Isis reports Phase II SMA data
Isis Pharmaceuticals Inc. (NASDAQ:ISIS) reported data from an ongoing Phase II trial of ISIS-SMNRx to treat infants with spinal muscular atrophy (SMA). The median event-free age of the infants who completed a three-dose course was 16.3 months for the 6 mg group and 11.6 months for the 12 mg group. Isis said that the data "compare favorably" with a Pediatric Neuromuscular Clinical Research Network natural history study, in which the median event-free age of infants with SMA was 10.5 months.
The antisense oligonucleotide, which modulates splicing of survival of motor neuron 2 centromeric ( SMN2) mRNA, has Orphan Drug designation in the U.S. and EU. Isis began the Phase III ENDEAR trial to treat infants with SMA in August, and plans to start the Phase III CHERISH trial in children by year end. ...