September 30, 2026
Bonus Content: uniQure Lost 37% in a Day. Here Is What You Own at $24.
Dear Reader,
Back when I was in Congress, I issued a warning that disturbed a lot of people.
I said:
“We can expect a continuous and dangerous march toward corporatism and even fascism with even more loss of our liberties.”
I’m reaching out to you today because we’re now further down this road than ever before.
In fact, a dangerous experiment is under way within our stock market, which has already seen at least two popular stocks drop by 50%.
Get the name of both of them right here.
Today many Americans feel that something isn’t right. We’ve taken a turn down a path that’ll lead to ruin. But very few people in Washington, D.C. will ever tell you the truth – or explain what you need to do with your money.
That’s why I’m warning:
Every American family needs to seriously consider taking these THREE critical financial steps today.
It might surprise you to hear I’m not talking about gold or silver here.
I’ve long told folks to buy and hold precious metals. And I stand by that.
But gold and silver alone won’t be enough to survive what’s happening in D.C. today.
To explain what’s happening, I recently sat down and recorded an interview with one of the most respected financial research firms. They’ve posted the footage on their website free of charge. It’s right here.
If you have money in the stock market – or in a 401k, I recommend you watch it today.
Regards,
Dr. Ron Paul
Former U.S. Congressman and Presidential Candidate
P.S. Please get the facts for yourself and protect your family and your savings. No one in D.C. is telling you the truth. I want to help you wake up and open your eyes to what’s happening to our country.
There’s still time to prepare, but you don’t have long. Here are the three steps I recommend you take, while you still can.
uniQure Lost 37% in a Day. Here Is What You Own at $24.
Hey there, bargain hunter. A gene therapy company just handed you a 37% discount on the same stock that was up roughly 75% three months ago. The question is not whether that is painful. It obviously is. The question is whether the thing underneath the price is worth more than $24 and change.
Scoreboard
Shares of uniQure opened down sharply on September 29 after the company’s 48-month AMT-130 Huntington’s disease data showed a smaller treatment benefit than the one reported at three years. The stock touched a session low around $22.5 on volume of about 29.6 million shares. It clawed back significantly and closed near $24.5. The 52-week high, reached on October 28, 2025 after the company’s September 2025 Phase 1/2 topline release, was $71.50.
What Actually Happened
At 36 months, the updated high-dose analysis showed 80% slowing on cUHDRS and 67% slowing on Total Functional Capacity, with nominal p-values of 0.005 and 0.011, respectively. At 48 months, the primary cUHDRS endpoint showed 44% slowing, missing statistical significance with a p-value of 0.144, while TFC showed 61% slowing with a nominal p of 0.008.
That divergence is the whole story. The drug’s functional benefit held up; the composite scale did not. uniQure also argued that missing data in the external control group increased over time and that dropouts appeared to be progressing faster than those who stayed, suggesting the 48-month comparison could understate the treatment effect. The market, understandably, took the headline p-value and sold.
Why the BLA Filing Still Matters
The company has said its BLA is anchored on the 36-month dataset, and that the 48-month results were not included in that submission. The weaker four-year data are not, procedurally, part of what the FDA is required to review inside the submitted package. Whether the agency weighs them informally is the real open question.
AMT-130 holds RMAT, Breakthrough Therapy, and Fast Track designations from the FDA, which matter because they can compress timelines and invite more active dialogue with regulators. That is not a guarantee of approval. It is a structural advantage that smaller biotechs rarely carry into a filing.
The Numbers You Are Buying
- Cash, cash equivalents, restricted cash, and investment securities: $811.9 million as of June 30, 2026, which management said should fund operations into 2030.
- Free cash flow for the twelve months to June 30, 2026: not disclosed as a single figure in the company’s quarterly filing, but net cash used in operating activities was $66.8 million for the first six months of 2026.
- Market cap at about $24.5: roughly $1.7 billion.
- At that price, you are paying roughly $900 million above the cash pile for the entire AMT-130 program, the epilepsy asset AMT-260, and the pipeline.
That is cheap relative to where the stock was. It is not cheap in any absolute sense. The burn continues. Cash runway into 2030 cushions the risk considerably, but only if the spend stays disciplined and no large confirmatory trial is required before the FDA acts on the BLA.
The Legal Overhang
Kehoe Law Firm opened a securities investigation on September 29 on behalf of uniQure investors after shares declined sharply following the 48-month data release. This is the second litigation wave. A class action filed on February 10, 2026 in the Southern District of New York alleged false and misleading statements about the Phase 1/2 study and BLA timing, covering investors who bought shares between September 24 and October 31, 2025. That case is active. A new wave of plaintiffs focusing on the 48-month data could add to it. Settlements in biotech securities cases can be meaningful. Against $811.9 million in cash and investment securities, that is manageable but not free.
Bull / Base / Bear
Bull: The FDA accepts the BLA, grants accelerated approval based on the strong 36-month data, and the confirmatory study design using a standard-of-care control proceeds without a sham arm. AMT-130 becomes the first approved gene therapy for Huntington’s disease, which has no disease-modifying treatments. The $71 high is not unreasonable in that scenario.
Base: The FDA issues a complete response letter requesting additional data or a longer follow-up, uniQure runs a confirmatory study, and approval comes in 2028 or later. Cash holds. The stock trades sideways in the $20-$35 band for two years.
Bear: The FDA is spooked by the 48-month data degradation, rejects the BLA outright, and litigation costs compound against a now-stalled pipeline. The stock revisits the low $20s.
Action Plan
This is speculative biotech, not a value stock. Position sizing is everything. If you are adding here, the only rational approach is a small starter position, sized to survive a complete loss, with a plan to add on FDA acceptance of the filing or a positive agency meeting, not before.
Do not average down into the lawsuits. Wait for the motion-to-dismiss ruling on the existing class action, which gives you a cleaner read on the legal exposure before you commit more capital.
Cheap Investor Checklist
- FDA accepts the BLA for review: watch for a 60-day filing decision, expected by early November 2026.
- Confirmatory study design agreed with the FDA: no sham arm is the key concession to monitor.
- 48-month data presented at a scientific conference: peer review will either validate or destroy the missing-data argument.
- Class action motion-to-dismiss outcome: a dismissal reduces legal tail risk materially.
- Quarterly cash burn: any acceleration above $45 million per quarter shortens the runway.
- AMT-260 epilepsy data: early seizure-reduction results are promising and represent optionality the market is ignoring today.
- Insider buying post-crash: management purchasing shares at $24 would signal conviction that the three-year data holds regulatory water.
Bottom Line
If the FDA treats the 36-month data as the regulatory anchor, uniQure at $24 is materially undervalued relative to its cash position and the optionality of the first Huntington’s approval in history. If the 48-month miss gives the agency pause, you are holding a cash-rich shell with a long road and an active lawsuit. The BLA submission came before the 48-month data update. That sequence matters. Watch for FDA acceptance of the submission over the next 60 days: that single data point will tell you whether this discount is a gift or a warning.
