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A312.5
ACE-031 Research
Mostly mechanism / observationalLimited safety
5 peer-reviewed studies
What the evidence says
Mostly mechanism / observational
Most ACE-031 studies are mechanism or observational rather than RCTs that measure a clinical effect — keep findings provisional.
Most evidence is from mixed-quality randomised trials published 2001–2026.
Based on 5 studies · 2 RCTs
Confidence
Low
By outcome
Lean body mass & muscle growth
Mostly mechanism / observational5 studies
Safety profile
Too few graded studies2 studies
Muscle strength & power
Too few graded studies1 study
Therapeutic & clinical
Too few graded studies1 study
Steady research
1 study in the last 5 years
200120132026
1RCT2013
Single-dose ACE-031 treatment was generally well-tolerated and resulted in increases in muscle mass in healthy postmenopausal women.
Attie KM, Borgstein NG, Yang Y, Condon CH, Wilson DM, Pearsall AE, Kumar R, Willins DA, Seehra JS, Sherman ML. · Muscle Nerve (2013)
The pivotal human pharmacodynamic study: a double-blind, placebo-controlled single-ascending-dose trial in 48 healthy postmenopausal women given ACE-031 (0.02-3 mg/kg subcutaneously) or placebo
Statistically significant increases in mean total-body lean mass (3.3%; P=0.03 by DXA) and thigh-muscle volume (5.1%; P=0.03 by MRI) at day 29 in the 3 mg/kg group
Generally well tolerated; the main adverse event was injection-site erythema; mean half-life was 10-15 days
The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias.
Campbell C, McMillan HJ, Mah JK, Tarnopolsky M, Selby K, McClure T, Wilson DM, Sherman ML, Escolar D, Attie KM. · Muscle Nerve (2017)
The pivotal patient trial — randomized, double-blind, placebo-controlled, ascending-dose — in ambulatory boys with Duchenne muscular dystrophy, dosed subcutaneously every 2-4 weeks
HALTED after the second dosing regimen because of bleeding-related safety concerns: epistaxis (nosebleeds) and telangiectasias (dilated surface blood vessels); gum bleeding was also reported
Efficacy showed only non-significant trends: maintained 6-minute-walk distance, increased lean body mass and bone mineral density, and reduced fat mass — none statistically significant
By the end of treatment, mean body weight of the ACE-031 group was 16% greater than that of the control group, and wet weights of soleus, plantaris, gastrocnemius, and extensor digitorum longus muscles increased by 33, 44, 46 and 26%, respectively.
Foundational preclinical efficacy study: C57BL/6 mice treated with ACE-031 or vehicle for 28 days
Mean body weight was 16% greater than controls; individual muscle wet weights increased 26-46%
Muscle growth occurred independent of fibre type — distinct from myostatin-only inhibition, which predominantly targets type II fibres; fibre cross-sectional area rose in both type I and II fibres
Marmosets administered ACE-031 were greater at euthanasia compared to baseline; this was not observed in the vehicle-treated controls.
Cadena SM, Bogdanovich S, Khurana TS, Pullen A, Pearsall RS, Curran E, Faucette R, Lane J, Seehra J, Lachey JL, Mizener AD, Pistilli EE. · PLoS One (2026)
Non-human-primate efficacy study: marmosets given ACE-031 or vehicle for 14 weeks, with body composition tracked weekly and muscle assessed terminally
Significant time × treatment interaction for lean body mass — ACE-031 animals gained lean mass while controls did not
Biceps brachii showed increased cross-sectional area of both type I and type II fibres; EDL muscle showed increased absolute and specific force production
Independent transgenic mouse lines for each construct exhibited dramatic increases in muscle mass comparable to those seen in myostatin knockout mice.
Lee SJ, McPherron AC. · Proc Natl Acad Sci U S A (2001)
Mechanistic foundation: purified myostatin binds the activin type II receptors ActRIIB (and, less, ActRIIA), and this binding is inhibited by follistatin and the myostatin propeptide
Transgenic mice expressing follistatin or a dominant-negative ActRIIB in muscle showed dramatic hypertrophy comparable to myostatin-knockout mice
Establishes ActRIIB as the receptor that ACE-031's soluble decoy is designed to mimic and block — the scientific basis for the whole approach