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uniQure N.V. (QURE)

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Neutralpattern detected
Previous Close33.14
Bid33.00 x 1100
Ask35.90 x 1400
Day's Range32.72 - 34.93
52 Week Range28.58 - 71.45
Avg. Volume484,353
Market Cap1.569B
Beta (5Y Monthly)1.31
PE Ratio (TTM)N/A
EPS (TTM)-2.81
Earnings DateMar 01, 2021
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est67.50
  • uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington’s Disease Showing Safety of Administration in NHPs and Widespread Long-Term HTT-Lowering in the Brain

    uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington’s Disease Showing Safety of Administration in NHPs and Widespread Long-Term HTT-Lowering in the Brain

    Publication in Science Translational Medicine shows local striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering in Huntington’s disease minipigsLEXINGTON, Mass. and AMSTERDAM, April 08, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that three manuscripts on preclinical data from its gene therapy candidate AMT-130 in Huntington’s disease have been accepted for publication, in the journals Science Translational Medicine, Brain Science, and Brain Communications. The publications show the safety and efficacy of AMT-130 in the deep brain structures of a large animal model and outline a promising novel efficacy biomarker for AMT-130. “Taken together, these publications demonstrate widespread biodistribution and strong, durable efficiency of AMT-130 in disease-relevant regions in a large brain,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “The data provide further support for the potential therapeutic value of AMT-130, and we remain enthusiastic about our Phase I/II clinical trial of AMT-130 in patients with Huntington’s disease.” Widespread and Sustained Target Engagement in Huntington Disease Minipigs The paper published this week in Science Translational Medicine examines the translatability and long-term durability of AMT-130 in transgenic Huntington’s disease minipigs, which were used to assess the biodistribution and target engagement in a larger brain. The minipig model is the largest diseased animal model available, generally weighing up to 300 pounds. AMT-130 was administered by MRI-guided convention-enhanced delivery (CED) at a single dose, bilaterally in the caudate and putamen. Vector DNA distribution and transgene expression in minipig brains demonstrated extensive brain coverage comparable at the interim sacrifice timepoints of 6- and 12-months post administration, leading to significant lowering of mutant huntingtin (mHTT) protein in the brain. At 12 months, the most pronounced mHTT protein lowering was observed in the putamen (85%), caudate (80%) and amygdala (78%), followed by thalamus (56%) and cerebral cortex (44%). The publication, “Widespread and Sustained Target Engagement in Huntington Disease Minipigs upon Intrastriatal MicroRNA-based Gene Therapy,” is available online in the journal Science Translational Medicine (DOI: 10.1126/scitranslmed.abb8920). Well-tolerated in non-human primates and rats In addition, a GLP toxicity study of AMT-130 in non-human primates and rats was published in January 2021 in the journal Brain Science. The study demonstrated an excellent safety profile and biodistribution after MRI-guided CED of AMT-130 in the treated animals. One-time bilateral administration in the caudate and putamen resulted in widespread vector DNA and miHTT transgene distribution in the brain, particularly in areas associated with HD pathology. Intrastriatal administration of AAV5-miHTT was well tolerated, with no clinically relevant changes in either species. The publication, “Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology,” is available online in the journal Brain Science (DOI: 10.3390/brainsci11020129). Monitoring Durability of MicroRNA-based Therapies A third manuscript was published last week in the journal Brain Communications, examining the potential use of measuring therapeutic HTT microRNA (miHTT) in extracellular vesicles in CSF as sources to monitor the expression and durability of gene therapies in the brain. After AAV treatment in non-human primates, the secretion of mature engineered microRNA molecules was confirmed, with extracellular microRNA levels correlating with viral dose and cellular microRNA expression in neurons. In investigating the detection of engineered microRNAs over time in the CSF of non-human primates after a single intrastriatal injection of AAV5-miHTT, quantifiable engineered microRNA levels enriched in extracellular vesicles were detected in the CSF up to two years after brain infusion. The results confirm the long-term expression (up to two years) of AAV5-delivered microRNAs in non-human primates and provide further support for the potential use of extracellular vesicle-associated microRNAs as novel biomarkers in ongoing clinical trials of gene therapies for neurodegenerative diseases, including AMT-130. The publication, “Secreted therapeutics: Monitoring durability of microRNA-based gene therapies in the central nervous system,” is available online in the journal Brain Communications (DOI: 10.1093/braincomms/fcab054). About AMT-130 AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that lowers Huntingtin protein in Huntington’s disease patients. AMT-130 is uniQure’s first clinical program incorporating its proprietary miQURE™ platform. miQURE is designed to degrade disease-causing genes without off-target toxicity and induce silencing of the entire target organ through secondary exosome-mediated delivery. About Huntington’s Disease Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington’s disease, there are no currently approved therapies to delay the onset or to slow the disease’s progression. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to whether we will advance our Phase I/II gene therapy clinical trial of AMT-130 in Huntington’s disease. uniQure’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, the regulatory approval of that transaction, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s periodic securities filings, including its Annual Report on Form 10-K filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and uniQure assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Maria E. CantorChiara RussoTom MaloneDirect: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558Mobile: 617-680-9452Mobile: 617-306-9137Mobile:339-223-8541m.cantor@uniQure.comc.russo@uniQure.comt.malone@uniQure.com

  • Is QURE Stock A Buy or Sell?
    Insider Monkey

    Is QURE Stock A Buy or Sell?

