Spinraza: Additional Ph III ENDEAR data

Additional data from the 13-month, double-blind, international Phase III ENDEAR trial in 121 patients with infantile-onset SMA showed that intrathecal Spinraza met the co-primary endpoint of reducing the percentage of patients who died or required permanent ventilation vs. sham procedure (39% vs. 68%, p<0.01). Data were presented at the British Paediatric Neurology Association meeting in Cambridge. Last August, Biogen stopped the trial early after a pre-specified interim analysis showed that Spinraza met the co-primary endpoint of a greater proportion of motor milestone responders per the motor component of HINE vs. sham procedure (see BioCentury, Aug. 8, 2016)...