QURE uniQure N.V.

31.71
-0.73  -2%
Previous Close 32.44
Open 31.66
52 Week Low 28.58
52 Week High 71.45
Market Cap $1,459,218,762
Shares 46,017,621
Float 45,738,505
Enterprise Value $1,337,409,625
Volume 249,111
Av. Daily Volume 467,976
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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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AMT-130
Huntington's disease
Phase 1/2
Phase 1/2
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Latest News

  1. ~ Data Show No Clinically Significant Correlation Between Pre-Existing NAbs and FIX Activity ~

    ~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients --

    ~ Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting ~

    LEXINGTON, Mass. and AMSTERDAM, May 13, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, yesterday presented 26-week clinical data from the pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between…

    ~ Data Show No Clinically Significant Correlation Between Pre-Existing NAbs and FIX Activity ~

    ~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients --

    ~ Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting ~

    LEXINGTON, Mass. and AMSTERDAM, May 13, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, yesterday presented 26-week clinical data from the pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between AAV5 NAbs and the tolerability of the therapy.

    The data were featured on Wednesday, May 12, 2021 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in an oral presentation by Michael Recht, M.D., Ph.D., M.B.A., professor of pediatrics, division of hematology and oncology at the Oregon Health & Science University School of Medicine. These data were previously presented at a medical meeting late last year.

    "The ASGCT presentation highlights these initial HOPE-B data that demonstrate the successful treatment with an AAV5 gene therapy of patients with pre-existing NAbs," stated Ricardo Dolmetsch, Ph.D., president of Research and Development at uniQure. "Patients in the trial who may not have been eligible for other gene therapies because of pre-existing neutralizing antibodies have achieved similar results with etranacogene dezaparvovec compared to those who did not have pre-existing NAbs. We believe this distinguishes etranacogene dezaparvovec as the only hemophilia gene therapy shown in a clinical trial to have the potential to treat nearly all patients, regardless of NAb levels in the generally prevalent range."

    Patients in the Phase III HOPE-B clinical study were initially enrolled into a prospective, observational lead-in period of at least six months during which bleeding events and FIX replacement therapy usage were monitored. All patients required prophylactic routine FIX replacement prior to entering the clinical trial, and patients were not excluded from the trial based on pre-existing NAbs to AAV5.

    Fifty-four participants were dosed and completed 26 weeks of follow-up, 31 of whom (57.4%) had no pre-existing NAbs to AAV5. Of the 23 participants (42.6%) with pre-existing NAbs, the median titer was 56.9, with a distribution representative of the general population.

    No clinically significant correlation of pre-existing NAbs with FIX activity was observed up to a titer of 678, a range expected to include more than 95 percent of the general population. Mean FIX activity at 26 weeks was 32.7 percent in participants with NAbs versus 41.3 percent in those without. A single participant with a NAb titer of 3,212 did not respond and remained on prophylaxis; it is expected that less than 1 percent of the general population have a pre-existing NAb titer of more than 3,000. All other participants, both with and without NAbs, discontinued prophylaxis and remained prophylaxis-free at 26 weeks.

    The data presentation from ASGCT can be found on the Investors & Newsroom section of uniQure's corporate website.

    About Etranacogene Dezaparvovec

    Etranacogene dezaparvovec (AMT-061) is an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure and CSL Behring have entered into a commercialization and license agreement providing CSL Behring exclusive global commercialization rights to etranacogene dezaparvovec. The collaboration combines uniQure's differentiated gene therapy candidate in hemophilia B and CSL Behring's strong global reach and commercial infrastructure in hematology in an effort to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

    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 etranacogene dezaparvovec or AAV5-based gene therapies generally can provide clinical benefit to patients with pre-existing neutralizing antibodies.  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, 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 Quarterly Report on Form 10-Q filed on May 10, 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. ~ 52-week follow-up data from HOPE-B pivotal study expected to be presented later this quarter ~

    ~ Announced closing of the global commercialization and license agreement with CSL Behring for hemophilia B gene therapy ~

    ~ Completed enrollment of first dose cohort of U.S. Phase I/II clinical trial of AMT-130 for Huntington's disease, with initiation of second dose cohort and European Phase Ib/II expected in second half of 2021 ~

    ~ Pierre Caloz to be appointed Chief Operating Officer ~

    ~ Research & Development Day to be held Tuesday, June 22, 2021 ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 10, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with…

    ~ 52-week follow-up data from HOPE-B pivotal study expected to be presented later this quarter ~

    ~ Announced closing of the global commercialization and license agreement with CSL Behring for hemophilia B gene therapy ~

    ~ Completed enrollment of first dose cohort of U.S. Phase I/II clinical trial of AMT-130 for Huntington's disease, with initiation of second dose cohort and European Phase Ib/II expected in second half of 2021 ~

    ~ Pierre Caloz to be appointed Chief Operating Officer ~

    ~ Research & Development Day to be held Tuesday, June 22, 2021 ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 10, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2021 and highlighted recent progress across its business.

