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.
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.