Biogen (Maidenhead, UK) have announced new results from the NURTURE study, which demonstrated that pre-symptomatic infants treated with Spinraza® (nusinersen) achieved motor milestones not before seen in the natural history of the disease, including 100% of children sitting without support and 88% walking independently.
NURTURE is an ongoing, Phase II, open-label study of 25 pre-symptomatic patients with spinal muscular atrophy (SMA; most likely to develop SMA Type 1 or Type 2) who received their first dose of nusinersen before 6 weeks old.
These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with nusinersen in comparison to the natural history of the disease. These new data also revealed that patients treated with nusinersen had continuous improvement in sitting and walking independently, with an overwhelming majority of patients achieving motor milestones in a normal timeframe.
“These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones,” commented Darryl De Vivo (Columbia University, NY, USA). “Nusinersen is setting patients on a path toward survival, greater mobility and independence from the start of treatment, which is helping improve outcomes for patients of all ages.”
The reported results from NURTURE (as of March 2019) include:
- 100% of patients were alive without a need for permanent ventilation
- The median age of study participants was nearly 3 years old (majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation)
- 100% of infants were sitting independently (in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance)
- 88% of the infants were walking independently with many of them doing so in the normal timeframe for a toddler (in the natural history of SMA Type 1 or Type 2, patients are never able to walk independently)
- Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function – 63.4 for patients with three SMN2 copies (n = 10) and 62.1 for those with two SMN2 copies (n = 15), demonstrating the impact of early treatment
- Nusinersen demonstrated efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function
- Nusinersen was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment
“It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types,” concluded Kenneth Hobby, President of Cure SMA (IL, USA).
Additional presentations from Biogen will highlight results from the ongoing open-label SHINE extension study of children with infantile and later-onset SMA. The company will also continue to explore the value of pNF-H and will present data on the ongoing evaluation of its potential as a biomarker in SMA.
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