Protalix BioTherapeutics, Inc. recently announced that on June 12, 2015 Dr. Yosehp Shaaltiel, EVP of Research and Development, presented pre-clinical data on the company’s AIR DNaseTM (PRX-110) for cystic fibrosis (CF) treatment at the 38th European Cystic Fibrosis Conference, held in Brussels, Belgium. The oral presentation was entitled “AIR DNaseTM: Actin Inhibition Resistant Plant Cell Recombinant Chemically Modified Deoxyribonuclease I (DNase I) for the Treatment of Cystic Fibrosis.”
CF is a rare, life-threatening genetic disease in which a defective gene (CFTR) induces a salt imbalance, causing the body to form unusually thick, sticky mucus that can obstruct the airways and promote dangerous lung infections resulting in serious respiratory and also gastrointestinal manifestations. The majority of the CF patients die due to respiratory failure. There is no cure for the disease and it is estimated that almost 75,000 individuals worldwide suffer from CF.
AIR DNase is the company’s inhaled, chemically-modified, recombinant form of human deoxyribonuclease I (DNase I) that is resistant to actin — a potent inhibitor of DNase that is found in high concentrations in the lungs of CF patients. AIR DNase was developed as a strategy to control the amount and thickness of mucus in CF patients in order to improve their lung function and reduce the susceptibility to recurrent respiratory tract infections.
“AIR DNase works by cleaving extracellular DNA and thinning the thick mucus that accumulates in CF patients’ lungs,” explained Dr. Shaaltiel in a news release.
The research group found in in vitro assays that the concentration of actin and DNase remains high in CF patients after treatment with AIR DNase, with an inhibitory effect of only 15% in comparison to 85-100% for Pulmozyme®, an approved drug to improve lung function in CF patients. AIR DNase was found to reduce by 70% the viscosity of the mucus, while Pulmozyme treatment induced a 30% reduction.
“We are very optimistic about the results thus far and are excited to proceed to the clinic. We believe that AIR DNase has the potential to address some of the significant unmet medical needs of the cystic fibrosis community,” concluded Dr. Shaaltiel.
Protalix BioTherapeutics is currently studying clinical sites to conduct a proof of concept trial for AIR DNase. The company is planning to launch this trial by the end of the year, and expects to have results in the first half of 2016.