Biogen reports on successful Phase III end of study of Spinraza (nusinersen) data in children with spinal muscular atrophy.

-Biogen will present Phase III end of study Spinraza (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of Spinraza across a broad range of individuals with SMA. The Spinraza development program represents the largest body of clinical data of its kind in SMA. Spinraza data will be presented at the American Academy of Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.

CHERISH is a Phase III, multicenter, randomized, double-blind, sham-procedure controlled study to assess the efficacy and safety of Spinraza in children with later-onset SMA. The 15-month study investigated Spinraza in 126 non-ambulatory children 2 to 12 years old who experienced symptom onset at greater than 6 months of age. In the CHERISH end of study analysis, children on Spinraza demonstrated a highly statistically significant and clinically meaningful improvement in motor function, as observed by the treatment difference of 4.9 points in the mean change from baseline to Month 15 in the Hammersmith Functional Motor Scale Expanded (HFMSE) score (p=0.0000001). The HFMSE is a validated tool specifically designed to assess motor function in children with SMA. When measuring changes from baseline, children who received Spinraza (n=84) achieved a 3.9 point mean improvement at Month 15, while children who were not on treatment (n=42) experienced a mean decline of 1.0 point. Primary endpoint results of the end of study analysis were consistent with results observed at the interim analysis. Data from the other endpoints analyzed, including attainment of new motor milestones and upper limb motor function, were consistently in favor of children who received treatment. Spinraza demonstrated a favorable safety profile. Treatment-emergent adverse events (AEs), severe AEs and serious AEs (SAEs) were reported less frequently in children treated with Spinraza than those not on treatment. The majority of the AEs were considered to be either related to SMA disease, common events in the general population, or events related to the lumbar puncture procedure. No children discontinued the study due to AEs.