Phase III ENDEAR study of Spinraza (nusinersen) shows reduction in the risk of death in infants with spinal muscular atrophy- Biogen

Biogen presented new data from the Phase III ENDEAR study of Spinraza (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in Spinraza-treated infants with spinal muscular atrophy (SMA) compared to untreated infants. 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. The data were presented at the British Paediatric Neurology Association (BPNA) annual conference.

Comment: In August 2016, Biogen reported that ENDEAR met its pre-specified primary endpoint at the interim analysis, the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). Following the positive interim analysis, Biogen ended the study early so that all participants could have the option to receive SPINRAZA in an open-label extension study. Now Biogen has provided the first presentation of the pre-specified primary endpoint, time to death or permanent ventilation, from the end of study (EOS) analysis.