Abstract
Myostatin, an important negative regulator of muscle mass, is synthesized as an inactive precursor, requiring two proteolytic cleavage steps to release the active growth factor. Apitegromab is an investigational, fully human, monoclonal antibody that specifically binds to the proforms of myostatin, which include promyostatin and latent myostatin, thereby inhibiting myostatin activation. To further characterize the relationship between levels of serum latent myostatin and response to apitegromab, we measured serum latent myostatin concentrations in patients with SMA during the TOPAZ phase 2 clinical trial (NCT03921528) and in previous studies in healthy humans and animals as well as in an SMA mouse model.
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