Table 1. Summary of clinical development of myostatin inhibitors for treatment of muscle wasting associated with cancer and other disorders.

Name	Type of inhibitor	Sponsor	Condition	Patient population	Phase of development	Current status	Outcome	CT identifier/Ref.
LY2495655	Myostatin antibody	Lilly	–	Healthy volunteers	Phase 1 SAD	Completed	Well tolerated, increased TMV	[66▪▪]
			–	Healthy Japanese volunteers	Phase 1; SAD, MAD	Completed		NCT01341470
			Muscular atrophy	Hip arthroplasty	Phase 2	Actively recruiting		NCT01369511
			Muscle weakness	Older weak fallers	Phase 2	Active, not recruiting		NCT01604408
			Advanced cancer	Cancer patients	Phase 1	Active, not recruiting	Well tolerated, increased muscle volume (interim report)	[66▪▪]; NCT01524224
			Advanced cancer	Pancreatic cancer	Phase 2	Recruiting		NCT01505530
MYO-029	Myostatin antibody	Wyeth	–	Healthy volunteers	Phase 1	Completed		NCT00563810
			Adult muscular dystrophy	BMD; Facioscapulohumeral; muscular dystrophy; Limb-Girdle muscular dystrophy	Phase 1/2	Terminated	Increase in LBM; no effects on strength or function; skin hypersensitivity at highest doses	[70▪▪]; NCT00104078
ACE-031/Ramatercept	ActRIIb-Fc	Acceleron/Shire	Muscle loss	Healthy postmenopausal women	Phase Ia (SAD)	Completed	Generally well tolerated, increased LBM and TMV	[70▪▪]; NCT00755638
			Muscle atrophy	Healthy postmenopausal women	Phase Ib (MAD)	Terminated	Common AE: nosebleed. Increased LBM, TMV	Borgstein et al. 2010, WMS, Japan; NCT00952887
			DMD	DMD boys	Phase 2; MAD	Terminated	Reversible telangiectasia and nosebleed; increased LBM, attenuated TMV and 6MWD	[71▪▪]; NCT01239758; NCT01099761
PF-06252616	Myostatin antibody	Pfizer	–	Healthy volunteers	Phase I; SAD, MAD	Recruiting		NCT01616277
BYM338	ActRIIB antibody	Novartis	Muscle wasting	Healthy volunteers	Phase 1 SAD		Well tolerated, increase in TMV	D. Rook, Intl conference on Sarcopenia research, Orlando, Dec, 2012
			sIBM	sIBM	Phase 2; Single dose	Completed	Well tolerated, increase in TMV, LBM, quadriceps strength and 6MWD	Amato et al. MDA meeting San Diego, 2013; NCT01423110
			COPD	COPD patients with cachexia	Phase 2	Recruiting		NCT01669174
			Skeletal muscle	Sarcopenic adults	Phase 2	Recruiting		NCT01601600
			Cachexia	Cancer cachexia (lung or pancreas)	Phase 2	Recruiting		NCT01433263
REGN1033/SAR391786	Myostatin antibody	Regeneron/Sanofi	Rehabilitation postorthopedic surgery	Healthy volunteers	Phase 1 SAD, MAD	Active, not recruiting		NCT01507402 NCT01720576
FS344	Follistatin-AAV gene therapy	Nationwide Children's Hospital/Milo Biotech	BMD and SIBM	BMD and SIBM	Phase 1	Enrolling by invitation		NCT01519349
AMG-745	Myostatin peptibody	Amgen	Age-associated muscle loss	Age-associated muscle loss	Phase 2	Withdrawn prior to enrolment		NCT00975104

BMD, Becker muscular dystrophy; COPD, chronic obstructive pulmonary disease; DMD, Duchenne muscular dystrophy; CT identifier, clinical trial identifier at ClinicalTrials.gov; LBM, lean body mass ; MAD, multiple ascending dose; SAD, single ascending dose; sIBM, sporadic inclusion body myositis; TMV, thigh muscle volume; 6MWD, six minute walk distance.