Eteplirsen Updates from the World Muscle Society Meeting

Sarepta provided an 144 week update on eteplirsen at the World Muscle Society meeting. Here are some brief notes.

• Neutral charge as it passes through kidney; cleared through kidney
• IV QW
• Inclusion: >7 years to 13 years (original cohort 7-11 yr)
• ITT: 12 pts (4 each placebo, 30 & 50 mg/kg)
• 2 pts stopped walking prior to 24 week time point
• Data at 144 weeks for modified ITT: 10 pts (6 pts eteplirsen and 4 placebo delay)
• At 144, delta 75 m (-107 vs –32)
• Both groups start producing dystrophin after 12 wk Tx, increasing over time
• No complete resolution, plateau and then slow decline; imperfect drug
• Slower decline in walking than natural history
• Pulmonary function test; remain stable (no stat diff)
• MIP and MEP stable
• No AE at 144 wk; no liver enzymes, kidney fx, coagulation, etc.
• No ISRs, etc.
• Pooled the doses in the data; no diff between dose levels