$qure $clpt
New video of Deborah Hall at Rush, the lady that had the 5 year patient. Not 100 percent sure if this was a newer interview or when technically it was given. She did say first patient was dosed 6 years ago and is speaking about the 3 year results. I have never seen this one so thought I would post it.
“You can imagine someone that gets the disease in their 30’s or 40’s they may actually have a normal life expectancy if they were able to receive a gene therapy like this.”
“If this does work for the majority of the patients the hope is that we can give it to earlier patients so that technically they never get the disease and wouldn’t that be the goal? That would be the closest to the cure.”
She has been advocating with Congress. Talks about the price.
https://t.co/roiy6VH3b9
This year, my friend won the HDSA Person of the Year Award. She's in a wheelchair. Today, I saw a post from her husband that she is now in the advanced stages and declining very rapidly. She's losing the battle to HD.
We are about the same age. Our children are close in age. And in the 13 years between the first photo and the last, she's losing her battle and I've started mine. That is the nature of this beast. I don't know why God gave me the opportunity to be able to communicate and to advocate on the level I do, but I realize how blessed I am and I will ALWAYS fight for my HD family, my fellow HD warriors who can't.
Never ever look at me and think we have time. We don't. This is proof.
Let's all do better and be better. #TimeMatters #ProgressionDoesntPause
#raredisease #DyingToLive
@FDA_KyleD@FDA@FDACBER@fdacder@WhiteHouse
@deepvalueco Nobody is sacrificing 10 million jobs to sell extra cheese. You’ve invented the most economically suicidal version of an embargo and assumed that’s the only way the U.S. can exercise leverage. And you’re right there is little point in discussing, let’s see what happens.
@deepvalueco If you genuinely believe Canada’s economic dependence on the U.S. gives us comparable leverage because their supply chains would also be disrupted, then there’s probably nothing left to debate. Let’s see how that thesis ages.
You keep arguing about whether the U.S. would tolerate the maximum possible self-inflicted damage. They don’t need to. They can apply 20% of the pressure and stop when Canada capitulates because our marginal pain is greater than theirs. That’s what leverage is.
The solution isn’t “be brave and call their bluff.” It’s spend the next decade building pipelines, ports, interprovincial trade and alternative markets so the bluff actually becomes callable.
This is bigger than Trump. U.S. trade policy has structurally shifted in a protectionist direction across both parties. There is no reason to assume the next agreement is the final agreement, or that the administration after Trump suddenly restores the old free-trade consensus. That’s exactly why Canada needs diversification, infrastructure and a domestic economy capable of giving us leverage. “Just stop conceding” isn’t a strategy when the other side can tolerate the breakdown far better than you can.
That analogy is backwards. If hurting us 10x costs them 1x, you’ve just described leverage. They also aren’t “shooting themselves in the head.” They can selectively squeeze sectors until our pain exceeds our willingness to resist. “They’d suffer too” is not a rebuttal to asymmetric leverage.
You’ve conceded my point. They have the leverage; your argument is that they won’t use it because it would hurt them too. Maybe they won’t. But “our largest trading partner could devastate our economy, so let’s hope their domestic politics restrain them” is precisely the strategic vulnerability I’m criticizing.
You’re confusing “this would hurt the U.S.” with “Canada therefore has leverage.” Those aren’t the same thing. We spent decades concentrating our economy around one vastly larger customer while failing to manage our resources, build export infrastructure or even eliminate our own interprovincial barriers. An embargo would be painful for the U.S. too but would be existentially worse for us. That asymmetry is the entire point.
A BLA for a potential first-ever disease-modifying therapy for Huntington’s disease (HD) is moving forward.
More than 200,000 Americans are at risk. HD represents an extraordinary unmet medical need.
A fatal disease. No approved treatment that slows progression. Families who cannot afford more delay.
@FDA, is the CNPV program still moving forward?
This is exactly the kind of urgency the program was created to address.
💙💜
#HuntingtonsDisease
If you’re wondering why $QURE is down after filing the BLA, here’s my opinion on it (worth exactly what you paid for it).
The last time QURE really moved on something the company itself did was the September data.
Since then the big moves have come from the FDA.
November rug pull.
March Type A.
Then the big reversal in June after the Type B.
To me June was the real BLA catalyst.
That was when FDA went from questioning whether the 3-year data could even support a filing to allowing QURE to move forward and getting aligned on the basic confirmatory trial: randomized AMT-130 vs SOC, no sham.
We also started hearing more from management about how they could deal with SOC patients dropping out once AMT-130 is commercially available, including potentially using Enroll-HD as a supplement if attrition becomes a problem. I don’t think we know exactly what FDA has agreed to there yet, but clearly they have discussed the issue.
That was a massive change from where we were a few months earlier. The stock rerated in June because the FDA changed the story.
So when QURE actually filed the BLA this week, what really changed? Not much. It was obviously an important milestone, but the big regulatory change had already happened.
That’s also why I’m not convinced good 4-year data moves the stock as much as people expect.
Bad 4-year data would obviously matter a lot. But if it shows what I think it will, continued efficacy and maybe even better separation with time, then it mostly confirms the story we already have.
I’m more interested in what FDA does next.
How fast do they accept the filing? If they do it well inside the 60-day window, I think that matters.
Does AMT-130 get a CNPV?
And there is one company catalyst that I think is getting overlooked: the peer-reviewed manuscript.
Sung seemed pretty excited about it in his recent presentation and hinted there is a lot more in the full dataset than what we have seen in the topline.
We already know imaging/sensitivity work should be in there. Now EHDN also has a presentation specifically on 3-year Q-Motor outcomes after AMT-130.
Maybe Q-Motor is in the manuscript, maybe it isn’t. But it makes me wonder what else is sitting in that dataset that QURE hasn’t really shown us yet.
That’s why I think the manuscript could actually be more interesting than the 4-year topline. Four-year data probably tells us whether the same story is still holding up. The manuscript may show us there was more to the story the whole time.
For the last year the market hasn’t really cared when QURE tells us FDA agrees with them.
It moves when FDA tells us FDA agrees with them.