Efmitermant alfa

Efmitermant alfa (ACE-083) is a locally acting, follistatin-based fusion protein. Efmitermant alfa also is a muscle-promoting agent. Efmitermant alfa can be used for the research of muscle disorders[1][2].

Research Areas >> Others

Signaling Pathways >> Others >> Others

ACE-083 (0-60 nM) 对肝素和细胞外基质具有高亲和力[1]。 ACE-083（0- 20 µg/mL）结合并有效中和肌肉生长抑制素、激活素 A、激活素 B 和生长分化因子 11 (GDF11) [1]。

ACE-083（肌肉注射；0-100 µg；每周两次，持续 4 周）在野生型小鼠和 Charcot-Marie-Tooth (CMT) 病和 Duchenne 肌营养不良症小鼠模型中引起注射肌肉的局部、剂量依赖性肥大 [1]。 ACE-083（肌肉注射；0-100 µg；每周两次，持续 4 周）还增加了野生型小鼠和疾病模型中注射的胫骨前肌的原位等长收缩力，并增加了 CMT 小鼠的踝关节背屈扭矩[1]。 Animal Model: Wild-type mice; CMT mice; mdx mice[1] Dosage: 1, 3, 10, 30 and 100 μg Administration: intramuscular; twice weekly for 4 weeks Result: Increased the weight of the injected muscle in a dose-dependent manner and caused focal growth of skeletal muscle in wild-type mice without evidence of systemic effects. Caused focal muscle hypertrophy accompanied by increased generation of absolute force, increased ankle dorsifexion torque and a benefcial change in a major biomarker of muscle atrophy. Caused focal growth of the injected muscle and increased generation of absolute force.

[1]. R S Pearsall, et al. Follistatin-based ligand trap ACE-083 induces localized hypertrophy of skeletal muscle with functional improvement in models of neuromuscular disease. Sci Rep. 2019 Aug 6;9(1):11392.  

[2]. Jeffrey M Statland, et al. Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy. Muscle Nerve