Ataluren update

Some further analysis has come out from the Ataluren/PTC124 study. Here is the document if you want all the technical details: ATALUREN . Basically, they found that the boys that were on the lower dose actually did BETTER than those on the higher dose, and in fact, those on the high dose performed almost the same as those on the placebo. The last 48 weeks of the study, all the boys were on the high dose, so when it turned out that this was not showing any improvement, they shut it all down (it was a double blinded study so nobody knew which boy was taking what for the first parts of the study when they were doing low, high, and placebo doses. They must have just assumed that the boys that were responding were on the high dose, so that's why the 2b phase was all high dose?).

So it's promising at least. I'm not a stats person, but they were looking for 95% confidence with whatever system of analysis they were using, but they only got 85%. So I have no idea what that means for the future of the drug. Will they have to do more studies on the lower dose? Will they be able to present for FDA approval based on what they have? I don't think so on that one, because of that whole 95%/85% thing. I think they are still going to look a little deeper to see if whether where the stop codon is on the gene or which amino acids are switched around has anything to do with response.

It's at least encouraging that a lot of boys showed some improvement on the drug. Now, when I say improvement, they were looking at the walk distance, and the low dose boys' walk distance decreased LESS than the high dose/placebo. So there was still decline noticed, it was just much slower. If it's all there is, I'll take it. Maybe it would at least slow the progression enough that something else would come up in the meantime. Maybe it means he can stop taking the steroids and grow normally and not worry so much about breaking bones. I just don't think it will be approved as soon as we thought it would (later this year), but at least I think the company will continue development since it is showing some promise. Fingers crossed that it will move along quickly.