A first-in-human, phase 1, double blind, placebo-controlled, single ascending dose study examined the safety, tolerability, and exploratory efficacy of intravenous infusion of a recombinant growth factor, cimaglermin alfa, in patients with heart failure and left ventricular systolic dysfunction (LVSD). In these patients on optimal guideline-directed medical therapy, cimaglermin treatment was generally tolerated except for transient nausea and headache and a dose-limiting toxicity was noted at the highest planned dose. There was a dose-dependent improvement in left ventricular ejection fraction lasting 90 days following infusion. Thus, cimaglermin is a potential therapy to enhance cardiac function in LVSD and warrants further investigation.
Keywords: AE, adverse event; AUC, area under the curve; DLT, dose-limiting toxicity; GGF, glial growth factor; HF, heart failure; LVEF, left ventricular ejection fraction; LVSD, left ventricular systolic dysfunction; NRG, neuregulin; NYHA, New York Heart Association functional class; TEAE, treatment-emergent adverse event; cardiac repair; growth factor; neuregulin; systolic dysfunction.