San Diego-based Mast Therapeutics, a clinical-stage biopharmaceutical company leveraging its molecular adhesion and sealant technology (MAST) platform to develop novel therapies for sickle cell disease, heart failure, and stroke, has initiated its phase II study of vepoloxamer for the treatment of patients with chronic heart failure.
Dr. Edwin Parsley, Mast’s chief medical officer, said, “Previously announced positive results from multiple randomized, placebo-controlled studies of vepoloxamer in a well-established model of chronic heart failure, including a repeat-treatment study, as well as recommendations from medical experts in the field, strongly support clinical development of vepoloxamer in this setting. If the promising results observed in non-clinical studies translate to patients with chronic heart failure, vepoloxamer may offer a novel way of directly improving left ventricle contractile function by restoring cardiomyocyte membrane integrity and increasing cardiomyocyte survival.
“Significantly, in this phase II study, vepoloxamer will be administered over three hours in an outpatient setting, as opposed to the 49-hour administration in our sickle cell disease studies, which will help demonstrate its practical utility for chronic heart failure patients. We intend to collect echocardiographic data on parameters such as ejection fraction as well as biomarkers associated with clinical outcomes such as ultra-high sensitivity troponin I and NT-proBNP, as these metrics were improved in a statistically significantly manner in previously announced heart failure models.”
Mast CEO Brian Culley noted, “We are pleased to have initiated this 150-patient phase II study, which, notably, is testing a new formulation of vepoloxamer. We designed the new formulation to be more suitable for heart failure patients and have filed a provisional patent application. We now have multiple pending patent applications, including a new formulation application and a new composition of matter application, which would provide market protection for vepoloxamer in the setting of heart failure. In addition, the new formulation is distinct from our sickle cell formulation, which may provide important commercial advantages.”
The phase II study is a randomized, double-blind, placebo-controlled, multicenter study (both within and outside the U.S.) of a new formulation of vepoloxamer in ambulatory patients, ages 18 to 74, diagnosed with chronic heart failure (New York Heart Association functional Class II or III) who are stable and well-managed, on an established guideline-based medication regimen for at least four weeks, have a documented left ventricular ejection fraction (LVEF) ≤35% as the most recent value within the last 12 months, and have an elevated cardiac troponin I (cTnI) blood level. About 150 patients will be randomized into one of three approximately 50-patient study arms and receive one of two dose levels of vepoloxamer or the placebo control, administered in addition to standard medical therapy as a single intravenous infusion over three hours in an outpatient setting or short-stay inpatient unit depending on local practice and resource availability.
The purpose of the clinical study is to evaluate whether vepoloxamer can provide both a functional and biochemical benefit to damaged heart muscle cells, which will be evaluated by measurement of echocardiograms and blood-based laboratory markers, and to evaluate the safety and pharmacokinetics of vepoloxamer in chronic heart failure patients compared to placebo control.