Ann: Investor Presentation, 14 March 2023, page-39

On the offensive thread which was created yesterday to which many were “sucked in", a poster highlighted the fact that Neuren has a patent for autism in Israel. He said that this and the fact that all four indications of NNZ-2591 are “in the autism spectrum” seems to have escaped everyone’s intention. As I don’t wish to contribute to the other thread, I’ve chosen to address this on a different thread.

TB, as per your 2021 post, it was trofinetide, not NNZ-2591, that was granted a patent in Israel for autism, but that was in 2020, not 2018. The Israeli patent application, by the way, was filed in 2013.

That Neuren has a patent in Israel for autism isn’t a secret, because Neuren announced in April 2020 that the first patent for tofinetide in Israel had been granted, covering trofinetide to treat Rett syndrome, Fragile X syndrome and autism. In the same announcement it was also stated that patents derived from the same international patent application had been previously granted in the US, Europe, Japan and Australia and were still under examination in Canada and Brazil (these jurisdictions have since been granted).

But, yes, NNZ-2591 has also had patent applications filed for treating autism spectrum disorders and neurodevelopmental disorders.  To date, patents have been granted in the United States, Japan and Denmark, but not Israel, and are pending in multiple other countries.

That all four NNZ-2591 indications have overlap with autism won’t be news to longer-term holders on this forum - this has been previously noted and discussed numerous times.

The “elephant in the room” for me is Acadia’s current Phase 2/3 study of its only marketed drug to date, Nuplazid, in the treatment of irritability associated with Autism Spectrum Disorder. This is a 12 month, open-label study in 228 patients aged 5-18 years and it uses the ABC-1 and CGI-I (both endpoints used previously in trofinetide trials) as secondary efficacy endpoints. The study commenced last November and is estimated to complete in mid-2025.

I have still not seen seen mention of this study on Acadia’s website or in its SEC filings, presentations or Earnings Calls, which is highly unusual given that it’s a Phase 2/3 study. It’s fairly clear that Acadia doesn’t want attention drawn to this study.

While it makes sense that Acadia would want to broaden Nuplazid indications and leverage its trofinetide clinical development experience and sales force, there has to be more to this, I believe. My thinking is that Acadia’s ambition is to build an “autism franchise”, similar to what it calls its “Rett syndrome franchise”, which consists of the now-approved DAYBUE as well as its pre-clinical antisense asset licensed from Stoke Therapeutics”.

The question to me is whether Acadia wishes to do that with trofinetide or with 2591.

https://hotcopper.com.au/threads/share-price.6359591/page-3328?post_id=65792854

https://clinicaltrials.gov/ct2/show/NCT05523895?term=acadia pharmaceuticals&draw=2&rank=14