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Isis Pharmaceuticals to Investigate a Higher Dose of ISIS-SMN Rx in Children with Spinal Muscular Atrophy
Date:11/22/2013

160;In the ongoing Phase 1b/2a study in children with SMA, all patients have completed dosing in the initial three dose cohorts (3 mg, 6 mg and 9 mg) and ISIS-SMNRx has been well tolerated over multiple doses.  Patients who have participated in the Phase 1b/2a study are eligible to enter an open-label extension study, which is designed to provide a single additional dose of 12 mg to the more than 50 children with SMA who are eligible to roll over into this study.  The investigation of the 12 mg dose in both studies is anticipated to begin in December or January.

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. 

Isis acknowledges support from the following organizations for ISIS-SMNRx: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

ABOUT SMA
SMA is a severe genetic disease that affects approximately 30,000-35,000 patients in the United States, Europe and Japan. SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in the survival motor neuron (SMN) protein. SMN is cr
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SOURCE Isis Pharmaceuticals, Inc.
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