The company has acquired the rights from an Austrian firm that has made a vital contribution to the development of new approaches to treating the disease. The core element of the platform technology involves human proteins which - despite offering sufficient functionality - are subjected to enzymatic digestion in untreated cells due to genetic mutations in their structure.

Axentis Pharma AG (Switzerland) today announced that, as part of its takeover of an Austrian research company, it has also acquired two patent families that incorporate a series of international patent applications. The company is taking over these rights from Austrian-based BioDevelops Pharma GmbH, which three years ago identified the therapeutic potential offered by a new approach to the treatment of cystic fibrosis and secured the relevant rights.

The technology directly targets the functional cause of cystic fibrosis - the process whereby the body destroys mutated proteins which were originally designed to regulate the concentration of salt in the epithelial cells of the lungs and other organs. In patients with cystic fibrosis, a genetic defect means that many such proteins are produced with minor structural mutations. Although the mutated proteins still offer sufficient functionality, they are eliminated by quality control functions in the endoplasmic reticulum of the cells. The therapeutic approach now under development at Axentis Pharma enables these proteins - which are still functional despite their mutated structures - to escape enzymatic digestion.

Dr. Gergely Lukacs who guided the experimental set-up in his labs at the Sick Kids Hospital in Toronto explains: "In patients with cystic fibrosis, the protein CFTR (cystic fibrosis conductance regulator) with mutated structure is marked with ubiquitin. These ubiquitin-marked proteins are degraded by multiple mechanisms prematurely. If this marker is missing, the protein evades destruction and can regulate the concentration of water and salt secretion in the lung despite its minor structural defect.

Joerg Zielasek, president of Axentis Pharma AG, comments on what the acquisition of these rights means for the company: "Just six months ago, we reached agreement with aRigen Pharmaceuticals, Japan, on terms and conditions regarding an exclusive license for developing and marketing a patented system for the liposomal encapsulation of an active ingredient designed to combat lung infections in patients with cystic fibrosis. By acquiring the rights to BioDevelops' technology platform, Axentis Pharma is both enhancing its development pipeline and offering both patients and investors extensive prospects for the treatment of this disease."

Cystic Fibrosis

Cystic fibrosis is the most common life-threatening hereditary disease among Caucasian population. 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.

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). The company is in final preparation to initiate a phase II clinical trial using tobramycin liposome formulation against lung infections in Cystic Fibrosis patients.

Patent Families

The inventions which have now been acquired by Axentis Pharma describe the use of molecules for the enzymatic removal of ubiquitin markers in incorrectly folded proteins. The inventions also protect the use of the proteasome inhibitor Bortezomib (Velcade (R)) for second medical use in cystic fibrosis (Velcade (R) has already been approved for use in humans to treat other indications).

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