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uniQure N.V. (NASDAQ:QURE) is a great speculative biotech play to look into. The reason why I state that is because it was able to actually receive FDA approval for its gene therapy known as Hemgenix (etranacogene dezaparvovec). This was the first gene therapy to be approved for the treatment of patients with Hemophilia B. What makes this approval first and foremost is for the patients who needed a new treatment option.
For uniQure, it provides two positive developments. The first positive development is that it allows uniQure to have its first FDA-approved product on the market. The second positive development is that it provides the biotech with the ability to earn up to $1.5 billion in commercial milestone payments and tiered double-digit royalties on net product sales. In addition, the biotech has been able to move forward with being able to test out higher dosing relating to the phase Ib/II study evaluating AMT-130 for the treatment of patients with Huntington’s Disease (HD). Not only does this move the program forward, but it provides traders/investors with a catalyst opportunity to look forward to. That catalyst being the release of results from the U.S. phase I/II study using AMT-130 for the treatment of patients with HD, which is expected to be released in Q2 of 2023.
AMT-130 For Huntington’s Disease Moves Forward
What makes uniQure a far more interesting look now is that it has already received FDA approval of Hemgenix for the treatment of patients with Hemophilia B, which is what I will go over briefly below. However, the most recent news is that it has been able to move forward with the use of its AMT-130 gene therapy, which is being developed for the treatment of patients with Huntington’s Disease (HD). In Huntington’s Disease, neuron cells in the brain start to die off. This leads to random movement events, thinking impairment, and psychiatric symptoms as well. The age bracket where this disease starts to take place is between the age of 30 to 40 and is quite a devastating disease. Over time, the brain cells described above, start to die off.
Well, the loss of such brain cells doesn’t only cause a host of problems or symptoms for the patient. In addition, this disease can be fatal after a period of up to 20 years. It is a pretty addressable market size, matter of fact it is expected that the global Huntington’s Disease Treatment market is expected to be worth up to $1.7 billion by 2030. While the market opportunity is great here for uniQure, there is a more humane aspect to consider. That aspect being there are no approved treatment options for patients with HD. An eventual approval of AMT-130 for Huntington’s Disease would not only help the company achieve marketing success, but it would create a first as well. Meaning, at the moment it holds the potential to have the first approved treatment option for this patient population.
The ability to move forward was thanks to a positive comprehensive safety review of the Data Safety Monitoring Board (DSMB), which took a look at safety issues. After having looked at safety, biomarker and imaging data, it concluded that the higher dose of the ongoing phase I/II study using AMT-130 for HD, could proceed as planned. The goal now is to complete the recruitment of HD patients in this higher dose cohort in the coming year. That doesn’t mean that traders/investors will be left with no news in the early part of next year. That is, uniQure intends to release results from the U.S. phase I/II study using AMT-130 for the treatment of patients with HD, in Q2 of 2023. This provides a huge catalyst opportunity to look forward to in the 1st half of 2023.
FDA Approval Of First Gene Therapy For Adults With Hemophilia B
As I described above, uniQure and its partner CSL Behring have received FDA approval of Hemgenix, which as approved as a one-time gene therapy for the treatment of adults 18 and older with Hemophilia B. What makes this regulatory approval so special is that it is the first gene therapy to be approved for Adults with Hemophilia B. Why is this an important approval for both uniQure and its partner CSL Behring? Well, it now provides the opportunity to target a large market for starters. It is expected that the global Hemophilia treatment market could reach $26.9 billion by 2031.
However, it is important to note that the Hemophilia B market is not as large as the Hemophilia A market. Still, even only accounting for Hemophilia B alone, it is still expected to be a multi-billion dollar market opportunity. Secondly, it provides an opportunity for patients to get relief. That is because, current treatment options are Factor IX prophylaxis therapy. Even with this type of treatment option, Hemophilia B patients still have excessive spontaneous bleeding episodes.
The reason why I wanted to bring up the recent FDA approval of Hemophilia B is for two particular reasons. The first reason being is that there is another opportunity to receive regulatory approval for Hemgenix for the treatment of patients with Hemophilia B. What do I mean by this? Well, an application for review was already submitted to the committee for advanced therapies for accelerated assessment of this gene therapy for this patient population. Despite not receiving expedited approval for Europe, the review has switched over to a standard one for the European Union. As such, a possible catalyst to watch would be European approval of Hemgenix for the treatment of adult patients with Hemophilia B in 2023. The second reason is because uniQure is eligible to receive up to $1.5 billion in commercial milestone payments and tiered, double-digit royalties of net product sales, ranging up to a low-twenties percentage.
Financials
According to the 10-Q SEC Filing, uniQure N.V. had cash, cash equivalents, and restricted cash of $443.4 million as of September 30, 2022. It believes it has enough cash on hand to fund its operations into the 1st half of 2025. However, this is going to be contingent upon the ability to achieve specific sales milestones as it relates to the partnership with CSL Behring. That is, uniQure intends to achieve the first commercial sale milestone in the United States during this time, to provide it with up to $100 million. Then, it expects to achieve the ability of first commercial sale in any of the five contractually defined European countries as of or prior to July 1, 2023. With these being achieved in the coming years, then its projection of cash into the 1st half of 2025 should be on target.
Risks To Business
There are several risk factors that investors should be aware of before investing in this biotech. The first risk to consider would be with respect to the ability for both uniQure and its partner CSL Behring to successfully market Hemgenix for Hemophilia B. Receiving FDA approval for this gene therapy for this patient population is only half the battle. Now, both companies need to see how this treatment does on the market.
The good news is that there is potential to expand the market to Europe, should the European approval in 2023 go well. A second risk to consider would be with respect to the sales milestones being achieve. The ability for uniQure to maintain its projection of cash into the 1st half of 2025, is that it achieves specific sales milestones. There is no guarantee that such sales milestones will be achieve and as such, the company may need to raise cash by some other means. A third risk to consider would be with respect to the ongoing higher dose cohort of AMT-130 in the phase I/II study, which is treating patients with Huntington’s Disease (HD). Even though there was a green light to continue this study, there is no guarantee that there won’t be any newly identified safety issues.
Conclusion
The final conclusion is that uniQure is a great speculative biotech play to look into. It has not only already received FDA approval of Hemgenix for adults with Hemophilia B in the United States, but it can also do so for the European Union as well in 2023. In addition, should sales of this gene therapy go well, it would be entitled in up to $1.5 billion in commercial milestone payments and tiered, double-digit royalties of net product sales, ranging up to a low-twenties percentage. There is also a catalyst opportunity for investors/traders to look forward to. In that, the biotech intends to released results from the U.S. phase 1/2 study using AMT-130 for the treatment of patients with Huntington’s Disease in Q2 of 2023.
With U.S. FDA approval of Hemgenix for the treatment of adults with Hemophilia B, plus the ability to continue higher dosing of AMT-130 in the phase I/II study for HD, these are the reasons why I believe uniQure N.V. is a great speculative biotech play to look into.
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