The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for AB569 from Arch Biopartners Inc. as a treatment for Pseudomonas aeruginosa (P. aeruginosa) pulmonary infections in patients with cystic fibrosis. The Orphan Drug Designation is a crucial step for the development of new medicines for the safe and effective treatment of chronic P. aeruginosa infection in cystic fibrosis patients.
Cystic fibrosis (CF) is a genetic disease that affects various organs and is characterized by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CF patients are susceptible to lung infections due to abnormal mucus production in the lungs and airways, which results in regular infective pulmonary exacerbations with opportunistic pathogens, such as Pseudomonas aeruginosa (P. aeruginosa). In cystic fibrosis patients, the mucoid form of P. aeruginosa is highly resistant to antibiotics and phagocyte-mediated killing by innate immune cells, inducing epithelial inflammation that ultimately causes tissue damage, which leads to poor prognosis.
In the United States, 40% of CF patients from ages 6 and 10 years old are infected with P. aeruginosa, while the frequency of infection increases to 60% and 75% by the age of 17 and between the ages of 25 and 34, respectively. As a result, there is an urgent clinical need for the development of novel successful therapies for cystic fibrosis.
AB569 is a combination of two compounds, sodium nitrite and ethylenediaminetetraacetic acid, and is an novel potential nebulized (inhaled) treatment to deal with mucoid and non-mucoid P. aeruginosa pulmonary infections that are resistant to conventional antibiotics. This drug was invented by Professor Dr. Daniel Hassett from the University of Cincinnati.
During 2015, AB569 successfully completed pre-clinical in vivo and in vitro validation studies. During these studies, AB569 showed significant efficacy against P. aeruginosa. This included a recent study using a mouse model of chronic pulmonary infection. The Arch Biopartners research team is convinced these results provide the proof-of-concept for a human clinical trial to test the safety and efficacy of AB569 for chronic P. aeruginosa infection in cystic fibrosis (CF) patients. As a result, Arch management is evaluating the adequate toxicology and regulatory guidelines for the first human trial for AB569.
“The individual active ingredients of the drug have been approved as safe for use in humans in the past. Our team at the University of Cincinnati looks forward to working with Arch to advance AB569 into clinical trials for cystic fibrosis patients who urgently need more effective treatments for their P. aeruginosa respiratory infections,” stated Dr. Hassett in the press release.