Biogen today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained unparalleled results compared to the natural history of the disease. As of March 2019, all participants were alive, without the need for permanent ventilation and experienced continuous improvements with the majority achieving motor milestones in timelines consistent with normal development. The results also demonstrated durability of effect with children making progress throughout the study.

NURTURE is an ongoing, Phase 2, open-label study of 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA in the pre-symptomatic stage and before six weeks old. The study, conducted at 15 sites in seven countries, has results up to 45.4 months. When compared with the natural history of the disease, the results are dramatic in their impact on changing the course of SMA.

As of March 2019, all infants in the study were 25 months or older, past the typical age of symptom onset for SMA Type 1 and Type 2, and were alive without the need for permanent ventilation. In comparison to the natural history of SMA, many of these infants would likely have passed away or require permanent ventilation on average by 13.5 months. In both the children with two and three copies of SMN2, treatment with SPINRAZA demonstrated rapid onset of improvement and durability of effect with their mean Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) score of motor function reaching the maximum mean score of 64 for all participants with three copies of SMN2 (n=10) and a mean of 62.1 for those with two copies of SMN2 (N=15).

Additional highlights include:

  • The majority of study participants achieved motor milestones in timeframes consistent with the World Health Organization (WHO) standards, with 100 percent sitting independently and 88 percent walking independently.
  • Hammersmith Infant Neurologic Examination, Section 2 (HINE-2) development of motor function scores increased for all participants with the mean score for both those with two or three SMN2 copies approaching the maximum score of 26 points at the last assessment.
  • SPINRAZA demonstrated longer term efficacy up to nearly four years, with participants continuing to make progress.
  • SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly four years of treatment.

These published results from the NURTURE study were previously presented at the 2019 Cure SMA Annual SMA Conference and the 5th Congress of the European Academy of Neurology.

Cure SMA Provides Seed Funding for Spinraza

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach behind SPINRAZA. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.