Molecular profile
- Entry type
- Peptide research profile
ACE-083
ACE-083 is a locally acting fusion protein built by Acceleron Pharma from the follistatin FS344 domain joined to an IgG2 Fc region.
Dosing & scaling, explained
Amounts reported for ACE-083 are listed under Reported protocols, each with its source and study kind. No amount ladder is inferred from them. This is educational source context, not a protocol or recommendation.
No source-supported amount ladder is shown
Enter a target amount only from your own reviewed source. The calculator can convert it into concentration, volume and device markings, but it will not infer an amount from product availability or another compound.
Only mcg or mg amounts are retained. The calculator derives mL, syringe units or pen clicks from the current vial strength, water volume and device.
No independently verified schedule
Clinical context
Human study rows are described per source under Reported protocols; they belong to their trial populations and are not a general amount.
Storage, handling and interpretation
Product identity
Verify the exact compound, formulation and amount against the product label or certificate before calculation.
Storage & handling
Follow the product label, certificate and validated laboratory SOP; no universal storage assumption is applied here.
Prepared solutions
Label concentration and preparation date, use sterile technique where applicable, and follow a validated stability protocol.
Reported protocols for ACE-083
What published studies and recent community write-ups report about amounts, timing and course length. 7 clinical and 1 preclinical rows, each linked to its source. These are descriptions of what others report, not instructions, and nothing here is medical advice.
| Context | Dose | Frequency | Route | Course | Source |
|---|---|---|---|---|---|
| Clinical7 rows | |||||
| Phase 1 first-in-human dose-ranging study58 healthy postmenopausal women, 42 on ACE-083 and 16 on placebo | 50-200 mg per muscle | One dose, or two doses 3 weeks apart | Intramuscular injection into the rectus femoris or the tibialis anterior, one side only | Up to 3 weeks between the two dosesNot applicable | Muscle and Nerve (PubMed Central)2018 |
| There were no serious adverse events, no dose-limiting toxicities and no withdrawals for adverse events. The largest mean volume increases were 14.5% in the rectus femoris and 8.9% in the tibialis anterior. No significant change in mean muscle strength was observed, which is the pattern that repeated in every later trial: the muscle got bigger without getting stronger. | |||||
| Registry record for the phase 1 study58 healthy subjects | 50-200 mg per muscle | One or two doses | Intramuscular | Single-cycle studyNot applicable | ClinicalTrials.gov2014 |
| The registry lists this first-in-human study as completed with 58 participants under Acceleron Pharma, which the entry now records as a Merck subsidiary. It is the only ACE-083 record of the four on the registry that was not terminated. | |||||
| Phase 2 trial in facioscapulohumeral muscular dystrophy37 adults in the open-label part 1 and 58 in the randomised part 2, all with FSHD1 or FSHD2 | 150, 200 or 240 mg per muscle in part 1; 240 mg per muscle in part 2 | Once every 3 weeks | Intramuscular, bilaterally into the biceps brachii or the tibialis anterior | 27 weeks blinded, that is nine doses, followed by 24 weeks open-label at the same amountNone described within the trial | Muscle and Nerve (PubMed Central)2022 |
| Total muscle volume rose 16.4% against placebo in the biceps group and 9.5% in the tibialis anterior group, contractile muscle volume rose in both and fat fraction fell in the tibialis anterior. There were no consistent improvements in function, strength or patient-reported outcomes over up to 12 months of treatment. Mild to moderate injection-site reactions were the commonest adverse event. The paper states that the FSHD programme was discontinued on these results. | |||||
| Registry record for the FSHD trial95 patients with facioscapulohumeral muscular dystrophy | 240 mg per muscle in the randomised part; 150 and 200 mg per muscle in the dose-escalation part | Once every 3 weeks | Intramuscular, bilaterally | Roughly 6 months of blinded dosing, nine dosesTerminated | ClinicalTrials.gov2016 |
| The registry records this trial as terminated with the reason given as the study being discontinued because it did not achieve functional secondary endpoints, with a completion date of 9 October 2019. | |||||
| Phase 2 trial in Charcot-Marie-Tooth disease63 adults with CMT1 or CMTX; 18 in open-label part 1 and 44 treated in randomised part 2 | 240 mg per muscle | Once every 3 weeks | Intramuscular, bilaterally into the tibialis anterior | 6 months blinded, then a 6-month open-label extensionNone described within the trial | Neurology (PubMed Central)2022 |
