According to a story from BioSpace, the biopharmaceutical company AzuRx BioPharma Inc. recently announced that the first patient has been treated in the company’s phase 2 clinical trial. This trial is testing the experimental drug candidate MS1819-SD combined with porcine enzyme replacement therapy (PERT) as a treatment for patients with cystic fibrosis who experience severe exocrine pancreatic insufficiency. Such patients have difficulties with the malabsorption of fat even when taking the maximum PERT dosage.
About Cystic Fibrosis (CF)
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 Study
While most known for its impact on the lungs, exocrine pancreatic insufficiency is an example of another way in which cystic fibrosis can negatively affect the health of patients. The phase 2 clinical trial will include a total of 24 patients and will feature dose escalation, with dose levels of 700 mg, 1120 mg, and 2240 mg that will be administered on a daily basis. This treatment will be provided alongside a standard dose of PERTs. This combination treatment is intended to resolve of symptoms of maldigestion, allow patients to obtain optimal nutrient absorption with their typical diet, and ultimately correct maldigestion.
Not all cystic fibrosis patients have difficulties with exocrine pancreatic insufficiency, but those that do face a substantially diminished quality of life. Hopefully the addition of MS1819-SD to standard PERT therapy will prove to be an effective method for resolving this problem.
