uniQure NV (NASDAQ:QURE) – A Prognosis

In early Dec 22, I wrote about uniQure’s latest development on their AMT-061 gene therapy treatment. They have made headlines as the world’s most expensive drug.

For this post, I take a more in-depth look into this company to see what are the current developments on AMT-061 and their research pipeline.

Company Overview

uniqure NV is a biotechnology company focused on developing gene therapies for rare diseases. The company was founded in 1998 and is headquartered in Amsterdam, Netherlands.

uniQure uses an Adeno-associated (AAV) virus delivery system for the foundation for all their therapeutic genes. They use the AAV5 variant, or serotype, and have exclusive, worldwide rights to its use in therapeutic products delivered to the brain or the liver.


You can visit the link above to get a complete understanding of how the system works. There is a Youtube video which explains it pretty well.

Product Pipeline

Uniqure’s product pipeline includes several gene therapies in various stages of development for a range of rare diseases, including hemophilia B, Fabry disease, and Huntington’s disease. These therapies are designed to target the underlying genetic causes of these diseases, with the goal of providing long-term symptom relief and potentially even cure.

uniQure N.V. (QURE) Investor Presentation – Slideshow Sept 22

Although the company has many diverse candidates, there are now only two clinical projects active. That does come with some elevated risk and stakes, especially as these programs could be compared to the launch of UniQure’s gene therapy platform.

One of Uniqure’s most advanced products is its gene therapy for hemophilia B, which is a rare bleeding disorder caused by a deficiency of clotting factor IX. The therapy is designed to deliver a functional copy of the factor IX gene to patients, allowing them to produce their own clotting factor and potentially eliminating the need for regular infusions of factor IX protein. The therapy is currently in Phase 3 clinical development and has shown promising results in early clinical trials.

As we can see, Hemophilia B is quite close to complete Phase 3, which could result in commercialization in the near future and significantly reduce risk. Despite this, we shouldn’t ignore the company’s very broad portfolio. It also has many opportunities in the upcoming years, but at the moment the business is based on a few gene therapies, so investors should keep a very close check on them.

Overall, Uniqure is committed to developing innovative treatments for rare diseases that have a significant impact on patients’ lives and offer the potential for long-term symptom relief or even cure. The company’s diverse product pipeline and focus on gene therapies make it a leader in the field of rare disease treatment.

AMT-061 Hemophilia B

AMT-061 is a gene therapy developed by Uniqure NV for the treatment of hemophilia B, a rare bleeding disorder caused by a deficiency of clotting factor IX. The therapy is designed to deliver a functional copy of the factor IX gene to patients, allowing them to produce their own clotting factor and potentially eliminating the need for regular infusions of factor IX protein.

AMT-061 is administered through a single intravenous infusion and is delivered using a modified adeno-associated virus (AAV) vector. The AAV vector carries the functional copy of the factor IX gene and is designed to target liver cells, where the gene is expressed and the protein is produced.

Once delivered, the therapy is designed to provide long-term symptom relief and potentially even cure the disease by allowing patients to produce their own functional clotting factor IX. This could potentially eliminate the need for regular infusions of factor IX protein and reduce the risk of bleeding episodes and related complications. The big pivotal study for the drug is the HOPE-B phase III study for AMT-061.

CSL Behring global licensing deal

In May 2021, uniQure and CSL completed their licensing deal for the gene therapy. Under the agreement, uniQure has received payments from CSL totaling ~$500M and is eligible to receive up to an additional $1.5B in commercial milestone payments, plus royalties.

In Nov 22, the U.S. Food and Drug Administration (FDA) approved Hemgenix for adults with hemophilia B who are currently undergoing Factor IX prophylaxis therapy or who have experienced recurrent bleeding episodes or fatal hemorrhages. CSL Behring and uniQure’s hemophilia B gene therapy Hemgenix debuts not only as the first gene therapy approved for the rare disorder, but as the new most expensive drug in the world. The drug will be given price tag of $3.5 million per dose.


On 16 Dec 2022, A committee of the European Medicines Agency (EMA) recommended granting conditional marketing authorization to CSL & uniQure’s  one-time gene therapy Hemgenix to treat severe and moderately severe Hemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. The positive opinion by the EMA’s Committee for Medicinal Products for Human Use (CHMP) was based on their incredible results from their HOPE-B pivotal trial, which shows that their drug works 96%, or 52 patients out of 54 patients.

Potential Income Stream

With the regulatory approval, I expect revenues potentially coming in by 2023.

