Apologies unable to open a new thread for this not sure why....

Apologies unable to open a new thread for this not sure why. Managed to catch the webinar. Summary as follows:

PRESENTATION:
Preamble/description of company/Daybue sales in US and ROW update - essentially nothing new if you are up to date with recent presentations.

NNZ-2591:
Jon discussed 3 indications only -

PMS - nothing new. Emphasised confidence in a positive result and that this would be a massive outcome for Neuren.

PTHS - FDA meeting this month. Hoping to get OK to proceed with a phase 2/3 approach with some flexibility around endpoints and control group.

HIE - pre IND meeting this month. Hoping to get OK to do a small PK study in 0-3yo age group then proceed into a single registrational trial.

QUESTIONS:
There were apparently a lot of questions submitted but only two were asked as many were similar and due to time constraints.

1. More info about PMS phase 3 trial/ first dosing/ delay?

Jon answered they only have one site open. A second site is very close to opening. There is a massive amount of training that has been occurring to get the investigators ready for the trial plus the month of observation pre dosing. So no patient has actually been dosed yet. It's a phase 3 trial with a huge amount riding on it so they are taking time to get everything executed perfectly. There won't be a patient by patient commentary but big milestones will be communicated.

2. Describe competitors to the 2591 indications.

PMS - 2 gene therapy companies (Jaguar and ??) one in preclinical one just commencing trials so a long way behind NEU.

PTHS - no competition

HIE - one company (Realta?) but not offering anything like the treatment paradigm NEU is undertaking.


Obviously these are just my recollections and summary so rewatch or read the transcript if you want 100% accuracy.