uniQure N.V. (NASDAQ:QURE) – World’s Most Expensive Medicine

uniQure N.V. (NASDAQ:QURE) is a biotechnology company developing gene therapies for rare genetic disorders. Its lead pipeline gene therapy is AMT-061 (etranacogene dezaparvovec) for the treatment of Hemophilia-B. In Nov 2022, regulators have approved this drug. Priced at $3.5million per dose, this is currently the world’s most expensive medicine.

In this investment case study, we will analyze the company’s financials, competitive landscape, and potential risks and rewards associated with investing in uniQure N.V.



Company Overview

UniQure N.V. is a biotechnology company based in Amsterdam, Netherlands. The company specializes in the development of gene therapies for rare genetic disorders. Its lead pipeline gene therapy is AMT-061 (etranacogene dezaparvovec) for the treatment of Hemophilia-B. In November 2022, regulators approved this drug, making it the world’s most expensive medicine, priced at $3.5 million per dose.

The gene therapy will be manufactured in Lexington, Massachusetts, by uniQure NV, which sold the commercialization rights for Hemgenix to CSL Behring in 2020.

$QURE was founded in 2012. The company has a strong management team, led by CEO Matt Kapusta, who has over 25 years of experience in the biotechnology industry.


Collaboration with CSL Behring

CSL Behring is a global biotherapeutics leader. CSL focus on serving patients’ needs by using the latest technologies. The company develop and deliver innovative therapies for treating coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company’s products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (CMXHF) (ASX:CSL;USOTC:CSLLY) delivers its life-saving therapies to people in more than 100 countries. Their headquarters is in Melbourne, Australia.

In 2020, CSL Behring agreed to acquire exclusive global license rights to commercialize the drug.


CSL Behring is seen as the ideal strategic partner with extensive commercial expertise in hemophilia. They have the capability to provide this drug to the greatest number of global patients as quickly as possible.

Etranacogene Dezaparvovec (AMT-061)

Hemophilia B is caused by mutations in the Factor IX gene, which provides instructions for making a protein called Factor IX. This protein is essential for blood clotting, and without it, people with hemophilia B are at risk for spontaneous bleeding and injury. Etranacogene Dezaparvovec is designed to correct the underlying genetic cause of the disorder by providing a functional copy of the Factor IX gene.

Etranacogene Dezaparvovec is administered through a one-time intravenous infusion. The therapy uses a harmless virus to deliver the functional Factor IX gene to the patient’s liver cells, where it can begin producing the necessary protein. Etranacogene Dezaparvovec has been shown to be safe and effective at reducing bleeding episodes and improving clotting function in patients with hemophilia B.


One of the key advantages of Etranacogene Dezaparvovec is its long-lasting effects. Etranacogene Dezaparvovec provides a lasting benefit with just a single infusion. This is unlike other treatments for hemophilia B, which must be administered regularly to maintain their efficacy. This can greatly improve the quality of life for patients as they no longer have to worry about frequent injections or infusions.

Etranacogene Dezaparvovec has the potential to reduce the need for regular blood transfusions. This is often required to treat severe cases of hemophilia B. By providing a functional copy of the Factor IX gene, Etranacogene Dezaparvovec allows the patient’s own body to produce the necessary protein, potentially reducing or eliminating the need for transfusions. This can not only improve the patient’s health and wellbeing, but also save money and resources.

Cost Savings

Blood transfusions can be expensive and time-consuming, requiring regular hospital visits and specialized medical equipment. Etranacogene Dezaparvovec has the potential to save healthcare systems and insurance companies significant amounts of money. . They do this by reducing the need for transfusions. Additionally, it can free up medical resources that can be used to treat other patients, further improving the efficiency of the healthcare system.

Despite its potential benefits, Etranacogene Dezaparvovec is not without its challenges. The development and production of gene therapies like Etranacogene Dezaparvovec are complex and costly, and the therapy is not yet widely available. Additionally, the long-term safety and effectiveness of Etranacogene Dezaparvovec and other gene therapies are still being studied and are not fully understood.

Overall, Etranacogene Dezaparvovec represents a significant advance in the treatment of hemophilia B. It has the potential to improve the lives of patients with the disorder. It also reduce their reliance on frequent blood transfusions. The development of Etranacogene Dezaparvovec is a promising step forward in the field of gene therapy.



