NEU 3.17% $20.86 neuren pharmaceuticals limited

How is everyone's confidence levels leading into the Phase 2...

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    How is everyone's confidence levels leading into the Phase 2 results for Fragile X?

    I think meeting the primary outcome measure (safety) is a given, and there are plenty of secondary outcome measures to assist Neuren in identifying and demonstrating clinical effectiveness.

    Everyone here would be fully aware that a successful mouse model does not guarantee success in human trials, however the statement below from Larry Glass at the 2013 Annual Shareholders’ Meeting does give me some extra confidence (on top of positive Rett Phase 2 results) leading into the Fragile X results:

    “The preclinical study in the Fragile X Syndrome model was intended to provide an opportunity to show an effect on the “too many” end of the spectrum. What we frankly did not anticipate was that the results of the preclinical FXS model would be so overwhelmingly positive and conclusive as to change the calculus of our strategy. At doses directly comparable to those planned for the first Rett trial, NNZ-2566 completely normalized behavioural and anatomic features of Fragile X Syndrome with statistical significance achieved in virtually every measure of effect. The results appear to be among the most profoundly positive results achieved with any molecule in a validated model”.

    http://www.asx.com.au/asxpdf/20130520/pdf/42fzq062vrf03b.pdf
 
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