This is the history of this masquerade:
In April 2012 a company named AVI BioPharma reports results of a 24 weeks Phase 2 double blind randomized placebo controlled trial in 12 DMD patients. The study shows a 22% increase in dystrophin levels as compared only in the lowest eteplirsen treated group (30mg) but not 50 mg. Supposedly 50 mg dystrophin levels were only measured at 12 weeks but we don’t know why. Importantly the study show’s no increase in 6 minute walk distance for treated patients as compared to placebo. AVI stock goes nowhere after release of the data.
In July 2012 the company changes its name to Sarepta. Same company, same product, same data.
In October 2012 Sarepta management introduces the concept of Modified Intent To Treat to analyze the data. May be they should also have changed their name to MAVI (Modified AVI), because this is what Sarepta is.
The key aspect of the modified analysis is that they now drop two of the 8 patients in the treated group who had a dramatic decline in walk ability following initiation of treatment with eteplirsen. By doing so the study now starts to look positive. It’s a miracle!
Wrong again. The eteplirsen trial primary endpoint was muscle dystrophin levels. There is no evidence that walking speed over 6 mintues was unaffected at 24 weeks, and the primary dystrophin endpoint was met.
I work with data analysis in Minitab on a daily basis. Sometimes outliers need to be dropped to make the data look better. It's pretty easy, just need a good excuse to drop the points. When you only have 8 points and you drop 2 outliers.....BAMMO.....perfect data! And SRPT is the perfect short at the moment.
Supposedly 50 mg dystrophin levels were only measured at 12 weeks but we don’t know why.
Everyone else knows why they didn't do a biopsy at 12 weeks.
I suggest you undergo the procedure a fourth time and then you will understand why they only did it three times.
This isn't a simple biopsy, but an opening to allow the surgeon to collect tissue samples from two muscles. There are pictures shown.
The reason they chose 12 weeks for the 50 mg/kg and 24 weeks for the 30 mg/kg was to understand the relationship between treatment time and dosing amount.
If one is familiar with the natural history studies of duchenne, it will be easy to understand how miraculous this drug is!