Scholar Rock To Highlight Apitegromab Data From Ambulatory Cohort In Muscular Dystrophy

Scholar Rock Holding Corp SRRK will showcase data from an exploratory analysis of the ambulatory cohort in the Phase 2 TOPAZ trial of apitegromab in individuals with later-onset spinal muscular atrophy (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 explored the relationships between the efficacy of apitegromab 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, a clinician-reported outcome measure assessing physical abilities.
• Related: Scholar Rock Outlines Pivotal Apitegromab Trial Design In Spinal Muscular Atrophy Patients.
• 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. 
• In addition, patients without joint contractures (n=10) had a mean change of +1.10 points from baseline in the RHS score, while patients with joint contractures (n=13) had a mean change of -1.46.
• Price Action: SRRK shares closed 5.47% lower at $13.83 on Friday.