According to a story from BioSpace, the biopharmaceutical company Aridis Pharmaceuticals, Inc., has recently announced that it has struck an agreement with the US Food and Drug Administration (FDA) in regards to its phase 2 clinical trial design. This trial will be testing the company’s experimental treatment AR-501 for chronic lung infections associated with cystic fibrosis, a rare disease that primarily impacts the lungs. Aridis is focused on the development of novel therapies to treat and prevent serious, life-threatening infections.
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. More useful treatments for the disorder have been introduced in recent years. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40’s and 50’s with good care. To learn more about cystic fibrosis, click here.
New Trial Design
AR-501 has already displayed positive safety findings in its earlier phase 1 trial, which included single dose and multi-ascending dose evaluations. The new phase 2 trial design is simplified in that it will only contain a multi-dose regimen. In addition, this new design will also cover phase 2a and phase 2b, whereas the earlier design was solely for phase 2a. Aridis aims to complete the clinical trial by the end of 2021.
About AR-501
AR-501 is an inhaled formulation of gallium citrate. This substance is expected to have the capability of preventing infections from a variety of microbes that commonly appear in the respiratory system of cystic fibrosis patients. The investigational therapy is currently being evaluated in a phase 1/2a clinical study. It is being developed alongside the CF Foundation and has earned Fast Track designation, Orphan Drug designation, and Qualified Infectious Disease Product designation from the FDA.
