Cystic fibrosis


Neupharma Srl


Cystic Fibrosis

Cystic Fibrosis (CF) is a recessive autosomic disease caused by a defect of CFTR gene. CFTR protein is normally localized in the apical membrane of several epithelial tissues and its main function is the transport of chloride ion, thus indirectly regulating sodium and water transport.

The main cause of the reduced life expectancy and life quality of patients suffering from CF lies in the pulmonary effects of the disease. In conductive airways, bronchi and bronchioles, the defect of CFTR protein reduces hydration of surface respiratory secretions and mucociliary clearance, causing a constitutive inflammatory status, thus favoring bacterial infections, caused by several bacteria, including Methicillin-resistant Staphylococcus aureus (MRSA), frequently chronic in the advanced stages of the disease. The presence of MRSA in the respiratory tree is a sign of a poor prognosis and is associated with a reduced life expectancy.

The disease affects 1 out of 2.500-3.000 newborns, with 1 healthy carrier out of 25-30 subjects in a general population of Caucasian origin. There are about 7.000 patients affected by cystic fibrosis in Italy, with a regional prevalence being between 4,4 and 10,4 patients out of 100.000 inhabitants. MRSA is present in about 20% of the EU patients and in about 25% of US patients.



Development of TeicoAir®, teicoplanin by inhalation

Rare Partners collaborates since many years with Neupharma Srl, an Italian pharmaceutical company highly specialized in the CF field and in particular in the development and marketing of antibiotics administered by inhalation.

The most advanced project concerns the clinical development of TeicoAir®, an innovative inhalatory formulation of teicoplanin, antibiotic of first choice for the treatment of lung infections due to Gram-positive bacteria and approved in most countries since 1989. The therapeutic indication pursued by Neupharma in collaboration with Rare Partners is the treatment of chronic lung infections due to MRSA bacteria in CF patients.

The rational basis of the project is the efficacy of teicoplanin towards the target and the advantage of the proposed route of administration, in terms of increased efficacy and reduced toxicity in comparison with i.v. administration.
Despite the fact that MRSA infections are relatively common, few agents are available for this severe respiratory diseases and none of them is given by the inhalatory route. 
After having confirmed the efficacy of the new formulation in an experimental murine model of MRSA infection, a first phase 1 clinical study has been completed at the University Hospital of Verona in CF patients.

The clinical trial showed after treatment by inhalation high levels of teicoplanin in the sputum despite almost negligeable systemic concentrations, together with a very good local tolerability.
After these promising results from phase 1 study, an experimental clinical protocol has been prepared for a trial in CF patients infected by Staphilococcus aureus (including MRSA), with the aim of collecting first efficacy data both on microbiological and clinical parameters after administration of TeicoAir®. The protocol has been approved by the Ethical Committee of the University Hospital of Verona (Principal Investigator Dr. Marco Cipolli), center coordinator and sponsor of the trial.

The second clinical center involved in the study is the Policlinic Hospital of Milan (Prof. Francesco Blasi).
In parallel, Neupharma and Rare Partners are exploring potential partnerships with companies wishing to complete the clinical development of the project and to bring the product to marketing authorization.

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