Mast Therapeutics Announces Positive Top-Line Results From Phase 2a Study Of AIR001 In Patients With Heart Failure With Preserved Ejection Fraction (HFpEF) Conducted At Mayo Clinic

SAN DIEGO., Mast Therapeutics, Inc. a biopharmaceutical company developing novel clinical-stage therapies for sickle cell disease and heart failure, today reported positive top-line results from a blinded Phase 2a study of AIR001 for the treatment of heart failure with preserved ejection fraction (HFpEF) conducted at Mayo Clinic by lead investigator Barry A. Borlaug, M.D., Associate Professor of Medicine and Director, Circulatory Failure Research, Division of Cardiovascular Diseases, Department of Internal Medicine. In the Phase 2a study, AIR001 showed statistically significant improvement for the pre-specified primary endpoint: change in pulmonary capillary wedge pressure (PCWP) at 20 Watts exercise after drug treatment relative to PCWP at 20 Watts exercise in the initial assessment prior to drug treatment, compared to placebo-treated patients. Study data show that nebulized AIR001 attenuates the hemodynamic derangements of cardiac failure that occur during exercise in HFpEF patients. AIR001 was generally well-tolerated. Detailed study results are expected to be submitted for presentation at a scientific conference later this year.

"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," stated Dr. Borlaug. "One factor that complicates treatment is that the hemodynamic perturbations causing morbidity such as high filling pressures and low cardiac output are typically present only intermittently—being absent at rest but observed during stress such as exercise. As such, an ideal therapy would become more effective during stress, without untoward effects on resting cardiovascular function. 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."

"These results are an important step in validating our second asset and establishing the potential clinical utility of AIR001 in HFpEF," stated Brian M. Culley, Chief Executive Officer of Mast Therapeutics. "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," continued Mr. Culley. "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."

Source: Mast Therapeutics, Inc.