NNZ-2591 development & commercialisation

There'sbeen a fair bit of discussion on how NNZ-2591 could be best developed andcommercialised across the hundreds of indications it could potentially beeffective for. I posted on this earlier in the year here, but with the recentsuccess I think it is timely to revisit.

https://hotcopper.com.au/threads/moonsoon-briefing-thurs-20-april.7327805/page-66?post_id=67397828

I do not believe NNZ-2591 would need to be out-licensed to a bunch ofdifferent pharma then a whole pile of clinical trials conducted for eachindication. This can, and I believe will, be achieved by use of a basket trial which could be conducted by a single company. Briefly, a basket trial would group patients together based on their common underlying brain pathophysiology and molecular features (ie in our case mutations that cause abnormal dendrites and synapsesand IGF-1 deficiency) rather than diagnosis of a particular syndrome. So, in a way, the 4 indications we are doing now can be thought of as 4 sub-groups of a mini basket trial.

This blog is a nice primer on the use of basket trials in neurodegenerative disease rather than neurodevelopmental disorders, but the principle is the same.

https://parkinsonsblog.stanford.edu/2019/12/research-summary-basket-trials-and-neurodegenerative-disease/

This is how BP will be thinking about it and if this approach is taken, and I believe it eventually will be, that makes NNZ-2591 a drug with bona fide blockbuster potential.