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Pharmaust - Media & News, page-59

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    Rat's Rant email today:

    What's Doing

    PharmAust Limited - PAA.asx

    I recently caught up withDr. Michael Thurn,Managing Director and CEO ofPharmAust Limited(PAA.asx), to discuss thecompany's recent milestone achievements.

    PharmAustis currentlytrading at 21.5c giving them anEV of approximately $97m, as of their last quarterly report, theyheld over $10.5m in the bank.

    This doesn't include arecentinstitutional placement to raise $10m @ 19calong with a recently completedshare purchase plan (SPP) which raised $7.8m @ 19c.

    This SPP was significantly in excess of the initial target of $2m,you wouldn't see many take ups like that in this market !!!

    The strong support by existing shareholdersis a vote of confidence for the exciting opportunity ahead for monepantel, and the recent refresh to the Board of Directors and management team which I won't go into,you can DYOR.

    PharmAust Limitedis a mid-stage biotechnology company focused on repurposing monepantel(MPL)for the treatment of amyotrophic lateral sclerosis(ALS), a form of motor neurone disease(MND).

    ALSis a devastating disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and eventually paralysis.

    According to Allied Market Research, theglobal ALS market size was valued at US$662.3m in 2022 and is projected to reach US$1,038.94m by 2032, growing at a compounded average growth rate (CAGR) of 4.6%.

    PharmAustaims to leverage MPL's potent inhibition of the mTOR pathway, which plays a central role in the growth and proliferation of degenerating neurons.

    Recently,PharmAust announced thatMPL has been selectedfor inclusion in the prestigious HEALEY ALS Platform Trial under a Clinical Research Support Agreement with Massachusetts General Hospital.

    This innovative Phase 2/3 trialallows multiple investigational treatments to be tested simultaneously, aiming to expedite development timelines, reduce study costs, and increase patient access.

    The inclusion in this platform trial is a significant validation of MPL's potential as an ALS treatment and offers PharmAust substantial exposure within the global ALS research community.

    The trial aims to commence in the second half of 2024 and couldpotentially lead to accelerated approval with the US FDA for MPL in 2026.
    Those poor West Coast Eagles will need more than MND beanies, Harley Davidson & a new coach to turn things around.

    How nice it must be to be a Freo supporter in Perth right now.....
    Back in February, PAA announced itsPhase 1 trial had metits primary safety and tolerability endpoints with monepantel (MPL) and also demonstrated a preliminary signal of potential efficacy.

    The safety and tolerability profile of MPL includedno treatment‑related deaths,with all 12 patientssuccessfully completing the study, and no dose limiting toxicities experienced.

    Treatment with monepantel for up to 12 months slowed the progression of MND/ALS by 39% in all 12 patients - 23% in Cohort 1 (Low Dose) and 58% in Cohort 2 (High Dose)- when compared to matched controls from the PRO-ACT historical database.

    There were also no significant changes between pre-dose and end of treatment in ALSFRS-R scores, ALS Quality of Life Questionnaire, Edinburgh Cognitive and Behavioural ALS Screen, and Slow Vital Capacity for all 12 patients (p=0.78), Cohort 1 (p=0.41), and Cohort 2 (p=0.88) further suggesting that treatment with monepantel over 8–12 months slowed the rate of disease progression.

    A 0.48 slope change as calculated for all 12 patients could provide an additional 13.5-56.5 months in median survival.

    Currentlyapproved treatments for ALS/MND provideapproximately 2-6 monthsin additional life expectancy, along with other unwanted side effects.

    Back in January, the boys and girls at Spark Plus wrote a long and detailed note on PAA (with a 97c price target),which you can find on the link below.

    https://bit.ly/3OEIFfh

    Keep an eye on this one as they progress towards offering a much-needed therapy for patients with ALS.

    I am not sure if will be able to make out the below but will leave it in there....

    Good luck to Michael & his team - interesting few months ahead, I wish them all the best & we will be watching my friend (popcorn in hand).
 
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