SLDB:
I heard the call. I am long SRPT (no SLDB) but think that post is misleading. The patient was given only 3 doses of Soloris. Furthermore (as I'll describe below) I HIGHLY doubt they were given a full go ahead and FDA has several concerns!
That being said I don't know how their GT will turn out but I don't trust management at all! Notwithstanding the IPO disclosure fiasco, this call had numerous concerns that management is being less than forthright in their disclosure. If one reads between the lines of what was said (and more importantly what wasn't) there are clearly some regulatory limitations. I suspect they have some serious concerns go ahead (if they really don't know the cause) or are misleading investors. Just a few things:
1-New Steroid course may affect efficacy (their answer was deliberately misleading IMO)
2-Now dosing just ambulatory children (instead of older non-ambulatory). Why? didn't say but could it because the total amount of dose is a concern with FDA possible safety concern?
3-Moved up one of the biopsies to as early as 6 weeks. Didn't say but I suspect they may be thinking efficacy is impacted by the new steroid protocol.
4-Didn't know when could move to the higher dose....That has to BS I suspect they know its dependent on certain safety criteria but they, of course, didn't disclose that.
5-They said that it was clearly an immune reaction suffered by the initial patient but they did not believe it was manufacturing related. This is catch 22 IMO. Mfg is very important in GT and a problem here would be a major setback (delay) but I think a less serious one if it's fixable...The alternative something with the vector/promotor/transgene which would basically end the program.
There were numerous other things I didn't like about management on that call too! These were just top of my head.