Acadia reports positive Phase 3 trofinetide results in Rett syndrome; NDA plan mid-2022

ACADIA PHARMACEUTICALS INC

Trofinetide met co-primary endpoints: RSBQ change -5.1 vs -1.7 (p=0.0175); CGI-I 3.5 vs 3.8 (p=0.003).
Key secondary endpoint CSBS-DP-IT-Social also met: -0.1 vs -1.1 (p=0.0064); effect size 0.43.
Discontinuation due to AEs 17.2% trofinetide vs 2.1% placebo; diarrhea (80.6%) and vomiting (26.9%) most common.
Pre-NDA meeting with FDA planned Q1 2022; NDA submission expected mid-2022; Fast Track, Orphan, RPD designations granted.
More than 95% of completers rolled into open-label Lilac extension; Acadia licensed trofinetide from Neuren Pharmaceuticals.

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