Mast Therapeutics Reports Top-Line Results from Phase 2 Study for AIR001

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 Mast Therapeutics, Inc.
MSTX
today reported top-line results from a Phase 2 study of AIR001 (sodium nitrite) inhalation solution for the treatment of pulmonary arterial hypertension (PAH).  Mast obtained the rights to the AIR001 program through its acquisition of privately-held Aires Pharmaceuticals, Inc. earlier this year. In the primary efficacy analysis of the Phase 2 study, all doses showed improvement in median pulmonary vascular resistance (PVR). In the secondary efficacy analysis, all doses showed improvements in the median distances obtained in the 6-minute walk test, including clinically-meaningful improvements at the highest dose level.  Additionally, AIR001 was well-tolerated, with no treatment related serious adverse events. In particular, methemoglobin levels remained normal (< 1.5%), which distinguishes AIR001 from safety concerns associated with intravenously-administered nitrite.  Edwin L. Parsley, D.O., interim Chief Medical Officer, stated: "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." Brian M. Culley, Chief Executive Officer, stated: "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." About the Phase 2 Study (AIR001-CS05) The Phase 2 study was a multi-center, open-label, randomized, parallel-dose study to determine the safety and efficacy of AIR001 in subjects with PAH.  Subjects were randomized into one of three treatment arms and treated with AIR001 for 16 weeks: 80 mg once daily after a 2-week "run-in" period of 46 mg once daily; 46 mg four times daily after a 2-week run-in period of 46 mg four times daily; or 80 mg four times daily after a 2-week run-in period of 46 mg four times daily.  The primary objective of the study was to evaluate the efficacy of inhaled nebulized AIR001 as determined by change in pulmonary vascular resistance (PVR) from baseline to week 16, measured immediately post-completion of AIR001 nebulization.  Secondary endpoints included change from baseline to week 16 in: 6‑Minute Walk Distance (6MWD) assessed immediately post-completion of AIR001 nebulization (peak), but no more than 40 minutes after completion of AIR001 nebulization; hemodynamic measurements of cardiac output, mean right atrial pressure and pulmonary capillary wedge pressure at peak; N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP); hemodynamics and 6MWD at trough; and quality of life measures. The study was powered to enroll 90 patients, however, prior to its acquisition by Mast, Aires discontinued the study due to capital constraints.  Data is available from 29 patients who enrolled in the study.
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