Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in Spinal Muscular Atrophy at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference
Both presentations will include six-month interim analysis data from the TOPAZ Phase 2 clinical trial evaluating the potential of apitegromab, a highly selective inhibitor of latent myostatin, in improving motor function in patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Top-line clinical data from TOPAZ for the full 12-month treatment period are expected in the second quarter of 2021.
Oral presentation details:
- Title: Apitegromab, a Novel High-Affinity Anti-proMyostatin Monoclonal Antibody for Treating Spinal Muscular Atrophy: Results of a Phase 2 Interim Analysis
Date and Time:
Thursday, March 18, 2021, 2:00-2:15 pm EST
Amy Place, PhD, MBA, MS, RD, CLT on behalf of the apitegromab development team
Poster presentation details:
- Title: Apitegromab, a Novel High-Affinity Anti-Promyostatin Monoclonal Antibody for Treating Spinal Muscular Atrophy: Results of a Phase 2 Interim Analysis (Poster #51)
Dates: Virtual poster presentations are available to view on the virtual conference platform from
March 15-18, 2021
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.
A Phase 2 clinical trial in patients with Type 2 and Type 3 SMA is ongoing (NCT03921528). The
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