Isis Reports Preliminary Data From Phase 1 Study Of ISIS-SMNRx - Quick Facts

(RTTNews) - Isis Pharmaceuticals Inc. (ISIS) announced that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy or SMA, show that most SMA children receiving the two highest doses of the drug, 6 mg and 9 mg, continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.

Lynne Parshall, chief operating officer at Isis, said, "Our ongoing Phase 2 program is proceeding well. The 6 mg dose group in our Phase 2 study in infants with Type I SMA has completed dosing. We have amended the infant study to increase the dose from 9 mg to 12 mg dose. In our Phase 1b/2a multiple-dose study in children with Type II and Type III SMA, we have completed dosing in all three dose cohorts (3 mg, 6 mg and 9 mg), and we are considering adding a 12 mg dose cohort to this study."