Emerging Drug Developer: Nile Therapeutics

Nile navigates its way to Phase III for AHF

Nile Therapeutics is a small developer targeting a very big market.

Founded five years ago, San Mateo, CA-based Nile--which is operating with only four employees--has been primarily focused on CD-NP, a carefully crafted molecule that was hatched by a top investigator at the Mayo Clinic. And Nile CEO Joshua Kazam was at the BIO CEO & Investor conference in New York early Monday morning to discuss the company's plans.

CD-NP is in a class of natriuretic peptides used for acute decompensated heart failure patients with decreased cardiac function. That class of drugs has had a positive pharmacological effect, but is linked with a decrease in renal function, a big issue for co-morbid patients with chronic kidney disease. John C. Burnett--a professor of medicine at the Mayo Clinic College of Medicine and head of the cardiorenal research lab-and a colleague designed a molecule that Nile in-licensed, believing it can not only help heart failure patients but also improve renal function in patients with chronic kidney disease.

The program has successfully passed through a Phase Ia dose-escalating trial, a Ib in stable heart failure patients and a IIa proof-of-concept trial. Investigators are currently enrolling AHF and renal failure patients in a Phase II trial, where researchers will look for an ideal dose or doses that can be taken into a late-stage study.

Once they get into Phase III, says Kazam, recent regulatory guidance indicates that if they can establish an improvement in symptoms of dyspnea (breathlessness common in AHF), improved renal function - offering a positive end organ effect for regulators to consider--and demonstrate an improvement in cardiac filling pressure the company will have the necessary data to gain an approval to use the drug in combination with standard of care.

For now, the company (NLTX, trading for $1.23 a share this morning) is focused on harvesting Phase II data mid-year, with the long-term plan calling for Phase III data and a regulatory approval perhaps in 2013.

It hasn't all been clear sailing for Nile, though, since the company raised $20 million in financing and went public in 2007 in a reverse merger. The developer earned a ‘going concern' alert in early 2009 from its auditor. And about a year ago the FDA put a clinical hold on its lead program, asking for more data from the Phase IIa. The hold was lifted just a couple of months later.

Kazam, a Wharton grad and a vice president of the venture capital group Two River, says the program is still unpartnered but adds that the developer is in ongoing talks. Nile raised $3.4 million in a private placement last July, and a pact could be struck at the end of Phase II, provided the data looks promising, or after a planned Phase IIb. The CEO added that Nile has enough cash to get through Phase II, when it can decide whether or not to go to the market to raise additional cash.

There is a second program in the pipeline--another natriuretic peptide dubbed CU-NP. But that program is still in its preclinical phase, with Nile's virtual staff concentrating heavily on its mid-stage prospect.

Like any good numbers man, Kazam is careful to point out the size of the market: 5.3 million Americans are affected by acute heart failure. The disease is responsible for one million hospital admissions each year. And AHF's close connection to CKD gives Nile a shot at taking its place in the list of therapies that are deployed to control an epidemic of symptoms in an increasingly overweight nation.

Emerging Drug Developer: Nile Therapeutics

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