We need to see how this will impact uniQure. Within the licensing agreement, we know that they will receive $1.6 billion for regulatory and commercial milestones on top of the $450 million they have already received. They will also get reimbursement on clinical and royalty costs. They also mention uniQure will also receive royalties, upwards of low-twenties percentage of net sales.

I do not have the breakdown on the specific milestones or requirements to achieve them. Nor do I have the percentage of the royalties.

But having access to the commercial infrastructure of CSL Behring could be very positive in the race to commercialize this product. It will also provide them plenty of cash to fund their pipeline all the way into 2026 and beyond, which uniQure needed for their research. Fintwit has a estimated milestone payment to be made by CSL. You can find the link below.

Fintwit’s estimated earnings from CSL deal

Next, we assume that the cost is $1.5m, that will be a net sales of $2m. A 20% royalty would mean that uniQure wlll earn $400,000 for each treatment.

The next step would be to estimate the number of patients that will be receiving this treatment. Hemophilia B occurs in approximately 1 in 25,000 male births. Although many hemophilia B carrier females do not have symptoms, an estimated 10-25% will develop mild symptoms and females have also been reported with moderate and severe symptoms. In 2021, there are about 1.874mil males births. This means it is expected that there will be around 75 diagnosed with Hemophilia B. Using data on the number of patients seen at Hemophilia Treatment Centers (HTCs). There are about 30,000 – 33,000 people with hemophilia in the US, of which 76.5% had hemophilia A and 23.5% were Bs. That would mean we have about 8,250 prevalent cases and 75 new cases yearly,

Projected Income from Hemgenix royalties

The question is how much royalties uniQure will receive in the future. It depends on the affordability, amount of eligible people and percentage given to uniQure.

This is only a rough estimate based loosely on an incremental of 200 treatments per year. Assuming uniQure’s cash burn rate is about $220mil, the royalties can fully fund their future pipeline from 2025 onwards. There are too many variables and there is not enough information to do a more accurate estimation.

However, we must remember that Hemegenix is worth $2billion plus royalties to CSL Behring. uniQure currently has a market cap of $1billion. If this therapy is a commercial success, uniQure would be a potential winner in the biopharma space.

AMT-130 Huntington’s Disease

uniQure is currently developing a gene therapy for the treatment of Huntington’s disease called AMT-130.

In August 2022, patient enrollment at the higher dose had been voluntarily paused following the reporting of suspected unexpected significant adverse reactions (SUSARs) in three patients shortly after they received the higher dose of AMT-130. This have since fully resolved.

In October 2022, the DSMB reviewed all available safety, biomarker and imaging data from the ongoing Phase I/II clinical trials of AMT-130 and recommended resuming treatment at the higher dose.

uniQure will probably provide another update on this gene therapy in 2Q23, where planned to announce one to two-years of follow up data from the U.S. Phase I/II clinical study.

The market size for treatment of Huntington’s disease is currently valued at around $1.7b. There is currently no approved treatment for patients diagnosed with Huntington’s disease and this provides a huge opportunity for uniQure if they are able to be the first approved treatment.


Other Research Pipeline

Looking at the R&D pipeline diagram above, we can see that the rest of uniQure’s pipeline is still in pre-clinical stage. AMT-260 and AMT-191 are currently working towards Investigational New Drug (IND) applications in 2023. AMT-260, acquired from Corlieve Therapeutics, is the treatment of refractory temporal lobe epilepsy while AMT-191 is the treatment of Fabry disease.


It is still fairly early to put a valuation on uniQure. Although it is difficult to quantify, we already have some data from their next prospective gene therapies. Disregarding those in preclinical stage, the 2 therapy treatment AMT-130 and AMT-061 could be worth more than $3bil (AMT-061 $2b, AMT-130, $1B). This estimate is solely based on what uniQure can monetize from these 2 treatments after they reached commercialisation stage. As mentioned earlier, uniQure’s market cap is around $1b, so I am seeing upside from here.

I think uniQure N.V. is an attractive biotech play to look at because of Hemgenix’s U.S. FDA approval for the treatment of adults with hemophilia B and the opportunity to keep using higher doses of AMT-130 in the phase I/II study for HD.

However, there is a risk that AMT-130 fail to get approval, uniQure would then be solely relying on Hemgenix’s royalties to fund their pipeline research and that may be insufficient if the take-up rate of this treatment is slow. uniQure would then need to look for other funding options.

Cheers and Merry Christmas.

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