QURE uniQure N.V.

18.03
-0.23  -1%
Previous Close 18.26
Open 18.11
52 Week Low 18.22
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Market Cap $833,242,819
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Enterprise Value $361,799,819
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Upcoming Catalysts

Drug Stage Catalyst Date
Etranacogene dezaparvovec (AMT-061) - (HOPE-B)
Hemophilia B
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
AMT-130
Huntington's disease
Phase 1/2
Phase 1/2
Phase 1/2 treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up. A total of 19 patient procedures have been performed, noted December 16, 2021. Higher-dose cohort is expected to be completed in mid-2022.
AMT-130 (Europe Trial)
Huntington’s disease
Phase 1/2
Phase 1/2
Phase 1/2 screening initiated, noted December 16, 2021.
AMT-191
Fabry disease
Phase 1
Phase 1
IND filing 2023.

Latest News

  1. ~ Treatment was well tolerated with no significant safety issues

    related to AMT-130 in first two treated patients through one year of follow-up ~

    ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery and
    returned to baseline in treated patients ~

    ~ A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial,
    with higher-dose cohort enrollment expected to be completed by mid-2022 ~

    ~ Screening initiated in European open-label Phase I/II study ~

    ~ Conference call today at 8:30 a.m. ET ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with…

    ~ Treatment was well tolerated with no significant safety issues

    related to AMT-130 in first two treated patients through one year of follow-up ~

    ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery and

    returned to baseline in treated patients ~

    ~ A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial,

    with higher-dose cohort enrollment expected to be completed by mid-2022 ~

    ~ Screening initiated in European open-label Phase I/II study ~

    ~ Conference call today at 8:30 a.m. ET ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced initial observations on the first four patients enrolled in the lower-dose cohort of its ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. Two of the four enrolled patients received AMT-130, and two patients experienced an imitation (sham) surgery in this randomized, blinded clinical trial being conducted in the United States.

    "We are pleased with these data that support the tolerability of AMT-130, a potential one-time gene-therapy approach for Huntington's disease," stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. "Neurofilament light chain, a key biomarker of injury in the brain, trended downward and returned to baseline levels in the treated patients, and structural MRI data are consistent with the safety profile of AMT-130. Based on recommendations of the DSMB and steering committee, we will disclose efficacy data including volumetric MRI changes once all patients in the first two cohorts are unblinded. We look forward to evaluating safety, biomarker and functional data on a larger number of patients and over a longer period during the ongoing study."

    "AMT-130 appears to be generally well tolerated both immediately after surgical administration and at one year of follow up in these four patients," stated Jody Corey-Bloom, M.D., PhD, professor of neurosciences at University of California, San Diego and chairperson of the Data Safety Monitoring Board (DSMB) for AMT-130. "We are encouraged by the early data in two treated patients and will continue to provide comprehensive safety oversight of the program as additional patients are enrolled and the trial expands into Europe.  The DSMB will continue to oversee the conduct of this important program, and will evaluate safety, biomarker, imaging, and functional data as the program advances."

    One-Year Observations on AMT-130

    The first four patients were a heterogeneous group representing a spectrum of Stage I-II early manifest disease with 41-44 CAG repeats, baseline Total Functional Capacity scores of 10-13 and Total Motor Scores of 7-23.

    • AMT-130 was generally well tolerated in the treated patients at the lower dose of 6x1012 vector genomes (vg).
    • There have been no serious adverse events (SAEs) to date related to AMT-130 in these patients.
    • NfL increased as expected immediately following the AMT-130 surgical procedure and returned to baseline in the two treated patients. NfL remained relatively constant in the two untreated control patients.
    • Structural magnetic resonance imaging did not reveal any clinically meaningful safety findings in either treated or control patients at one year of follow-up.
    • Measurements of total and mutant HTT protein in the cerebral spinal fluid of the four patients were highly variable and inconclusive.
    • The safety profile of AMT-130 in the low-dose cohort to date supports continued enrollment of patients in the higher-dose cohort of 6x1013 vg. Nineteen patients have been enrolled in the clinical trial to date, including 9 of 16 in the higher-dose cohort.

    A clinical update on the low-dose cohort of ten patients, largely focused on safety, is expected in the second quarter of 2022 after patient unblinding. Full safety and efficacy data from the first two cohorts in the Phase I/II clinical trial are expected in the first half of 2023 after all patients in the higher-dose cohort have achieved one year of follow-up.

