Isis Pharmaceuticals, Inc. announced 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.
“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 study supports that commitment and ensures that patients who completed the controlled portion of the Phase 3 program have continued access to ISIS-SMNRx.”
Ongoing Clinical Trials
In addition to SHINE, four ongoing trials are testing ISIS-SMNRx. The others are:
- ENDEAR, a Phase 3 clinical trial testing infants with SMA type I
- CHERISH, a Phase 3 clinical trial testing children age 2-12, with onset of symptoms at age six months or later
- NURTURE, a Phase 2 clinical trial testing very young infants who are genetically predisposed to SMA but not yet showing symptoms
- EMBRACE, a Phase 2 clinical trial testing a small subset of children and infants who do not meet ENDEAR or CHERISH criteria