Ann: Positive topline results from Phase 3 trial in Rett syndrome, page-53

Had to go back to the initial Lavendar trial results to understand the substantial long term treatment benefit for those in both the Lavendar and Lilac Rett trials.

Lavendar results from 25 Feb 2022 NEU presentation.
Rett Behaviour Questionnaire change from base line at 12 weeks:

Placebo -1.7 Trofinetide -5.1 p=0.0175

In the Lilac study that followed Lavendar those on both placebo and Trofinetide were offered trofinetide for another 92 weeks. Total of 77 females completed both the Lavendar and Lilac studies, some took Trofinetide for 104 weeks, those initially on placebo took the drug for 92 weeks.

The mean change in Behaviour Questionnaire from base at week 104:

Initial placebo patients -13.8 Trofinetide patients -9.8
So there was about a doubling of performance benefit over the 2 years compared to the initial 12 weeks of the Lavendar trial.

These long term results were a great endorsement for me of efficacy so purchased more shares last Friday.