    Planned Expansion of Phase I/II Studies of AMT-130

    The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease is exploring the safety, tolerability, and efficacy signals in a planned 26 total patients with early manifest Huntington's disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose cohort; patients will be randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. A total of 16 patients in the clinical trial will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).

    uniQure also plans to initiate a third cohort in the ongoing U.S. Phase I/II clinical trial in the second half of 2022 that will explore the use of alternative stereotactic navigation systems to simplify placement of catheters for infusions of AMT-130. This will be explored in two steps and include up to 18 additional randomized patients who will receive the higher dose of 6x1013 vg.  

    The European, open-label Phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntington's disease across two dose cohorts. Screening has been initiated, and the first procedures in the lower-dose cohort are expected to be complete in early 2022. 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 is uniQure's first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE® platform.

    Investor Conference Call and Webcast Information

    uniQure management will host an investor conference call and webcast today, Thursday, December 16, 2021, at 8:30 a.m. ET. The conference call may be accessed by dialing (833) 962–1471 for domestic callers and +44 0800 0288 438 for international callers. The conference call ID: 2866185. Please specify to the operator that you would like to join the "uniQure Conference Call." If you are joining the conference call, please dial-in 15 minutes before the start time. The webcast of the conference call may also be accessed through the Investors & Newsroom section of the uniQure website. Following the live webcast, a replay of the call will be archived for several weeks.

    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 temporal lobe epilepsy, Alzheimer's, Parkinson's and ALS. 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 be able to evaluate data on a larger number of patients or over a longer period during the ongoing study; whether safety data on the first cohort of ten patients will be available in the second quarter of 2022 or ever; whether full safety and efficacy data from the Phase I/II clinical trial will be available for cohorts 1 and 2 in the first half of 2023 or ever; whether 16 patients or any patients in the clinical trial will receive a higher dose administration of AMT-130; whether we will initiate a third cohort in the ongoing U.S. Phase I/II clinical trial in the second half of 2022 or ever; whether we will enroll 15 patients or any patients in a European, open-label Phase Ib/II study of AMT-130; whether we will complete the first procedures in the low-dose cohort of the European study in early 2022 or ever; whether the European study will establish safety, proof of concept, or the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study or for any purpose; and whether an accelerated registration pathway will be feasible or available at all. Our 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, our and our collaborators' clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, 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 and Quarterly Report on Form 10-Q filed on October 25, 2021. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume 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 Malone
    Direct: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558
    Mobile: 617-680-9452Mobile: 617-306-9137Mobile: 339-223-8541
       


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  2. KING OF PRUSSIA, Pa., Dec. 15, 2021 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.

    The CHMP grants accelerated assessment when a medicinal product is expected to be of major public health interest, particularly pertaining to therapeutic innovation. Accelerated assessment potentially reduces the review timeline from 210 days to 150 days once the MAA is filed and validated.

    "CSL Behring has been dedicated to improving the lives of patients with Hemophilia B for decades and the acceptance of an accelerated regulatory review underscores the high unmet need for a long-term, innovative treatment option for these patients," said Emmanuelle Lecomte Brisset, Head of Global Regulatory Affairs at CSL Behring. "We look forward to submitting our Marketing Authorization Application in the first half of 2022 so that we can make this novel therapy option available to patients as soon as possible."

    Brahm Goldstein, MD, MCR, Vice President, Research and Development, Hematology added, "Accelerated regulatory review supports our commitment to advanced research and providing pioneering treatment options.  Gene therapy, and its associated near-normal blood clotting potential, could be life changing for people living with hemophilia B who are vulnerable to spontaneous bleeding in their muscles, internal organs and joints.  This milestone underscores our excitement about the promise of gene therapy for people living with hemophilia B."

    Along with global partner, uniQure (NASDAQ:QURE), the company recently announced positive topline results from the HOPE-B pivotal trial of etranacogene dezaparvovec.  The study achieved its primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose.  It also achieved a secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy. uniQure led the multi-year clinical development of etranacogene dezaparvovec prior to entering into a Commercialization and License Agreement with CSL Behring in June 2020 for exclusive global rights to etranacogene dezaparvovec.

