SRPT related predictions:
But first, a note - I am not trying to tweak anyone here. This is just the right board on which to post such things and SRPT has been in the news over the last month or two with significant articles in major newspapers. So it seems timely...
1) I predict that meaningful SAE will show up for Etep or its cousins for other exons. All but one of the oligonucleotides run in big RCTs have shown bad SAE related to oligo technology that was not readily apparent in the smaller, single arm or shorter ph2s. (interesting note - the only oligo that did not show oligo related toxicity in their ph3 RCT was miplomersen. It's interesting because it has really unpleasant SAE of another form - huge injection site reaction - and it has target releated SAE in liver enzymes. Finally note that in the REMs it has apparently shown thrombo, so it is not actually immune to standard oligo SAE.)
2) CG, the ex-CEO under whom most of the really poor science decisions were made, will become CEO of another company (he is now running an incubator) when he finds one that meets 3 of the following 4 conditions: a) treats devestating childhood disease, b) already a very strong/vocal parent advocacy group, c) the 'treatment' has an MOA that sounds, in a soundbite, compellingly like it treats root cause, d) any side effects are minimal or not observable in a small population. (Having these characteristics make for good politics to avoid running a meaningful RCT, but still be able to force approval.)
Random other note on oligo's: I now think it more likely than not that ALNY's TTR drug will show excess bone breaks (due to osteonecrosis - they had two in their fairly small, single arm, ph2).
AI
