Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.

Mark McIntosh,'s picture
PubMed URL:
Attie KM
Author List: 
Campbell C
McMillan HJ
Mah JK
Tarnopolsky M
Selby K
McClure T
Wilson DM
Sherman ML
Escolar D
Attie KM
Muscle Nerve
PubMed ID: 
ACE-031 is a fusion protein of activin receptor type IIB and IgG1-Fc, which binds myostatin. It aims to disrupts its inhibitory effect on muscle development and provide potential therapy for myopathies like Duchenne muscular dystrophy (DMD).ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending dose trial. The primary objective was safety evaluation. Secondary objectives included characterization of pharmacokinetics and pharmacodynamics.ACE-031 was not associated with serious or severe adverse events. The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias. A trend for maintenance of the 6 minute walk test (6MWT) distance in ACE-031 groups compared to decline in placebo (not statistically significant) was noted, as was a trend for increased lean body mass, bone mineral density (BMD), and reduced fat mass.ACE-031 demonstrated trends for pharmacodynamic effects on lean mass, fat mass, BMD, and 6MWT. Non-muscle-related adverse events contributed to the decision to discontinue the study. Myostatin inhibition is a promising therapeutic approach for DMD. This article is protected by copyright. All rights reserved.
Published Date: 
July, 2016

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