UniQure N.V. has seen a steady rise in its financials over the past few years.

In 9 months 2022, the company reported total revenues of $3.7 million, down from $466 million in 2021. This decrease was due to the license revenue of $450mil and $12.4mil received from CSL Behring. The company’s research and development expenses also increased from $101 million in 2021 to €139 million in 2022. The company reported net loss for the year at -$2.86 per share.

UniQure N.V.’s balance sheet seems healthy. The company’s total assets stands at $638mil. The company has $440mil in cash and cash equivalents. The company’s total liabilities also increased from $213 million in 2021 to $217 million in 2022.

UniQure N.V.’s stock price has also seen a steady increase over the past few years. The company’s stock price has increased from $7.00 per share in 2019 to around $25.00 per share in 2022.

Potential Returns from AMT-061

The cash flow that UniQure N.V. will earn from AMT-061 will depend on a number of factors. These include the size of the market, the pricing and the cost of manufacturing and distributing the therapy.

The size of the market for AMT-061 will depend on the number of people with hemophilia B in the United States and Europe. Approximately 6,000 of these people living in the United States. This suggests that the potential market for AMT-061 is relatively small.

From their press release, they are expected to receive double digit royalty payments for each dose administered by CSL. 20% of $3.5mil would be $700,000.00

Assuming that 5,000 from both United States and Europe are eligible for the treatment. $QURE will receive about $3.5B in royalty payments.

$QURE has a market cap of $1.2B. This means they have some catching up to do.


Regulatory Risk

The regulatory environment for the biotechnology industry is complex and constantly evolving. In order to bring a new treatment to market, a biotechnology company must navigate a maze of regulations and obtain approval from various government agencies. This process can be lengthy and costly, and there is no guarantee that a company will be successful in obtaining the necessary approvals.

Despite these risks, there are also potential rewards for investors who are willing to take on the challenges of investing in $QURE. If the company is successful in developing and commercializing new treatments, it could potentially generate significant revenue and provide strong returns for its investors.

Clinical Risk

Clinical trials are a crucial step in the development of new treatments. They test the potential drug’s safety and efficacy. However, there is always a risk that a treatment will not perform as well in clinical trials as it did in preclinical studies, and this could have a negative impact on a company’s financial performance.

Furthermore, even if a treatment is found to be safe and effective in clinical trials, there is still a risk that it will not be approved by regulatory agencies. The approval process for new treatments is rigorous and can be lengthy, and there is no guarantee that a company will be successful in obtaining the necessary approvals to bring its treatment to market.

For investors in $QURE, this means that there is a risk that the company’s treatments will not be successful in clinical trials or will not be approved by regulatory agencies. This might impact on the $QURE’s financial performance and the value of their investment.

Cash Burn Rate

The cash burn rate refers to the rate at which a company is using up its available cash. It is an important metric to consider when evaluating the financial health of a company. A high cash burn rate can indicate that a company is spending a significant amount of money on its operations. This can be a cause for concern for investors.

In the case of $QURE, the company’s cash burn rate may be a concern for potential investors. The biotechnology industry has high research and development costs, and $QURE is no exception. The company is currently conducting clinical trials for several of its treatments, and these trials can be expensive. Additionally, the regulatory process for bringing a new treatment to market can also be costly. This means that $QURE may be burning through its cash reserves at a significant rate. This means that it could be a cause for concern for investors.

However, it is important to note that the cash burn rate is just one factor to consider when evaluating the financial health of a company. Investors should also consider a company’s overall financial performance, as well as its future growth prospects, before making any investment decisions.


In conclusion, investing in $QURE carries a high level of risk, but it also offers the potential for significant rewards. They are set to enjoy the upcoming milestone payments and royalties from their AMT-061. Potential investors should carefully weigh the risks and rewards of investing in the company and consult with a financial professional before making any investment decisions. While there are no guarantees in the world of investing, those who are willing to take on the risks of investing in $QURE may be rewarded with strong returns.


Hope you have enjoyed this article. If you are looking for more of my case studies, I have shared a few ideas here.

4 thoughts on “uniQure N.V. (NASDAQ:QURE) – World’s Most Expensive Medicine

Leave a Reply