Based on our request for an update, Biogen has provided us with the following information about new results from their NURTURE study, a study of pre-symptomatic patients with SMA, recently presented at the virtual Cure SMA Research & Clinical Care Meeting, which took place from 10th to 12th June 2020.
NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of nusinersen before 6 weeks of age. The study has been extended by an additional three years, enabling Biogen to evaluate the longer-term efficacy and safety of nusinersen through to 8 years of age and further understand the impact of early treatment.
These new results of nearly a year of additional follow-up, show that pre-symptomatic SMA patients treated with SPINRAZA® (nusinersen) continue to demonstrate sustained benefit from the treatment. After up to 4.8 years of continuous treatment with nusinersen, 100 percent of children treated pre-symptomatically (25 patients) were alive and none required permanent ventilation. Patients continued to maintain and make progressive gains in motor function compared to the natural history of the disease, with 96 percent now able to walk with assistance.
All children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.
Nusinersen was well-tolerated, with no new safety concerns identified over the extended follow-up period. No children have discontinued the study due to adverse events associated with treatment.
Additional results from the updated interim analysis as of February 2020 show:
- All study participants who were previously able to walk with assistance (92%) and walk independently (88%) maintained that ability over the 11 months since the last data cut¹
- Over the 11 months of follow-up, one child gained the ability to walk with assistance (increasing to 96% of all study participants) and also reached the maximum score on the Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) scale, increasing the total number of study participants who achieved the maximum score to 21 of 25 (84%).
- Patients with two copies of SMN2 were able to score and advance on the Hammersmith Functional Motor Scale Expanded scale (HFMSE), which is atypical to the natural history of the disease.
1De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842‐856.