Mast Therapeutics has announced that data from an aborted Phase 2 study of AIR001 sodium nitrite inhalation solution for the treatment of pulmonary arterial hypertension (PAH) demonstrated improvement in median pulmonary vascular resistance (PVR) for all doses. Improvements in median distance in a 6-minute walk test were also seen for all doses, “including clinically-meaningful improvements at the highest dose level.”
In February 2014, Mast acquired Aires Pharmaceutical, which was developing AIR001, also known as Aironite. Prior to the acquisition, Aires discontinued the open-label, randomized, parallel-dose study “due to capital constraints” after enrolling 29 patients; the study was originally set to enroll 90 patients with PAH.
Mast CEO Brian M. Culley commented, “We are encouraged by the results seen in the Phase 2 study of AIR001 and believe they further validate our acquisition of Aires. The data from the study show benefits consistent with prior studies and support further development of AIR001. We look forward to proceeding with the Phase 2a studies in PH associated with left heart disease and anticipate reporting preliminary study results as early as the second half of 2015.”
Interim Chief Medical Officer Edwin L. Parsley said, “These results are promising and consistent with earlier findings of AIR001 as an agent that can have a positive effect on hemodynamic parameters in a PH population. To date, more than 120 individuals have received AIR001, including patients who have received repeat administration for as long as 12 months, patients who were treatment naive, and patients on PAH disease-specific background therapy. Given the hemodynamic improvements observed, we feel AIR001 may be uniquely suited to address the serious unmet need facing the large number of patients with pulmonary hypertension associated with left heart disease. Consequently, we will be pursuing clinical development of AIR001 in that indication and plan to support multiple, institution-sponsored Phase 2a studies that will evaluate 1) acute hemodynamic effects, 2) acute effects versus placebo on maximum oxygen consumption and exercise hemodynamics, and 3) inhaled versus intravenous administration of nitrite, as well as the safety of multiple doses of AIR001, in patients with PH associated with left heart disease.”
The FDA and EMA have both granted orphan drug status to AIR001 for the treatment of PAH.
Read the Mast Therapeutics press release.
