Favus reported that out of the 12 subjects in the clinical trial, 4 were receiving placebo. Out of the 8 subjects receiving treatment, two were dropped from the study due to clinical decline, despite the fact that they were producing dystrophin.
This might suggest the dystrophin produced is non-functional?
Interested in hearing what you think of the Favus report, and how this will effect approval, etc. How much will this hurt the timeline, etc. Is the pullback overdone, or will it continue?
The next bogey to dispute is the small sample size.
My math is approximate here but kept simple on purpose:
Out of roughly 4MM births each year in the U.S., roughly 1/2 are boys and 1 in 3,500 of those will have DMD. That’s 571 boys per year. Of those 13% could benefit from skipping exom 51; that’s 71. The boys chosen for the clinical trial were by design ones that were in decline or near that point, so let’s say 7, 8 and 9 years old. So the potential population for a trial was about 210 boys. So 12 were chosen or 6% of the proper subset of the DMD population.
However, when this is approved, there are a potential 1,500 boys that will be helped by etep immediately and an 71 additional each year to help; not counting the ROW of course.
The 8, then 12, (6% roughly of the age cohort) show remarkable stabilization and possible improvement at a time when they should be well into a rapid decline. The population is small and the sample is actually large compared to the population and that is why consistent stabilization has such a strong statistical significance against the population.
Then of course, the other exoms are easy to fix when this is approved, you do the math on that.
Answering myself again..
SRPT is trashed for having a small sample size; however GSK/RNA is not. Consider this,
GSK/RNA drew their trial participants from the world. So 5B/300MM is 16.6. 16.6 x 12 is 200. GSK/RNA's sample size given their DMD populaion to draw from was 182 I believe. So in terms of sample precentage based on DMD population picked from, SRPT was a larger % but very similar %; AMAZING.
You guy are fixated on the absolute #, when population and sample size of that populaton is what's important. These trials were designed by experts in statistics, not hacks.
You need to read the 96 week data from the co.
4 on placebo were switched to drug and stabilized.
2 started on drug were older and lost ambulation after the trial started, before the drug took effect. They have since stabilized upper body strength.
They were dropped because they became unable to walk. This happened before any of the subjects started to show dystrophin production. What would be the point of measuring a 6 minute grovel?
The jury is out on these two in my book. It is possible that with continued treatment they may walk again.
Every pullback has been met with a rally with new data, leaving an overall uptrend in place. There's no reason to give credence to a hackneyed report. Pullback is just normal consolidation at the levels of old resistance. Longs took profit, shorts added positions; life is good.
He didn't suggest anything, the Favus report stated it. The report has already affected stock price.
The small group study, and the dropping of two of those involved may very well pose a problem with approvals. If the tredleon brain is functioning, then discuss the report without making Joy like attacks.