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Post-hoc analysis of plasma from the study assessed ATL1102’s effects on proteomics as measured by the Somascan® assay, a large scale, aptamer-based assay, using normalized relative fluorescence units (nRFU). Parametric mixed effect longitudinal analysis was conducted to determine the average percent change over time, p-value and Benjaminin-Hochberg false discovery rate (FDR) adjusted p-value. At 24 weeks, statistically significant mean increases of LTBP4 (20.7%), sCXCL16 (29.9%), and sVCAM-1 (18.0%) were observed compared to baseline levels (FDR p-value <0.0005).
ATL1102 modulation of VLA-4 ligand VCAM-1 is comprehensible and indirectly CXCL16, the latter with a reported role in muscle regeneration. LTBP4, which sequesters TGF-beta, a genetic modifier of DMD involved in early loss of ambulation, interacts with fibronectin, another VLA-4 ligand. These effects have a potential role in the positive stabilization of function and strength observed in the Phase II study.
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