Clinical trial advances cystic fibrosis therapy

Axentis Pharma AG has initiated a clinical phase IIa trial to assess the safety and tolerability of a new therapeutic formulation for the treatment of severe pulmonary infection in cystic fibrosis patients. The new formulation allows an established therapeutic agent to be delivered directly to the site of infection. The forthcoming trial will also compare the effects of two different doses of the new drug.

Initial results are expected in summer 2009. Axentis Pharma acquired all the necessary rights for this formulation from international partners just eight months ago. In addition to these advances, the company has also succeeded in appointing two renowned experts to its Scientific Advisory Board.

Axentis Pharma AG (Switzerland) announced today that all the necessary requirements for a clinical phase IIa trial have been fulfilled. The objective of this trial is to assess the safety and tolerability of an inhalable tobramycin, a well characterised and established drug for the treatment of pulmonary infection in cystic fibrosis patients. The product ARB-CF0223 - also known as Fluidosome® tobramycin - is a liposomal formulation of tobramycin, delivered directly to the site of infection via standard nebulizers. ARB-CF0223 has an improved safety profile and higher efficacy compared to current treatments for infections of the respiratory tract in patients with cystic fibrosis. It can be used in lower doses and also reduces the frequency and severity of side effects for pulmonary infections. The company expects to begin recruiting patients at its four international trial centres by the end of the year.

Dr. Hans Schreier, Chief Scientist of Axentis Pharma on the advances the company has made: "It was only very recently that we obtained all the necessary legal rights including transfer of sponsorship of the EMEA Orphan Designation to further develop our current lead product, Fluidosome® tobramycin. However, we are already in a position to initiate clinical trials to fully assess its safety and tolerability and gather information on appropriate doses. I am very pleased for patients suffering from cystic fibrosis, who will benefit directly from this promising once-a-day treatment: a significant improvement both in treatment and patient management. This is also great news for our investors, who have helped us to advance so far in a very short time."

Fluidosome® technology is based on the well characterised drug tobramycin. Utilising synthetic liposomes containing tobramycin, a standard nebulizer delivers the drug directly to the endobronchial sites of infection in cystic fibrosis patients. This results in prolonged, high local drug concentration, which in turn achieves higher efficacy and enables lower doses.

The phase II study will be carried out in 4 international centres. A total of 24 patients will receive treatment: Eight will receive a twice-daily 300 mg dose of the current tobramycin formulation over 28 days; another eight will be given a twice-daily 150 mg dose of Fluidosome® over two weeks and later a third group will receive one 300 mg dose of Fluidosome® per day for two weeks.

The company has also announced the appointment of two renowned experts to its Scientific Advisory Board. Prof. Adriano Aguzzi, Director of the Institute of Neuropathology at University Hospital Zürich, Switzerland and Prof. Gergely C. Lukacs, Canada Research Chair, Department of Physiology at McGill University Montreal, Canada will in future advise Axentis Pharma on scientific and medical issues related to the company's product pipeline.

Dr. Schreier on the latest additions to the team: "Axentis Pharma is very happy to have such outstanding experts on its Scientific Advisory Board. Just as our investors provide us with the trust and financial means that are essential to the ongoing development of our products, Prof. Aguzzi and Prof. Lukacs will contribute to the intellectual capital of Axentis Pharma."

Cystic fibrosis is the most common life-threatening hereditary disease amongst Caucasian populations. The disease is caused by a mutation in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene found on chromosome 7. This mutation causes increased secretion deposits on mucous membranes. Lung complications represent the most serious manifestation of the disease - and the reason for the high mortality rate amongst patients.

Such complications often involve infection of the bronchi by the bacteria Pseudomonas aeruginosa. Chronic inflammations then cause lung functions to become blocked. Besides the break-down of lung tissue, this also leads to bronchiectasis and lung failure.

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