Biotech Values

[mcbio]

SRPT -

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.

What about their PPMO program? They have talked that up quite a bit. I believe it is set to enter clinic by end of this year. Do you think that can overcome the issues that you predict will plague etep and the other related more advanced exon skipping programs?