Apitegromab Explained

Drug Name:	Apitegromab
Type:	mab
Target:	Promyostatin
Synonyms:	SRK-015
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]}

Notes and 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 . 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 . 2021 . 38 . 6 . 3203–3222 . 10.1007/s12325-021-01757-z . 33963971 . 8189951 . 0741-238X.
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 . Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005) . Neurology . 3 May 2022 . 98 . 18 Supplement . en . 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 . 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 . 2021 . 429 . 118388 . 10.1016/j.jns.2021.118388. 238481481 .