Chemistry:Apitegromab
From HandWiki
| Monoclonal antibody | |
|---|---|
| Type | ? |
| Target | Promyostatin |
| Clinical data | |
| Other names | SRK-015 |
| Identifiers | |
| CAS Number | |
| UNII | |
Apitegromab (SRK-015) is a fully human monoclonal antibody developed to treat spinal muscular atrophy. It works by binding to and inhibiting promyostatin, a precursor to myostatin, which limits the size of skeletal muscle tissue, as well as inactive myostatin. It does not bind to active myostatin, activin A, active BMP9/10 or TGFβ1 that all operate on the activin type 2 receptors.[1][2][3]
References
- ↑ Barrett, Doreen; Bilic, Sanela; Chyung, Yung; Cote, Shaun M.; Iarrobino, Ryan; Kacena, Katherine; Kalra, Ashish; Long, Kimberly et al. (2021). "A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy". Advances in Therapy 38 (6): 3203–3222. doi:10.1007/s12325-021-01757-z. ISSN 0741-238X. PMID 33963971.
- ↑ Crawford, Thomas; Darras, Basil; Day, John; Song, Guochen; Nomikos, George; Place, Amy; Barrett, Doreen; Bilic, Sanela et al. (3 May 2022). "Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005)" (in en). Neurology 98 (18 Supplement). ISSN 0028-3878. https://n.neurology.org/content/98/18_Supplement/1859.
- ↑ Crawford, Thomas; Place, Amy; Barrett, Doreen; Cote, Shaun; Nomikos, George; Song, Guochen; Bilic, Sanela; Kalra, Ashish et al. (2021). "Relationship of pharmacokinetics and pharmacodynamics to apitegromab efficacy in patients with later-onset spinal muscular atrophy (Types 2 and 3 SMA): Results from the TOPAZ study". Journal of the Neurological Sciences 429: 118388. doi:10.1016/j.jns.2021.118388.
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