Isis advances ENDEAR study of ISIS-SMNRx for infants with spinal muscular atrophy-Isis + Biogen IDEC

Isis Pharma in collaboration with Biogen IDEC is advancing an ongoing pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy. The study known as ENDEAR is a randomized, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with SMA. The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx with a primary endpoint of survival or time to permanent ventilation. Additional efficacy endpoints are also included in the study.

Comment:SMA is a severe genetic disease that affects approximately 30,000-35,000 patients in the United States, Europe and Japan. SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in the survival motor neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function. One in 50 people, the equivalent of about 6 million people in the United States, are carriers of a defective SMN1 gene, which is unable to produce fully functional SMN protein. Carriers experience no symptoms and do not develop the disease. However, when both parents are carriers, there is a one in four chance that their child will have SMA.

The ENDEAR study and CHERISH study are the two Phase III studies that Isis and Biogen IDEC plan with FDA agreement to be adequate to meet the requirements for a new drug application.