Monepantel MPL is a breakthrough drug for the treatment of ALS. Pharm Aust just delivered a master class in delivering complex biotech information and clinical results to the market.
A trading halt followed by release of top line results after market close with an investor webinar immediately following for Q and A and detailed explanation.
To summarize the drug dosage for cohort 2 had a ALFRS-S score of -0.6 showing the rate of decline in symptoms slowed in ALS patients and their survival trajectory just expanded up to 56.5 months.
Remembering the nearest drug competitor Rylevrio extended life expectancy by 8-9 months.
The drug was well tolerated with minimal side- effects and no significant adverse events related to the drug.
MPL successfully crosses the blood brain barrier. Implications for MS, Alzheimer’s, Parkinson’s are promising as @Quiltman pointed out.
No significant difference in lung capacity, cognitive scores and quality of life scores between pre-clinical and after 15 months on the drug.
The stronger dosing is more effective and will be carried through to the phase 2/3 trial. Remember they anticipate a price tag of around $20- $30 million to run this trial. Michael Thurn states there are multiple non dilutive funding options that they are currently exploring. @Rick clinical trials will be run in Australia. This drug brings some much needed hope back to the ALS community.
I was going to attach the latest investor presentation but the webpage keeps crashing as there is too much demand for the site.
Needless to say I will be continuing to build my position in PAA and I am very excited to see where MPL can lead. The company is aiming for accelerated FDA approval and this drug is possibly going to be rolled out and treating ALS patients by mid 2025 with full approval targeted for 2026.
Well done investors who found this company early on Strawman a very exciting day ahead.