Matan flank

[Matan flank @matan_flank]

At 3y cUHDRS was -0.38 vs -1.52 NH (75% slowing). If NH is ~-2.0 at 4y, 40–50% slowing implies ~-1.0 to -1.2: a year-4 decline of ~0.6–0.8 vs ~0.5 NH. If that is the case the drug just stopped working and made patients worse at 4 years. Impossible that she meant that. $QURE

[Matan flank @matan_flank]

@mike98572986 There is no way it goes down to 40/50 now. That would effectively mean that the treated population progressed faster than NH from 3 to 4 years. She absolutely meant long term. $QURE

[Matan flank @matan_flank]

$QURE isn’t $REPL under CDER? Why is this relevant to $QURE?

[Matan flank @matan_flank]

$QURE wouldn’t it be great if Nicole was VP replacement!

[Matthew Herper @matthewherper]

An eye-catching -- and curious -- detail from @LizzyLaw_'s FDA story:

Nicole Verdun, the official who was marched out of the FDA earlier this year, "may be returning from administrative leave after [an] investigation apparently found that her behavior did not merit permanent removal."

I do wonder if this is a signal that not being careful about legal matters -- something that has led previous FDA officials to walk on eggshells -- could lead to a lot of FDA decisions later being walked back. It's only one data point, of course.

https://t.co/N9rKRiIQDm

[Matan flank @matan_flank]

Understand the point about randomization, but it how would have to be the case that there was slow progressing bias for high dose and fast progressing bias for low dose to explain the difference in slowing between the doses. Seems highly improbable. Do we know if there were different recruitment criteria between doses?

[Matan flank @matan_flank]

@HarrieScarlet @mike98572986 I assume there would need to be some meeting before submission, but if they say file tomorrow, i assume $QURE is ready

[Matan flank @matan_flank]

Please explain dose dependency. Look at difference between high and low dose at 36 months $QURE -0.38 hi vs -1.65 low. For reference the NH match control for hi dose was -1.52 and -1.72 for low dose. So low dose barely showed any slowing. I don’t know enough about this, but couldn’t they even use the low dose as the placebo?

[Matan flank @matan_flank]

@anish_koka @johnkmason How would you explain the difference between the high and low dose then? Low dose showed significantly less disease slowing compared to high dose…

[Matan flank @matan_flank]

@Lucy3370 I say he doesn’t last the weekend! $QURE

[Matan flank @matan_flank]

You know the “senior FDA official” is none other than @VPrasadMDMPH. What an embarrassment to this agency. Tell me what the point of double blind sham surgery where the sham is 30 min with “tiny nicks” made in the scalp vs a 12 hour actual procedure where several burr holes and catheter are inserted into the deep regions of the brain, cmon please admit that this is insanity. And to suggest that $QURE and the FDA were never aligned in an accelerated approval pathway!!!?

[Matan flank @matan_flank]

@BiotechCH FDA has not rejected $QURE they didnt even review!

[Matan flank @matan_flank]

@adamfeuerstein So the head of CBER is spewing blatant lies about a company to the media. Forget $QURE for a minute, this is actually scary that this man is in this position!!

[Matan flank @matan_flank]

Someone needs to call out the FDA on these straight out lies. How is it that @uniQure_NV has been talking about their accelerated approval alignment with the FDA since December 2024, including having 3 additional meetings with the FDA discussing details of the submission package. Why would these meeting take place if there was no official accelerated approval alignment. I won’t get into all the other lies the FDA is spewing

[Matan flank @matan_flank]

@LongVollllll Where is this from?

[Matan flank @matan_flank]

Adam, have you ever seen the FDA behave like this? Do you think they are trying to cover something up here? They have made unpopular decisions before, but never do I recall something like this. It’s highly unprofessional and discrediting…can $QURE take legal action against these remarks if they are outright lies?

[Matan flank @matan_flank]

Why the sudden focus on the statistically insignificant results of the 1 year RCT. This was over two years ago and it was never mentioned until now. Notably, the 1 year data vs external control, while slightly better than vs placebo was also statistically insignificant and very little improvement vs comparator.

Also what about the dose dependency, the low dose showing way less effect and not stat sig. how can that be explained away?

[Matan flank @matan_flank]

Some of the accusations being made are frankly hard to reconcile. For example, questioning $QURE previous AA alignment with the FDA. @uniQure_NV has been talking about this alignment publicly for well over a year. Why on earth would they lie about this and how would it make past all the legal and financial due diligence since? Do you think it’s at all possible that the FDA has seriously messed up here?

[Matan flank @matan_flank]

@RxRegA @mike98572986 But surely within the dataset of 30 thousand HD patients we can find an appropriate comparator?! Why require a whole new study? $QURE