Clinical Trial for Experimental Cystic Fibrosis Drug Begins

According to a story from, the specialty pharmaceutical company Santhera Pharmaceuticals has recently announced of its Phase Ib/IIa clinical trial that will test its experimental product POL6014. This therapy is in development for the treatment of cystic fibrosis. This trial will be a multiple ascending dose trial, with three different dosages being evaluated. Santhera specializes in the development of therapies for rare orphan diseases and other urgent unmet medical needs.

About Cystic Fibrosis

Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.

About The Trial

This clinical trial is expected to involve around 40 patients with cystic fibrosis. The treatment period will last for 15 days. Patients will either receive a placebo or three ascending doses of POL6014. These doses will be administered either once or twice per day. The treatment sites for this trial are located in Poland and Germany, and it is expected to be completed by the first half of 2019.

About POL6014

POL6014 is a unique inhibitor of human neutrophil elactase (hNE), a substance that researchers have found is often present in high levels in a number of chronic lung conditions in which inflammation plays a role. Inflammation is not the primary concern in cystic fibrosis, but it contributes to the worsening of lung function and can make the lungs even more susceptible to infections. Santhera hopes that POL6014 will be an effective treatment for other lung diseases as well.


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