Isis Pharmaceuticals receives $2.15 million milestone payment to advance ISIS-SMN Rx study in children with SMA

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $2.15 million milestone payment from Biogen related to advancing the ongoing pivotal Phase 3 study (CHERISH) evaluating ISIS-SMN_Rx in children with spinal muscular atrophy (SMA). To date, Isis has generated more than $120 million in payments from Biogen related to the development of ISIS-SMN_Rx.

CHERISH, a Phase 3 study of ISIS-SMN_Rx, is a randomized, double-blind, sham-procedure controlled fifteen month study in approximately 120 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 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. Additional efficacy endpoints are also included in the study.