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news.Isis Pharmaceuticals has earned a $2 million milestone payment from Biogen Idec related to the advancement of the ongoing open-label extension study of ISIS-SMN in children with spinal muscular atrophy (SMA).
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The open-label extension study of ISIS-SMN is offered to those children with SMA who have completed dosing in Isis’ previous studies.
In this study, children with SMA are receiving a 12 mg dose of ISIS-SMNRx every six months for the duration of the study.
ISIS-SMN is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.
The United States Food and Drug Administration granted orphan drug status and fast track designation to ISIS-SMN for the treatment of patients with SMA. Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMN, to treat all types of SMA.
Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study or the completion of two Phase 2/3 studies.
Isis acknowledges support from the following organizations for ISIS-SMN: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.