Clinical Data
SPINRAZA met the pre-specified primary endpoint at the ENDEAR EOS, demonstrating a statistically significant 47% reduction in the risk of death or permanent ventilation (p<0.01). In the EOS analysis, a greater percentage of untreated infants (68%) died or required permanent ventilation compared to infants treated with SPINRAZA (39%).SPINRAZA demonstrated a favorable safety profile, with commonly reported adverse events including respiratory events and constipation, consistent with those expected in the general population of infants with SMA. Further EOS efficacy and safety results from ENDEAR will be presented at a future medical congress.ENDEAR was a randomized, double-blind, sham-controlled study in patients with infantile-onset (most likely to develop Type 1) SMA. The EOS efficacy analysis included all patients (n=121) who had their final study visit after the interim analysis (n=78) and had the opportunity to attend the six-month study visit assessment.