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uniQure: A High-Risk, High-Reward Investment Opportunity

Apr. 07, 2021 9:32 PM ETuniQure N.V. (QURE)IONS, RHHBY, WVE15 Comments
Sage Advisors profile picture
Sage Advisors


  • uniQure has submitted data to the FDA that suggest that etranacogene dezaparvovec (AMT-061) is highly unlikely to be the cause of liver cancer in a patient in the pivotal trial.
  • FTC review of the transaction with CSL Behring is ongoing and is projected to close during the second quarter of 2021.
  • Wave Life Sciences and Roche/Ionis reported disappointing results in Huntington’s disease clinical trials.
  • uniQure has made significant progress in their Huntington’s program and data readouts in late 2021 will give investors a glimpse into AMT-130’s efficacy and safety profile.

uniQure (NASDAQ:QURE) is on track to read out data from its pivotal trial of AMT-061 for patients with hemophilia B. Given the gene therapy appears safe, the company can complete the pivotal trial and eventually file for a regulatory approval. The closing of the deal with CSL Behring may provide significant cash flow to fund the pipeline. uniQure remains a high-risk, high-reward investment due to the Huntington's disease program.

AMT-061 appears to have a favorable safety profile

uniQure released findings regarding their investigation into the cause of hepatocellular cancer that was diagnosed in a patient in the HOPE-B gene therapy trial for patients with hemophilia B. The company concluded that its gene therapy for hemophilia B is not likely to be related to the patient developing hepatocellular cancer. The patient who developed the cancer had multiple risk factors including hepatitis B and C infections which are associated with 80 percent of all hepatocellular cancers.

According to a statement released by uniQure, "The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC. Moreover, whole genome sequencing of the tumor showed that this patient had large abnormalities on chromosomes 1 and 8 that are commonly associated with HCC, as well as mutation of TP53 and several other potentially oncogenic genes." Discussions with the FDA about these findings should occur in Q2 and based on this data, uniQure seeks to have the clinical hold lifted.

The clinical hold placed on the HOPE-B trial due to the hepatocellular cancer case resulted in QURE's stock falling significantly in December. QURE shares had been trading in the high $40 range and fell as low as $28 as investors waited for a resolution to the issue. bluebird bio (

This article was written by

Sage Advisors profile picture
M.A. Science. I use my science background, the knowledge acquired while working for a major pharmaceutical company and access to scientists with domain specific knowledge to find the best opportunities in biotech.

Analyst’s Disclosure: I am/we are long QURE. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

This article is not a recommendation to buy or sell any security.

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Comments (15)

joezapp profile picture
Can't see anything bad about the early data reported today.
What is the market seeing that I'm not?
Sage Advisors profile picture
@joezapp The company previously guided that they would have biomarker and MRI data. They had technical difficulties with the assay testing mHTT so that data was not available. Even if they had this data it would not tell us anything more than a guess at whether there was target engagement. The CSF levels may not correlate with brain levels.

QURE was advised if they gave out MRI data showing potential improvements it would potentially unblind the study and the data could not be used for regulatory purposes so they did not give this data. This perhaps is the most important data (in my opinion) because it is a clear suggestion of efficacy to see reduced brain atrophy. So, this was a disappointment not to be able to see this data.

One piece of good news was they saw Neurofilament light chain levels initially rise as a result of the impact of surgery and then return to baseline at one year. Recall this is a measure of neuro-axonal damage. This was exactly what was hoped for. This was clearly good news.

In addition, Huntington's disease progresses slowly so they need time to elapse w placebo patients and will also use natural disease progression controls to assess whether treated patients progress differently. The market is impatient and science is slow.

So, we know very little other than the AMT-130 is safe and they will have a real indication about efficacy in 2023. This is good news but they have been given the green light on safety previously so it was not new news.

Science moves slowly and what the market perceives (in my opinion) is that we do not have enough data to show an efficacy signal. I would add ( in my opinion) there was no negative data just an absence of data.
joezapp profile picture
@Sage Advisors Thank you for this thorough explanation! So it's more waiting, and investors don't like waiting. I can wait, so I'll add at these levels.
Forsitan profile picture
Any feedback on today's readout? Thank you
Sage Advisors profile picture
@Forsitan The data was excellent. It showed a statistically superior reduction in ABR in treated patients compared to the lead in period. (p=0.0002) Uniqure will be due milestone and royalty payments which they can add to the $700 m in cash which is huge stash considering the market cap. HD safety has been excellent thus far. HD biomarker data due for first treated patient soon and although it will be difficult to ascertain whether there is an efficacy signal, in 2022 there will be more data.
joezapp profile picture
@Sage Advisors Thanks again for your updates. We knew this would become a waiting game, but I didn't foresee the stock price getting so low. Investors have no patience!
Sage Advisors profile picture
@joezapp Biotech has been absolutely crushed this year. The fundamentals for QURE are good despite the share price. The share price does not reflect a clinical setback. In fact, they have excellent safety updates not just in hem B but also in their early stage HD program which has been expanded.

In addition, if you followed their investor day update they have also done a lot to expand their pipeline. It is early stage and high risk but they have the cash needed to execute.

It is difficult to have patience but generating data in these types of diseases takes years in the clinic which also creates a nice moat around their business.
KevinJohnston profile picture
Great article on QURE! I love your style :)

Also, when you say the "likelihood of success is low", does your feeling/research give you the impression that the probability of success is "Above 25%", or "Above 33%", or below the following 2?

Thank you
Sage Advisors profile picture
@KevInvesting Until there is biomarker data and clinical data, it is a very low probability in my opinion.
KevinJohnston profile picture
@Sage Advisors Ok, thank you! And when you refer to the low probability are you speaking of FDA Approval after they conduct a Phase 3 trial or of the chances of the current Phase 2 trial posting positive data (i.e., mHTT CSF levels)? If it is the former, what are your feelings on the Phase 2 trial results?
Sage Advisors profile picture
@KevInvesting Uniqure has only treated 10 patients in a Ph 1/11 so they are very early stage in the clinic. My opinion re: the probability of success is based on the current stage/current data.
joezapp profile picture
Thank you for another nice piece on QURE.
biogenius profile picture
Great objective article. I agree with everything you say. Near term price movement will depend more on the hemophilia B program. I am long.
Boston Biotech Investors profile picture
Thank you & well done @Sage Advisors You have a truly unique ability to take complex value propositions and translate them into simple English...late 2021...got it.

I recommend investors interested in biotech take Sage's guidance very seriously. Because Sage generally analyzes clinical stage biotech companies, the target profiles are much riskier than (easier to understand) commercial-stage biotechs. Sage has picked some real winners over the years & is, arguably, the best (most qualified & competent) clinical stage biotech analyst on Seeking Alpha.
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