$QURE A remarkable valuation fact:
At QURE’s all-time high of $82.49 in June 2019, the company had only ~37.84m shares outstanding.
ATH market cap: ~$3.12bn.
What did investors own then?
AMT-130 had never been administered to an HD patient. There was no human efficacy data, no durability data, no clinical proof of disease modification and obviously no regulatory filing.
Fast forward to today.
QURE is worth roughly the same ~$3bn.
Except today:
• AMT-130 has shown 75% slowing on cUHDRS at 3Y, p=0.003
• TFC slowing of ~60%, p=0.033
• NfL below baseline
• FDA BLA for accelerated approval SUBMITTED
• UK MAA SUBMITTED
• Priority Review requested
• Breakthrough Therapy + RMAT + Fast Track
• 4Y efficacy/durability data due this month
The dilution explains why comparing $82 then with ~$46 today is misleading.
Compare the market caps.
The market valued QURE at ~$3.1bn when AMT-130 was essentially a pre-human clinical proposition.
Seven years later, it is assigning approximately the same equity value to QURE with a potentially disease-modifying HD therapy already submitted for approval in both the U.S. and UK.
Today we announced the submission of a BLA to the U.S. FDA for the accelerated approval of AMT-130, an investigational #genetherapy for the treatment of #HuntingtonsDisease. We also announced that an MAA for AMT-130 has been submitted to the U.K.’s MHRA. https://t.co/fM1w1kKF0j
@PatternRouaj@zerohedge Majority of canada’s trade deficits are from oil… canada exports dirty oil to the US, they refine it, then sell it to Asia, Europe and some back to Canada for a profit. US benefits from Canada from the deficit lol… stop drinking the koolaid
@muddywatersre Damn, you guys must be down bad with this one that now, you’ve resorted to just spamming so you can unload your dogshit onto them. Unfollowed
The FDA Commissioner role is open. This is a pivotal moment for American medicine — & for American patients.
Here’s what I believe the next Commissioner should stand for. Not as a wish list. As a baseline.
🔬 1. BRING BACK THE ADCOMS — AND MAKE THEM COUNT.
Advisory Committee meetings are one of the FDA’s most powerful tools for transparency. They should return in full force. Open. Public. And real.
Patients, doctors, scientists, advocates, & skeptics should all be able to speak. But if you want to speak, you fill out a financial conflict-of-interest form under penalty of perjury — & you read it out loud at the podium before you say another word. Every single person.
And if the issues are complex and the science requires more than one day, then take the time. Don’t cut people off because the schedule says so. The public deserves to see exactly how these decisions are made. Real transparency builds real trust.
⚖️ 2. STOP ASKING ONLY “IS IT SAFE ENOUGH TO APPROVE?” — START ASKING “WHAT HAPPENS IF WE DON’T?”
For rare diseases. For serious conditions with no good options. For patients who don’t have ten years to wait for traditional trials that may never be feasible.
Every regulatory decision carries two risks: the risk of approving something, and the risk of not approving it. Both are real. Both affect real people.
When traditional gold-standard trials aren’t practical, we should still be able to move with urgency — but only when paired with strong post-approval commitments and rigorous safety monitoring. We can give desperate patients a chance without abandoning scientific integrity.
🇺🇸 3. THE FDA’S ONLY CLIENT IS THE AMERICAN PATIENT. FULL STOP.
The FDA exists to serve patients — not outside interests or external pressures of any kind.
It must continue protecting the public from products that carry real, known risks but offer no meaningful clinical benefit.
At the same time, when there is credible evidence that a treatment can help patients with serious conditions, Americans and their physicians should be trusted to make informed decisions once they have complete and honest information about the risks, benefits, and alternatives.
The FDA’s job is to make sure the science is sound and the information is clear. Then let patients and doctors do what’s best for them.
These principles matter because the FDA’s decisions affect every family in this country.
Note: This is a simple social media post and not a massive policy document. The issues are FAR more nuanced, there are challenges and risks with each of the things I said above, and I recognize that. But it's intended to serve as a discussion starter. We must always strive to improve, and we can when we have open debate and dialogue.