uniQure N.V.: 2 Possible Regulatory Approvals Makes This A Must Watch

Aug. 30, 2022 2:26 PM ETuniQure N.V. (QURE)HTGC3 Comments


  • uniQure N.V. has U.S. and European approvals possible of etranacogene dezaparvovec for treatment of Hemophilia B before end of 2022 and early 2023 respectively.
  • uniQure N.V. has already tapped a partner that is licensed and will help commercialize the drug by the name of CSL Behring.
  • There are three catalysts for AMT-130, which is being developed as gene therapy for the treatment of patients with Huntington's Disease; One in Q4 2022 and the rest in 2023.
  • uniQure N.V. had $500.5 million in cash as of June 30, 2022; Potential to fund its operations into 1st half of 2025.
  • This idea was discussed in more depth with members of my private investing community, Biotech Analysis Central. Learn More »

Experiments in a chemistry lab. conducting an experiment in the laboratory.

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uniQure N.V. (NASDAQ:QURE) is a biotech that should be watched closely. That's because it has already submitted two regulatory applications to the FDA and EMA for use of its gene therapy, known as etranacogene dezaparvovec (AMT-061), for the treatment of patients with Hemophilia B. If the company receives regulatory approvals for this treatment, it would be the first gene therapy to be approved for this specific disease.

In other words, uniQure holds the potential to have the very first approved gene therapy option for Hemophilia B patients. The FDA BLA was accepted in May of 2022 with Priority Review, therefore I believe such approval could happen before the end of 2022. The European Medicines Agency (EMA) had it as a speedy review, but notified the company that it needs to change it to a standard review instead because it can't reach the timetable necessary to review it. The EMA review was validated in March.

Due to the change from speedy review to standard review for the European Union, I believe such approval is now likely in early 2023 for etranacogene dezaparvovec for the treatment of patients with Hemophilia B. UniQure doesn't even have to much of the heavy lifting, because it has already found a partner to focus on regulatory talks with agencies. The partner it found to move the drug forward with is CSL Behring, which is a huge powerhouse pharma company with 27,000+ employees. With an expanding gene therapy pipeline, plus two possible regulatory approvals for etranacogene dezaparvovec for the treatment of patients with Hemophilia B, these are the reasons why I believe that this biotech should be watched closely.

Etranacogene Dezaparvovec For The Treatment Of Patients With Hemophilia B

The fist program to go over involves the use of etranacogene dezaparvovec for the treatment of patients with Hemophilia B. The global Hemophilia market is expected to reach $18.88 billion by 2028. Hemophilia A is more common than Hemophilia B, but still even if you only account for let's say 20% of the entire Hemophilia market, it is still a huge opportunity. As the name suggests, severe Hemophilia B involves a situation where these patients experience a large deficiency of Factor IX. This means they have frequent spontaneous hemorrhage and abnormal bleeding events. Yes, that even included minor stuff like small injuries or following surgery/tooth extraction. How bad are these patients? These patients are in rough shape, that's because they have Factor IX below 1%. Just to give you an idea of what this entails, it is important to note the severity types of Hemophilia B:

  • Mild Hemophilia B patients have Factor IX levels between 5% to 40% of normal
  • Moderate Hemophilia B patients have Factor IX levels have between 1% to 5% of normal
  • Severe Hemophilia B patients have Factor IX levels less than 1% of normal as noted directly above.

You may ask right away, does this mean there is a small market opportunity for severe Hemophilia B? The quick answer is "No." That's because, believe it or not, but Hemophilia B accounts for 20% of all cases.

The phase III HOPE-B trial, is an open-label single-arm study which used etranacogene dezaparvovec for the treatment of patients with Hemophilia B. A total of 54 patients were recruited into this study, but had to first go through a lead in treatment period. That is, they took uniQure's gene therapy along with current standard of care (SOC) therapy in order to establish annualized bleed rate (ABR). After that, patients had received a single intravenous dose of 2x10^13 gc/kg of etranacogene dezaparvovec. Out of the 54 patients recruited into this late-stage study, about 53 patients had completed at least 18 months of follow-up. The primary endpoint for this study was 52-week Annualized Bleed Rate (ABR) after having achieved stable FIX expression compared to 6-month lead in period.

What does this mean? It means that the measure of ABR didn't actually start until month 7 and then out to month 18. Then this period was compared to the 6-month lead in period. It was noted that etranacogene dezaparvovec achieved a reduction of adjusted ABR by 64%. In addition, it was shown to be superior to prophylaxis treatment at 18 months post-treatment compared to the 6-month lead in period. This measure was achieved with a statistically significant p-value of p=0.0002. The bottom line is that this gene therapy achieved statistical superiority in reducing ABR compared to baseline FIX prophylactic treatment.

With this data on hand, uniQure was able to submit a Biologics Licensing Application (BLA) to the FDA for the potential approval of etranacogene dezaparvovec for the treatment of patients with Hemophilia B. The FDA accepted the application of this gene therapy for Priority Review in May of 2022. As I have stated on many occasions the FDA review time with this designation is cut down from 10 months to 6 months. At the latest update, the notion is that the FDA is still reviewing etranacogene dezaparvovec for patients with Hemophilia B as a Priority Review submission. I mention this, because it's now different when it comes to the European application. The European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for this gene therapy for this patient population back in March of 2022. However, since then things have changed slightly. It was noted by the Committee for Advanced Therapies (CAT) in Europe in July of 2022 that they will not be able to complete their review of this gene therapy with accelerated assessment. As such, they had to switch it to a standard review which will take it longer to complete.

