Scholar Rock to Present Apitegromab TOPAZ Phase 2 Trial Results Highlighting Pharmacokinetic (PK) and Pharmacodynamic (PD) Data at the 2021 European Academy of Neurology Congress
Details for the virtual EAN oral e-presentation are as follows:
- Title: Apitegromab, an Investigational Anti-proMyostatin Monoclonal Antibody for Spinal Muscular Atrophy: Phase 2 Results
Muscle and Neuromuscular Junction Disease 2 Session: Virtual e-presentation #EPR-184 on
Saturday, June 19from 14:00 to 14:45 CEST( 8:00-8:45am ET) in Room Paris
Amy Place, PhD, MBA, MS, RD, CLT on behalf of the apitegromab development team
The e-presentation will highlight the 12-month top-line TOPAZ clinical results, including PK/PD data as these relate to the efficacy of apitegromab, an investigational selective inhibitor of myostatin activation. In the TOPAZ trial, apitegromab showed therapeutic potential to further improve motor function in patients with non-ambulatory Type 2 and 3 SMA who were already receiving chronic maintenance therapy with a background SMN upregulator (nusinersen). Two doses of apitegromab (20 mg/kg and 2 mg/kg) were evaluated in the TOPAZ trial and a dose response was observed based upon clinical efficacy (improvements in Hammersmith Functional Motor Scale Expanded scores) and pharmacodynamics (target engagement).
Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans.
Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children. An estimated 30,000 to 35,000 patients are afflicted with SMA in
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