    About Hemophilia B

    Hemophilia B is a life-threatening degenerative disease. People with the condition are particularly vulnerable to bleeds in their muscles, internal organs, and joints, leading to pain, swelling, and joint damage. Current treatment includes life-long prophylactic infusions of FIX to temporarily replace or supplement low levels of the blood-clotting factor. This can reduce joint bleeding events, prevent life-threatening bleeds, and preserve joint function. However, infusions can be cumbersome, painful and veins can fibrose over time, making ongoing treatment difficult. A person's immune system may also generate inhibitors against the replacement factor, negating its benefit. In addition, many people receiving prophylaxis are forced to plan their lives around the highs and lows of their FIX levels, which rise immediately after an infusion but drop over time -- leaving them especially vulnerable to bleeds and pain in the days before their next infusion. Most troubling, prophylactic FIX replacement therapy sometimes fails to control unobservable micro-bleeds in the joints, meaning that the degeneration can continue despite regular infusions. Missing an infusion may also the increase their likelihood of a life-threatening bleed or even premature death.

    About Gene Therapy in Hemophilia B

    Gene therapy has the potential to make a functional cure possible in hemophilia B.  Gene therapy achieves this with modified non-infectious viruses called "vectors" that can enter certain cells.  Vectors act as delivery trucks, carrying a package of genetic instructions to specific cells. Once delivered, the package acts like a generator that plugs into the cellular machinery, allowing a person to produce their own stable levels of FIX. A certain type of vector, called an adeno-associated virus, or AAV, dissolves after delivering its package. The genetic instructions remain, but never actually become a part of a person's own DNA.

    About Etranacogene Dezaparvovec

    Etranacogene dezaparvovec (also known as CSL222 and EtranaDez, previously known as AMT-061) uses a specific type of AAV, called AAV5, as its delivery vehicle. The AAV5 vector carries the patent-protected Padua gene variant of Factor IX (FIX-Padua), which generates FIX proteins that are 5x-8x more active than normal. Preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in the 95 percent of hemophilia B patients with pre-existing antibodies to AAV vectors, thereby potentially increasing patient eligibility for treatment compared to other AAV gene therapy product candidates. 

    About CSL Behring

    CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

    CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited ((ASX:CSL, OTC:CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people worldwide, and delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/csl-behring-receives-accelerated-chmp-assessment-for-etranacogene-dezaparvovec-for-european-patients-living-with-hemophilia-b-301445080.html

    SOURCE CSL Behring

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  3. ~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~

    ~ Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy ~

    ~ Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months ~

    ~ Manufacturing operations supporting process validation of etranacogene dezaparvovec successfully completed by uniQure ~

    LEXINGTON, Mass. and AMSTERDAM…

    ~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~

    ~ Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy ~

    ~ Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months ~

    ~ Manufacturing operations supporting process validation of etranacogene dezaparvovec successfully completed by uniQure ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands and KING OF PRUSSIA, Pa., Dec. 09, 2021 (GLOBE NEWSWIRE) -- CSL Behring, a global biotherapeutics leader, and uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately severe hemophilia B, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy in the pivotal Phase III HOPE-B gene therapy trial. The study also successfully achieved a secondary endpoint demonstrating statistical superiority in reduction of ABR compared to baseline FIX prophylactic therapy.

    The primary endpoint in the pivotal study was 52-week ABR after achievement of stable FIX expression compared with the six-month lead-in period, considering all bleeds regardless of investigator adjudication as true bleeds. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented likely steady-state FIX transgene expression. Secondary endpoints included assessment of FIX activity and statistical superiority of ABR after dosing.

    "We are very pleased with these top-line results from what is the largest and first pivotal trial of a gene therapy for patients with hemophilia B," stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. "The HOPE-B data not only achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate following 12 months or more of stable FIX expression, but also the secondary endpoint of superiority in reduction of annualized bleeding, while continuing to demonstrate durability and stability in FIX levels and other benefits to this point in the study."

