Heart failure

Safe, tolerable, experimental mRNA injection for patients with heart failure


Injection of mRNA encoding vascular endothelial growth factor (VEGF-A) into the myocardium of patients undergoing elective coronary artery bypass grafting (CAP) was both safe and tolerable in patients with heart failure, based on the results of the EPICCURE Phase 2a trial. Called AZD8601, the mRNA injection is intended to help repair heart damage following a heart attack.

“Over a billion heart cells can be lost in a heart attack,” said Mene Pangalos, PhD, executive vice president of BioPharmaceuticals R&D at AstraZeneca, in a press release. “These early results indicate the potential of mRNA-based therapies to stimulate the production of VEGF-A to provide restorative and disease-modifying options for patients with heart failure and other ischemic vascular diseases.”

According to the researchers, VEGF-A is an important paracrine factor for the formation of new blood vessels. It has also been shown to stimulate the division of progenitor cells, descendants of stem cells which, when stimulated, differentiate to create specialized cell types that can help repair and regenerate the heart.

The randomized, double-blind, multicenter, placebo-controlled study observed 11 patients with stable coronary artery disease and moderately reduced left ventricular ejection fraction (LVEF) undergoing coronary artery bypass grafting. Seven of these patients received AZD8601 treatment and 4 received placebo injections.

The primary endpoint was safety and tolerability, which were achieved. The researchers also observed trends in 3 exploratory efficacy endpoints: LVEF, NT-proBNP (a biomarker measuring the level of a hormone, which is elevated in patients with heart failure) and the functional results reported by the patients, compared to placebo.