Individuals treated with nusinersen in the Phase 3, randomized, double-blind CHERISH study could transition to the open-label SHINE extension study (NCT02594124). Plasma pNF-H levels were measured using the pNF-H SimplePlex ELLA assay (ProteinSimple); motor function was assessed using the Hammersmith Functional Motor Scale – Expanded (HFMSE) and Revised Upper Limb Module (RULM). Linear multivariate regression models were applied to determine any relationship between log pNF-H levels at baseline and change from baseline in HFMSE or RULM to Day 450, 690, 930, 1170, or 1410.

pNF-H data were available for up to 75 participants with HFMSE/RULM data. After controlling for age at treatment initiation and baseline motor function, higher baseline log pNF-H level was not significantly associated with change in HFMSE or RULM at Days 450, 690 or 930. Higher baseline log pNF-H level was significantly associated with greater improvements in HFMSE and RULM at Days 1170 and 1410. The relationship between higher baseline pNF-H and improvement in HFMSE and RULM increased over time. Additional analyses will be presented.

These results show that participants with higher pNF-H levels at baseline are expected to have higher HFMSE and/or RULM scores relative to baseline at later visits after receiving nusinersen. pNF-H may be a useful indicator of future motor function and warrants further evaluation as a biomarker of treatment response in SMA.

Supported by: Biogen