Table 2.
Status of myostatin/ActRII-attenuating drugs
| Drug, status | Identification | Stage | Participants, age, n | Results summary |
|---|---|---|---|---|
| ACE-031, suspended | NCT00755638 | P1 | Healthy, 45-75, 48 | Completed, no results posted |
| NCT00952887 | P1 | Healthy, 45-75, 70 | ||
| NCT01099761 | P2 | DMD, 4+, 35 | 39% TAEs, terminated | |
| ACE-083, suspended | NCT02257489 | P1 | Healthy, 45-75, 58 | No TAEs |
| NCT02927080 | P2 | FSHD, 18+, 58 | Muscle function unaffected | |
| NCT03943290 | P2 | FSHD and CMT, 18+, 150 | Terminated | |
| NCT03124459 | P2 | CMT, 18+, 42 | Terminated | |
| ACE-2494, suspended | NCT03478319 | P1 | Healthy, 45-75, 48 | Completed, no results |
| AAV1:FS344 | NCT01519349 | P1/2a | BMD, 18+, 6 | BMD: larger myofibers, possibly improved function (6MWT) |
| IBM, 18+, 9 | IBM: larger myofibers, improved function (6MWT) | |||
| NCT02354781 | P1/2 | DMD, 7, 3 | Completed, no DLTs. | |
| AVGN7 | PC | IBM | PoC complete, GLP/tox ongoing | |
| BIIB110 | No entry | ? | ||
| Bimagrumab (BYM338), unclear | NCT01423110 | P2 | IBM, 40-80, 14 | Same frequency of SAEs in treatment and placebo groups. Enhance LBM. No or minor effect on muscle function (6MWT, sIFA) |
| NCT02250443 | P2/3 | IBM, 40-75, 10 | ||
| NCT01925209 | P2/3 | IBM, 36-85, 251 | ||
| NCT02573467 | P3 | IBM, 36+, 211 | ||
| NCT01601600 | P2 | Sarcopenia, 65+, 40 | Increased measures of muscle mass, no or minor improvement of muscle function (eg, SPPB, 6MWT) | |
| NCT02333331 | P2 | Sarcopenia, 70+, 217 | ||
| NCT02468674 | P2 | Sarcopenia, 70+, 160 | ||
| NCT01669174 | P2 | COPD, 40-80, 67 | ||
| NCT01433263 | P2 | Cancer cachexia, 18+, 57 | Muscle mass and function unaffected | |
| NCT02152761 | P2 | Hip fracture, 60+, 251 | Increased LBM, function unaffected | |
| NCT03005288 | P2 | Obesity/T2DM, 18-75, 77 | Increased LBM, decreased fat mass, and improved insulin sensitivity | |
| Domagrozumab (PF-06252616), unclear | NCT01616277 | P1 | Healthy, 18-64, 86 | Completed, no results posted. Published (405) |
| NCT02841267 | P1/2 | LGMD2I, 18-99, 19 | No SAEs, no effect on multiple muscle function tests | |
| NCT02310763 | P2 | DMD, 6-15, 121 | No SAEs. Function unaffected | |
| NCT02907619 | P2 | DMD, 6-18, 59 | No efficacy. Terminated | |
| Landogrozumab (LY2495655), unclear | NCT01341470 | P1 | Healthy, 24-85, 47 | No SAEs |
| NCT01604408 | P2 | Older fallers, 75+, 201 | LBM preserved; muscle function improved (358) | |
| NCT01369511 | P2 | Hip arthroplasty, 50+, 400 | Small increase in LBM, muscle function unaffected (360) | |
| NCT01524224 | P1 | Advanced cancer, 18+, 29 | SAEs in 25%-67% of subjects in 5 highest dose groups (8 total) | |
| NCT01505530 | P2 | Pancreatic cancer, 18+, 125 | Muscle volume and function unaffected, survival concerns (359). Terminated | |
| PINTA 745 (AMG 745), suspended | No entry | P1 | Pancreatic cancer | Small LBM increase, no SAEs (364) |
| NCT01958970 | P1/2 | ESRD, 18-85, 51 | LBM unaffected | |
| NCT00975104 | P2 | Sarcopenia | Withdrawn | |
| RO7239361 (BMS-986089), suspended | NCT03100630 | P1 | Healthy | Completed, no results posted |
| NCT02515669 | P1/2 | DMD, 5-10, 43 | Failed preplanned futility analysis for efficacy. Terminated | |
| NCT03039686 | P2/3 | DMD, 6-11, 166 | Completed; no effect on muscle mass or strength | |
| SRK-015 | NCT03921528 | P2 | SMA, 2-21, 55 | Active, not recruiting |
| Stamulumab (MYO-029), suspended | NCT00563810 | P1 | Healthy, 18-80, 72 | Completed, no results posted |
| NCT00104078 | P1/2 | BMD/FSHD/LGMD, 18+, 108 | Completed, no results posted. Published (357) = SAEs same in treatment and placebo; no effect on muscle mass or strength | |
| Trevogrumab (REGN1033, SAR391786), unclear | NCT01507402 | P1 | Healthy, 18-85, 76 | Completed, no results posted |
| NCT01720576 | P1 | Healthy, 60+, 60 | ||
| NCT01910220 | P1 | Healthy, 60+, 125 | ||
| NCT02741739 | P1 | Healthy, 18-65, 28 | ||
| NCT02943239 | P1 | Healthy, 35-70, 82 | ||
| NCT01963598 | P2 | Sarcopenia, 70+, 253 | ||
| NCT03710941 | P2 | IBM | Withdrawn |
Data and results obtained from clinicaltrials.gov unless otherwise referenced.
Abbreviations: 6MWT, 6-minute walk test; BMD, Becker muscular dystrophy; CMT, Charcot-Marie Tooth disease; COPD, chronic obstructive pulmonary disease; DMD, Duchenne muscular dystrophy; ESRD, end-stage renal disease; FSHD, facioscapulohumeral muscular dystrophy; GLP/tox, good laboratory practices/toxicology; IBM, inclusion body myositis; LBM, lean body mass; LGMD, limb-girdle muscular dystrophy; P#, clinical trial phase; PC, preclinical; PoC, proof of concept; SAE, ; sIFA, sIBM physical functional assessment; SMA, spinal muscular atrophy; SPPB, short physical performance battery; SAE, serious adverse event; TAE, treatment-related adverse event; “suspended,” formally announced; T2DM, type 2 diabetes mellitus; “unclear”, not listed on corporate website’s pipeline