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Investor2014

07/08/19 1:03 PM

#200056 RE: Steady_T #200052

The n of the Rett trial is very small even if placebo controlled, so the much larger PDD trial would yield a more reliable indication of future success or not.

Doc328

07/08/19 2:23 PM

#200086 RE: Steady_T #200052

IMO, the Rett trial is too small and too short to give us a good indication about future success or failure of 2-73. With 15 patients, there are probably 5 placebo patients, 5 low dose and 5 high dose. Likely 2-3 girls dropped out. 7 weeks is not a lot of time for any separation to occur between treated and untreated and also adults (age 16-45 Rett) might respond less than pediatric populations. As an example trofinetide had a trend for adults in a larger study (age 16-45 n = 67) but was significant for age 5-15 for Rett (n=82). So, maybe we see a difference which can be positively PR'd but significance is unlikely. Therefore, I feel a big pop large enough to do an offering is unlikely, though a 1.00-2.00 might still be seen.

PDD is much larger so a positive outcome could greatly move the stock and would also give a better indication of AD outcomes.