QURE uniQure NV

H.C. Wainwright reitd $QURE Buy-$80/said—AMT-130's four-year cUHDRS result complicates the durability case, although the sustained TFC benefit and the three-year data underlying the submitted BLA remain encouraging. $TEVA $NBIX PTCT RHHBY WVE ANNX ALNY - REGN H.C. Wainwright added—In 12 high-dose patients, the prespecified four-year CUHDRS analysis showed 44% slowing versus an updated external control but missed nominal statistical significance (p = 0.144). However, Total Functional Capacity (TFC) showed 61% slowing (nominal p = 0.008 ) which management and a treating clinician emphasized reflects patients' ability to work, manage finances, and live independently, making its sustained four-year benefit an important part of their interpretation of the data alongside the cUHDRS result. The four-year results were not part of the submitted BLA. We reiterate our Buy rating, with the BLA already submitted on the earlier three-year data and FDA acceptance for review the next regulatory milestone.

$QURE just want to thank Sara Tabrizi for her rock bottom medical opinion that allowed me to buy a couple thousand more shares 🙏

$QURE Barclays halved its uniQure target this morning, cutting it to 28 from 60 and downgrading to equal weight from overweight. The stock gapped down, opening at 22.28 after closing at 23.46 on Friday. This is the durability debate from last week's four-year Huntington's data, now in sell-side form. Barclays is pricing the fade in the 48-month effect; the cut follows Goldman trimming to 31 and RBC to 48 last week. The Street is now genuinely split on the same data cut. Wells Fargo restated overweight with a 75 target last Tuesday, while Barclays sits at 28. That is nearly a 3x spread between two large banks. The stock still carries a moderate-buy consensus with a 53.73 average target (MarketBeat). The approval case, as before, is the 36-month data anchoring the BLA; the debate is whether the four-year fade matters.

$QURE uniQure's AMT-130, its one-shot gene therapy for Huntington's disease, fell more than 39% to $23.71 (IBD) this morning after the 4-year update made the effect look like it is fading. The headline: progression slowed 44% on the cUHDRS scale at 48 months (12 patients), vs 80% at the 36-month mark (15 high-dose patients). The 4-year cut was not significant. The nuance: the BLA rests on the 36-month data (80% slowing, statistically significant), and the FDA agreed that cut could anchor the approval filing. The 48-month readout used a new external control set (5,000 more patients, added Sept 2025); Leerink's Joseph Schwartz notes faster-progressing patients dropped out of the dataset, which flatters the control. Against the original control set, the 4-year effect reads 53.5% slower, not 44%. Safety: 5 patients had serious treatment-related CNS inflammation; all recovered fully. The approval case was always the 3-year data. Today is a durability debate, not a rejection.

$QURE my ass thesis . By the time uniQure submitted the BLA, management did not necessarily know the final four year efficacy result. But remember, because the study is open label and the four year clinical observations were already being collected in the EDC, some folks inside the company could plausibly have had considerable visibility into the underlying patient trajectories. Bare minimum the clinical trial physician. Also, I assume they probably had monthly meetings, maybe even more, going over each patient as it’s such a small n. Probably know them each one by name. Therefore, filing the three year BLA shortly before releasing the four year analysis is modest evidence against a year four efficacy collapse. Now, for the run up, we need to see more or around 70% cUHDRS slowing. Fingers crossed and much love to all my longs

$QURE this is a massive overreaction on qure today and it will rebound. i opened up a large position earlier today.

$QURE If you are scared about small intraday movements, you should get out of small-cap biotechnology investing forever. No news, no new data, just paper hands longs that have no understanding of the situation at hand. Frankly I should buy more but I have enough "Skin in the Game." BUCKLE UP!

$QURE It will be approved eventually. I believe this will be like GRAL story eventually. Serious unmet need and theres clear evidence of disease modifying effect. Zoom out.

Stifel on $QURE updated data said -- We're reiterating our Buy rating on QURE on the back of updated ph1/2 data for AMT-130 in Huntington's Disease. Most importantly, the 3-year data, after including 3 additional patients where results have matured, continues to look robust, now showing 80% diseases slowing on cUHDRS and 67% slowing on TFC as compared to natural history. Both of these are slight improvements vs. the prior data cut, and each are statistically significant. Meanwhile, QURE also disclosed 4-year data for 12 patients--this continues to clearly support a drug effect, though there is regression in efficacy on cUHDRS (44% slowing) whereas TFC data (61% slowing) hold up. We caught up with QURE who argues that as they follow patients for longer, using the natural history control can underestimate benefit due to survivorship bias (discussed below). The bottom line though is we view the big picture as unchanged ahead of an AMT-130 regulatory review.

$QURE wowee this is the drop that was expecting on $KOD , where the tard that said thats why you dont hold short into data

$QURE UNABLE 2 SELL 200 AT 38.60 ON 9/25. SOLD NOW 9/28 38.995 WOW ! PAID JUST Type Margin Shares +200.000 Price $38.44 Amount -$7,688.00 Settlement Date Sep-25-2026 BOT ON 9/24

$QURE If I am running a business, and I have 37 open job listings for this business, one would conclude that I have a product ready to sell, right? Now replace "Business" with "UniQure" and replace $40 a share with $140 a share. Simple stuff here folks!

$QURE $CGON $AVBP might be worth doing cheap lottos on these in case they drop data before 9/18 opex. Not sure if Uniqure will move much though.

$QURE 50-72 is what I told you. Data drop before end of September $CLPT lagging wayyyy behind but what do I know

$QURE does this stupid ass ticker show up on everyone else's CNBC satellite radio display EVERYDAY for no fn reason?

$QURE stock is a bargain. the fda will approve and durability doesnt seem to be an issue. results were heavily impacted by dropouts.

$QURE market being like “oh right it’s the only therapy with disease modifying effect for Huntington that has a very high chance of being approved next year, first to market”

$QURE (NP) TFC (primary for confirmatory) is unequivocal... the miss was on cUHDRS (control arm issues) Nfl 4% above baseline, 8% below for low dose (vs 10%- 15% annual increase for early HD patients) Context is important as well- Huntington's comes w/ 12x higher suicide risk

$QURE not sure how a drug works so well in year 3 and degrade in year 4. Still the best hope for huntington. Should be approved and there will be demand as no other option exists.

$QURE Down -38% but up more than 210% from premarket bottom if you were lucky to catch that. $LABU $LABD $BBH $IBB