Wir verwenden standardmäßig essenzielle Cookies (Anmeldung, deine gespeicherten Ziele/Stacks). Mit deiner Erlaubnis aktivieren wir außerdem datenschutzfreundliche Analytik (Vercel Web Analytics, anonyme Ladezeit-Metriken) und Fehler-Replay-Diagnostik (Sentry — DOM-Snapshots nur, wenn ein Fehler auftritt), damit wir Bugs schneller beheben können. Mehr über Cookies erfahren
A312.5
ACE-031 – Forschung
Überwiegend Mechanismus / Beobachtung
8 begutachtete Studien
Was die Evidenz sagt
Überwiegend Mechanismus / Beobachtung
Die meisten Studien zu ACE-031 sind mechanistisch oder beobachtend statt RCTs, die einen klinischen Effekt messen — betrachte die Ergebnisse als vorläufig.
Die meiste Evidenz stammt aus mittelwertigen randomisierten Studien, veröffentlicht 2001–2026.
Basierend auf 8 Studien · 2 RCTs
Konfidenz
Geringe Konfidenz
Nach Outcome
Lean body mass & muscle growthEine einzelne Humanstudie zeigte eine erhöhte Magermasse, doch die Phase-2-Studie wurde wegen Blutungs-/Gefäßsicherheitsbedenken abgebrochen; Muskelzuwächse stammen größtenteils aus Tierversuchen. · Nicht etabliert (Entwicklung eingestellt)
Überwiegend Mechanismus / Beobachtung6 Studien
Muscle strength & powerEine einzelne Humanstudie zeigte eine erhöhte Magermasse, doch die Phase-2-Studie wurde wegen Blutungs-/Gefäßsicherheitsbedenken abgebrochen; Muskelzuwächse stammen größtenteils aus Tierversuchen. · Nicht etabliert (Entwicklung eingestellt)
Zu wenige bewertete Studien2 Studien
Therapeutic & clinical
Zu wenige bewertete Studien2 Studien
Safety profile
Zu wenige bewertete Studien2 Studien
Aktives Forschungsgebiet
4 Studien in den letzten 5 Jahren
200120132026
1Systematische Übersicht2026
We further discuss the implications of MSTN in muscle hypertrophy, inflammation, and sports performance, highlighting future research directions in precision sports genomics and translational muscle biology.
Chandel S, Uvarajan D, Iyer M, Reddy DH, Siama Z, Giridharan B, Narayanasamy A, M J, Wander A, Vellingiri B. · Comprehensive Physiology (2026)
Specific polymorphisms, including rs1805086 and rs11333758, have been associated with variations in muscle strength, hypertrophy, and endurance capacity.
Despite extensive research, a comprehensive evaluation of the relationship between MSTN signaling, skeletal muscle mass, and athletic performance remains limited.
This review provides an integrated overview of myogenesis, MSTN-mediated signaling pathways, genetic polymorphisms, endocrine interactions, and therapeutic modulation strategies.
Pathways such as SMAD signaling, in which SMAD moves to the nucleus by making a complex and leads to tissue fibrosis in CKD, STAT3, which drives renal fibroblast activity and the production of ECM, Kidney injury molecule (KIM-1) in the synthesis, deposition of ECM proteins, SERCA2a (sarcoplasmic reticulum Ca 2+ ATPase) in cardiac dysfunction, and NF-κB (Nuclear factor kappa-light-chain-enhancer of activated B cells) in inflammation are involved in Act A signaling, have also been discussed.
Kundra S, Kaur R, Pasricha C, Kumari P, Gurjeet Singh T, Singh R. · International immunopharmacology (2024)
Act A also governs the immunological and inflammatory responses of the body, as well as cell death.
Moreover, Act A levels have been observed to elevate in several disorders like renal fibrosis, CKD, asthma, NAFLD, cardiovascular diseases, cancer, inflammatory conditions etc.
This study contributed in fully comprehending the significance of genetic and epigenetic alterations in BMP receptors and BMP signaling in metastatic breast cancer cells for the development of breast cancer treatment plans.
Hermawan A, Putri H. · Biochemical genetics (2024)
Oncoprint analysis showed genetic alterations in BMPR1A (39%), BMPR1B (13%), BMPR2 (34%), ACVR2A (14%), ACVR1 (7%), ACVR2B (13), ACVR1B (35%), HJV (40%), and ENG (33%) across the patients with breast cancer in The Metastatic Breast Cancer Project.
Functional mutation prediction showed that mutants in BMPR2 (R272L, E274K, and L685F), ACVR2A (S127L), and ACVR1B (R484H), are deleterious, probably damaging, and possess a cancer phenotype.
ROC plot revealed no BMP receptors correlated with endocrine therapy sensitivity.
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