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Isis Reports Follow-Up Data From ISIS-SMN Rx Phase 1 Study In Children With Spinal Muscular Atrophy
Date:9/19/2013

we are in ongoing discussions with them.  We plan to report data from both of these ongoing studies late this year or early next year.  We also plan to begin our Phase 3 clinical program early next year."                                             

The Phase 1 study was an open-label, single-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of ISIS-SMNRx in medically stable children from age 2-14.  In this study, children with Type II or Type III SMA received ISIS-SMNRx as a single dose of 1, 3, 6, or 9 mg administered intrathecally.  In addition to measurements of drug concentration in plasma and cerebral spinal fluid, exploratory analyses of changes in motor function were conducted.  Gross motor movements were measured using the HFMSE, a modified version of the Hammersmith Functional Motor Scale.  The HFMSE is used to assess responses on 33 motor function tasks, each scored on a scale from 0 to 2 and allows for assessment of any SMA patient aged 2 or older.  HFMSE has demonstrated good test-retest reliability in other studies.

Data from this study was reported at the American Academy of Neurology in March 2013, showing that a single-dose of ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in HFMSE scores in a number of children, with a mean increase in HFSME scores for the 9 mg cohort at three months was 3.1 points.  The data to be reported at the World Muscle Congress in October is a follow on analysis of HFMSE in 24 children who had completed the Phase 1 study.

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SOURCE Isis Pharmaceuticals, Inc.
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