Release Today Neuren Valuation Estimate Raised, page-69

The P3 format agreed with the FDA is simple. As per the company announcement below endpoints and RSBQ have been agreed for one active dose group for a period of 6 months. To me we need to repeat what was already successful in P2 with a more accurate dosing regimen based on weight. IMO the success rates quoted in the above posts are very conservative.

"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. "