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]
^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.
Apitegromab (SRK-015) is a fully human monoclonal antibody developed to treat spinal muscular atrophy. It works by binding to and inhibiting promyostatin...
clinical trial in adolescent and adults with SMA types 2, 3, and 4. Apitegromab (SRK-015) is monoclonal antibody that blocks the activation of the skeletal...
Monoclonal antibodies have been developed that disable myostatin, including apitegromab, domagrozumab, landogrozumab, and stamulumab. Another form of myostatin...