    "We have made great strides with our clinical trial execution across both our hemophilia and Huntington's disease programs, with all patients in the HOPE-B pivotal trial completing their 52-week follow-up visits and all ten patients in the first dose cohort of our U.S. clinical trial in Huntington's disease completing their procedures," stated Matt Kapusta, chief executive officer at uniQure. "Looking at important near-terms catalysts, we look forward to announcing top-line, 52-week follow-up data from the HOPE-B pivotal trial, initiating our collaboration with CSL Behring and holding an R&D Day to provide updates on the research pipeline, including new product candidates," he added. "In the second half of the year, we expect to begin patient enrollment in the second cohort of AMT-130 and to initiate our open-label European study of AMT-130. Later this year, we expect to submit the BLA for etranacogene dezaparvovec and announce early data on the first four patients in the Phase I/II study of AMT-130."

    Recent Company Progress

    • Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B



      • The Company expects to announce top-line data during the second quarter of 2021 related to additional clinical trial endpoints in the HOPE-B pivotal trial of etranacogene dezaparvovec in severe to moderately severe hemophilia B patients, including 52-week annualized bleeding rates and FIX activity.



      • The Company announced the closing of the transaction with CSL Behring for its hemophilia B gene therapy following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 on May 5, 2021.   In accordance with the agreement, the upfront payment of $450 million was paid to uniQure on May 7, 2021.



      • In April 2021, the FDA removed the clinical hold on the Company's hemophilia B gene therapy program regarding a diagnosis of hepatocellular carcinoma (HCC), a form of liver cancer, in one patient in the HOPE-B trial. This followed a review of data from a comprehensive investigation that found it was highly unlikely etranacogene dezaparvovec was the cause of the HCC in this patient.

    • Advancing the clinical development of AMT-130 for the treatment of Huntington's disease



      • The ten-patient, first dose cohort of the Phase I/II clinical trial of AMT-130 was fully enrolled in the second quarter of 2021.   The second, higher-dose cohort is expected to begin enrollment in the third quarter of 2021 after a review by the independent Data Safety Monitoring Board (DSMB). Early imaging and biomarker data from the first four enrolled patients in this first-in-human AAV-gene therapy trial in Huntington's disease are anticipated by year-end 2021.



      • In February 2021, the DSMB recommended proceeding with patient enrollment in the Phase I/II clinical trial of AMT-130 after finding no significant safety concerns based on its review of the six-month data from the first two enrolled patients and the 90-day data from the next two enrolled patients.



      • The Company also announced plans to initiate an open label, Phase Ib/II clinical study of AMT-130 in Europe with enrollment beginning in the second half of 2021.

    • Expanding the Pipeline and Strengthening the Platform



      • In February 2021, the Company began an expansion of its Amsterdam site to build additional laboratories to support growing research and development activities, as well as a cleanroom designed for manufacturing cGMP materials at a 500-liter scale.



      • The Company plans to host a virtual Research & Development Day on June 22, 2021 to highlight new investments in its expanding gene therapy pipeline focusing on CNS and rare, liver-directed disorders, as well as new advancements in platform technology and manufacturing.

    • Expansion of the leadership team



      • Pierre Caloz will join the leadership team as Chief Operating Officer, effective May 17, 2021, overseeing all manufacturing operations, global CMC development and innovation, supply chain and facilities. Mr. Caloz joins the Company with nearly 20 years of global operations experience in the biopharma industry, including CSL Behring, Merck-Serono, Abgenix and Amgen. Most recently, he served as Senior Vice President and General Manager of European Union and Asia Pacific Operations at CSL Behring where he managed a team of more than 4,000 FTEs and an operating budget of more than EUR 1 billion.  Mr. Caloz earned a BSc degree from the University of Geneva, a MSc degree from Swiss Federal Institute of Technology, and an EMBA from the Ashridge Business School.



    • Strong cash position to advance the Company's programs



      • As of March 31, 2021, the Company's cash position was $260.8 million, which did not include any financial impact associated with the commercialization and license agreement with CSL Behring. Including the receipt from CSL Behring of the $450 million upfront payment, the Company expects cash and cash equivalents will be sufficient to fund operations into the second half of 2024.

