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Re: InTheTrenches post# 14502

Wednesday, 09/04/2019 3:01:34 PM

Wednesday, September 04, 2019 3:01:34 PM

Post# of 21531
That would be my take. There are many different measurements or endpoints for Rett but certainly a P3 will be needed. Truthfully I don't how many patients it would take for a pivotal trial if Neuren puts out stat significant results. My guess is there will be some flexibility because there are so few actual patients to start with so I doubt it would take 1000 to be honest. I think after years of AVXL holders hoping, due to a worldwide Rett data base, that they could just immediately throw 15 people into a trial, that it simply isn't how it works. Rett is bad news and I hope 273 will work, but simply hoping someone will see a girl doing better in a double blind placebo controlled trial being the end all, be all when Neuren is showing what it will take per the FDA, I think is more than wishful thinking.

Alkon chose the path that no one else is choosing, moderate to severe AD. It's quickest and most cost effective and we'll know soon. When your career is in AD, you don't gamble the company on a small indication as some seem willing to do. We'll get our crack at multiple CNS diseases if this trial pans out. I think it will be partners that pursue that however as we continue to push for AD approval.
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