Spinraza: Interim Ph III data

The double-blind, international Phase III CHERISH trial in 126 non-ambulatory patients with later-onset SMA was stopped early after a pre-specified interim analysis showed that intrathecal Spinraza met the primary endpoint of improving mean HFMSE score from baseline to 15 months vs. sham control (improvement of 4 points vs. a reduction of 1.9 points, p=0.0000002). The trial enrolled patients ages 2-12 with later-onset SMA and included patients who experienced onset of SMA signs and symptoms at >6 months of age. Patients in CHERISH and the Phase III ENDEAR trial of Spinraza in infantile-onset SMA are eligible to enroll in the open-label SHINE extension study (see BioCentury, Aug. 8)...