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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

of the disease provides the greatest opportunity to reap substantial improvements in muscle strength and function, potentially resulting in a lifetime of benefit. ISIS-SMNRx targets the underlying primary cause of SMA, taking advantage of the backup gene, SMN2, present in all SMA patients," said Kathryn J. Swoboda, M.D., professor, department of neurology and director of pediatric motor disorders research program at the University of Utah, School of Medicine.  "Preliminary results from Phase 1 clinical trials reveal a favorable safety profile, and appear to indicate that a single dose of the medication at the higher doses tested to date may result in sustained benefit over many months in some children. Thus, while early, we are excited to continue to work closely with Isis in further studies of ISIS-SMNRx in SMA."

"We are pleased with the progress we are making on ISIS-SMNRx.  Although there was no placebo group, the continuing improvement for up to a year after a single dose observed in this study is encouraging, particularly when considered within the context of the dose response," said B. Lynne Parshall, chief operating officer at Isis.  "Our ongoing Phase 2 program is proceeding well. The 6 mg dose group in our Phase 2 study in infants with Type I SMA has completed dosing.  Based on the safety, pharmacokinetic and pharmacodynamic profile of ISIS-SMNRx we have observed to date, we have amended the infant study to increase the dose from 9 mg to 12 mg dose. We plan to start dosing this cohort soon.  In our Phase 1b/2a multiple-dose study in children with Type II and Type III SMA, we have completed dosing in all three dose cohorts (3 mg, 6 mg and 9 mg), and we are considering adding a 12 mg dose cohort to this study.  The FDA has expressed reservations about increasing the exposure in children with Type II and Type III SMA, and
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SOURCE Isis Pharmaceuticals, Inc.
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