I suspect that Sanofi Genzyme is a lead candidate for acquisition or partnership. Dr. Jean-Paul Kress, the head of Sanofi Genzyme's Specialty Care Unit, which is the rare-disease arm of Sanofi Genzyme, joined Sarepta's board of directors within days of his leadership appointment at Sanofi Genzyme. From his Sanofi units webpage: "Every part of our Rare Disease franchise serves small patient populations through personalized care and highly specialized products." Sounds like Sarepta. No coincidence there.
Nippon Shinyaku just completed a two-year trial in Japan skipping Exon 53 in DMD children using morpholino chemistry. Sarepta will have its first real competition in exon skipping in the United States. I've expected a patent fight for years by Japanese companies since their duchenne exon skipping patents seem to pre-date both Sarepta's and Prosensa's patents. Now Nippon Shinyaku is testing the US/FDA waters with Sarepta's drug, but claimed as their own drug. Sarepta's exon 53 trial has not yet started in the US, though it seems to be moving along nicely in Europe.
An amendment this week to study 202 now caps the study duration at 236 weeks, or sooner if eteplirsen is approved. The amendment also changes the time for determining primary functional efficacy endpoint (6MWT), to be from baseline to 212 weeks (instead of to "end of study").