Mast Therapeutics says that top-line results from a Phase 2a study of its AIR001 sodium nitrite inhalation solution for the treatment of heart failure with preserved ejection fraction (HFpEF) demonstrated statistically significant change in pulmonary capillary wedge pressure (PCWP) during exercise compared to placebo, which was the primary endpoint.
Mast acquired AIR001 in early 2014 when it acquired Aires Pharmaceutical and later that year, the company announced positive results from a Phase 2 trial of AIR001 for the treatment of pulmonary arterial hypertension which had been terminated by Aires due to budget constraints.
Lead investigator Barry A. Borlaug of the Mayo Clinic commented, “Heart failure with preserved ejection fraction is a major public health problem that has no proven effective treatment, yet currently afflicts 2 to 3 million Americans. [. . .] The results observed with AIR001 in this study support our hypothesis that acute administration of nebulized inhaled sodium nitrite unloads the heart during exercise without excessive reduction in resting pressures or arterial blood pressure.”
Mast Therapeutics CEO Brian M. Culley said, “These results are an important step in validating our second asset and establishing the potential clinical utility of AIR001 in HFpEF. Mayo Clinic is a well-known leader in the characterization and treatment of heart failure and we thank Dr. Borlaug for working with us and leading this study. We look forward to advancing AIR001 in this area of high unmet medical need for which there is no FDA-approved therapy available. We also anticipate reporting interim data from a second investigator-sponsored Phase 2a study of AIR001 in HFpEF patients around the middle of this year.”
Read the Mast Therapeutics press release.
