@pawcio2009 +ve This management team have a history of selling / getting acquired. -ve I am not sure that their drug is especially potent and think $NGNE might win in Rett. Like the CEO but CMO doesn't know hoe to answer a question straight.
@LogicalValueMx@LuckyPenguin10 The problem is @johnarnold seems to also have influence over UK, and lack of sites in the UK/Europe to administer the therapy. But I agree it will be approved there, but much better outcome if US is first.
The significance of this can't be overstated re: $QURE AMT-130.
Punchline β AMT-130 will get approved overseas and the U.S. will continue to fall behind in the biotech race globally.
First, some relevant dates:
- This presentation: Last week, Mar 30, 2026
- 4y cohort cutoff: Jun 2026
- 4y data readout: ~Sep 2026
Why this presentation is so important:
> Dr. Tabrizi obviously, and of course, hasn't seen the 4y dataset yet...
> ...BUT, she's a clinician β she sees these patients on an *ongoing* basis.
> Dr. Tabrizi would not be speaking w/ this level of conviction if she were seeing deterioration leading into the 4y assessment.
> Remember β she's been involved in many HD trials, and she doesn't sugarcoat results. For example, in Mar 2021, she publicly stated that Rocheβs P3 tominersen (Gen HD1) did not work. [1]
Dr. Tabrizi β one of the world's leading HD researchers β continues to pound the table:
"... and two people [from the high-dose AMT-130 cohort] β and this is unheard of β went back to work. Having medically retired, and then gone back to work." [2]
This is how the U.S. falls behind.
Cc @fjeg1987@JTLonsdale
β
@SenGillibrand | @lukaske@SenRickScott | @ClareLattanze@SenRonJohnson | @GraceCarnathan@RepAuchincloss | @GeorginaBurros
β
#FDA $XBI