Mast announces plans for development of Vepoloxamer in heart failure

Consistent with prior guidance, earlier this year, Mast Therapeutics submitted an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA), together with the protocol for a Phase 2 clinical study in patients hospitalized for acute decompensated heart failure.

FDA has completed its 30-day safety review and informed the Company that it may proceed with its proposed clinical study. The Company is finalizing the study protocol and expects to begin recruiting patients in the Phase 2 study in the first half of 2015.

Brian M. Culley, Chief Executive Officer, said: "Since we first announced data from our nonclinical proof-of-concept study in the first quarter, vepoloxamer data has been presented at meetings of the European Society of Cardiology, the Heart Failure Society of America and the American Heart Association. Interest from the leading medical conferences and thought leaders in this field reflects the potentially novel way that vepoloxamer may improve heart failure.

We believe its membrane-sealing activity helps restore damaged cardiac cell membranes, thus minimizing calcium overload injury, preserving cardiomyocytes and directly improving heart contractility and function. In contrast to most approved drugs, which have not been shown to improve long-term outcomes, by salvaging cardiac tissue, vepoloxamer may have the potential to reduce the high rates of near-term hospital readmission and mortality associated with heart failure hospitalization."

Mr. Culley continued: "Our planned study will evaluate the safety and efficacy of multiple doses of vepoloxamer in patients hospitalized for acute decompensated heart failure, including its effect on markers of cardiac injury (troponin) and wall stress (NT-proBNP), as well as clinical outcomes. As demonstrated by our rapid progress in this area of significant unmet medical need, we continue to identify new ways to create additional value from our vepoloxamer franchise."

Heart failure is a chronic, progressive condition in which heart muscle is unable to pump sufficient blood to meet the body’s needs. It is estimated that more than 20 million worldwide, including five to six million in the U.S., suffer from heart failure, which is the most common diagnosis for hospital admission in the U.S. for patients over age 65.

The American Heart Association estimates that total medical costs of heart failure in the U.S. will increase from approximately $21 billion in 2012 to approximately $53 billion in 2030, with the majority (80%) of such costs related to hospitalization.

In March 2014, the Company announced that the United States Adopted Names (USAN) Council assigned "vepoloxamer" as the unique non-proprietary (generic) name for the active pharmaceutical ingredient (API) in MST-188.

The Company sought a unique name for its API to clearly identify it as different from non-purified poloxamers. In support of its application, the Company submitted proprietary data showing that drug products containing non-purified poloxamers may have serious toxicity consequences and should not be substituted for or confused with MST-188.