The data are excellent...perhaps this more common sense item might have been missed in all of the emotions...
This is terrific news. Stabilization of performance over more time. Don't let the shorts and day traders scare you out of your shares.
Something I realized about the 6MWT numbers themselves. Consider the average ADULT brisk walking rate is about 3.3 mph or equivalently 5 km/h. This means, given that the kids are not allowed to run during the test and, in addition, have shorter stride length than the average adult (since their legs are shorter) I would not expect even healthy kids to WALK much more than 500 meters in 6 minutes. These kids were mostly walking 300-400 m in 6 minutes. So these kids were partially chosen so as to have a large sensitivity to the expected drug therapeutics because they were at a point at the start of the trial where they were walking near the expected amount for their age--even if healthy---but also chosen at a time point where the disease caused dystrophin degradation would show the most observable negative effect for the 6 MWT as well as other tests. ( Apologies to any DMD patients or parents--I imagine the other effects not measured are heartbreaking as well). In other words, the drug trial was designed for maximum signal sensitivity which makes complete sense. You might expect some small improvement in 6 MWT distance but not likely more than 50-100m--perhaps 10-20%. On the other hand, without the drug you could, sadly, expect a number of them to non-ambulatory. The sensitivity of the trial, regarding 6 MWT, was weighted toward preventing the negative outcome, not necessarily showing huge positive increases in distance. I think Max's ability to walk 3 miles shows what huge positive increases are possible and were not really tested in 6 MWT--and that is muscle endurance. I hope all of the trial boys can improve their endurance like that!
So, my point here is that improvement from a fairly high level of 6 MWT performance (for even healthy kids) to start with may be unrealistic. While just maintaining that performance is a HUGE deal. To reiterate what others have said but perhaps in a slightly different way--the slight decline in 6MWT is a minor noise term in the significance of the data and is, of course, being exploited by those that want cheaper shares after eteplirsen has been completely de-risked and proven to have excellent efficacy.
Remember, the big picture is outstanding. The drug is safe and effective. There are no competing treatments for these sufferring boys and they need eteplirsen yesterday! As we have seen, every piece of new data bolsters the results and thesis behind the drug and the entire DMD treatment platform for the remaining exons. IMHO.
The issue the market has is that the gains in 6 mwt from 36 to 48 wks were lost from 48 to 62 weeks.Your post skirts around that.See chart on pdf of backup material for Dr. Mendell's talk
yesterday.Did he address that?
Sorry, but you cant measure what was lost by the treated group relative to the treated group, you have to measure it related to the untreated group. In this case, the difference is immensely positive. Much of the treated group (and the placebo crossovers -check out the trendline, and published natural history data) would be non-ambulatory by now. But even compared to the placebo crossovers, the initial treatment group haven't lost - they are stable.
Next time think before expressing. That way you will be expressing your thoughts.