I'm posting this for those who seem fixated on 'end of trial numbers' and their impact on the "other" drug. What lines are people drawing for the end of the phase 2 trial for eteplirsen versus the extension study numbers?
Not the full PR, just paragraphs
Jul 24, 2012 (Marketwire via COMTEX) --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).
Out of order paragraph to give additional context: "There was no statistically significant difference between the cohort of patients who received 30mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort."
When the dystrophin numbers came out after the 48 week biopsies, I think the 30 mg boys did better, not sure though.
Also in 2012 this was in one of Sarepta's updates, "Sarepta is optimizing sequences for two preclinical exon-skipping compounds, Exon 45 and Exon 50, and expects to file an investigational new drug application by the end of 2013." Sometime in 2013, it appears exon 50 was a business decision to hold off developing right now and may be the reason the Secklers are now highlighting the need for platform approval. Business decisions are very different animal in the biotech world.