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uniQure N.V. (QURE)

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Previous Close35.47
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  • 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
    GlobeNewswire

    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

  • uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
    GlobeNewswire

    uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

    ~ Enrollment of second dose cohort expected to begin in 3Q 2021 ~ ~ Company announces plans to initiate a second clinical study of AMT-130 in Europe in the second half of 2021 ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 05, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of patient enrollment in the first dose cohort of a randomized, double-blinded, Phase I/II clinical trial of AMT-130 for the treatment of early stage Huntington’s disease. The Company also announced plans to begin an open-label clinical trial of AMT-130 in Europe later this year. “This is an important milestone in our ongoing clinical development of AMT-130,” stated David Cooper, M.D., vice president, clinical development at uniQure. “Nine U.S. study sites are now active to support enrollment in the next cohort, which is expected to start after the Data Safety Monitoring Board’s review in the middle of the year. Completing enrollment of the first, 10-patient cohort ahead of schedule highlights the high level of interest among the Huntington’s disease patient and clinical community, and the collaboration between our participating HD Centers of Excellence and the expert neurosurgical sites performing the MRI-guided procedures. We also look forward to initiating a new clinical study of AMT-130 in Europe later this year. It is estimated that there could be as many as 75,000 Europeans affected by Huntington’s disease.” The ongoing Phase I/II clinical trial of AMT-130 is a randomized, sham controlled, double-blinded study to explore the safety, tolerability, and proof of concept of AMT-130 in patients with early manifest Huntington’s disease. The study, which includes two dose cohorts, will randomize a total of 26 patients to either treatment with AMT-130 or an imitation surgical procedure. The first dose cohort includes 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery. The second dose cohort is planned to include 16 patients, of which 10 patients will receive treatment with AMT-130 and six patients will receive imitation surgery. The trial consists of a blinded 12-month study period followed by unblinded long-term follow-up for 5 years after administration of AMT-130. Patients receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The planned Phase Ib/II study of AMT-130 will be conducted in Europe and is expected to begin enrolling patients in the second half of 2021. This open-label study will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts. Together with the U.S. study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible. 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, the enrollment of patients in, or Data Safety Monitoring Board review of, our Phase I/II gene therapy clinical trial of AMT-130 in Huntington’s disease, including whether we will be able to fully enroll the second dose cohort as currently planned, and whether we will initiate our P1b/II clinical study of AMT-130 in Europe later this year or ever. 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

  • uniQure to Participate in Multiple Upcoming Industry Conferences in April
    GlobeNewswire

    uniQure to Participate in Multiple Upcoming Industry Conferences in April

    LEXINGTON, Mass. and AMSTERDAM, April 01, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in the following upcoming virtual investor and scientific conferences: Virtual Guggenheim Healthcare Talks│2021 Genomic Medicines & Rare Disease, April 1, 2021 Members of uniQure’s management team will participate in virtual one-on-one investor meetings throughout the day on Thursday, April 1.Matt Kapusta, chief executive officer, will participate on a panel discussion entitled, “The Low-Hanging Fruit in Gene Therapy Might Be Hard to Reach – Challenges for Hemophilia A and B” on Thursday, April 1 from 10:00 to 10:50 a.m. ET. The live webcast of the fireside chat can be accessed through the link displayed in the Investors & Newsroom section of the uniQure website. Ricardo Dolmetsch, Ph.D., president of research and development, will participate in a panel discussion entitled, “An Idea Whose Time has Come – Disease Modifying Therapies for Huntington’s Disease” later the same day from 3:00 to 3:50 p.m. ET. Wells Fargo Corporate Access Days, April 6-8, 2021 Members of uniQure’s management team will participate in virtual one-on-one investor meetings throughout the day on Tuesday, April 6. Professional Patient Advocates in Life Sciences, April 13, 2021 Daniel Leonard, senior director of global patient advocacy, will participate on a virtual panel discussion entitled, “Building an Internal Patient Advocacy & HR Relationship Series – Part 1: Journey to Patient Advocacy” starting at 2:00 p.m. ET on Tuesday, April 13. 20th Annual Needham Virtual Healthcare Conference, April 12 - 15, 2021 Members of uniQure’s management team will participate in virtual one-on-one investor meetings throughout the day on Thursday, April 15.A company presentation with Matt Kapusta will take place the same day from 3:45 to 4:25 p.m. ET. The live webcast of the fireside chat can be accessed through the link displayed in the Investors & Newsroom section of the uniQure website. Chardan 5th Annual Genetic Medicines Manufacturing Summit, April 26 - 27, 2021 Members of uniQure’s management team will participate in virtual one-on-one investor meetings on Tuesday, April 27.A fireside chat with Matt Kapusta will take place the same day from 1:45 to 2:25 p.m. ET. The live webcast of the fireside chat can be accessed through the link displayed in the Investors & Newsroom section of the uniQure website. Kempen Life Sciences Conference – 2021 Thematic Virtual Series “Cell, Gene & RNA based companies,” April 28, 2021 Members of uniQure’s management team will participate in virtual one-on-one investor meetings on Wednesday, April 28. CHDI’s 16th Annual Huntington’s Disease Therapeutics Conference, April 27-29, 2021 David Cooper, M.D., vice president of clinical research CNS, will present in the clinical stage interventional programs session on Thursday, April 29 between 10:00 a.m. and 12:30 p.m. ET. His presentation is entitled, “Updates on HD-GeneTRX-1: A Phase 1-2 Clinical Trial of CNS-Administered Gene Therapy (AMT-130) for Early-Stage HD.” Following the pre-recorded presentation, Dr. Cooper will be available for live Q&A. Astrid Valles-Sanchez, Ph.D., senior scientist at uniQure, will have a poster presentation entitled, “Lowering the Pathogenic Exon1 HTT Fragment by AAV5-miHTT Gene Therapy,” during the conference dates. World Orphan Drug Congress, April 28, 2021 Nick Li, Ph.D., M.B.A., senior director, global/US market access lead, will participate on a panel entitled “Pricing & Reimbursement for Rare Diseases in the United States” starting at 3:00 p.m. ET on Wednesday, April 28. 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 Contacts: FOR INVESTORS: FOR MEDIA: Maria E. CantorChiara RussoTom MaloneDirect: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558m.cantor@uniQure.comc.russo@uniQure.comt.malone@uniQure.com