| Total muscle volume rose 13.5% against placebo and contractile muscle volume and ankle dorsiflexion strength also separated from placebo, but fat fraction and every other functional outcome did not improve. The authors conclude that despite the volume gain, patients did not show greater functional improvement than those on placebo. Injection-site reactions were the commonest adverse event and there were no grade 3 or worse events in part 1. The trial is registered as NCT03124459, which the registry lists as terminated because the investigation was discontinued after the functional secondary endpoints were missed. | |||||
| Sponsor announcement discontinuing the programme44 randomised patients with Charcot-Marie-Tooth disease in part 2 of the trial | The optimal dose level selected in part 1, injected into the tibialis anterior once every 3 weeks | Once every 3 weeks | Intramuscular | 3-month dose escalation, then 6 months blinded and 6 months open-labelProgramme ended | Acceleron Pharma press release filed with the US Securities and Exchange Commission2020 |
| On 9 March 2020 Acceleron reported that the increase in mean total muscle volume was statistically significant but did not translate into a significant improvement in any functional or quality-of-life secondary endpoint, and that it was therefore discontinuing development of ACE-083. The chief executive described the myostatin-plus hypothesis as not having produced the functional outcomes needed for clinically meaningful benefit in neuromuscular disease. Adverse events were mostly mild to moderate and largely injection-site related. | |||||
| Registry record for the long-term extension study62 patients with FSHD or CMT rolling over from the two phase 2 trials | 240 mg per muscle | Every 4 weeks in the loading phase, then randomised to every 4 or every 8 weeks | Intramuscular, bilaterally | 6-month loading phase, then a maintenance phase planned for 24 monthsTerminated in March 2020 | ClinicalTrials.gov2019 |
| This extension is the only place a maintenance schedule for ACE-083 was described, with patients randomised between a 4-weekly and an 8-weekly interval. The registry records it as terminated because the functional secondary endpoints were not achieved in the parent trials, ending on 11 March 2020, two days after the sponsor's announcement. | |||||
| Preclinical1 row | |||||
| Mouse models that justified local injectionWild-type mice and mouse models of Charcot-Marie-Tooth disease and Duchenne muscular dystrophy | Up to 100 mcg per injection, the highest amount tested | Twice weekly in the 4-week tibialis anterior experiment | Intramuscular injection into the gastrocnemius or tibialis anterior | 4 weeks in the repeated-dosing experimentNot applicable | Scientific Reports (PubMed Central)2019 |
| ACE-083 was shown to bind heparin and extracellular matrix strongly, which is the design feature meant to keep it in the muscle it is injected into, and to neutralise myostatin, activin A, activin B and GDF-11 in surface plasmon resonance and cell assays. At 100 mcg the injected gastrocnemius gained 85% mass against vehicle, and twice-weekly dosing for 4 weeks raised injected tibialis anterior weight by roughly 75%, with no effect on other muscles, body weight or follicle-stimulating hormone. Isometric force in situ and ankle dorsiflexion torque also rose in CMT mice. That functional gain in mice is exactly what the human trials failed to reproduce. | |||||
How a course is commonly described
- Dose findingClinical
Both phase 2 trials opened with an open-label dose-escalation part, 150 to 240 mg per muscle given unilaterally or bilaterally every 3 weeks for up to five doses, to pick the amount used in the randomised part. 240 mg per muscle was the figure selected both times.
- Months 1-6, blinded treatmentClinical
240 mg per muscle bilaterally every 3 weeks, nine doses, against placebo. This is where the muscle volume gain appeared and separated clearly from placebo on MRI.
- Months 7-12, open-labelClinical
Everyone continued on ACE-083 at the same amount for a further 6 months. Up to 12 months of treatment still produced no consistent improvement in function, strength or patient-reported outcomes in the FSHD trial.
- Maintenance, planned but endedClinical
The extension study planned 24 months of maintenance dosing at 240 mg per muscle, randomised between a 4-weekly and an 8-weekly interval. It was terminated on 11 March 2020 when the programme was discontinued.
- ACE-083 has no PubChem compound record because it is a recombinant fusion protein, the follistatin FS344 domain joined to an IgG2 Fc region, not a small molecule or a synthesisable peptide. PubChem does hold a record carrying ACE-083 as a synonym, CID 14293713, but that is a 345 g/mol piperidine small molecule with an unrelated CAS number and nothing to do with this compound; the chemistry card here is deliberately empty rather than wrong.
