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Nektar Announces Inhaled Amphotericin B Product in Human Clinical Trials for Preventing Fatal Pulmonary Fungal Infections in Immunosuppressed Patients

Posted on: Thursday, 29 September 2005, 15:00 CDT

Designed to Prevent Pulmonary Aspergillosis Incidence, Morbidity, Mortality and High Cost of Treatment in Immunosuppressed Patients

Nektar Therapeutics (Nasdaq:NKTR) today announced the company has developed an inhaled amphotericin B product for preventing fatal pulmonary fungal infections in immunosuppressed patients to reduce the incidence, morbidity, mortality and high cost of treating these infections. Using a small proprietary pocket size product, the company has conducted two Phase I trials and has long-term toxicity studies underway to support the planned pivotal trials.

"More than 150,000 patients annually in the US and Europe are at risk of developing often fatal and costly fungal infections in the lungs. Nektar's inhaleable amphotericin B potentially could be the first prophylactic therapy to prevent these serious infections," said Dr. Michael Weickert, Nektar Senior Program Executive.

Clinical Need

"Aspergillosis in immunosuppressed patients has a mortality of approximately 50% and, although newer treatments have improved side effects, they are still only 50% effective most of the time. Further, the cost of the disease is severe. Prevention may be more effective than treatment," said Dr. Thomas Patterson, Professor of Medicine in Infectious Diseases, Director of the Infectious Diseases Fellowship Training Program, University of Texas Health Sciences Center, San Antonio, Texas.

There are several ways in which a patient can become vulnerable to aspergillosis infection. The treatment of acute leukemias and hematological malignancies require chemotherapy that can lead to significant immunosuppression, especially if a bone marrow or stem cell transplant is required. In addition, organ transplantation requires immunosuppression therapy to prevent rejection of transplanted organs. Immunosuppressed patients are vulnerable to many infections, and some therapies exist to help prevent bacterial, viral and yeast infections. Currently, however, there are no approved pharmaceutical therapies to prevent fungal infections like aspergillus, a widely prevalent genus responsible for infecting the lungs and invading the body, eventually causing systemic infections that are very difficult to cure and are associated with an extremely high mortality rate.

More than 150,000 patients in the US and Europe would benefit from this protection, based on the severity of their immunosuppression. In addition to patients who have had bone marrow or stem cell transplants, organ transplants, or chemotherapy for hematological malignancies, patients with certain genetic diseases, late stage AIDS with the associated neutropenia, and aplastic anemia are also at significant risk for pulmonary aspergillosis infections.

Studies show that the treatment cost is significant. A study by Dasbach et al (2000) reported that for cancer or leukemia patients, a secondary diagnosis of aspergillosis resulted in a 26 day longer hospital stay, $115,262 more in total costs (1996 health care dollars) and 4 times the mortality rate.(1)

The Nektar Solution

The Nektar solution enables the inhalation of a therapeutic concentration of amphotericin B directly to the lungs. Amphotericin B is a potent broad spectrum anti-fungal drug which has been used intravenously for decades. By delivering it directly to the site of potential infection, Nektar inhaled amphotericin B is designed to prevent potential life-threatening lung fungal infections, while limiting the systemic exposure and thereby preventing the common therapy-limiting toxicities associated with intravenous amphotericin therapy.

In addition to preventing pulmonary fungal infections, the Nektar inhaled amphotericin B product is designed to provide a delivery mode that will encourage compliance, as the prevention therapy could require many months of use both in an in-patient as well as an out-patient setting. Nektar has developed a powder formulation of amphotericin B and method of delivery, including a small, hand-held inhaler that will enable dosing in a single inhalation.

Pre-clinical and Phase I Clinical Data

Pre-clinical data indicate that the inhaleable amphotericin B:

-- Protects immunosuppressed animals from pulmonary fungal infections;

-- Has little or no pulmonary toxicity in animals at 10 times or more than the expected dose for humans;

-- Has no systemic toxicity and has very low amounts of drug in the bloodstream -- less than 5% the concentration generally regarded as toxic in blood in humans -- even when delivered at doses more than 10 times the level expected for humans.

Clinical data reinforce the pre-clinical data:

-- The highest doses expected to be delivered in the pivotal trial and commercial product have been safely administered to volunteers;

-- Inhaled amphotericin B was well tolerated in the lungs;

-- No serious adverse events have been seen in subjects to date; and

-- The highest doses resulted in less than 3% of the concentration generally regarded as the toxic concentration in the blood of humans.

Product Status

Inhaleable amphotericin B has been studied in two Phase I trials. There are two long-term toxicity studies underway to support the pivotal trial which Nektar believes will be sufficient for US registration with the Food and Drug Administration (FDA).

The announcement was made at the Nektar Investor Day 2005 in New York City today where the company outlined its proprietary programs and its strategy to reach profitability and achieve sustained revenue growth. Inhaled amphotericin B is the lead product in the company's proprietary products program. In a separate press release, Nektar also announced its Inhaled antibiotics product to prevent pneumonia in mechanically-ventilated patients.

About Nektar

Nektar Therapeutics enables high-value, differentiated therapeutics with its industry-leading drug delivery technologies, expertise and manufacturing capabilities. The world's top biotechnology and pharmaceutical companies are developing new and better therapeutics using Nektar's advanced technologies and know-how. Nektar also develops its own products by applying its drug delivery technologies and its expertise to existing medicines to enhance performance, such as improving efficacy, safety and compliance.

This release contains forward-looking statements that reflect management's current views as to Nektar's business strategy, product and technology development plans and funding, collaborative arrangements, clinical trials, and other future events and operations. These forward-looking statements involve uncertainties and other risks that are detailed in Nektar's reports and other filings with the SEC, including its Annual Report on Form 10-K, as amended, for the year ended December 2004 and its Quarterly Report on 10-Q for the quarter ended June 30, 2005. Actual results could differ materially from these forward-looking statements.

(1)Dasbach et al 2000, Clin Infect. Dis. 31:1524.

Source: Business Wire

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