Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease. These results are being presented at the 2020 Virtual SMA Research & Clinical Care Meeting taking place June 10-12, 2020.

The new data include nearly a year of additional follow-up for NURTURE study participants. As of February 2020, all patients treated (n=25; median age of 3.8 years) were alive and remained free of permanent ventilation. In the absence of treatment, most children with SMA Type 1 would, on average, not reach their second birthday. Additionally, 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.

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). These individuals received their first dose of SPINRAZA 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 SPINRAZA through 8 years of age and further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available on clinicaltrials.gov.

Additional results from Biogen on the updated interim analysis as of February 2020 show:

  • All study participants who were previously able to walk with assistance (92 percent) and walk independently (88 percent) maintained that ability over the 11 months since the last data cut.
  • Over the 11 months of follow-up, one child gained the ability to walk with assistance (increasing to 96 percent 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 percent).
  • 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.
  • SPINRAZA 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.