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Cystic Fibrosis – Orphan Drug Designation for Innovative Treatment Against Lung Infections by Axentis Pharma AG

April 30, 2009

ZURICH, Switzerland, April 30 /PRNewswire/ — An innovative treatment for
infections of the respiratory tract in cystic fibrosis patients has received
orphan drug designation in the US. Axentis Pharma of Zurich, Switzerland
announced today that this sought-after designation has been granted to its
product candidate Fluidosomes-tobramycin, a therapeutic that will soon be
tested in Phase II clinical trials. The company has now been granted orphan
drug designation for this candidate in both Europe and the US.

Axentis Pharma (Switzerland) announced today that the Office of Orphan
Products Development of the US Food and Drug Administration (FDA) has granted
the orphan drug designation to its lead product candidate
Fluidosomes-tobramycin. This drug is a liposomal formulation of tobramycin,
an innovative treatment for infections of the respiratory tract in patients
with cystic fibrosis that is delivered directly to the site of infection via
standard nebulizers. Pre-clinical and Phase I clinical studies support
improved safety and efficacy profiles for Fluidosomes-tobramycin as compared
to currently marketed treatments for infections of the respiratory tract in
patients with cystic fibrosis.

The orphan drug designation is granted with respect to treatment of
pulmonary infections caused by Pseudomonas aeruginosa, a bacterium that is
one of the most common causes of infections of the respiratory tract in
patients with cystic fibrosis. Axentis Pharmas product candidate received the
orphan drug designation for the US only two months after the application and
less than one year after orphan designation in Europe was transferred to the
company. Dr. Helmut Brunar, company CEO and President, comments: “The orphan
drug designation for the US is very good news for affected patients as well
as for Axentis Pharma’s shareholders. Together with the Orphan Drug
Designation that was already achieved last year in Europe, the US Designation
puts Axentis Pharma in a favourable position to register
Fluidosomes-tobramycin in two major world markets with substantial support of
the relevant authorities and at a cost advantage for the company. As a
result, we will be able to deliver the product at competitive prices to
patients once it has passed the final clinical test phase. In addition to
this, the orphan drug designation grants Axentis several years of exclusive
marketing rights once the product has been launched. That is a significant
strengthening of Axentis Pharma market position as well as the company’s
value.”

Fluidosomes-tobramycin combines the companys proprietary Fluidosomes
technology with the well-established generic 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 may result in prolonged high local drug concentration in the
lung, which in turn may lead to higher efficacy and may allow lower doses.

Currently, the company is initiating Phase II clinical trials that will
assess the safety and tolerability of a new therapeutic formulation as well
as the effects of two different doses of the new drug. Results of the
clinical trial are expected early 2010.

About Axentis Pharma AG (http://www.axentispharma.com)

Axentis Pharma is a respiratory specialty pharmaceutical company which
core competence is the application of a fully patented, encapsulating drug
delivery system to already established and well-characterized therapeutic
agents. Currently, the company is using this technology, named Fluidosome(R)
technology, for the development of its lead product, a clinical stage
treatment against cystic fibrosis (CF).

About Fluidosome technology

Axentis Pharmas Fluidosome technology uses biocompatible lipids
endogenous to the lung that are formulated into small liposomes. This
nanocapsule platform offers wide-ranging potential for unmet medical needs,
including other respiratory diseases. In the case of 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.

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
cystic fibrosis transmembrane conductance regulator (CFTR) gene found on
chromosome 7. This mutation causes increased secretion deposits on mucous
membranes. Lung complications represent the most serious manifestation of the
disease Ӛ­ 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. As well as the breakdown of lung tissue, this also leads to
bronchiectasis and lung failure.

    For further information, please contact:

    Dr. Helmut Brunar, Ph.D.,
    CEO Axentis Pharma AG
    Limmatquai 138 CH-8001
    Zurich, Switzerland,
    T +41-44-202-7878,
    E board@axentispharma.com,
    W http://www.axentispharma.com

    Copy Editing Distribution:
    PR - Public Relations for Research Education
    Campus Vienna Biocenter 2 1030 Vienna
    Austria,
    T +43-1-505-70-44,
    E contact@prd.at,
    W http://www.prd.at

SOURCE Axentis Pharma AG


Source: newswire



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