    "On behalf of uniQure, we extend our heartfelt gratitude to all the HOPE-B clinical trial patients and their families, as well as the trial investigators," he continued. "We now look forward to collaborating with CSL Behring on completing the regulatory submissions that we hope will advance etranacogene dezaparvovec one step closer to reaching hemophilia B patients around the world."

    uniQure led the multi-year clinical development of etranacogene dezaparvovec prior to entering into a Commercialization and License Agreement with CSL Behring in June 2020 for exclusive global rights to etranacogene dezaparvovec. Earlier this month, uniQure successfully completed manufacturing operations supporting process validation of etranacogene dezaparvovec.

    Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicine (PRIME) regulatory initiative by the European Medicines Agency. CSL Behring plans to submit regulatory applications for marketing approval of etranacogene dezaparvovec in the United States and European Union in the first half of 2022.

    Top-line Data Results

    A total of 54 patients received a single dose of etranacogene dezaparvovec in the pivotal trial, with 53 patients completing at least 18 months of follow-up.

    ABR for all bleeds after stable FIX expression, assessed at 18 months, was 1.51 compared with the ABR of 4.19 for the lead-in period of at least six months, achieving the primary non-inferiority endpoint and a secondary superiority endpoint (p=0.0002) in the HOPE-B trial. ABR for investigator-adjudicated FIX-treated bleeds was 0.83 compared with lead-in ABR of 3.65 (p<0.0001).

    Data from the HOPE-B pivotal trial showed that patients continued to demonstrate durable, sustained increases in FIX activity at 18 months post-infusion with a mean FIX activity of 36.9 percent of normal as measured by a one-stage APTT-based clotting assay, compared to mean FIX activity of 39.0 percent of normal at six months post-infusion.

    Etranacogene dezaparvovec was generally well-tolerated with over 80% of adverse events considered mild. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65-weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma (HCC) was identified in one patient. Independent molecular characterization and vector integration analysis of the HCC and adjacent tissue supported the conclusion by the investigator and company sponsor that the HCC was unrelated to treatment with etranacogene dezaparvovec. No inhibitors to FIX were reported. 

    "These encouraging results illustrate the potential that gene therapy has to be a long-term treatment option for patients living with hemophilia B and we look forward to sharing more detailed data with the medical community in the near future," stated Brahm Goldstein, MD, MCR, Vice President, Research and Development, Hematology at CSL Behring. "This milestone advances our efforts towards expected regulatory submissions in first half of 2022."

    HOPE-B Pivotal Trial Design

    The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult hemophilia B patients classified as severe or moderately severe (defined as less than or equal to 2% of normal FIX activity) and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline annualized bleeding rate. No prophylactic immunosuppression was provided to patients upon entering the study. After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5. Forty-three percent of patients in the study had pre-existing NAbs to AAV5 up to a maximum observed pre-dosing titer of over 3,200.

    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 temporal lobe epilepsy, Alzheimer's, Parkinson's and ALS. www.uniQure.com

    About CSL Behring

    CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

    CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. Our parent company, CSL Limited ((ASX:CSL, OTC:CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people, and delivers its lifesaving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSLBehring.

    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 CSL Behring will submit a BLA for etranacogene dezaparvovec in the first half of 2022, whether etranacogene dezaparvovec will reach hemophilia B patients around the world, and whether etranacogene dezaparvovec has the potential to provide well-tolerated, long-term clinical benefits, and whether AAV5-based gene therapies can provide clinical benefit to patients with pre-existing neutralizing antibodies. Our 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, our and our collaborators' clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, 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 2, 2020 and Quarterly Report on Form 10-Q filed on October 25, 2021. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume 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. Cantor

    Direct: 339-970-7536

    Mobile: 617-680-9452

    Chiara Russo

    Direct: 617-306-9137

    Mobile: 617-306-9137



    Tom Malone

    Direct: 339-970-7558

    Mobile: 339-223-8541



    CSL Behring Contact:

    Jennifer Purdue

    Mobile:   610-306-9355



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  4. ~ No Significant Safety Concerns Observed in First Four Patients Enrolled in Higher-dose Cohort ~

    ~ Enrollment Expected to be Completed by Mid-2022 ~

    LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 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 positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment.

    A…

    ~ No Significant Safety Concerns Observed in First Four Patients Enrolled in Higher-dose Cohort ~

    ~ Enrollment Expected to be Completed by Mid-2022 ~

    LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 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 positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment.

    A total of 14 blinded administration procedures have been completed as of August 2021. In the study to date, eight patients have been treated with AMT-130, and six patients have received imitation surgery. Full enrollment of the second, higher-dose cohort is expected to be completed in mid-2022.

