Careful_investor, you are not being very careful yourself when you publicly accuse a company of fraud/dishonesty. Sarepta can easily find out who you are through a subpoena issued to yahoo. Multi-million dollar judgments obtained by companies against individuals who have defamed them are not unheard of.
in the second half of 2015. According to its press release, Its nucleic acid drug is 25 fold more powerful than eteplirsen and reaches the diaphragm and heart muscles. Matthew Wood at Oxford is studying and promoting the drug: "The data we have seen to date using WAVE’s novel approach to exon-skipping in DMD is very promising. I believe that academia and industry, working together, may be on the verge of a veritable medical revolution where we can potentially effectively and durably treat genetically based diseases such as DMD.” The company says: "“Based on the strong preclinical data we’ve seen to date, we are highly encouraged that we are on track to develop exon-skipping medicines that maximize potency with a favorable safety profile. In addition, we plan to conduct rigorous, well-designed clinical trials that explore various predictive biomarkers and evaluate comprehensive endpoints. WAVE’s planned clinical trials will include both ambulatory and non-ambulatory patients in order to evaluate preservation of walking ability, heart and lung function."
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").