What do all these advancements for etranacogene dezaparvovec mean for investors/traders? It means that there are two catalysts on deck to look forward to. The first of which is possible European Medicines Agency (EMA) regulatory approval for this gene therapy for this indication in early 2023. This is because of the switch from an expedited review to a standard review. That's the bad news, in that European approval could take longer. The good news is that with no change on the FDA front in terms of Priority Review, FDA approval could happen before the end of 2022. The risk is that there is no guarantee that this gene therapy will be approved for Hemophilia B in one or either of these territories.

I would put uniQure in a good position at the moment, whereby it made the right decision to find a partner. That's because it doesn't have to do much of the hard work to reap a lot of the benefits. The partner that it found is known as CSL Behring and such a partnership was announced a few years ago back in June of 2020. However, this partnership could not close until the completion of antitrust reviews in the United States, United Kingdom and Australia. Having said that, the actual agreement didn't actually close until May of 2021. Under the terms of the agreement, uniQure received an upfront cash payment of up to $450 million, with potential to earn a few additional items such as:

  • Regulatory milestones
  • Commercial sales milestones
  • Royalties on net sales of a commercialized product.

The thing is that all uniQure really had to do was complete the phase 3 HOPE-b trial and then scale up manufacturing for the initial commercial supply to be sold. However, CSL Behring is responsible for all regulatory talks and commercialization of the gene therapy should it be approved. I believe this is a good thing, because a lot of biotechs tend not to succeed because they don't have the capabilities to market successfully on their own. With a huge partner like CSL Behring, at least it has a greater chance of success. It won't guarantee success, but it's nice having a partner with a lot of financial backing.


According to the 10-Q SEC Filing, uniQure N.V. had cash and cash equivalents of $500.5 million as of June 30, 2022. The reason for the amount of cash on hand has to do with the CSL Behring partnership for the company with respect to etranacogene dezaparvovec, which helped it obtain an upfront payment of $450 million. It had also enacted equity offerings to sell shares on the open market and through private placement agreements throughout the year. It believes it has enough cash on hand to fund its operations into the 1st half of 2025 if it can obtain the $175 million first commercial sales milestones with respect to the CSL Behring Agreement.

Despite the notion that it has enough cash until 2025, I believe it's possible it could raise earlier than expected, so please be aware of that. Why do I believe this to be the case? That's because it owes an outstanding loan amount to Hercules Capital, Inc. (HTGC) for $100 million as of June 30, 2022. The good thing is that it won't have to repay this $100 million amount until between December 2024 and December 2025 if one of two items are achieved before June 30, 2024. The first is if etranacogene dezaparvovec is approved by the FDA for the treatment of patients with Hemophilia B. The second is that AMT-130 is advanced into a pivotal study in the coming years.

Risks To Business

There are several risk factors that investors should be aware of before investing in this biotech. The first risk involves the BLA filing of etranacogene dezaparvovec for the treatment of patients with Hemophilia B. With Priority Review given, U.S. approval is expected before the end of 2022. Despite the FDA having given this gene therapy Priority Review, there is no assurance that it will approve it for this patient population.

The second risk lies with the European Medicines Agency (EMA) review of etranacogene dezaparvovec. That's because this agency originally had expedited review, but needed additional time to look over the filing. Once again, there is no guarantee that it will approve it for Hemophilia B as well. Even if both regulatory agencies decide that this gene therapy merits approval for this patient population, it still has to perform well once it is on the market. This means, future sales will highly depend on whether or not insurance will cover the cost and if Doctors prescribe it for their patients.


I believe that uniQure is a great speculative biotech play to look into. It has the ability to receive two regulatory approvals of etranacogene dezaparvovec for the treatment of patients with Hemophilia B. The expected possible approval timeframe for the FDA for this treatment is 6 months from May 2022, which should be around November/ December of 2022. The possible European approval of etranacogene dezaparvovec for Hemophilia B is expected in early 2023, because of the expedited designation being changed. Still, I think there is great opportunity here based on these two catalysts in the coming year.

Another item to watch closely would be the other gene therapy that it has in its pipeline, which is AMT-130 for Huntington's Disease (HD). There are several catalysts for this program as well. There is a safety review update for the high dose of AMT-130 in the phase 1/2 study expected Q4 of 2022. There then is phase 1/2 U.S. study data using this gene therapy for HD in Q2 of 2023.

Lastly, there will be phase 1b/2 European study data results for this very same indication in 2023 at some point. With two possible regulatory approvals approaching for etranacogene dezaparvovec for Hemophilia B, plus several other catalysts as noted directly above relating to AMT-130 for HD, these are the reasons why I believe it is a great speculative biotech play to look into.

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33.50% discount price of $399 per year.

This article was written by

Terry Chrisomalis profile picture
Actionable ideas on small-large cap biotech stocks through deep analysis.

I am the Founder of Biotech Analysis Central, A subscription service on Seeking Alpha's Marketplace. If you want to learn more about biotech investing or you want to check out my biotech analysis you can do so with a free 2-week trial to my service. Just hit the "Learn More" button on the bottom of the Marketplace Research Tab. I have a Bachelors of Applied Science Degree In Technology Management, Industrial and Business Services Management from St. Petersburg College Florida. I have been investing in biotech stocks for many years, and I prefer to invest as a long term investor. With that In mind I seek stocks that have long term value! I primarily Like to Invest In biotechnology stocks and I accept the risks. I Write for the Healthcare Sector and Stock market in general. I contribute to Seeking Alpha.

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