Apitegromab

Apitegromab

Monoclonal antibody
Type	?
Target	Promyostatin
Clinical data
Other names	SRK-015
Identifiers
CAS Number	2278276-46-1
UNII	UZ54216N0Y

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

1. 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.
2. 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.
3. 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.