Biogen, Isis Pursue Muscle Disease Drug

The biotech firm Biogen Idec has obtained an option to license an Isis Pharmaceuticals investigational drug intended to treat spinal muscular atrophy, a genetic disease that affects one out of 10,000 children worldwide. Children with SMA have a defect in the survival motor neuron 1 gene. The drug, an antisense compound called ISIS-SMNRx, works by altering the splicing of a related gene to increase production of survival motor neuron protein. Isis will receive an up-front fee of $29 million and could get $45 million in milestone payments associated with development of the drug. Isis could also receive a $225 million licensing fee and further milestone payments.