Shares in Carlsbad, CA-based Ionis Pharmaceuticals Inc. (formerly ISIS Pharmaceuticals) shot up by more than 30% to close at $38.01 on Aug. 1 when it announced the initial data from its ENDEAR study of the antisense candidate nusinersen plus a $75m license fee payment from Biogen Inc.,which will now assume responsibility for the drug's development. Biogen's shares also received a 4% boost.
A pre-specified interim analysis has shown that babies with infantile-onset (consistent with Type 1) spinal muscular atrophy (SMA) receiving nusinersen experienced a significant improvement in achieving motor milestones compared with those who did not, with what the companies described
