Pipeline-in-a-drug, page-189

The result this morning was really pleasing for shareholders and for patients - great for them to have some hope on the horizon!!

What really strikes me is the correlation we continue to see between mouse models and patient studies. Talk of "Pipelines in a drug" is brilliant and accurate in my view but what is equally brilliant is that there is a VERY STRONG argument to be made moving forward, that positive results in mouse models will very likely result in similar results in humans.

I appreicate there are always exceptions but this is not a replication of the mouse model efficacy once, its 3 times now and this touches on my question yesterday about PWS being the paternal "version" of Angelmans - it would very likely be 4 in December/January, if the trial was not on hold.

I think this point needs to be rammed home - the outstanding results we are seeing in mice models is being replicated in humans, irrespective of the underlying genetic conditions. There is enough evidence now to credibly make this argument moving forward, which bodes really well for the undisclosed conditions currently being explored. We can say to BP with confidence, that we think this will work in conditions XS, YS and ZS (amongst others), based on excellent mouse model results, as was the case with PMS, PHS and AMS, which then enables proper modelling and value attribution.

I am sure that most will appreciate this but I still think it is really important to highlight and I hope that it is something Jon and his team highlight this morning.