    Upcoming Investor Events (each to be conducted virtually)

    • 2021 RBC Capital Markets Global Healthcare Conference, May 18-20, 2021
    • Jefferies Virtual Healthcare Conference, June 1-4, 2021
    • 42nd Annual Goldman Sachs Global Healthcare Conference, June 8-10, 2021
    • Virtual Raymond James Human Health Innovations Conference, June 21-23, 2021
    • uniQure Research & Development Day, June 22, 2021

    Financial Highlights

    Cash Position: As of March 31, 2021, the Company held cash and cash equivalents of $260.8 million, compared to $244.9 million as of December 31, 2020. In January 2021, the Company and Hercules entered into an amended debt facility agreement increasing the aggregate principle to up to $135.0 million, of which the Company drew down an additional $35.0 million for a total of $70.0 million outstanding under the facility as of March 31, 2021. In March 2021, the Company entered into an Open Market Sale Agreement with SVB Leerink LLC providing for the sale of up to $200.0 million of its ordinary shares from time to time in ‘at-the-market' offerings under a shelf registration statement. Through March 31, 2021, the Company sold 859,885 ordinary shares pursuant to this agreement for gross proceeds of approximately $28.7 million.

    Revenues: Revenue for the three months ended March 31, 2021 was $0.5 million, compared to $0.1 million during the same period in 2020. 

    R&D Expenses: Research and development expenses were $32.7 million for the three months ended March 31, 2021, compared to $26.0 million during the same period in 2020. The change was primarily related to increased activities associated with our ongoing clinical studies of Huntington's disease, increased activity in programs in preclinical development and the recruitment of personnel to support the development of our product candidates.

    SG&A Expenses: Selling, general and administrative expenses were $12.4 million for the three months ended March 31, 2021, compared to $9.1 million during the same period in 2020. The change was primarily related to increases in personnel and consulting expenses to support our growth, increases in professional fees, and increased share-based compensation.

    Other non-operating items, net: Other income, net was $3.1 million for the three months ended March 31, 2021, compared to other income, net of $6.5 million during the same period in 2020. The decrease is primarily due to the termination of the Bristol-Myers Squibb warrants in December 2020, resulting in no recognition of changes in fair value of the derivative financial liability in the current period compared to a recognized in the same period in 2020.

    Net Loss: The net loss for the three months ended March 31, 2021 was $41.6 million, or $0.91 loss per share, compared to $28.0 million, or $0.63 loss per share during the same period in 2020.

    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, uniQure's expectations its plans to announce data from the HOPE-B pivotal trial, patient enrollment in the second cohort of AMT-130, the initiation of an open-label European study of AMT-130, the announcement of data on the first four patients in the Phase I/II study of AMT-130, the submission of the regulatory filing for marketing approval of etranacogene dezaparvovec, the employment of a Chief Operating Officer, the potential financial compensation we may be paid pursuant to our agreement with CSL Behring, the planned R&D Day or other updates on the research pipeline, and our plans to advance or expand our pipeline, accelerate research, identify business development opportunities, invest in technology, or scale our manufacturing capabilities. 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, 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. 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

     
         



    uniQure N.V.

    UNAUDITED CONSOLIDATED BALANCE SHEETS

        
     March 31, December 31,
    2021 2020
      
     (in thousands, except share and per share amounts)
    Current assets   
    Cash and cash equivalents$260,813 $244,932
    Accounts receivables 5,445  6,618
    Prepaid expenses 9,186  4,337
    Other current assets 6,886  3,024
    Total current assets 282,330  258,911
    Non-current assets   
    Property, plant and equipment, net 33,862  32,328
    Operating lease right-of-use assets 25,313  26,086
    Intangible assets, net 2,908  3,361
    Goodwill 518  542
    Restricted cash 2,716  2,748
    Deferred tax asset 16,206  16,419
    Total non-current assets  81,523  81,484
    Total assets$ 363,853 $ 340,395
    Current liabilities   
    Accounts payable$5,749 $3,772
    Accrued expenses and other current liabilities 20,896  18,038
    Current portion of operating lease liabilities 5,457  5,524
    Total current liabilities 32,102  27,334
    Non-current liabilities   
    Long-term debt 70,467  35,617
    Operating lease liabilities, net of current portion 29,487  30,403
    Other non-current liabilities 3,107  3,136
    Total non-current liabilities 103,061  69,156
    Total liabilities$ 135,163 $ 96,490
    Shareholders' equity   
    Total shareholders' equity 228,690  243,905
    Total liabilities and shareholders' equity$ 363,853 $ 340,395
        



    uniQure N.V.

