AIR001, a new treatment for Pseudomonas aeruginosa infection in patients with lung diseases such as cystic fibrosis (CF) and non-cystic fibrosis bronchiectasis, will be evaluated in an open-label Phase 1/2 clinical trial, according to Mast Therapeutics, the company developing the drug.
Mast’s subsidiary, Aires Pharmaceuticals, has entered into an agreement in which the University of Pittsburgh Medical Center will conduct the trial and Aires will be responsible for providing the drug and nebulizers for the study. The company will have the rights to use the study’s results for potential regulatory submissions.
“We are excited that CF experts at University of Pittsburgh wish to study the therapeutic potential of AIR001 in this patient population,” Brian M. Culley, CEO of Mast Therapeutics, said in a press release. “We believe this initiative opens an entirely new area of potential clinical and commercial opportunity for AIR001.”
AIR001 is a sodium nitrite solution designed to be inhaled via a nebulizer. Nitrite is a vasodilator, and it can form nitric oxide (NO), which ultimately results in a dilation of blood vessels and a reduction in inflammation.
The Phase 1/2 clinical trial aims to determine the safety of AIR001, administered in a dose escalation manner, in adults with cystic fibrosis and P. aeruginosa airway infection. The clinical trial will also examine the effectiveness of AIR001 on measures of lung function, exhaled airway nitric oxide, and bacterial density.
“AIR001 may represent a new therapeutic approach for the treatment of chronic infection in CF patients because it has demonstrated broad in vitro antimicrobial activity against P. aeruginosa and other airway pathogens,” said Edwin L. Parsley, DO, chief medical officer of Mast Therapeutics.
“The antimicrobial activity of nitrite increases under anaerobic and acidotic conditions such as those found in the CF airways, and in non-clinical studies, AIR001 has been shown to prevent P. aeruginosa biotic biofilm growth on the surface of primary CF airway cells,” Parsley added.
According to Mast Therapeutics, AIR001 may provide a novel therapy for chronic P. aeruginosa infection not only in CF patients, but also in patients with non-CF bronchiectasis.
Bronchiectasis is an abnormal and irreversible dilation of the airways of the lung. Symptoms typically include a chronic cough with mucus, shortness of breath, coughing up blood, and chest pain. Those with the disease often get frequent lung infections, including those caused by the Pseudomonas aeruginosa pathogen.
Bronchiectasis has both congenital and acquired causes. While CF is a cause in up to half of cases, between 10 and 50 percent of patients have non-CF bronchiectasis, in which the cause remains poorly understood.