In this randomized, double-blind, placebo-controlled, ascending dose trial, ACE-03, a myostatin inhibitor, was administered subcutaneously every 2-4 weeks to Duchenne muscular dystrophy (DMD). boys. The primary objective was safety evaluation and secondary objectives included characterization of pharmacokinetics and pharmacodynamics. ACE-031 was not associated with serious or severe adverse events. However, 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.
