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

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Apitegromab is an investigational, fully human, monoclonal antibody that inhibits myostatin activation by specific binding to myostatin proforms, promyostatin and latent myostatin. We will present data, from the Phase 2 TOPAZ clinical trial (NCT03921528), on the relationships of pharmacokinetics (PK) and pharmacodynamic (PD) (marker of target engagement, latent serum myostatin levels) to apitegromab efficacy.