Morning,As far as I know, those recruitment numbers and details...

Morning,

As far as I know, those recruitment numbers and details aren’t finalised yet - looking forward to them as well - but maybe I can shed some light on that second half with a very crude unscientific explanation.

Statistics and probabilities don’t translate at all sometimes between different situations. And for two reasons.

One is - 3.4 in 10,000 - is the chance of the results ‘failing’- by not being statistically significant.

Lotteries, Powerball etc. are Independent events - where the chance of winning is about the same every time you play. 1: blahMillion.

Independent meaning there’s no causal relationship or effect between one game and the next. Nothing changes, there are no connecting points. One game doesn’t depend on the previous games as to what’s going to happen next.

The OLE results are a Dependent event - and where the chance of ‘winning’, is the inverse of the trial results ‘failing’. And we have observed highly ‘winning’ statistically significant results.

Dependent meaning there are obvious and known linkages, connections, planned and adaptive changes etc to previous results. One result will depend on the changes made as to what’s going to happen next.

And that’s where it gets exciting because we know a lot of the links and changes that have happened already or will change, for example:

the patient population numbers and treatments,

the dosage upgrade to full-time blood saturation rate of 10mg/kg,

all patients on the prime dosage rate from day 1 onwards,

different times from diagnosis, ages,

genomes - the full set of demographics -

and the phase 2/3 is an adaptive trial where things may change according to developments,

including any possible insightful results of pre-clinicals etc.,

and we know the reliability and confidence levels, and the statistical significance levels.

So it’s a different situation,

and the probabilities of independent & dependent events;

have different mathematical equations,

so they can’t be correlated or evaluated against each other in any way that makes sense.

Maybe an AI could do it. Or a quant.

Putting it another way:

Powerball chance of losing: 1.339999M : 1.34M

PAA MPL chance of losing: 340 : 1M

I like our chances.

cheers,

Ice