The STR1VE-US study findings are being presented today during a virtual Clinical Trial Session conducted by the
Interim data from the ongoing SPR1NT study continue to show patients achieved age-appropriate motor milestones when treated with Zolgensma presymptomatically. Most patients (7/8) with two copies of SMN2 who achieved the ability to sit independently did so within the
'SMA is a disease that robs babies of the ability to talk, eat, sit up and even breathe. In complete contrast to the natural course of the disease, patients who received Zolgensma soon after birth before the onset of symptoms are achieving age-appropriate motor milestone development - an extraordinary outcome for SMA patients,' said
SMA Type 1 patients experienced rapid, sustained and clinically meaningful improvements in motor function in the completed pivotal STR1VE-US study. In STR1VE-US, nearly all (91%) patients met the co-primary efficacy endpoint of event-free survival at 14 months, and more than half (59%) of patients met the co-primary efficacy endpoint of sitting for 30 seconds at 18 months of age, a milestone never achieved in the natural history of SMA Type 1. Importantly, nine of 22 patients demonstrated the 'ability to thrive' at 18 months of age. As the goal of treatment for SMA Type 1 moves beyond survival and motor milestone achievement, the STR1VE-US trial is the first to incorporate this stringent composite endpoint - inclusive of functions of swallowing, feeding and age-appropriate weight maintenance - and demonstrate remarkable achievements in symptomatic patients with SMA Type 1, the most prevalent form of the disease accounting for 60% of SMA diagnoses.1,2
New data from the START long-term follow-up study continue to demonstrate the durability of a single, one-time dose of Zolgensma in patients now up to five years post-dosing and some patients more than five years of age. All patients in this study who received the therapeutic dose were alive and free of permanent ventilation and continued to maintain developmental milestones, including two patients who achieved the new milestone of standing with assistance during the long-term follow-up period.
Cumulative safety data from patients treated with intravenous Zolgensma in clinical trials,
'The bar for treatment efficacy in SMA Type 1 patients has been raised beyond event-free survival and motor milestone achievement, and the expectation is now that these patients maintain the ability to thrive, an unprecedented and challenging endpoint,' said
SPR1NT Data as of
SPR1NT is an ongoing Phase 3, open-label, single-arm, multi-center trial designed to evaluate the safety and efficacy of a one-time intravenous (IV) infusion of Zolgensma in presymptomatic patients with SMA and two or three copies of SMN2 who are
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