Biogen and Ionis shares leap as nusinersen meets Phase III goal in infantile-onset spinal muscular atrophy

2 August 2016

US biotechs Biogen (Nasdaq: BIIB) and Ionis Pharmaceuticals (Nasdaq: IONS) have announced that their investigational treatment nusinersen has met the primary endpoint in a Phase III trial of its efficacy infantile-onset spinal muscular atrophy (SMA).

Shares in both companies leapt on Monday after the good news about a potential treatment for a sometimes fatal childhood muscular disorder was revealed, with Ionis’ value increasing by more than 30%.

The ENDEAR trial found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment, and it also demonstrated an acceptable safety profile.

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