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07/20/23 10:06 AM

#423586 RE: Investor2014 #423570

The “20% more patients in the dose arms had to be down titrated” meaning at least 20% dosed patients didn’t reach the target dose, plus a portion of the 14.3% who stayed on 10mg obviously didn’t reach 30/50mg. The bottom line is in adult Rett p2b/3, 20% patients didn’t reach 30mg. In AD p2b/3, 20-34% patients didn’t reach target 30/50mg.

It’s not a safety issue, a tolerability issue to some extent, but efficacy suffers when 1/3 patients are not on targeted dose on an already small n. Why this was not caught in the 132-patient PD trials? The PD patients were even older and had similar dementia scores?