    At Insider Monkey, we pore over the filings of nearly 887 top investment firms every quarter, a process we have now completed for the latest reporting period. The data we’ve gathered as a result gives us access to a wealth of collective knowledge based on these firms’ portfolio holdings as of December 31st. In this […]

  • These 2 Stocks Look Poised for a Turnaround, Say Analysts

    These 2 Stocks Look Poised for a Turnaround, Say Analysts

    Stocks can fall for any number of reasons, but successful investors know to look under the hood before bailing out. Some stocks will sputter and stall, only to restart and accelerate later. If the underlying business is sound, even a sharp drop in share value may be an incident rather than a lasting hit. But before you buy a beaten-down stock, make sure to do your homework first, and find out what’s behind the stock’s share price drop. With this in mind, we set out on our own search for compelling investment opportunities trading at a discount. Using TipRanks database, we were able to find 2 stocks that are down from their recent peaks, while some Wall Street analysts believe a turnaround is on the horizon. Let's take a closer look. uniQure N.V. (QURE) We’ll start in the biotech sector, with uniQure, a gene therapy company, researching single-dose, potentially curative, treatments for patients with severe genetic diseases. uniQure’s two most advanced programs are treatments for hemophilia and Huntington’s disease, which are in Phase 3 and Phase 1/2 trials, respectively. The treatments are adeno-associated virus-based gene therapies (AAV), developed on a proprietary platform. The FDA has placed a hold on the company’s hemophilia B studies, following the mid-December safety report that identified a serious adverse event during the HOPE-B Phase 3 clinical trial of AMT-061. One patient was diagnosed with HCC (hepatocellular carcinoma, the most common liver cancer) during the study. That patient had several HCC risk factors, including a long history of hepatitis C and B, smoking, and non-alcoholic fatty liver disease. uniQure has since screened over 100 patients in all of its hemophilia B programs, including all 54 patients in the HOPE-B study for liver complications, with negative results; the company and the FDA are now evaluating this event. Preliminary indications are, the adverse event was not related to this specific gene therapy. uniQure’s other main pipeline project, AMT-130, is a potential treatment for Huntington’s disease, a severe, genetically related mental disorder. AMT-130 is undergoing Phase 1/2 clinical trials, with the second dose cohort due to start enrollment in 3Q21. A second clinical study of AMT-130 is scheduled to start in Europe in 2H21. Through all of this, QURE shares are down 26% since the FDA hold on AMT-061. However, analyst Difei Yang, of Mizuho, takes note of the investigation of the HCC event in the HOPE-B trial as a boost for investors. “[We] believe these analyses suggest that multiple risk factors independent of AAV vector integration likely contributed to the development of HCC. The company submitted these analyses to the FDA, and an update on the clinical hold status could be expected as early as in 2Q21,” Yang noted. Yang sees the stock’s current valuation and positive prospects as reason for optimism. “[We] see … a favorable risk/reward in the shares given: 1) a positive safety update on the lead HemB program which we see as a de-risking event for the company, and 2) initial efficacy data from the Huntington's program expected in late 2021/early 2022. We expect this data update to be a closely-watched catalyst following the recent failures of competing ASO programs,” Yang summed up. To this end, Yang gives QURE a $52 price target to go long with her upgraded stance, indicating a 45% upside potential for the year ahead. (To watch Yang’s track record, click here) The stumbling blocks that have hit QURE in recent months are a type familiar to followers of the biotech industry, and so the analysts have not deserted this stock – QURE shares have a unanimous Strong Buy analyst consensus rating, based on 5 recent positive reviews. The shares are priced at $35.78, with an average price target of $67.40 suggesting an 88% one-year upside. (See QURE stock analysis on TipRanks) Ontrak (OTRK) Ontrack is another stock related to the healthcare industry – but on the customer facing end rather than biotech research. Ontrak is in the telehealth niche, using an AI-powered platform to track and monitor patients with chronic disease conditions, recommending behavioral modifications to improve health outcomes. The company combines predictive analysis and human engagement in its program, and has delivered durable cost savings for more than half of its enrolled members. Shares in in Ontrak had been rising through the beginning of February, but started slipping in the middle of that month. In March, several headline events began impacting the stock, and shares dropped 63% from their peak. The first hit came when management, in the Q4 earnings pre-announcement, revealed that the company’s largest customer, Aetna, would be terminating its contract in June of this year. The news pushed the 2021 guidance down, making forward projections much worse than the Street had anticipated. The actual Q4 results, however, showed substantial year-over-year increases in revenue – Q4’s top line, at $29.3 million, was up 149% yoy. The quarterly results were followed on March 16 by the announcement that Jonathan Mayhew, Executive VP of CVS, would step up as CEO of Ontrak starting April 12. Mayhew is a former executive from Aetna, and there is hope that his connections will help Ontrak to regain its largest contract. The announcement of Mayhew’s accession to the top spot at Ontrak prompted 5-star Canaccord analyst Richard Close to upgrade his rating on OTRK shares from Hold to Buy. “[We] view this [the Mayhew hire] as a major positive for Ontrak and validation of the company's service offering. The addition of Mayhew could enable Ontrak to regain its Aetna Behavioral Health contract for substance use disorder (SUD) and potentially also add anxiety and depression conditions, while also opening up the potential for other populations at Aetna and CVS Health," Close opined. The analyst added, "We do note in 2021 the financial optics of losing Aetna still remain, but longer term this hire is very encouraging for Ontrak's growth, and we believe it will provide the opportunity for multiple expansion from the stock's historically discounted valuation.” Close’s comments back his upgraded stance, and his $46 price target suggests room for ~33% one-year upside. (To watch Close’s track record, click here) The rest of the Street leans to the bullish side. OTRK's Moderate Buy consensus rating is based on 3 Buys and 2 holds. There’s possible upside of 36%, should the target of $47 be met in the year ahead. (See OTRK stock analysis on TipRanks) To find good ideas for stocks trading at attractive valuations, visit TipRanks’ Best Stocks to Buy, a newly launched tool that unites all of TipRanks’ equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.