US-based Mast Therapeutics has begun its Phase II trial of new drug vepoloxamer for patients with chronic heart failure.
The company uses molecular adhesion and sealant technology (MAST) to develop new therapies for sickle cell disease, heart failure and stroke.
The randomised, double-blind and placebo-controlled trial will evaluate a new form of vepoloxamer in ambulatory patients, aged between 18 and 74.
Assessed patients will have been diagnosed with chronic heart failure and on a guideline-based medication regimen for at least four weeks.
They will also have a left ventricular ejection fraction (LVEF) documented at less than 35% in the last 12 months, and an elevated cardiac troponin level.
After being randomised into one of three study arms, 150 patients will receive one of two dose levels of vepoloxamer, or the placebo control.
Doses will be administered over 3h in an outpatient setting or short-stay inpatient unit, depending on local practice and resource availability.
Mast Therapeutics chief medical officer Dr Edwin Parsley said: "Previously announced positive results from multiple randomised, 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 3h in an outpatient setting, as opposed to the 49h 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."
Evaluation of echocardiagrams and blood-based laboratory markers will determine whether vepoloxamer would benefit damaged heart muscle cells.
Phase II will also assess the safety and pharmacokinetics of vepoloxamer in chronic heart failure patients compared to a placebo.
Mast Therapeutics chief executive officer Brian Culley said: "We designed the new formulation to be more suitable for heart failure patients and have filed a provisional patent application."
Vepoloxamer is also being tested in a Phase III trial to treat vaso-occlusive crisis in patients with sickle cell disease.
Next year, the company intends to initiate clinical development of vepoloxamer for treating ischemic strokes.