Biogen reports additional Spinraza data for SMA

Biogen Inc. (NASDAQ:BIIB) reported additional data from the Phase III ENDEAR and Phase II EMBRACE trials of Spinraza nusinersen (IONIS-SMNRx) to treat spinal muscular atrophy (SMA). Data were presented at the World Muscle Society meeting in Saint Malo, France.

In the 13-month, double-blind, international ENDEAR trial in 121 patients with infantile-onset SMA, Spinraza significantly increased the proportion of motor milestone responders per the motor component of the Hammersmith Infant Neurological Examination (HINE), a co-primary endpoint, in both patients with disease duration of ≤12 weeks (n=52) (75% vs. 0%, p<0.0001) and with disease duration of >12 weeks (n=69) (32% vs. 0%, p=0.0026) vs. sham procedure. In patients with disease duration of ≤12 weeks, Spinraza also significantly improved event-free survival (EFS), defined as time to death or permanent ventilation, vs. sham procedure (p=0.0004). Spinraza did not improve EFS in patients with disease duration of >12 weeks (p=0.5325)...