    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

        
     Three months ended March 31,
     2021 2020
      
     (in thousands, except share and per share amounts)
    Total revenues$ 454  $ 104 
    Operating expenses:   
    Research and development expenses (32,656)  (26,013)
    Selling, general and administrative expenses (12,375)  (9,072)
    Total operating expenses (45,031)  (35,085)
    Other income 352   857 
    Other expense (233)  (339)
    Loss from operations (44,458)  (34,463)
    Non-operating items, net 3,115   6,464 
    Loss before income tax expense  (41,343)  (27,999)
    Income tax expense (213)  - 
    Net loss$ (41,556) $ (27,999)
        
    Basic and diluted net loss per ordinary share$(0.91) $(0.63)
    Weighted average shares used in computing basic and diluted net loss per ordinary share 45,468,485   44,279,456 
        

     



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  3. KING OF PRUSSIA, Pa., May 6, 2021 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced the closing of its global Commercialization and License agreement with uniQure (NASDAQ:QURE) for etranacogene dezaparvovec (AMT-061), a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown  to result in near-normal levels of Factor IX (FIX) – the blood clotting protein lacking in people with hemophilia B. CSL Behring has been recognized as a global hematology leader for decades.

    The global licensing agreement was announced on June 24, 2020 and the closing follows the completion of antitrust reviews of the transaction in the US, the UK and Australia…

    KING OF PRUSSIA, Pa., May 6, 2021 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced the closing of its global Commercialization and License agreement with uniQure (NASDAQ:QURE) for etranacogene dezaparvovec (AMT-061), a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown  to result in near-normal levels of Factor IX (FIX) – the blood clotting protein lacking in people with hemophilia B. CSL Behring has been recognized as a global hematology leader for decades.

    The global licensing agreement was announced on June 24, 2020 and the closing follows the completion of antitrust reviews of the transaction in the US, the UK and Australia.  Under the terms of the agreement, uniQure will receive an upfront cash payment of US$450 million, with the potential for regulatory and commercial sales milestone payments and royalties as the therapy is developed and commercialized. uniQure will complete the Phase 3 HOPE-B trial and scale up manufacture for initial commercial supply.

    "We are continuing to build on our legacy of delivering lifesaving innovations in hematology with today's news. This agreement enables us to take forward a gene therapy that, if approved, has the potential to transform the lives of hemophilia B patients, sharply reducing or eliminating bleeds and routine infusions, life-limiting restrictions, and the constant fear of debilitating injury when blood clotting levels are low," said CSL CEO Paul Perreault. "Etranacogene dezaparvovec has the potential to be the first-ever gene therapy approved for hemophilia B and to help CSL Behring deliver on our ongoing commitment to improving the lives of those living with hemophilia B."

    Gene therapy has the potential to be life-changing and transformative, offering functionally curative benefits to people with hemophilia B with years of functional FIX levels generated by their own bodies.

    "Hemophilia B patients live with the knowledge that they are at constant risk of bleeds, and that every bleed can mean that tissue or joints are irreparably damaged," said CSL Behring's Executive Vice President, Head of Research and Development and Chief Medical Officer Bill Mezzanotte, M.D., MPH. "Imagine what it might mean to be freed from that fear, secure in the knowledge that your self-generated FIX levels will be high enough to protect you today, tomorrow, and every day, ideally for years to come. This is the essence of great science bringing hope to patients." 

    The acquisition complements both CSL Behring's cell and gene therapy scientific platform and its hematology product portfolio, which include other treatments for hemophilia B as well as therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions.

    Current Results from the Etranacogene Dezaparvovec Clinical Program  

    Ten participants in a Phase 1/2 safety and dosing study were treated five years ago using AMT-060, a gene therapy highly similar to etranacogene dezaparvovec (AMT-061).  AMT-061 uses the same delivery vehicle as AMT-060 but contains the high-activity Padua FIX gene instead of the FIX gene more commonly found in the general population.

    As of December 2020, the latest report available, all 10 participants treated with AMT-060 continue to express stable FIX levels, have reduced usage of FIX replacement therapy, and have decreased bleeding frequency, demonstrating its long-lasting durability.

    In December 2020, uniQure also announced that data from its Phase 2b dose-confirmation study of etranacogene dezaparvovec (AMT-061) showed that all three patients stabilized and sustained FIX activity at functional levels two years after a single dose – with increases in FIX activity of up to 51.6% of normal and a mean of 44.2%. Patients experienced zero bleeds and no longer needed routine prophylaxis with a FIX replacement therapy.  

    The Phase 3 HOPE-B study currently underway includes 54 study participants, with FIX activity increasing to a mean of 37% at 26 weeks (reported November 19, 2020). In all but one patient, these improvements occurred irrespective of pre-existing neutralizing antibodies. FIX activity levels will be assessed again at 52-weeks (mid-2021) to confirm that activity levels stabilized over time as they did in the Phase 2b study.

