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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

CARLSBAD, Calif., Sept. 19, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA), show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.  The Phase 1 data, including these preliminary follow-on data, will be presented at the International Congress of the World Muscle Society by Dr. Kathy Swoboda on Oct. 3, 2013.  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.  ISIS-SMNRx is an antisense drug designed to treat all types of SMA.

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The preliminary data reported today is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx.  Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the Hammersmith Functional Motor Scale-Expanded (HFMSE).  The improvements in HFSME scores were dose dependent with the largest improvements observed in children in the highest dose cohort (9 mg, mean = 5.75). Most children in the 9 mg dose cohort showed continuing improvements during follow up, with no children declining.

"SMA is a devastating disease that results in severe muscle weakness and respiratory compromise in the majority of affected patients. Treating children early in the course
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SOURCE Isis Pharmaceuticals, Inc.
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