AGM, page-6

Certainly don’t want to cut the grass of regular posters, but here are a few of my thoughts after attending the AGM. This is from memory and a few notes I took, so I’d certainly welcome refinements from other attendees.

1. I was impressed by recent progress in paediatric Rett trial. The increase in hospitals, enrolled patients and patients with first appointments scheduled was very good news and imo reflected NEU’s effort and the encouragement of US based Rett support groups.
2. Not sure how to rationalise the impact of dealing with two different FDA divisions in trials of the same drug candidate. Given that the FDA Division of Psychiatric Products has requested additional tolerability and toxicity data before approval of the next FX trial, to me that all suggests that FX may fall more than 12 months behind Rett in terms of trial progress.
3. From previous NEU releases I’d concluded that TBI/Concussion testing might go onto the back burner, but that appears to now not necessarily be the case. The use of higher doses, possibly up to three times what was used in the last trial, and stringent screening of candidates to recruit a more homogeneous sample, will almost certainly be part of any future TBI trial design. Nonetheless, the future of these programs clearly depends on the provision of US Army funding. Discussions with the US Army expected to be ongoing this year.
4. Future funding for me was the key takeaway from the meeting. The board are ‘very much mindful of cash burn’ and are considering whether to raise funds through a simple capital raising from shareholders, from third party strategic investors or a partnering agreement. And, if it’s the latter, whether to sign up pre or post Rett paediatric trial results. I personally got no hint of which way or when the Board might go with this.
5. It sounded to me that the main benefit of the alliance with Leerink up till now had been to introduce NEU to potential capital partners and investors.
6. NEU has not applied for Breakthrough Designation in FX. The first direct FX related discussions with the FDA apparently only took place last week.
7. Any reapplication for BTD for Retts will depend on a review of the topline Rett results due Q1, 17.
8. The AGM was told that obvious effects of the existing ODD/Fast Track designations for Trofinetide included smooth channels of communication and very full turnouts from the FDA to meetings with NEU.
9. Re 2591- apparently the main focus has been to prosecute patents and build up and protect the IP portfolio in the U.S. and Europe. I agree with Ajostu’s assessment; for now the Board are clearly ‘focusing on trofinetide’.

All the best. Herro