Ann: Neuren FDA meeting confirms Phase 3 plan for Rett syndrome, page-2

Neuren confirms Phase 3 plan for Rett syndrome at FDA Meeting Melbourne, Australia, 13 October 2017: Neuren Pharmaceuticals (ASX: NEU) today announced that at its End of Phase 2 Meeting, the US Food and Drug Administration agreed with Neuren’s proposal for the key elements of its clinical development program to support a New Drug Application for trofinetide to treat children and adults with Rett syndrome. Neuren will conduct a single pivotal Phase 3 trial, using the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression of Improvement (CGI-I) as co-primary efficacy endpoints. The double-blind, randomized, placebo-controlled trial will test one active dose group with a treatment duration of 6 months. The dosing regimen has been designed to achieve consistent drug exposure in subjects regardless of their weight. Neuren Executive Chairman Richard Treagus commented: “We are pleased to have held a very constructive meeting with the FDA Division of Neurology Products. It has provided necessary confirmation on the key issues relating to our proposed Phase 3 trial in Rett syndrome. We are now able to progress the final stages of development with full confidence.”


Boom! Great result that primary end points with clinical significance are the P3 agreed outcomes.