I'm not sure why people are so excited that the competition's drug helped 72% of patients, that is a pretty high number - and when dosing was decreased/intermittent it resulted in fewer patients showing increased dystrophin production - establishing a direct correlation between the dosing of their drug and dystrophin e production.
I must be missing something here. Sure SRPTs drug is better/more effective, but the competition's drug was just shown to be effective. Not to mention that the FDA rejected our attempt to use dystrophin increases as a surrogate endpoint.
I'd much rather have had the competition's drug be completely ineffective (in terms of SRPT share price - it is of course good for patients). 72% is an incredibly high number.
What am I missing?
Clearly the board doesn't want to hear anything different from their line of thinking. That is OK, and I admire their zeal. Be careful though, complacency is a dangerous thing.
Still, nothing changes the fact that NDA is a year away, and FDA approval 6-12 months after that. The FDA cannot approve an NDA never filed, so this part is on the company.
I disagree. The data released by the prosensa trial helped SRPT by establishing the connection between dystrophin and clinically meaningful result..walking test. The continuously treated arm saw waking benefit while the intermittently treated cohort did not. The continuously treated arm saw 72% of boys register higher dystrophin while the intermittently treated arm saw 59%. There is an apparent minimum dystrophin threshold below which no clinically meaningful result exists. My take on the FDA and SRPT is this...SRPT has good data but it is limited. I believe the FDA just needs to see more of the same compelling data to creat a larger body of work to support eventual approval, and that will be when SRPT continues to show the strong correlation between dystrophin and walking function over the next 6 months...with no adverse safety concerns.
Intermittent is what has to be used due to toxicity. These numbers are weak at best. Also since no real baseline is established in these younger boys the failed stat sig for 6mwt gives more evidence that it is weaker in the 59% that responded, and they responded slower. It is win win win for all measured of efficacy. Safety was already known. Now efficacy advantage is clear as well
You're missing the most basic point. With this disease, 72% doesn't cut it, especially when it is accompanied by serious side effects. What parent or doctor is going to want to put their child on a drug where there is a 1-in-4 chance it won't work and leave the child deteriorating and dying? This is a death blow to Prosensa's drug - they have no chance of marketing this drug against a drug that is 100% effective with a pristine safety profile.
C'mon, System Architect, don't be obtuse. Yes, Prosensa's drisapersen shows hints of a dosing effect, but side effects have FORCED Prosensa to test only suboptimal doses. Their dose is one-fifth of Sarepta's low end (6 vs. 30). And there's nothing Prosensa can do about it, unless they want to start a new trial in which they risk frying kidneys!
Make no mistake about it, if it were my child, I'd have them on Eteplirsen but as an investor, I have to realize that there will always be some people, for whatever reason, who will opt for a lesser alternative.
I'd also hate the delivery method of Drisapersen, but some will still do it.
What your missing is that it is not 100% like Sarepta... and anyone who though their delivery method was quicker than our intravenous delivery is now wrong.
We know that they have not reached statistical significance in 6mwt.... WE HAVE. This is good for our drug