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Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy
Date:8/1/2014

Hobby, president of Families of SMA.  "SMA is a devastating disease with no current therapeutic options.  Families of SMA applauds Isis for progressing its development program in an expedient manner, and looks forward to additional trials in patients with SMA beginning later in 2014."

ENDEAR, a Phase 3 study of ISIS-SMNRx, is a randomized, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with SMA.  The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx with a primary endpoint of survival or permanent ventilation.  Additional efficacy endpoints are also included in the study.  For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMNRx or the identifier number NCT02193074 or visit the ISIS-SMNRx study site at www.smastudy.com

ABOUT ISIS-SMNRx
ISIS-SMNRx 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-SMNRx for the treatment of patients with SMA.  Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMNRx, 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-SMNRx: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licen
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SOURCE Isis Pharmaceuticals, Inc.
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