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Isis Pharmaceuticals Earns $1.5M from the Advancement of the Phase 2 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy
Date:11/12/2013

CARLSBAD, Calif., Nov. 12, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $1.5 million milestone payment from Biogen Idec related to the advancement of the ongoing Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA).

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The Phase 2 study of ISIS-SMNRx is an open-label, multiple-dose, dose-escalation pilot study in infants who have been diagnosed with Type I SMA. To meet enrollment criteria, infants must be between the ages of three weeks and seven months, live in close proximity to a study site and pass screening evaluations conducted at study sites. The study is being conducted at centers in the United States and Canada. For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMNRx.

ABOUT ISIS-SMNRx
ISIS-SMNRx is an investigational compound 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 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-SMNRx is currently being evaluated in two Phase 1b/2a multiple-dose, dose-e
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SOURCE Isis Pharmaceuticals, Inc.
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