Acadia Pharma: Positive Phase 2 Pediatric Study in Neurology Journal

— Statistically Significant Improvement Indicates Trofinetide’s Potential for Treating Rett Syndrome

— Trofinetide for Rett Syndrome has Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in Europe

ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD), Neuren Pharmaceuticals Limited (ASX: NEU), and Rettsyndrome.org (RSO) announced today that the positive results from a Phase 2 study conducted by Neuren, which evaluated the safety and efficacy of trofinetide in females with Rett syndrome (RTT), have been published in Neurology^®,the medical journal of the American Academy of Neurology.

ACADIA plans to initiate a 12-week Phase 3 double-blind, randomized, placebo-controlled study evaluating trofinetide in the second half of 2019 following completion of additional manufacturing scale-up activities. This study will evaluate efficacy and safety of trofinetide and placebo in approximately 180 females ages 5 to 20 years with Rett syndrome. Half of the study participants will receive trofinetide and half will receive placebo. The study will use the RSBQ and the CGI-I syndrome specific efficacy measures as co-primary efficacy endpoints. The Phase 3 study will be followed by a nine month open label extension study in which all participants, including those on placebo in the Phase 3 study, will be eligible to receive trofinetide. In the open label extension study, all participants will be followed to evaluate long term tolerability and safety of trofinetide.

https://www.marketscreener.com/ACADIA-PHARMACEUTICALS-IN-8222/news/ACADIA-Pharmaceuticals-Positive-Phase-2-Study-Results-of-Trofinetide-in-Pediatric-Rett-Syndrome-Pu-28248796/