Phase 3 trial of trofinetide will take place in the US during the latter half of 2019

4 February 2019 – Neuren Pharmaceuticals advised the Australian Stock Exchange, Melbourne, of a statement released by ACADIA Pharmaceuticals about its plans for the Phase 3 trial of trofinetide in Rett syndrome. As with Phases 1 and 2, the trial will be held in the US. In part, ACADIA stated that

We would like to share our plans for the Phase 3 study of trofinetide for Rett syndrome starting in the second half of 2019 which have now been confirmed. 

 ACADIA plans to conduct a three-month Phase 3, double-blind, randomized, placebo-controlled study to 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 Phase 3 study will be followed by an 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………………………. The ACADIA Rett Team“.


In the beginning
Distinguished Professor Dame Margaret Brimble, co-creator of NNZ-2566 which was to be later renamed trofinetide, gave a presentation on the drug at Rett New Zealand’s bi-annual family conference which was held in Auckland in May 2014. A recording of her talk can be found by clicking on the following link which is contained on the Rett New Zealand’s website

February 08, 2019
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RETT Syndrome Association of Australia