Scholar Rock Holding Corp (NASDAQ: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.
