A Phase I, Single Ascending Dose Study of Cimaglermin Alfa (Neuregulin 1β3) in Patients With Systolic Dysfunction and Heart Failure

• Main Authors: Daniel J. Lenihan, MD, Sarah A. Anderson, ACNP, Carrie Geisberg Lenneman, MD, Evan Brittain, MD, MSCI, James A.S. Muldowney, III, MD, Lisa Mendes, MD, Ping Z. Zhao, PhD, Jennifer Iaci, MS, Stephen Frohwein, MD, Ronald Zolty, MD, PhD, Andrew Eisen, MD, PhD, Douglas B. Sawyer, MD, PhD, Anthony O. Caggiano, MD, PhD
• Format: Article
• Language: English
• Published: Elsevier 2016-12-01
• Series: JACC: Basic to Translational Science
• Subjects: cardiac repair; growth factor; neuregulin; systolic dysfunction
• Online Access: http://www.sciencedirect.com/science/article/pii/S2452302X16301619
• ISSN: 2452-302X

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.