Interim results from NURTURE, an ongoing efficacy and safety study of Spinraza in 25 presymptomatic infants with SMA. Biogen

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 (spinal muscular atrophy). The data are being presented 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 a fatal, early-onset form of SMA known as Type 1, and infants with three copies of the SMN2 gene (n=10) who typically develop SMA Type 2 or 3. People living with SMA Types 2 and 3 may never be able to walk or will lose that ability over time. 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.