Biogen, Inc. announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25 presymptomatic infants with SMA. The data were presented Sunday in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina.
The interim analysis evaluated survival and respiratory intervention rates in infants (n=25) who were genetically diagnosed with SMA and began treatment in the presymptomatic stage of the disease. As of May 2018 all patients in the study were alive and none required tracheostomy or permanent ventilation. Additionally, 22 of the 25 participants were able to walk either with assistance or independently according to the motor milestone standard of the World Health Organization and all 25 were able to sit without support.
The motor skills of study participants were also evaluated using the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), an assessment which considers 16 different types of movement to create an overall score between zero and 64. The mean CHOP INTEND scores were 62.6 for study participants with three copies of the SMN2 gene and 61.0 for those with two copies of the gene.
All NURTURE study participants were 14 months or older at the time of the analysis. Participants included infants with two copies of the SMN2 gene (n=15) who are likely to develop SMA Type 1, and infants with three copies of the SMN2 gene (n=10) who typically develop SMA Type 2 or 3. No new safety concerns were identified.
Additional research presented at WMS compared levels of phosphorylated neurofilament heavy chain (pNF-H) in plasma in more than 300 patients from SPINRAZA clinical trials, including those in the NURTURE study, and a control group of people without SMA. The data demonstrated that treatment with SPINRAZA is associated with a rapid decline followed by stabilization of pNF-H in plasma at levels close to those of healthy controls. The results are part of Biogen’s ongoing work to identify and validate biomarkers that could provide insight on the disease progression of SMA.
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.
For More Information
To learn more about Spinraza please visit www.curesma.org/spinraza or the links below: