Neuren Pharmaceuticals Limited

ASX:NEU

Neuren Pharmaceuticals Limited

ASX:NEU
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$19.75 (+3.5%)
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#19
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35
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Forum Topics NEU NEU #News

Pinned straw:
Nnyck777
Added 8 months ago

https://endpts.com/small-biotech-oak-hill-will-bring-roches-angelman-drug-back-to-life/?utm_medium=email&utm_campaign=891+-+Roches+Angelman+drug+is+revived+JJ+holds+firm+on+guidance+Basic&utm_content=891+-+Roches+Angelman+drug+is+revived+JJ+holds+firm+on+guidance+Basic+CID_8f18afd3731e9e3ed3610bc1d942e8bb&utm_source=ENDPOINTS+emails&utm_term=Exclusive+A+small+biotech+will+bring+Roches+Angelman+drug+back+to+life


Another Angelman competitor. Starting phase 3 1st qrt 2026. So that is Ultagenix and the new Roche drug. Very understandable why Phelan McDermid went first. Might be tough to recruit for Angelman unless Neuren steps on the gas. I would suspect they might tackle HEI or Pitt Hopkins next to mitigate first mover advantage. I also suspect that Neuren’s FDA primary endpoints agreement has just made other companies end point pathways a lot easier.
mikebrisy
Added 8 months ago

@Nnyck777 my reading of how Jon has always spoken about Angelman is that it is third of the current three in their batting order? The only value I’ve given it, is the extra data it gives on safety and side effects derisking PMS and PH (to a lesser extent).

You make a very good point, however, that FDA decisions on end points in new therapy areas, reduces uncertainty for competing candidates.

We lost 4-6 months on PMS, so hopefully it progresses smoothly from here.

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Nnyck777
Added 8 months ago

Hi @mikebrisy,

Agreed likely 4th in order now. I hope Roche and Ultragenyx send Neuren a fruit basket for their ground breaking FDA meetings. Communication scales will likely be a a primary end point for all these neurodevelopmental disorders.

Jon and team are very impressive. Maybe I am naive but I feel they are very trustworthy and I have felt very confident adding to my position at these lows.

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mikebrisy
Added 8 months ago

@Nnyck777 I take it from your comment that you think their win was getting the FDA to accept just the communication measure from the Caregiver score, rather than the broader measure? If that’s true, then I agree that is a big win, as the p-value for the broader measure at Phase 2 was not good at all.

I also am thinking about adding more. However, I’m expecting a poor winter quarter result from DAYBUE in early May when $ACAD announce, and the dumb money might give us a further pullback opportunity then. Still mulling this over,

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