    "The AMT-130 program continues to advance at a steady pace, and with this positive recommendation from the DSMB, we are eager to enroll the final 12 patients in the higher-dose cohort," said David Cooper, M.D., vice president of clinical development at uniQure. "We also look forward to expanding our efforts with the initiation of a separate open-label study of AMT-130 in Europe and to sharing preliminary imaging and biomarker data from the initial four patients in the U.S. clinical trial before the end of the year."

    About the Phase I/II Clinical Program of AMT-130

    The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease is exploring the safety, tolerability, and efficacy signals in a planned 26 total patients with early manifest Huntington's disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose cohort; patients will be randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. A total of 16 patients in the clinical trial will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details are available on www.clinicaltrials.gov (NCT04120493).

    The European, open-label Phase Ib/II study of AMT-130 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 is uniQure's first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE™ platform.

    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 commence the enrollment of the final 12 patients in the second dose cohort in the fourth quarter of 2021 or ever, whether we complete enrollment of the U.S. Phase I/II clinical trial of AMT-130 by the middle of 2022 or ever, whether we initiate dosing in our European open-label Phase Ib/II clinical trial in the second half of 2021 or ever, whether we are able to enroll the currently planned number of patients in the U.S. Phase I/II or the European open-label Phase Ib/II clinical trials, , and whether we share initial imaging and biomarker data towards the end of the year or ever.  Our 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 clinical development activities, clinical results, collaboration arrangements, regulatory oversight, manufacturing activities, 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 2, 2020 and Quarterly Report on Form 10-Q filed on October 25, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume 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 Malone
    Direct: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558
    Mobile:  617-680-9452Mobile: 617-306-9137Mobile: 339-223-8541



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  5. LEXINGTON, Mass. and AMSTERDAM, Nov. 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 investor and scientific conferences:

    • The 28th Annual Meeting of the Huntington Study Group (Virtual), November 4 - 6, 2021

      • David Cooper, M.D., vice president of clinical research CNS, will present an overview of the ongoing clinical trials of AMT-130 in Huntington's Disease, HD-GeneTRX-1 and -2, and participate in a Q&A session on Friday, November 5th at 3:15 p.m. ET.

      • An encore poster presentation entitled, "Demographics and healthcare resource utilization (HRU) in US patients with Huntington's…

    LEXINGTON, Mass. and AMSTERDAM, Nov. 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 investor and scientific conferences:

    • The 28th Annual Meeting of the Huntington Study Group (Virtual), November 4 - 6, 2021



      • David Cooper, M.D., vice president of clinical research CNS, will present an overview of the ongoing clinical trials of AMT-130 in Huntington's Disease, HD-GeneTRX-1 and -2, and participate in a Q&A session on Friday, November 5th at 3:15 p.m. ET.



      • An encore poster presentation entitled, "Demographics and healthcare resource utilization (HRU) in US patients with Huntington's Disease: data from the Huntington's Disease Burden of Illness (HDBOI) study" will also be available to view at the Poster Pavilion to registered participants.



    • Virtual Neuroscience 2021 – 50th Annual Meeting, November 8 - 11, 2021



      • Corlieve academic collaborator, Valeria Crepel, Ph.D., will present an encore presentation "CL002, An AAV9 vector expressing engineered miRNA targeting knockdown of GluK2-containing kainite receptors as a novel gene therapy approach for treating intractable temporal lobe epilepsy" on Thursday, November 11th at 10:00 a.m. ET, Session P152: Antiepileptic Therapies IV.



    • Stifel 2021 Virtual Healthcare Conference, November 15 - 17, 2021



      • Members of uniQure's management team, including Matt Kapusta, chief executive officer, will participate in virtual one-on-one investor meetings on Wednesday, November 17th.



      • A fireside chat with Mr. Kapusta will take place the same day from 10:40 to 11:10 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.

    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 severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in hemophilia B and Huntington's disease and preclinical candidates in Fabry disease, spinocerebellar ataxia Type 3, temporal lobe epilepsy, Alzheimer's, Parkinson's, and ALS. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS: FOR MEDIA:
       
    Maria E. CantorChiara RussoTom Malone
    Direct: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558


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