Cystic Fibrosis – Axentis Pharma Initiates Clinical Trial for Lung Infections

Zurich, 4. September 2008. 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.”

About cystic fibrosis
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.

About Axentis Pharma AG (
Axentis Pharma AG is a Swiss biotechnology company. The company is using a
patent-pending platform technology to develop therapies for diseases caused
by incorrect protein folding in the endoplasmic reticulum. The most
prominent example of such diseases is cystic fibrosis (mucoviscidosis).

About Fluidosome® technology
Axentis Pharma’s Fluidosome® technology uses biocompatible lipids endogenous
to the lung that are formulated into small liposomes. This nanocapsules
platform opens broad applicability for unmet medical needs including other
respiratory diseases. In the case of the Fluidosome tobramycin®, the
interaction between tobramycin and the microbial cell is triggered when the
liposomes attach to the outer cell membrane. Tobramycin then leaches into
the inner cell compartment, which leads to rapid cell death.

For further information, please contact:

Axentis Pharma AG
Brandschenkestrasse 60
8027 Zürich
T +43 1 505 70 44 (PR&D)
E [email protected]

Copy Editing & Distribution:
PR&D – Public Relations for Research & Education
Campus Vienna Biocenter 2
1030 Vienna
T +43 1 505 70 44
E [email protected]

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