I guess the difference now is that, post-FDA approval, Jon has identified “commercial partnerships ex-North America for Rett syndrome” as an expected catalyst in 2023.
With respect to NNZ-2591, I think that if the Board is confident, any potential sale of the whole company should not be considered until after read out of the four Phase 2 trials.
Note that all four trials will not be reporting results this year.
Guidance has been that only Phelan McDermid is likely to have results reported this year. Pitt Hopkins and Angelman may well see trial completion this year, but results are not expected before next year. Given that Prader-Willi hasn’t yet commenced, I don’t expect results before H2 2024.
The more I consider Neuren's choice of pipeline for NNZ-2591, the more I think that the four indications have been carefully chosen with a clever, multi-prong strategy in mind.
Obviously, choosing orphan diseases comes with numerous development and financial benefits. Also, by limiting the selection to neurodevelopmental disease, shorter trials can be used, unlike the long trials that come with neurodegenerative diseases.
But, as importantly, the chosen indications have the potential to provide an insight into what NNZ-2591 might be capable of well beyond these four specific indications – the potential to provide a broad proof of concept for NNZ-2591 in an affordable and timely way, if you like.
Firstly, results from these trials may hold relevance for any disease that comes under the umbrella of Autism Spectrum Disorder (all four diseases have overlap with autism) or Neurodevelopmental Disorder.
If impact on aspects of brain function, such as memory, learning, cognition, hypoactivity, hyperactivity and sociability is seen, this may have relevance to multiple other diseases and conditions of the brain.
Any proven impact on seizures (one NNZ-2591 preclinical study showed 60% reduction in seizures while another study saw elimination) is likely to have broader application.
And finally, any impact on metabolism (such as reduction in obesity and insulin levels seen in PWS pre-clinical) may also hold relevance for other therapeutic areas.
It's seeking out data that gives you more "bang for your buck”.
But first, Neuren’s four Phase 2 trials will need to provide convincing data. If they do, and taking into account what Jon Pilcher has said about this before, I believe that the company is likely, from that point, to be put up for sale.
The broad potential of NNZ-2591 would now have been demonstrated. The Board would have achieved its stated aim of maximising return for shareholders. NNZ-2591 would now advance to the next stage of its journey under the stewardship of a large pharma which has the resources to more fully exploit NNZ-2591’s potential and to protect and extend its valuable IP.
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