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OFP

02/13/18 9:32 AM

#140904 RE: Investor2014 #140900

Why base the dosing of Rett girls on mouse data when we have data from multiple dosing schemes in humans?

I suspect they did try to base it on human data with using the PK data to target blood levels that were associated with better response in the P2a AD trial. The FDA rejected on the basis that that dosing level was not tolerated by the majority of subjects in that trial. Just to anticipate...that is all speculation.

They could have used mouse data as well but the equivalent doses would have been even higher and therefore likely even more out of reach.
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F1ash

02/13/18 9:33 AM

#140906 RE: Investor2014 #140900

Why base the dosing of Rett girls on mouse data when we have data from multiple dosing schemes in humans?



Rett is not Alzheimer’s, Alzheimer’s is not Rett.

You can use the human “safety data” from Alzheimer’s, but should probably check to see if that dosage (translated to mouse dosing) was effective in the Rett mouse models, don’t you think?