Scholar Rock Holding ((SRRK)) announced an update on their ongoing clinical study.
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Scholar Rock Holding (SRRK) is currently conducting a Phase 2, double-blind study titled A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA). The study aims to assess the pharmacokinetics, pharmacodynamics, efficacy, and safety of Apitegromab in infants under two years old diagnosed with Spinal Muscular Atrophy (SMA), a condition characterized by delayed motor milestones.
The intervention being tested is Apitegromab, a fully human anti-proMyostatin monoclonal antibody designed to inhibit myostatin activation. It is administered via intravenous infusion every four weeks, alongside standard SMN therapies like Nusinersen or Risdiplam, to enhance muscle function in SMA patients.
The study employs a randomized, parallel intervention model with quadruple masking, ensuring that participants, caregivers, investigators, and outcomes assessors remain unaware of treatment assignments. The primary purpose of this study is treatment-focused, aiming to improve outcomes for young SMA patients.
The study began on April 16, 2025, with primary completion expected shortly after. The most recent update was submitted on September 11, 2025. These dates are crucial for tracking the study’s progress and anticipating results that could influence market dynamics.
This study update could significantly impact Scholar Rock’s stock performance and investor sentiment, given the potential breakthrough in SMA treatment. Investors should also consider the competitive landscape, as advancements in SMA therapies could shift market positions.
The study is ongoing, with further details available on the ClinicalTrials portal.