QURE Stock Overview
uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases.
uniQure N.V. Competitors
Price History & Performance
|Historical stock prices|
|Current Share Price||US$19.53|
|52 Week High||US$36.55|
|52 Week Low||US$12.52|
|1 Month Change||-5.47%|
|3 Month Change||-6.29%|
|1 Year Change||-37.20%|
|3 Year Change||-54.21%|
|5 Year Change||84.77%|
|Change since IPO||33.68%|
Recent News & Updates
uniQure: Clinical Risk, A Critical Upcoming Catalyst And A Cash Cushion
Summary uniQure outlicensed AMT-061, EtranaDez, to CSL Behring who will market the gene therapy for hemophilia B. EtranaDez has a PDUFA date in November and if approved, uniQure will collect a $175 m milestone payment upon commercial launch strengthening their cash position. The AMT-130 program in Huntington’s disease (HD) had a setback but the study will continue and the company is on track to report data in 2023. While target engagement was confirmed, the AMT-130 program remains a high risk program given the poorly understood biology. The pipeline continues to advance and 2023 will be pivotal for the company where success in HD could dramatically reset the valuation. Summary uniQure (QURE) shepherded AMT-061 through two iterations and produced a best in class gene therapy for hemophilia B which may be FDA approved in November. After securing a licensing deal, uniQure is positioned to collect milestones and royalties potentially providing a revenue stream. Despite a setback with AMT-130, the company is on track to present proof of concept efficacy data in 2023. If positive, this could reset the valuation for uniQure. The AMT-130 program remains very high risk but the pipeline will expand in 2023 with 2 news INDs providing a backup plan. While AMT-130 may not pan out, should AMT-130 show efficacy, uniQure will be on track to produce the first disease modifying treatment for HD which may open up a $7B market and reset the valuation. AMT-061 AMT-061, EtranaDez, has a PDUFA date in November and an EU decision will follow. Given the treatment met the primary endpoint and demonstrated non-inferiority in annualized bleeding rate compared to baseline Factor IX, approval is likely. uniQure is on track to receive a milestone payment of $175 upon commercial launch. The licensing deal with CSL entitles uniQure to $1.6B in milestone payments and royalties that reach as high as the low 20 percent range. A draft ICER report noted that a gene therapy for hemophilia A provided significant benefit and a $2.5m price was warranted. The EU pricing of this gene therapy produced by BioMarin (BMRN) was announced to be approximately 1.5m Euros in the EU. While uniQure's gene therapy is for hemophilia B rather than hemophilia A, it may command a similarly high price and royalty payments may be very significant given they can reach the low 20 % range. AMT-130 The AMT-130 program seeks to deliver a disease modifying treatment for Huntington's disease. This is uniQure's lead program and it recently encountered a setback. The focus remains on whether the gene therapy will show efficacy in Huntington's disease. Huntington's Disease Huntington's disease ((HD)) is a devastating autosomal dominant genetic disease. It is progressive, debilitating and inexorably fatal. Involuntary movements, impaired gait and posture, difficulty speaking along with mood and memory problems occur as the disease progresses. There are no disease modifying treatments for Huntington's disease. Many people at high risk of having the disease chose not to be tested as there is no treatment. Symptoms usually begin between age 30 and 50 and the cognitive and functional declines manifest over 15 to 20 years. There are approximately 30,000 people in the US diagnosed with Huntington's disease and an additional 75,000 at risk of developing it. It is more prevalent in people of European ancestry and it is estimated there are 40,000 Europeans with the disease. The biology underlying HD is poorly understood. The genetic mutations in HD results in mutant huntingtin protein, mHTT, aggregating in the brain. The disease manifests as the selective destruction of cells in the caudate and putamen. There is debate as to whether the disease and the symptoms are caused by the absence of functional wild-type HTT, (wtHTT) the presence of mHTT or a combination of both. In addition, the accumulation of a short, potentially toxic fragment known as exon1 HTT protein may also be a cause of the disease. This fragment is only found in Huntington's patients suggesting its causal involvement. Studies in mice show the presence of only HTT exon 1 results in all of the characteristic disease manifestations. Scientists writing in Nature noted that, "inhibiting the production of exon 1 HTT…might pose a very promising strategy in treating HD." A key question for developers of HD treatments is whether non-allele specific huntingtin lowering will benefit patients. While there is evidence that individuals lacking wtHTT can be healthy, debate remains as to whether reducing wtHTT could negatively impact clinical outcomes. A Competitor Faces a Setback Roche's tominersen is an antisense oligonucleotide and has shown mHTT lowering in clinical studies. Roche announced results for the tominersen phase 3 trial that suggest that non-allele specific HTT lowering may not have a positive impact on patients. Unfortunately, the phase 3 trial was halted after a review concluded that patients receiving the higher dose of tominersen showed worse outcomes than placebo patients. While the reasons for these results were not clear, Roche recently decided to move forward with a new trial using lower doses and younger patients with less advanced disease. Vicki Wheelock, director of the Huntington's Disease Society of America Center of Excellence at University of California, Davis provided context on the results. "It doesn't mean that [antisense medicines] or huntingtin lowering doesn't work for Huntington's disease…It means that in this study, in this population of patients, with this dose, at this interval, it didn't work." The lack of understanding of the underlying biology in HD including the normal function of HTT protein has hindered progress in identifying drug targets. AMT-130 an extremely high risk program given it is unknown what an ideal drug needs to achieve to positively impact patients. AMT-130 AMT-130, is a gene therapy candidate which utilizes an AAV5 that is designed to deliver a piece of micro RNA to silence the huntingtin gene. The goal of