@mikebrisy @Nnyck777 FWIW this out from JP Morgan this morning - it basically highlights things you've already surfaced in your recent posts

Angelman results establish NNZ-2591 platform potential

Neuren has now delivered its third set of promising efficacy data for NNZ-2591, extending the range of rare genetic neurodevelopmental diseases potentially treatable with its key pipeline drug. The data presented for Angelman syndrome (AS) suggests it has potential to progress to the next clinical trial stage. However, current competitive dynamics in AS means it will be prioritised behind other indications. Of immediate focus will be NNZ-2591’s next steps in Phelan-McDermid, where Neuren will be engaging with the FDA next month in a post- phase 2 meeting, which we are hopeful will yield the progression of NNZ-2591 to a phase 3 trial. We continue to see upside in the NNZ-2591 platform and have lifted our DCF-based price target to $23, Overweight retained

NNZ-2591 “multi-indication platform” viability strengthened with promising Angelman results. NNZ-2591 delivered CIC and CGI-I mean scores which were clinically meaningful in its phase 2 trial of AS. This is the third neurodevelopmental disease to have shown early efficacy signs with NNZ-2591, following Phelan-McDermid in Dec-2023 and Pitt Hopkins syndrome in May-2024. The promising data set suggests the potential for other genetic neurodevelopmental diseases could be treated with this platform treatment

Two other competitors ahead in Angelman. As previously covered NNZ-2591 faces competition in AS with the potential of two competitors entering phase 3 clinical trials within the next 12 months (Ultragenyx & Ionis Therapeutics). It is too early to assess which treatment is leading and we caution comparing results across earlier trials. However, should NNZ-2591 progress, it has the administrative advantage of being an oral liquid dose vs the competitors intrathecal (in the spine) dosage

Competitive market dynamics mean Phelan-McDermid and Pitt Hopkins will be prioritised. Neuren have indicated it plans to focus on Phelan-McDermid and Pitt Hopkins after the good phase 2 trial results recognising the lack of competing therapies currently in trials. Neuren’s existing cash reserves will allow it to support two phase 3 trials itself (each to cost US$50-100m)

What’s ahead? While the newsflow has slowed with no further phase 2 trial read-outs for NNZ-2591, next month’s post-phase 2 meeting with the FDA for NNZ-2591 in Phelan-McDermid will be important. An ideal outcome would be the announcement of a phase 3 clinical trial noting Neuren will only comment once the meeting minutes are released. On Daybue, although the weaker guidance was a disappointment, we look to market expansion as sources of upside in the year ahead starting with the Canadian market later this year and Europe in early CY25

Retain Overweight. We lift our probability weighted valuation for Angelman syndrome which brings our Jun-25 price target to $23 

DISC: Held in RL & SM