Ionis completes target enrollment in phase 3 CHERISH study in children with spinal muscular atrophy

The study is designed to support marketing approval of nusinersen in children with spinal muscular atrophy (SMA).

Ionis Pharmaceuticals COO Lynne Parshall said: "Achieving our target enrollment number in CHERISH brings us one step closer to a marketing application for children with SMA. We also plan to complete enrollment in ENDEAR, our Phase 3 study evaluating nusinersen in infants with SMA, in the first half of this year. This progress sets the stage for Phase 3 data from both of these programs in 2017.

"We would like to thank the parents and families for their participation in CHERISH as well as the investigators and sites for the achievement of this milestone. We also appreciate the support from the broader SMA community. We, along with Biogen, are committed to advancing nusinersen toward the market as rapidly as possible."

CHERISH, a Phase 3 study of nusinersen, is a randomized, double-blind, sham-procedure controlled fifteen month study in approximately 117 children who are non-ambulatory with SMA between the ages of 2 to 12.

The study will evaluate the efficacy and safety of 12 mg doses of nusinersen 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. Additional efficacy endpoints are also included in the study.