We announced preliminary positive data from the Phase 2a study in October. The interim data showed the following:
- Statistically significant reduction in pulmonary capillary wedge pressure
- Statistically significant increase in diuresis
- Trend toward reduction in right atrial pressure
- Trend toward increase in cardiac output
- No symptomatic hypotension
- No observed change in renal function
We expect to complete enrollment in the Phase 2a study and announce the full study results in early 2009.
These positive results and insights learned to date from these two studies are supportive of further study to fully validate proof-of-concept of CD-NP in a Phase 2b clinical trial planned to begin in 2009.
Also in 2008, we in-licensed CU-NP, a rationally designed natriuretic peptide. CU-NP was designed by researchers to combine the favorable hemodynamic venodilating effects of CNP generated via NPR-B receptor agonism, with the beneficial renal effects of URO generated via NPR-A receptor agonism. We are proceeding with the preclinical development of CU-NP.
We intend to continue to aggressively pursue opportunities to present our data at scientific conferences and through publication and one-on-one meetings. In 2008, data relating to Nile compounds was presented at the American College of Cardiology (ACC), European Society of Cardiology (ESC), Heart Failure Society of America (HFSA) and the American Heart Association (AHA).
From a scientific perspective, we have made clear progress in our development efforts. However, from a business perspective, our progress is viewed though a broader economic lens and against a backdrop of current events. At Nile we are aware of this reality and adjust our strategy and tactics accordingly. This translates into developing a focus on core value creating activities such as CD-NP development; rededicating ourselves to the most efficient and cost effective operations possible; and conserving resources.
Developing new drugs is not an easy task but we believe in the promise of innovation and are committed developing the next generation of important therapies to address unmet medical needs. Through a focused effort of pre-clinical, product, process and clinical development, we look to continue to build the value of our compounds. We look forward to another productive year in 2009 and to communicating with you in the future with respect to our progress. We appreciate your continued support of Nile Therapeutics.
Sincerely,
Peter M. Strumph
Chief Executive Officer
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