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jb_118

09/05/18 1:04 PM

#220710 RE: DewDiligence #220700

RE: SGMO

Selloff seems to be related to this line from the PR:

"At baseline and for the first 16 weeks post-dosing of SB-913, plasma IDS activity (measurements obtained at trough of weekly ERT dosing) was below the level of quantification of the current assay."

However, they showed a dose response decrease in glycosaminoglycans, dermatan sulfate and heparan sulfate (% change at 16 weeks of +9%, +4%, -23% respectively for first dose cohort vs. -50%, -42%, -51% for second dose cohort). Hard to say with just two doses and two patients per dose, but suggests there is enzymatic activity even though the enzyme itself is below level of detection. Second cohort was 2x the first cohort dose, and third cohort is 5x the second cohort dose. Third cohort has already been dosed.

Seems like 20% sell off could be overdone here, although being able to measure the enzyme directly would have given one more confidence. Third cohort dosing will be interesting.