NUZ -- New Investor thread, page-28

New investors — welcome to the extended NUZ family — warts and all — not exactly what I was hoping for when starting this thread — but realistically, the family does exactly what they want to do, and say what they want to say, and that’s how it is.

All of the above encapsulates the unfiltered broad history of NUZ & PAA with its diverse cohort of serious well-informed adults and the occasional petulant 12yo (in PAA terms) child, but that’s family, and I’m sure that new investors will be able to sort the different perspectives.

A note on timelines…

The FDA has yet to provide guidance on the updates for changes to ALL existing Master Protocols, from their draft December 2023 guidance, and their published March 2024 stakeholder feedback - covering scope, concerns about blinding, acceptable randomisation practices, control arms and informed consent — but no news on this yet.

https://www.raps.org/News-and-Articles/News-Articles/2024/3/Stakeholders-seek-to-reshape-FDA%E2%80%99s-guidance-for-ma

The original guidance for master protocols expired in March. More recent guidance covered cancer treatment, and then the relatively-recent master protocol guidance.

https://hallorancg.com/insights/2024/02/27/pulling-the-thread-on-fdas-master-protocol-draft-guidance

Bureaucracy wheels grind slowly.

So these timelines are not NUZ’s to determine - everyone involved is at the mercy of the FDA.

I suspect there may be significant, perhaps major, changes coming — all involved will have been working through all that — to the Healey Master Protocol as a determination of the updated FDA guidance, resulting in a built-in structural delay for the opening of both the NUZ and Healey IND’s, and for the NUZ regimen-specific design. Nonetheless, it has been stated that we are ‘closing in on that’ in a matter of weeks.

Minor elements like Relyvrio being taken out of the protocol, pale in comparison, to things like, appropriateness of the fixed-term 6 month duration; risk mitigation of data integrity deficiencies; construction and implementation of informed consent: documentation, training and implementation procedures across 73 trial sites.

@Densy

The TGA decision is another setback for LEQEMBI after the European EMA knocked back their approval on safety issues regarding serious adverse events. Nothing deters pharma. They are going back to EMA to pressure them to reverse the decision.

https://www.ema.europa.eu/en/medicines/human/EPAR/leqembi

Btw, back in the US, Clene are going in for a second bite of the cherry with the FDA re: CNM-Au8, after rejection of a potential submission for accelerated approval.

https://alsnewstoday.com/news/clene-granted-in-person-fda-meeting-cnm-au8-als-therapy/

Back to timelines…

In comparison, NUZ items in development or newly announced;

easily meeting the timelines / or ahead of timelines / or proactive on-the-front-foot items :

• Vital sweep of OLE Study patients are all alive today
• 2 year anniversary this month
• Liquid NUZ-001 formulation developed
• New Head of Marketing, IR & Corporate Affairs hired
• New web site implemented
• Product launch capability instituted
• NIH Expanded Access funding in progress
• US company Neurizon LLC. registered
• FDA Fast Track application planned
• TGA special access schemes being utilised
• TGA Provisional Approval being sought
• Automatic approval in other countries from unilateral agreement
• Tablet medication batches sorted.
• PTO MPL 6 Patent notification arrived
• Patent expiring in 2039
• Partnering meetings BIOJAPAN
• Partner Summit BOSTON
• Pre-clinicals >>> coming soon to a biotech near you …

cheers,

Ice