Isis Pharmaceuticals has earned a $2.15 million milestone payment from Biogen for advancing the ongoing Phase III study (CHERISH) evaluating ISIS-SMN_Rx in children with spinal muscular atrophy (SMA). To date, Isis has received more than $120 million in payments from Biogen related to the development of ISIS-SMN_Rx.
 
The Phase III study of ISIS-SMN_Rx, will evaluate the efficacy and safety of 12 mg doses of ISIS-SMN_Rx with a primary endpoint of a change in the Hammersmith Functional Motor Scale-Expanded (HFMSE), a validated method to measure changes in muscle function in patients with SMA.     
 
SMA is a severe genetic disease for which there are no approved treatments. The disease is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene, which is critical for the health and survival of nerve cells in the spinal cord that are responsible for neuromuscular growth and function. 
 
Isis and Biogen have a broad strategic alliance focused on leveraging antisense technology to advance the treatment of neurological and neuromuscular disorders.