1. Locally acting ACE-083 increases muscle volume in healthy volunteers
- Author
-
Matthew L. Sherman, Barry Miller, Michael R. Gartner, Chad E. Glasser, Kenneth M. Attie, and Dawn Wilson
- Subjects
0301 basic medicine ,medicine.medical_specialty ,Neuromuscular disease ,Physiology ,Myostatin ,Muscle volume ,Placebo ,Muscle hypertrophy ,03 medical and health sciences ,Cellular and Molecular Neuroscience ,0302 clinical medicine ,Physiology (medical) ,Internal medicine ,medicine ,Muscular dystrophy ,Adverse effect ,biology ,business.industry ,medicine.disease ,030104 developmental biology ,Endocrinology ,biology.protein ,Neurology (clinical) ,business ,030217 neurology & neurosurgery ,Follistatin - Abstract
Introduction ACE-083 is a locally acting follistatin-based therapeutic that binds myostatin and other muscle regulators and has been shown to increase muscle mass and force in neuromuscular disease mouse models. This first-in-human study examined these effects. Methods In this phase 1, randomized, double-blind, placebo-controlled, dose-ranging study in healthy postmenopausal women, ACE-083 (50-200 mg) or placebo was administered unilaterally into rectus femoris (RF) or tibialis anterior (TA) muscles as 1 or 2 doses 3 weeks apart. Results Fifty-eight postmenopausal women were enrolled, 42 ACE-083 and 16 placebo. No serious adverse events (AE), dose-limiting toxicities, or discontinuations resulting from AEs occurred. Maximum (mean ± SD) increases in RF and TA muscle volume were 14.5% ± 4.5% and 8.9% ± 4.7%, respectively. No significant changes in mean muscle strength were observed. Discussion ACE-083 was well tolerated and resulted in significant targeted muscle growth. ACE-083 may have the potential to increase muscle mass in a wide range of neuromuscular disorders. Muscle Nerve 57: 921-926, 2018.
- Published
- 2018