Scholar Rock to Present Data from TOPAZ Ambulatory Cohort Analysis at the 2022 M… – Press Release


Scholar Rock SRRK, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that it will present a poster at the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 13 – 16, 2022).

The poster showcases data from an exploratory analysis of the ambulatory cohort in the Phase 2 TOPAZ trial evaluating apitegromab, a selective inhibitor of the activation of latent myostatin, in individuals with later-onset spinal muscular atrophy (SMA).

“We are enthusiastic about developing and investigating the therapeutic potential for apitegromab in both ambulatory as well as non-ambulatory forms of SMA,” said George Nomikos, M.D., Ph.D., Senior Vice President of Medical & Clinical Sciences and Head of the Muscle Therapeutic Area at Scholar Rock. “Hypothesis-generating insights from this analysis identify factors that may be more likely to be associated with motor function gains following apitegromab treatment in ambulatory patients with SMA.”

The TOPAZ study assessed the safety, tolerability, PK/PD and efficacy of apitegromab in Types 2 and 3 SMA. This post hoc analysis of the 12-month treatment data from the ambulatory type 3 SMA cohort explored the relationships between the efficacy of apitegromab (pooling data from both patients receiving it as monotherapy or in addition to background nusinersen) and factors such as pharmacokinetics and pharmacodynamics (PK/PD), age, body mass index, and the presence of scoliosis or joint contractures. The presence or absence of scoliosis and/or joint contractures at baseline were associated with differential responses in the Revised Hammersmith Scale (RHS) score after 12 months of treatment. Patients without scoliosis (n=12) had a mean change of +0.67 points from baseline in the RHS score, while patients with scoliosis (n=11) had a mean change of -1.45 from baseline in the RHS score. In addition, patients…