PAA 8.89% 24.5¢ pharmaust limited

I believe some focus on the content of this presentation is...

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    I believe some focus on the content of this presentation is required:

    MT has a page dedicated to explaining the ALSFRS-R .
    I'm assuming this will be the focus of the presented topline results and most likely what the FDA requires to make an ODD decision.
    We are shown that slowing the decline by 16.5% increases median survival by 4-5 months.
    Assuming MPL results for the 12 patients will be shown against standard progression from first reading/dose to the final reading.
    AMX0035 with SOC achieved 25% slower decline ... so I'm guessing >25% slower decline moves us to the gold medal position, and then the Healey Trial with placebo to prove the results against a numerically significant cohort..

    While there are several candidate biomarkers, ALSFRS-R seems to remain the gold standard favoured by the FDA


    https://hotcopper.com.au/data/attachments/5937/5937160-c59a3e08ef3fe12ed57206f8e8f98031.jpg


    On the next chart, MT tries to give attendees/investors a feel for the chance of success/positive results from the MEND study.
    All timelines are from diagnosis of patient.

    - 1 year mortality rates from diagnosis 22% to 34%

    this is from the 22/8/23 ASX release :

    https://hotcopper.com.au/data/attachments/5937/5937195-e4f7acf27d7dae921a5336684daa1b40.jpg

    For these 5 patients ( out of the 12 in the trial ), time since diagnosis from 589 days to 934 days.
    5th Feb now, at least another 150 days survival, 739 days to 1084 days.
    Assuming the other 7 are at least > 16 months ( from chart beloe : Shortest time since diagnosis ~16 months compared to the longest ~49 months for the completed Phase 1 MEND Study ).

    So, of the 12 , 22% to 34% would have been expected to die. We should have , statistically, only 9 patients left.

    On top of that, all 12 have not progressed to the "difficulty swallowing" point on the progression timeline, as all 12 have enrolled in the open label extension study - no wonder they have opted for remaining in the study.

    https://hotcopper.com.au/data/attachments/5937/5937187-c4ec5fcc3a1972525837183cd29a371d.jpg

    The numbers are compelling.

    And I suspect strong ALSFRS-R scores was the reason for Prof Susan Mathers confidence early in the trial - the numbers were staying high and not declining at the expected rate.

    Guess we will know soon enough, but this looks very promising IMHO.
 
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