Isis Expands Phase 3 Clinical Trials

Isis recently announced that they have tested the first patient in CHERISH, a multi-center Phase 3 clinical study evaluating the efficacy and safety of the investigational compound, ISIS-SMNRx, in non-ambulatory children with spinal muscular atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled study in approximately 117 children with SMA at study centers in the US, Canada, Europe and Asia Pacific.

CHERISH is the second Phase 3 study for ISIS-SMNRx. In September, Isis announced the launch of ENDEAR, a Phase 3 clinical trial to study ISIS-SMNRx in infants with SMA type I.

Additional Phase 2 Studies

In addition, Isis announced today that Biogen Idec, their clinical partner, will be conducting two additional studies which could begin in the first half of 2015:

  • NURTURE will be a Phase 2 clinical study evaluating ISIS-SMNRx in up to 25 pre-symptomatic newborns that are genetically predisposed to the disease.
  • EMBRACE will be a Phase 2 clinical study evaluating safety and exploratory efficacy of ISIS-SMNRx in approximately 20 patients with infantile or childhood-onset SMA. This study will bridge the gap in a small subset of patients that do not meet the age and inclusion criteria of the current Phase 3 studies ENDEAR and CHERISH.

As the ongoing trials progress, Isis and Biogen will be evaluating their clinical program and communicate any changes to our plans at that point in time.

More information on both ENDEAR and CHERISH can be found at www.clinical or

In addition, you can download a list of frequently asked questions, or see our recent news articles about ISIS-SMNRx and other clinical trials.

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