Sarepta Therapeutics Eteplirsen Update
Posted by christinemcsherry on July 25, 2012
I’m sure many of you have already seen this, but we recently received this update from Sarepta Therapeutics:
Today, we announced the 36 week clinical outcome results from our on-going open-label Phase 2b study of eteplirsen in DMD. Treatment with eteplirsen, an investigational exon 51-skipping therapy, achieved a significant clinical benefit on the primary clinical outcome, 6-minute walk test (6MWT), over a placebo/delayed treatment cohort. Eteplirsen administered once weekly at 50mg/kg over 36 weeks resulted in a benefit of 69.4 meters over a cohort of patients who received placebo for 24 weeks, followed by 12 weeks of treatment with eteplirsen in the open-label extension. Please see the attached press release or click here for more details of the 6MWT results.
More from their press release:
Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne muscular dystrophy (DMD) patients. Eteplirsen administered once weekly at 50mg/kg over 36 weeks resulted in a 69.4 meter benefit compared to patients who received placebo for 24 weeks followed by 12 weeks of treatment with eteplirsen in the open-label extension. In the predefined prospective analysis of the study’s intent-to-treat (ITT) population on the primary clinical outcome measure, the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50mg/kg of the drug weekly (n=4) demonstrated a decline of 8.7 meters in distance walked from baseline (mean=396.0 meters), while patients who received placebo/delayed-eteplirsen treatment for 36 weeks (n=4) showed a decline of 78.0 meters from baseline (mean=394.5 meters), for a statistically significant treatment benefit of 69.4 meters over 36 weeks (p≤0.019).