Isis Pharmaceuticals Announces Open-Label Extension Study SHINE in Pediatric SMA Patients


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Isis Pharmaceuticals, Inc. recently announced the official launch of an open-label extension study, called SHINE, which will be continuing treatment of the same infants and children diagnosed with spinal muscular atrophy (SMA) who had previously participated in Phase III studies ENDEAR and CHERISH. The infants whi had participated in ENDEAR will continue to receive 12mg of investigational product ISIS-SMNRx every 4 months, while those from CHERISH will receive treatment every 6 months. Following the launch of SHINE, Isis will be receiving a milestone payment worth $11 million from Biogen.

“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.”

Partners Isis and Biogen are currently studying the performance of ISIS-SMNRx in an extensive clinical program. A pair of Phase III randomized, double-blind, sham-procedure controlled trials of ISIS-SMNRx (ENDEAR and CHERISH) are currently under the care of Isis, with the company expecting the first clinical report to come in later in 2016 or early in 2017. ENDEAR is a 13-month study involving about 110 infants with SMA, while CHERISH spans 15 months and counts with the participation of about 120 non-ambulatory children with SMA.

According to a company press release, aside from Phase III studies ENDEAR and CHERISH, Isis drug candidate is also being evaluated by the following Phase II investigations:

  • Biogen is evaluating ISIS-SMNRx in an open-label study, NURTURE, in approximately 25 pre-symptomatic newborns who are genetically diagnosed with SMA but presymptomatic.
  • Biogen is evaluating ISIS-SMNRx in a randomized, double-blind, sham-procedure controlled study, EMBRACE, in 21 patients who do not meet the age and inclusion criteria of ENDEAR and CHERISH studies.
  • Isis is evaluating ISIS-SMNRx in a Phase 2 open-label study in 20 infants with SMA. Infants in this study have been on treatment for up to 29 months. In June 2015, Isis reported that it had observed increases in median event-free survival and increases in muscle function scores as well as the achievement of developmental milestones in infants who received ISIS-SMNRx in its open-label Phase 2 study.
  • Isis is evaluating ISIS-SMNRx in a Phase 2 open-label extension study in 30 children who have completed dosing in one of the earlier ISIS-SMNRx studies. Patients in this study have been on treatment for up to 46 months. In June 2015, Isis reported that it had observed increases in muscle function scores and additional motor function tests in children who received ISIS-SMNRx.

The company has also finished dosing in several ISIS-SMNRx trials that sought to evaluate the efficacy of either single or multiple doses of the drug in 56 children with Type 2 and Type 3 SMA. The patients who have completed these dosing studies have the option to proceed with a Phase II open-label extension study.