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October 4, 2012

Sarepta Phase IIb duchenne muscular dystrophy study meets primary endpoint

Sarepta Therapeutics' Eteplirsen has met primary endpoint of increased novel Dystrophin and achieved significant clinical benefit on six-minute walk test after 48 weeks of treatment in Phase IIb Study in Duchenne muscular dystrophy.

Sarepta Therapeutics’ Eteplirsen, a treatment for duchenne muscular dystrophy (DMD), has met the primary endpoint of increased novel dystrophin and achieved significant six-minute walk test improvements after 48 weeks of treatment in a Phase IIb study.

Eteplirsen administered once weekly at either 30mg/kg or 50mg/kg for 48 weeks resulted in a statistically significant increase in dystrophin-positive fibres to 47.0% of normal.

Nationwide Children’s Hospital, the Centers for Gene Therapy and Muscular Dystrophy, director and Phase IIb study principal investigator Dr Jerry Mendell said; "By addressing the underlying cause of DMD, eteplirsen has demonstrated unparalleled effects on enabling dystrophin production and slowing the progression of the disease as measured by the 6-minute walk test, with no treatment associated adverse events."

The placebo/delayed treatment cohort, which received 24 weeks of eteplirsen at either 30mg/kg or 50mg/kg following 24 weeks of placebo, also showed a statistically significant increase in dystrophin-positive fibres to 38.3% of normal.

Eteplirsen administered once-weekly at 50mg/kg over 48 weeks demonstrated an 89.4m benefit in six-minute walk test (6MWT), compared to those who received placebo for 24 weeks followed by eteplirsen for 24 weeks in the open-label extension.

"By addressing the underlying cause of DMD, eteplirsen has demonstrated unparalleled effects on enabling dystrophin production and slowing the progression of the disease as measured by the 6-minute walk test, with no treatment associated adverse events."

The predefined prospective analysis of the study’s intent-to-treat population reported that the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50mg/kg of the drug weekly demonstrated an increase of 21.0m in distance walked from baseline (mean=396.0m).

Patients who received placebo/delayed-eteplirsen treatment showed a decline of 68.4m from baseline (mean=394.5m), for a statistically significant treatment benefit of 89.4m over 48 weeks, the company said.

No statistically significant difference between the cohort of patients who received 30mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort was observed.

Sarepta Therapeutics president and CEO Chris Garabedian said; "On a broader scale, these results signify the promise and tremendous potential of our RNA-based technology to impact and modulate disease at the genetic level, which may lead to first-ever opportunities to target serious and life-threatening rare conditions at the origin of disease."

The study observed no treatment-related adverse events, no serious adverse events, and no discontinuations across all the subjects through 48 weeks.

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