Basel - AveXis, a Novartis company, today announced a one-time infusion of Zolgensma (onasemnogene abeparvovec-xioi) showed rapid, significant and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated presymptomatically, and sustained durability in patients now up to five years post-dosing and some patients more than five years of age.

The STR1VE-US study findings are being presented today during a virtual Clinical Trial Session conducted by the Muscular Dystrophy Association (MDA), scheduled after the 2020 MDA Annual Conference was cancelled due to COVID-19. The accepted posters for SPR1NT, START long-term follow up and cumulative safety data will be published online by MDA in the coming weeks.

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 World Health Organization window of normal development. The six remaining patients in this cohort of 14 patients have not yet passed the developmental window. The importance of independent sitting is that it allows for the potential development and integration of the cognitive, sensory and motor skills that are important for functional independence and social development. Additionally, nearly all patients were fed orally and required no feeding support. Most remained within the age-appropriate weight range. No patients required ventilatory support of any kind.

'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 Olga Santiago, M.D., Chief Medical Officer, AveXis. 'These SPR1NT data demonstrate the truly transformational impact a one-time dose of gene therapy can have, and further underscore the importance of newborn screening and early intervention to alter the course of the disease.'

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, U.S. managed access program, the RESTORE global registry and commercial experience were consistent with previously-reported safety information. Reported adverse events (AEs) were monitorable and manageable, and the overall benefit-risk safety profile remains favorable.

'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 Lisa Deschamps, Chief Business Officer, AveXis. 'Further, with hundreds of patients now treated, including some more than five years post-treatment and more than five years old, these new data further reinforce the profound benefit a one-time dose of Zolgensma has on SMA patients.'

SPR1NT Data as of December 31, 2019

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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