FDA grants Fast Track designation to Nile’s cenderitide

SAN MATEO, Calif.—Biopharmaceutical company NileTherapeutics, Inc. announced today that the U.S. Food and Drug Administrationhas granted its post-acute development program for cenderitide with Fast Trackdesignation. The program's goal is to reduce cardiovascular mortality andcardiovascular re-hospitalization in the post-acute period among patientssuffering from acute decompensated heart failure. Nile's plan is to developcenderitide as an outpatient therapy for acutely decompensated heart failure(ADHF) patients. The therapy would be delivered continuously for up to 90 daysfollowing a patient's discharge from the hospital, a novel therapeuticindication that is referred to as "post-acute."

"We are very pleased that the FDA has recognizedcenderitide's potential to address an important unmet medical need for heartfailure patients," says Joshua Kazam, Nile's Chief Executive Officer.

The FDA reserves the Fast Track designation for productsthat have demonstrated potential in addressing unmet medical needs for seriousor life-threatening conditions, and the process is intended to assist in thedevelopment and expedite the review of those products. Drugs that receive FastTrack designation have the option of submitting sections of the New DrugApplication (NDA) for review as they are completed, whereas normally, NDAreview doesn't begin until the entire application has been submitted.Additionally, a drug with Fast Track designation can be considered for PriorityReview, which can reduce the NDA review time from ten months to six months.

According to the FDA website, Fast Track designation andPriority Review "do not compromise the standards for the safety andeffectiveness of the drugs that become available through this process." The FDAalso asserts that the new review approaches "have yielded tangible results inbringing safe and effective drugs to patients with serious diseases morequickly." Applying for Fast Track designation is up to a drug company, andreflects their belief in their product's ability to either surpass currenttreatment options or offer options for unmet medical needs, as is the case withNile's cenderitide program.

Cenderitide currently holds most of Nile's focus, in keepingwith their cardiovascular leanings, and falls into the category of drugs callednatriuretic peptides. Preclinical and clinical data on the natriuretic peptidehave shown that the peptides can act on multiple disease processes thatcontribute to the negatives outcomes that go along with heart failure. Twonatriuretic peptides are already on the market for the treatment of ADHF,Natrecor in the United States and hANP in Japan.

Clinical results so far have shown that cenderitide might bea superior treatment solution thanks to the therapeutic benefits it offers, includingreduction in cardiac pressure, preservation/enhancement of renal function,improved diuresis and managed blood pressure reduction. A short-term infusionof cenderitide has demonstrated positive effects on patients' cardiovascularand renal parameters, and Nile feels that continuous and extended infusionsthrough a subcutaneous pump could provide sustained symptomatic relief,contributing to fewer post-acute hospitalizations and continued improvement incardio-renal functions.

Hospitalization during the post-acute period, 90 days afteradmission for ADHF, is a substantial issue, as the American Heart Associationnotes that there are more than 1.2 million ADHF admissions per year in theUnited States and 40 percent of those patients return to the hospital. It isexpected that cenderitide will improve post-acute re-hospitalization in thefollowing ways:

*Prolonged reduction of wedge pressure and blood pressure

*Additional diuretic and natriuretic effects on top of standard oral diuretics

*Improved medication compliance with a continuous subcutaneous pump delivery

*Possible prevention of the progression of maladaptive ventricular hypertrophy

* Suppression of cardiacfibroblast proliferation and a reduction in scarring

Nile has plans for a Phase I clinical trial in the secondquarter of 2011 to assess the pharmacokinetics and pharmacodynamics ofcenderitide when delivered via a subcutaneous pump, and expects the trial to becomplete by the first quarter of 2012.

"If our post-acute cenderitide program is successful, thenwe may be able to reduce the annual number of hospital visits for ADHF,potentially saving the health care system billions of dollars," says Kazam.