PharmAust media thread, page-163

Morning @illdrink2that

thanks and I just am not sure where this ph 2 / 3 trial will go. I do expect clearly that it will be good news or else i
would not be here!

As I mentioned, we theoretically need all 210 participants to reach the 24 week mark to hit the interim collective data point but………… PharmAust would not logically wait until every single person has concluded that time to conduct testing.

Note, they will definitely batch the results as batching is far more valuable in terms of data consistency as well as commercially it just makes sense. Now…. Food for thought and why the trial is adaptive..

By way of example: What if the first 30, 60, 90 (for the 90 example 30 in AU, 30 in USA, 30 in EU) etc show that there is daylight between placebo and dosed for efficacy ?

What if the age demographic plays into the results and show something we have not seen in the initial trial?

What will happen with higher dosing from day 1 rather than what we did with the cohorts in ph1 where they ramped up to target dose.

What if early onset patients see better (or worse :-/)initial results? Note all the patients on ph1 were not so early onset/early after diagnosis.

what if the results in the EU (or AU or US) are better than other locations?

what if race plays into results?

what if we see fast recruitment at the connected sites to Healey (I am like you that I think we are an extremely good contender for becoming a trial arm of Healey given the stellar ph1 data!) so we see the first batch in the USA far faster than anywhere else?

An important extreme probability. We do need to expect deaths during the trial. This is a fatal and fast moving (for many) disease. Sadly, deaths are common during larger scale trials in MND/ALS and we will learn from these patients and their selflessness. Personally, I hope we get none but we have placebo and we have terminally ill participants.. We also don’t know everything about how MPL interacts in many scenarios.

Another is that we are extremely likely to see some more adverse effects. Some may be severe. This is one of the key data points because these patients will be closely monitored. The large volumes of patients will help us learn what we need to do to protect folks in the future. Having adverse effects is not specifically a critical issue. It is about understanding how to best manage. They can be but given the safety profile of MPL, we can be reasonably confident that we wouldn’t hit a show stopper.

We all have much to learn and this next trial is so critical. I am leaning towards better results than Ph1 because of some of the what ifs above could actually present easier wins (younger patients, earlier treatment, better up front dosing etc).

One more high probability ‘what if’ - a commercial deal is presented & accepted by shareholders and they come with deep pockets to expedite proceedings or they just take it over. Hmm….


Cashie is rarely wrong… so if you read his post once every 29 days, he is bound to be right from that day (at some point :-D.

adreamer