- Every amount above is per muscle and given directly into that muscle. The compound was engineered to bind extracellular matrix and stay put, so these figures describe a local injection into a single named muscle and bear no relation to a systemic dose.
- This is a discontinued programme with a consistent result: the injected muscle grew in healthy volunteers, in FSHD and in CMT, and in none of the three did strength or function follow. The sponsor stopped development on 9 March 2020. ACE-083 has never been approved by any regulator, and follistatin-based myostatin binders are prohibited in sport under WADA section S4.3.
Sources for this section (9)
- Locally acting ACE-083 increases muscle volume in healthy volunteers
- Phase 1 Study of ACE-083 in Healthy Subjects (NCT02257489)
- Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
- Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (NCT02927080)
- Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
- Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (NCT03124459)
- Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Charcot-Marie-Tooth Disease
- Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With FSHD and Charcot-Marie Tooth Disease Types 1 and X (NCT03943290)
- Follistatin-based ligand trap ACE-083 induces localized hypertrophy of skeletal muscle with functional improvement in models of neuromuscular disease
Overview
ACE-083 is a locally acting fusion protein built by Acceleron Pharma from the follistatin FS344 domain joined to an IgG2 Fc region, designed to be injected into one muscle and stay there, where it neutralises myostatin, activin A, activin B and GDF-11. PubChem has no compound record for it: it is a recombinant protein, not a small molecule, so this page carries no formula, molar mass or structure, and a PubChem entry that happens to carry the synonym ACE-083 is an unrelated 345 g/mol small molecule rather than this protein. Across a phase 1 study and two phase 2 trials the injected muscles reliably grew, by 9% to 16% in volume, but strength, walking tests and quality-of-life scores did not follow, and Acceleron announced on 9 March 2020 that it was discontinuing the programme.
What researchers are exploring
These themes describe recurring questions in published research. They are not claims of effectiveness or recommendations for use.
- 1
Muscle & performance
Androgen receptor modulators, myostatin pathways and exercise-mimetic research.
- 2
Phase 1 first-in-human dose-ranging study
There were no serious adverse events, no dose-limiting toxicities and no withdrawals for adverse events. The largest mean volume increases were 14.5% in the rectus femoris and 8.9% in the tibialis anterior. No.
- 3
Registry record for the phase 1 study
The registry lists this first-in-human study as completed with 58 participants under Acceleron Pharma, which the entry now records as a Merck subsidiary. It is the only ACE-083 record of the four on the registry that.
- 4
Phase 2 trial in facioscapulohumeral muscular dystrophy
Total muscle volume rose 16.4% against placebo in the biceps group and 9.5% in the tibialis anterior group, contractile muscle volume rose in both and fat fraction fell in the tibialis anterior. There were no.
- 5
Registry record for the FSHD trial
The registry records this trial as terminated with the reason given as the study being discontinued because it did not achieve functional secondary endpoints, with a completion date of 9 October 2019.
- 6
Phase 2 trial in Charcot-Marie-Tooth disease
Total muscle volume rose 13.5% against placebo and contractile muscle volume and ankle dorsiflexion strength also separated from placebo, but fat fraction and every other functional outcome did not improve. The authors.
What the current evidence can support
Study models remain visible so laboratory and animal findings are not mistaken for established human outcomes.
- Human
Human studies and registrations
7 protocol rows on this page come from human trials, trial registrations, regulator labels or reviews of human data. Approval status and populations are stated per row.
- Animal
Preclinical models
1 protocol row describe animal, cell or toxicology work. Amounts from these rows do not translate to people.
Identity, limitations and unknowns
Important limitations
- Every amount on this page is a report of what a source describes, not a protocol to follow.
- Preclinical and community rows do not establish a human amount, schedule or route.
- Formulation, purity and presentation vary by supplier and are not covered by the dossier.
Sources
8 curated references, each with its journal, year and study model.
- Locally acting ACE-083 increases muscle volume in healthy volunteers
- Phase 1 Study of ACE-083 in Healthy Subjects (NCT02257489)
- Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
- Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (NCT02927080)
- Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
- Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (NCT03124459)
- Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Charcot-Marie-Tooth Disease
- Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With FSHD and Charcot-Marie Tooth Disease Types 1 and X (NCT03943290)