Polyphor successfully completes clinical Phase I study with POL6014 targeting life threatening lung diseases including cystic fibrosis

Polyphor Ltd today announced the successful completion of its clinical Phase I study with inhaled POL6014 in healthy volunteers. The macrocycle drug candidate POL6014 is a highly selective, potent and reversible inhibitor of human neutrophil elastase (hNE). Many severe respiratory diseases are frequently associated with increased hNE levels which may lead to life-threatening deterioration of lung function. The rare lung diseases cystic fibrosis (CF) and alpha-1 antitrypsin deficiency (AATD) represent just two examples of such conditions.

This randomized, double-blind, placebo-controlled dose-escalation Phase I study was designed to investigate the safety, tolerability and pharmacokinetics of inhaled single doses of POL6014 in healthy volunteers. The compound was very well tolerated in all treatment groups. No serious adverse events were reported, and the measured pharmacokinetic parameters increased proportionally with the dose administered using a customized Pari eFlow(R) aerosol inhaler.

"We are very pleased with the outcome of the Phase I clinical study with our macrocycle drug candidate POL6014. The data confirms that the drug can be safely delivered to the human lung by inhalation," said Dr. Leon Hooftman, CMO of Polyphor. "Following the successful completion of this study, we have now initiated two clinical Phase Ib trials investigating the safety, pharmacokinetics, and pharmacodynamics of POL6014 in individuals with cystic fibrosis and non-cystic fibrosis bronchiectasis."

Polyphor is also pleased to announce that it has reached the first two milestones in its recently announced collaboration with Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), Bethesda, Md. The collaboration with CFFT enables Polyphor to accelerate the clinical development of the inhaled elastase inhibitor POL6014 in cystic fibrosis.

About Cystic Fibrosis:
Cystic fibrosis (CF) is a life-threatening disease that affects the lungs and digestive system and impacts about 70,000 people worldwide. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which results in either no CFTR protein or an abnormal CFTR protein that does not function properly. The presence or absence of this dysfunctional protein causes the body to accumulate excessive levels of unusually thick mucus in the lungs. This excessive sticky mucus in the lungs is a site for infections that can require hospitalization. Respiratory distress in CF - defined as acute difficulty in breathing, infection and/or hospitalization - is most commonly related to lung infections and the resulting inflammation that damages lung tissue. The damage caused by inflammation can be attributed to an overwhelming and dysfunctional response by deregulated neutrophils. Treatment of this lung inflammation is, therefore, thought to be key to improving CF affected individuals' lung health and well-being. For more information on CF, go to www.cff.org.