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]
| Monoclonal antibody | |
|---|---|
| Type | ? |
| Target | Promyostatin |
| Clinical data | |
| Other names | SRK-015 |
| Identifiers | |
| CAS Number | |
| UNII | |
References
edit- ^ Barrett, Doreen; Bilic, Sanela; Chyung, Yung; Cote, Shaun M.; Iarrobino, Ryan; Kacena, Katherine; Kalra, Ashish; Long, Kimberly; Nomikos, George; Place, Amy; Still, James Gordon; Vrishabhendra, Leela (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. PMC 8189951. PMID 33963971.
- ^ Crawford, Thomas; Darras, Basil; Day, John; Song, Guochen; Nomikos, George; Place, Amy; Barrett, Doreen; Bilic, Sanela; O‘Neil, Janet; Kertesz, Nathalie; Cote, Shaun; Patel, Jagdish; Chyung, Yung (3 May 2022). "Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005)". Neurology. 98 (18 Supplement). ISSN 0028-3878.
- ^ Crawford, Thomas; Place, Amy; Barrett, Doreen; Cote, Shaun; Nomikos, George; Song, Guochen; Bilic, Sanela; Kalra, Ashish; Sadanowicz, Mara; O'Neil, Janet; Iarrobino, Ryan; Kertesz, Nathalie; Chyung, Yung (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. S2CID 238481481.