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Biogen have recently announced the latest data from the RESPOND trial, which is a phase 4 study designed to assess the safety and efficacy of Spinraza (nusinersen) in infants and children with SMA who have unmet clinical needs following previous treatment with Zolgensma (onasemnogene abeparvovec).

In a presentation given at the 2023 SMA Research & Clinical Care in Florida, Biogen provided interim results showing that Spinraza treatment resulted in motor function gains in most participants.

Initiated in July 2020, RESPOND is an actively recruiting (not in the UK) two-year, open label study that seeks to understand if the increased and continuous production of SMN protein by nusinersen provides any additional benefits to patients previously treated with Zolgensma.

Motor function of treated individuals was assessed using the Hammersmith Infant Neurological Examination Section 2 (HINE-2) score. 24 participants with two copies of the SMN2 gene showed an average improvement of more than 5 points on HINE-2 compared to their baseline score before treatment. Furthermore, all participants with three copies of SMN2 also showed improvements in HINE-2 – however, the average change in score was not calculated due to only three participants in this category being included in the trial thus far.

In addition, no Spinraza-related safety concerns were reported, and no serious adverse events led to withdrawal from the study.

Biogen aim to recruit a total of 60 participants in the RESPOND trial, which has an estimated completion date of September 2024.
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