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

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