- Main point is that once a patient starts to decline, that predicts future decline
- Most of his natural history data is from Atalauren trial placebo group
- Time to 10% progression (10% lower 6MWD) is predicted by being 410 meter initial 6MWD, you have a 40-50% of a 10% decline over 48 weeks.
- Figure 10a. ~40-50% of patients with initial 6MWD greater than 410 meters had 10%, or greater, decline over 48 weeks – 0/6 Sarepta patients (average initial 6MWD 389M) who received eteplirsen have declined 10% since producing dystrophin.
- Also Figure 10a. ~90% of patients with initial 6MWD 10% and had average initial 6MWD of 328M at time before dystrophin confirmed (@36 weeks) have now stabilized & none have even declined 5% over the next 48 weeks. (1 patient didn’t get 6MWD at the last timepoint due to broken bone, but was stable, and will participate in future 6MWT).
These numbers make it clear that it would be incredibly unlikely that patients that were on the decline, 7 years old, & with low initial 6MWD, would remain stable for 48 weeks.
As others have said, for a lethal, degenerative disease, with patients willing to tolerate some risk in treatment, Eteplirsen has demonstrated impressive clinical efficacy versus the expected natural history, and has had very little indication of any toxicity.