Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN Rx for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies

Isis Pharmaceuticals, Inc.

announced today that it has initiated an open-label extension study, SHINE.  The SHINE study provides ISIS-SMNRx to infants and children with spinal muscular atrophy (SMA) who have completed their participation in the Phase 3 ENDEAR and CHERISH studies and are eligible to participate in SHINE. All patients in the SHINE study will receive a 12 mg dose of ISIS-SMNRx every four months for infants who completed ENDEAR or every six months for children who completed CHERISH.  Isis has earned an $11 million milestone payment from Biogen for the initiation of the SHINE study.  "Both the ENDEAR and CHERISH studies are enrolling on track.  We are pleased to initiate the SHINE study; thus making ISIS-SMNRx available to the patients who have completed one of our Phase 3 studies, ENDEAR and CHERISH," said B. Lynne Parshall, chief operating officer at Isis Pharmaceuticals.  "We sincerely appreciate the dedication and support of the SMA community, the patients and their families for participating in and helping us complete the ISIS-SMNRx Phase 3 program.  Together with Biogen, we are committed to advancing ISIS-SMNRx toward the market as rapidly as possible.  The initiation of the SHINE