    To date, no serious treatment-related adverse events have been reported in the clinical studies of etranacogene dezaparvovec or its predecessor AMT-060. 

    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 and protective 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 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 work 8x harder 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 its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.

    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 27,000 people, 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:http://www.prnewswire.com/news-releases/csl-behring-announces-closing-of-global-commercialization-and-license-agreement-with-uniqure-for-etranacogene-dezaparvovec-301285426.html

    SOURCE CSL Behring

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  4. LEXINGTON, Mass. and AMSTERDAM, May 06, 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 the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired on May 5, 2021, and the agreement became fully effective today, May 6, 2021. The agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom. The antitrust review process is now complete in all three countries.

    "As a global leader…

    LEXINGTON, Mass. and AMSTERDAM, May 06, 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 the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired on May 5, 2021, and the agreement became fully effective today, May 6, 2021. The agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom. The antitrust review process is now complete in all three countries.

    "As a global leader in hematology and thrombosis, CSL Behring is an ideal commercial partner, and we are excited to embark on our relationship together with a shared goal of delivering this potentially transformative therapy to patients around the world living with hemophilia B," stated Matt Kapusta, chief executive officer of uniQure. "This transaction positions etranacogene dezaparvovec to be made available to the largest number of hemophilia B patients as quickly as possible and provides uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates."

    As previously announced in June 2020, uniQure and CSL Behring entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec. Under the terms of the agreement, uniQure will receive a $450 million cash payment from CSL Behring by May 13, 2021 and is eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. uniQure will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percentage of net product sales arising from the collaboration.

    Additionally, uniQure will be responsible for the completion of the HOPE-B pivotal study, manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec until such time that these capabilities are transferred to CSL Behring. Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for regulatory submissions and commercialization of etranacogene dezaparvovec.  

    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

    About CSL Behring

    As a CSL Limited ((ASX:CSL, OTC:CSLLY) company, CSL Behring is a global biotherapeutics leader delivering lifesaving medicines to patients with rare and serious diseases. A global leader in treating bleeding disorders, CSL Behring has been delivering innovations for the hemophilia patient community for more than 30 years. The company reported more than $1 billion in sales of hemophilia-related medicines in 2020.

    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 uniQure will receive the upfront cash payment or any of the financial benefits of the agreement; whether the collaboration will benefit Hemophilia B patients worldwide or be made available to the largest number of hemophilia B patients as quickly as possible, and whether uniQure will be able to advance or expand its pipeline of innovative gene therapies or its technology platform. 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 activities and commercialization agreement with CSL Behring, our clinical development activities, clinical results, potential adverse events, collaboration arrangements, regulatory oversight, and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's Quarterly Report on Form 10-K filed on 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 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, The Netherlands, May 03, 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:

    • 7th Annual Truist Securities Life Sciences Summit, May 4 - 5, 2021

      • Members of uniQure's management team will participate in virtual one-on-one investor meetings throughout the day on Tuesday, May 4.

    • American Society of Gene and Cell Therapy (ASGCT) Virtual 2021, May 11 - 14, 2021

      • uniQure will have a significant presence at ASGCT with five data presentations, of which two are oral presentations including:

        • "Clinical Outcomes in Patients

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 03, 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:

    • 7th Annual Truist Securities Life Sciences Summit, May 4 - 5, 2021



      • Members of uniQure's management team will participate in virtual one-on-one investor meetings throughout the day on Tuesday, May 4.



    • American Society of Gene and Cell Therapy (ASGCT) Virtual 2021, May 11 - 14, 2021



      • uniQure will have a significant presence at ASGCT with five data presentations, of which two are oral presentations including:



        • "Clinical Outcomes in Patients With and Without Pre-existing Neutralizing Antibodies to the Vector: 6-Month Data from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec" on Wednesday, May 12, 6:15 – 6:30 p.m. ET.



        • "First Proof-of-Concept of miQURETM Based Gene Targeting in the Liver: Lipid Lowering and Atherosclerosis Suppression by AAV-miQURETM-Mediated ANGPTL3 Targeting" on Thursday, May 13, 6:30 – 6:45 p.m. ET.

    • 2021 RBC Capital Markets Global Healthcare Conference, May 18 - 19, 2021



      • Members of uniQure's management team will participate in virtual one-on-one investor meetings throughout the day on Tuesday, May 18.



      • A fireside chat with Matt Kapusta, chief executive officer, will take place the same day from 9:10 to 9:35 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 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 Malone
    Direct: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558



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