Biogen announced new data from the Phase 3 ENDEAR study that showed a statistically significant reduction in the risk of death or permanent ventilation in Spinraza (nusinersen)-treated infants with spinal muscular atrophy (SMA) vs. untreated infants. 

ENDEAR was a randomized, double-blind, sham-controlled study that initially met its pre-specified primary endpoint at the interim analysis, which was the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). The study was stopped early following the positive interim analysis to allow all patients to receive Spinraza in an open-label extension study.  

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Study data on time to death or permanent ventilation, the pre-specified primary endpoint of the end-of-study (EOS) analysis, was presented at the British Paediatric Neurology Association (BPNA) annual conference. A significantly greater number of infants treated with Spinraza survived and did not require permanent ventilation, with a 47% reduction in the risk of death or permanent ventilation (P<0.01). In the EOS analysis, a greater percentage of untreated infants died or required permanent ventilation vs. infants who received Spinraza (68% vs. 39%). 

Spinraza, an antisense oligonucleotide, treats SMA by causing mutations in the chromosome 5q that leads to SMN protein deficiency. It increases production of full-length SMN protein by altering the splicing of SMN2 pre-mRNA. 

Spinraza is administered intrathecally and is supplied as a 12mg/5mL strength solution in single-dose vials.

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