Let's start by reviewing some earlier news on RNA's drisapersen.
1) I'll start by reposting something I wrote after RNA released info on its last trial:
Prosensa doesn't work. Go to the PDF linked to in the comments section of the latest seeking alpha article on SRPT. Look at slide 30. There you will see individual outcomes for all twelve participants in the Prosensa trial. Five of the twelve Prosensa kids declined MASSIVELY from their original 6 mwt baseline. One definitely became non-ambulant. A second dropped from 310 to 75 before quitting the study.
Now stay on the same slide and move over another column or so to "change from original baseline to extension baseline." Only THREE, yes THREE, of the twelve actually improved (this after 7 weeks). By week 93 (tons of time to build dystrophin), only 6 of 12 had increased (final column). In other words, whoever said Prosensa is "just as good as SRPT except for kidney toxicity" is SIMPLY WRONG. But let's continue (slide 31, the scary one): ALL TWELVE experienced PERSISTENT INFLAMMATORY REACTIONS at the injection site. ALL TWELVE experienced higher levels of alpha-migroglobulin in the urine. ALL TWELVE experienced PROTEINURIA. SIX experienced higher cystatin-c levels in urine. THREE experienced liver problems (higher g-glutamyltransferase). THREE experienced abnormal changes to the blood (decrease in thrombocytes).
2) Let's add another piece of data: Apparently it was revealed yesterday, that those injection site reactions are NO JOKE. Some poor boy has chosen certain decline and death rather than endure the drisapersen injections any longer. You don't think we need to get that boy eteplirsen ASAP?
3) Some months ago, Ian Estepan (who stays in contact with Jenn McNary and other DMD moms) tweeted that some of the strongest advocates of eteplirsen are the mothers of kids in the drisapersen trial. He also stated that there was considerable anger against GSK for the way they treated the kids in the trial.
Which SA article is the PDF link posted to - couldn't find it? Your comments above are in conflict with the CEO's at Wedbush - he indicated that there were no drop-outs from the 12 boys in the Phase II study. Looking at the slides from the Wedbush presentation, it appears that the initial treatment phase of the study was only about 18 weeks - they had an initial treatment phase of 5 weeks of dosing followed by a "follow-up" period of 13 weeks, during which it is not clear if the boys were given any drug? After about a year anda half in the extension phase of the study, they switched to "intermittent" dosing, no doubt to minimize the safety issues. Obviously, the side effects would have to be severe for a parent to pull their child from the study - I'm sure once you drop there is no getting back in.
Hi, Tredleon, You will need to check with Ian Estepan on twitter for the source for the boy dropping out. He announced that yesterday, during the PPMD call, which unfortunately I did not hear. I gather it was discussed then. I pass it along because Ian is a professional, not an anonymous stockerjocker-type rumor monger. If the RNA CEO said there were no discontinuations during the p2 study, he was being intentionally cagey by not saying it about the p3 study. RNA has to be consistently obfuscatory to hide how poor their results are. Did you notice how he has to use terms like "in general" to describe dystrophin or side-effects? That's called equivocating. RNA will get its comeuppance. Eventually it all comes to light.
The SA article which linked to the outcomes was from Fall 2012, which is when I originally posted that information. I don't have the exact date handy, and, as you know, the inability to post links makes discussions like this difficult.