treatment is to non selectively inhibit the production of both mHHT and wtHTT proteins. AMT-130 differs from other treatment approaches in that it also targets the exon 1 HTT fragment. In a phase I/II trial, AMT-130 was administered in a neurosurgical procedure directly to the striatum of early stage HD patients. One year results were announced for low dose AMT-130. The procedure was well tolerated with no SAEs in any of the 10 patients in the low dose (6x1012vg) cohort. Mean mHTT reductions of 53.8% were observed in the CSF in evaluable AMT-130 treated patients compared to a 16.8% decrease in placebo patients. This data suggests target engagement but there were no cognitive or functional measure of disease progression available. Whether the target engagement will result in clinical benefit is as of yet undetermined. uniQure uniQure's approach has some key differences from the tominersen trial. The AMT-130 trial only enrolled patients early in the clinical course of the disease. AMT-130 was distributed directly and exclusively to the tissues affected in the striatum rather than intrathecally. Lastly, AMT-130 targets HTT Exon 1 while tominersen does not. uniQure announced on a recent call that regulators had been notified of SUSARs (suspected unexpected serious adverse reactions) which involved localized inflammatory responses in two patients and severe headache and other symptoms in another patient treated with the high dose AMT-130. One patient of the 14 treated at the higher dose, developed motor and behavioral symptoms in the 12 day window after the procedure. After steroid administration the patient recovered but subtle deficits in verbal fluency, memory and attention relative to his preoperative state persisted. A second patient experienced vomiting and raised intracranial pressure in the acute post-operative timeframe (12 days) and experienced relief after a lumbar puncture to remove 20 cc of cerebral spinal fluid was performed. The symptoms fully resolved. A third patient experienced severe headache and vomiting symptoms soon after high dose AMT-130 administration. A blood patch, which is a procedure by which blood is injected into the spinal canal to patch a hole and prevent cerebrospinal fluid leaking was performed. Imaging revealed edema in the striatum and the infusion tracts, which largely resolved by the patient's 30-day follow-up visit. If AMT-130 demonstrates the ability to slow decline, the risk benefit analysis may still be favorable given the certain outcome if patients remain untreated. The most recent setback resulted in the pausing of dosing of only the higher dose of uniQure's AMT-130 trial while the company and the IDSMC review these adverse events. An extensive review which will include CMC as well as a review of the administration and monitoring protocols will be conducted. Results should be available by Q422. Data in 2023 will likely be "make it or break it" for AMT-130 By mid 2023, uniQure will release further data on the first cohort treated with AMT-130. There will be data provided for 16 patients who received the high dose. This cohort includes 10 patients who were treated and 6 control patients with one year follow up. There will also be data on ten patients who received the low-dose and 4 control patients with two years of follow-up.
uniQure N.V.: 2 Possible Regulatory Approvals Makes This A Must Watch
Summary 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. uniQure N.V. (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. Financials 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.
uniQure N.V. (NASDAQ:QURE) Analysts Are Reducing Their Forecasts For This Year
The latest analyst coverage could presage a bad day for uniQure N.V. ( NASDAQ:QURE ), with the analysts making...
uniQure plunges 27% on Q2 revenue miss, postpones higher dose trial of Huntington’s disease therapy
uniQure (NASDAQ:QURE) is trading 27% down premarket after the company reported lower-than-expected Q2 revenue. The company also announced that it was postponing the trial of a higher dose of AMT-130 to treat Huntington’s disease procedures due to recent suspected unexpected severe adverse reactions. In July, the company reported to the health authorities suspected unexpected severe adverse reactions in three of the 14 patients treated with the higher dose of AMT-130. However, the company said lower-dose procedures were not affected and it continues to expect on anticipated data readouts in 2023. Revenue fell ~100% to $0.5M, and missed estimates by $8.71M, while a loss of -$0.84 beat by $0.11. Cost of contract revenues for the quarter was nil compared to $23.2M, a year earlier. Research and development expenses were $46.2M compared to $32.8M, due to preclinical development of temporal lobe epilepsy (AMT-260) advancing the clinical development of the company’s Huntington’s disease gene therapy program, and recruitment of personnel to support the development of product candidates.
|QURE||US Biotechs||US Market|
Return vs Industry: QURE underperformed the US Biotechs industry which returned -25.6% over the past year.
Return vs Market: QURE underperformed the US Market which returned -23.2% over the past year.
|QURE Average Weekly Movement||11.0%|
|Biotechs Industry Average Movement||11.1%|
|Market Average Movement||6.8%|
|10% most volatile stocks in US Market||15.5%|
|10% least volatile stocks in US Market||2.8%|
Stable Share Price: QURE is more volatile than 75% of US stocks over the past 3 months, typically moving +/- 11% a week.
Volatility Over Time: QURE's weekly volatility (11%) has been stable over the past year, but is still higher than 75% of US stocks.
About the Company
uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington’s disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-210, a product candidate for the treatment of Parkinson’s disease; AMT-260 for temporal lobe epilepsy; AMT-240, a preclinical product candidate for the treatment of autosomal dominant Alzheimer’s disease; and AMT-161 for the treatment of amyotrophic lateral sclerosis. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
uniQure N.V. Fundamentals Summary
|QURE fundamental statistics|
Is QURE overvalued?See Fair Value and valuation analysis
Earnings & Revenue
|QURE income statement (TTM)|
|Cost of Revenue||US$161.52m|
Last Reported Earnings
Jun 30, 2022
Next Earnings Date
|Earnings per share (EPS)||-2.44|
|Net Profit Margin||-184.06%|
How did QURE perform over the long term?See historical performance and comparison