Bimagrumab (BYM338) |
Human monoclonal antibody Activin receptor type 2B antagonist |
Sporadic inclusion body myositis (sIBM) [NCT02573467] Muscle wasting (atrophy) after hip fracture surgery [NCT02152761] Chronic obstructive pulmonary disease (COPD) patients with cachexia [NCT01669174] |
Novartis AG MorphoSys AG |
Bimagrumab withdrawn from Phase 2 trials for sIBM PMID: 25381300 |
Trevogrumab (REGN1033) |
Human monoclonal antibody Myostatin or activin blocker |
Sarcopenia [NCT01963598] sIBM [NCT03710941] |
Regeneron Pharmaceuticals Inc./Sanofi SA |
Regeneron canceled clinical trial of REGN2477 and REGN1033 for inclusion body myositis |
Garetosmab (REGN2477) |
Human monoclonal antibody Myostatin or activin blocker |
sIBM [NCT03710941] Fibrodysplasia ossificans progressiva (FOP) [NCT03188666] |
Regeneron Pharmaceuticals Inc./Sanofi SA |
Nearly 90% reduction in formation of new lesions in patients with FOP |
ACE-083 |
Follistatin-based fusion protein Myostatin or activin blocker |
Facioscapulohumeral muscular dystrophy Charcot-Marie-Tooth (CMT) disease [NCT03943290] |
Acceleron |
ACE-083 in patients with CMT did not show functional improvement in the Phase 2 trial. |
ACE-2494 |
GDF ligand trapping peptide Myostatin or activin blocker |
Healthy volunteers (for safety) [NCT03478319] |
Acceleron |
In April 2019, Acceleron discontinued ACE-2494, which was in Phase I development. |
Domagrozumab (PF06252616) |
Monoclonal anti-myostatin antibody Myostatin or activin blocker |
DMD [NCT02310763] |
Pfizer |
Pfizer announced, in August 2018, that the study did not meet the primary efficacy endpoint |
Reldesemtiv (CK-212710) |
Small molecule Slowing the rate of calcium release from the regulatory troponin complex |
Mobility limitation in old age [NCT03065959] Amyotrophic lateral sclerosis (ALS) [NCT03160898], spinal muscular atrophy [NCT02644668] |
Cytokinetics/Astella |
European Medicines Agency (EMA) granted orphan drug status to Cytokinetics’ Reldesemtiv for the treatment of ALS. |
ARM210 (S48168) |
Small molecule Binding to leaky RyR channels and repairs the leak |
RYR-1 myopathy [NCT04141670] |
ARMGO Pharma Inc. |
In 2018, the FDA granted orphan drug designation to ARMGO for ARM210 as a potential treatment for patients with RYR1-RM. |
AVGN7 |
rAAV6 (gene therapy) rAAV6:Smad7 contains a gene called SMAD7 |
IBM, cancer cachexia, etc. |
AAVogen Inc. |
In preclinical development |
Ligandrol (VK5211, LGD-4033) |
Small molecule Nonsteroidal oral selective androgen receptor modulator (SARM) |
Acute hip fracture study in patients 65 years or greater [NCT02578095] |
Viking Therapeutics |
Phase 2 study of VK5211 in patients recovering from hip fracture reported (1) dose-dependent increases in lean body mass and (2) 20-meter increase in 6MWD over placebo |
GSK2881078 |
Small molecule SARM |
Healthy older men and postmenopausal women [NCT02567773] Male subjects and postmenopausal female subjects with COPD and muscle weakness [NCT03359473] |
Glaxo-Smith-Kline |
GSK2881078 promotes a dose-dependent increase in lean body mass in healthy older men and women |
Enobosarm (MK-2866, ostarine) |
Small molecule SARM |
Subjects with non-small cell lung cancer [NCT01355497 & NCT01355484] |
GTx Inc. |
Clinical trials failed to meet the overall criteria for the co-primary responder endpoints of lean body mass and physical function |
Nicotinamide riboside |
Small molecule NAD+ booster |
Aging male subjects [NCT02950441] Sarcopenia/frailty in chronic kidney disease [NCT03579693] |
University of Birmingham University of Washington |
Phase 2, no results posted Phase 2, recruiting |
Nicotinic acid (Niacin) |
Small molecule NAD+ booster |
Myopathy patients [NCT03973203] Early-stage mitochondrial myopathy patients [NCT04538521] Older adults [NCT03325491] |
University of Helsinki |
Muscle strength and mitochondrial biogenesis increased in all subjects Active, not recruiting Failed to recruit enough participants |
ASP0367 (MA-0211) |
Small molecule PPARδ agonist |
Pediatric male patients with DMD [NCT04184882] |
Mitobridge/Astellas |
FDA grants fast track designation for development for the treatment of primary mitochondrial myopathies |
Urolithin A |
Small molecule Mitophagy enhancer |
Healthy adults, older adults [NCT04160312] |
Amazentis SA |
Urolithin A is safe and improves mitochondrial and cellular health in humans. |
Elamipretide |
Peptide Mitochondrial enhancer |
Primary mitochondrial myopathy subjects [NCT03323749] |
Stealth BioTherapeutics |
The study did not record its essential endpoints of changes in the 6MWD and primary mitochondrial myopathy symptom assessment total fatigue score. |