Mast Therapeutics Initiates Clinical Study of AIR001 for Treating Chronic Infection in CF Patients

Mast Therapeutics reported that its wholly-owned subsidiary, Aires Pharmaceuticals, Inc., has entered into an agreement with the University of Pittsburgh related to a Phase 1/2 open-label safety and proof of concept clinical trial of the Company’s lead product candidate, AIR001, for the treatment of Pseudomonas aeruginosa (P. aeruginosa) infection in cystic fibrosis (CF) patients. The study is being conducted by the University of Pittsburgh and the University of Pittsburgh Medical Center. Mast’s subsidiary will provide study drug and nebulizers for the study, but no direct financial support.

“We are excited that CF experts at University of Pittsburgh wish to study the therapeutic potential of AIR001 in this patient population,” stated Brian M. Culley, Chief Executive Officer of Mast Therapeutics, Inc. “We believe this initiative opens an entirely new area of potential clinical and commercial opportunity for AIR001 and enjoys synergy with the pipeline of our anticipated reverse merger partner, Savara Inc.”

“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,” stated Edwin L. Parsley, D.O., Chief Medical Officer of Mast Therapeutics, Inc. “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,” continued Dr. Parsley.

The objective of the open-label Phase 1/2 study is to determine the safety of AIR001, a sterile, proprietary sodium nitrite solution for intermittent inhalation, administered in a dose escalation manner to adults with CF and P. aeruginosa airway infection. The study also aims to explore the effects of AIR001 on measures of lung function, exhaled airway nitric oxide, and bacterial density. Under the agreement with the University of Pittsburgh, the Company has rights to use the de-identified data and study results for potential regulatory submissions.