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Ann: PAA completes Phase 1 and files for Orphan Drug Designation, page-393

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  1. 2,397 Posts.
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    What excites me about the potential of running a phase 2/3 trial against frontotemporal dementia is that there is no treatment available, and the mechanism of action, if proven effective against ALS/MND, would raise expectations that it could very well also be effective against FTD.

    If this was the case, I would expect FDA accelerated approval would be a real chance .... a rare disease with NO treatment.

    From there, we already have GMP tablet manufacture, shelf life testing etc. etc. , monepantel is ready to go.

    The path to market could be relatively rapid on this indication.
    And once available, other indications can be pursued by care givers.

    Given this, BP may want to move quicker than expected on PharmAust.
    Has the FDA already been approached about potential against FTD as well as ALS ? A tantalising proposition.
    Last edited by Quiltman: 25/01/24
 
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