1. ~ Entered into Exclusive Global License Agreement with CSL Behring for Development and Commercialization of uniQure's Gene Therapy Candidate for Hemophilia B ~

    ~ Completed First Patient Dosing in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease ~

    LEXINGTON, Mass. and AMSTERDAM, July 30, 2020 (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 second quarter of 2020 and highlighted recent progress across its business.

    "The first half of 2020 has been extremely productive for uniQure with significant achievements across our clinical-stage programs," stated Matt Kapusta, chief executive…

    ~ Entered into Exclusive Global License Agreement with CSL Behring for Development and Commercialization of uniQure's Gene Therapy Candidate for Hemophilia B ~

    ~ Completed First Patient Dosing in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease ~

    LEXINGTON, Mass. and AMSTERDAM, July 30, 2020 (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 second quarter of 2020 and highlighted recent progress across its business.

    "The first half of 2020 has been extremely productive for uniQure with significant achievements across our clinical-stage programs," stated Matt Kapusta, chief executive officer at uniQure. "The initiation of patient dosing in our Phase I/II study of AMT-130 marked the first AAV gene therapy for patients with Huntington's disease to enter the clinic, and we remain highly focused on advancing development of this important program. We also completed the dosing phase of our HOPE-B pivotal study for etranacogene dezaparvovec in patients with hemophilia B and remain on track to present topline data on all patients by the end of 2020 and submit a BLA in 2021."

    "Importantly, in June we announced a landmark global license agreement with CSL Behring to develop and commercialize our hemophilia B gene therapy candidate, with uniQure eligible following regulatory approval to receive more than $2 billion of economics, including upfront cash, milestone, and royalty payments," he added. "The collaboration ideally positions us to deliver our highly innovative gene therapy to the largest number of hemophilia B patients as quickly as possible, and we expect it will provide an exceptionally strong balance sheet to aggressively expand our pipeline, invest in technology innovation and further scale our manufacturing capabilities."  

    Recent Company Progress

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



      • In June 2020, the Company and CSL Behring entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec, the Company's investigational gene therapy for patients with hemophilia B.  Under the terms of the agreement, which is subject first to regulatory review in the United States, Australia and the United Kingdom, the Company will receive a $450 million upfront cash payment and be eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. the Company 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.  The agreement is one of the largest gene therapy deals announced to date and leverages CSL Behring's global hematology capabilities and infrastructure to benefit hemophilia B patients around the world.



      • The Company continues to conduct the HOPE-B pivotal trial of etranacogene dezaparvovec and remains on track to provide 26-weeks of Factor IX data for all 54 patients before the end of this year. 



      • Manufacturing process validation for etranacogene dezaparvovec continues in anticipation of the submission of a biologics license application in the United States and a marketing authorization application in the European Union, which the Company expects will be submitted in 2021. 



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



      • In June 2020, the first two patients were enrolled in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. The Phase I/II study is a double-blinded, randomized clinical trial being conducted in the United States.



      • The next two patients are expected to be enrolled in the fourth quarter of 2020, following a 90-day safety evaluation by the clinical trial's Data Safety Monitoring Board.  
    • Advancing research pipeline of gene therapy candidates toward the clinic



      • The Company expects to initiate IND-enabling studies for its Spinocerebellar Ataxia Type 3 (SCA3) program in the third quarter of 2020, which is expected to support the submission of an IND application planned for 2021. SCA3 patients experience brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There is currently no treatment available that slows the progressive course of this lethal disease. AMT-150 is a one-time administered AAV gene therapy incorporating the Company's proprietary miQURE™ silencing technology, similar to the technology used in the Company's AMT-130 program for the treatment for Huntington's disease, and is designed to halt ataxia in early manifest SCA3 patients.     

             
      • In May 2020, the Company had a significant presence at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting, with 22 data presentations: five virtual oral presentations and 17 poster presentations.



        • The Company presented in vivo preclinical data on AMT-150 for the treatment of SCA3 in six, non-human primates (NHP) that received a one-time injection of AMT-150 via the cisterna magna.  The goal of the study was to assess expression and distribution with samples taken after eight weeks showing widespread transduction of the brain and spinal cord.



        • The Company also presented additional preclinical studies evaluating AMT-150 in SCA3 mouse models and human induced pluripotent stem cell (iPSC)-derived neurons and astrocytes which represent the most disease-relevant cell type for therapeutic targeting of AMT-150. The goal of these studies was to evaluate potential off-target effects of the AAV5-miATXN3. A clear dose-dependent expression of miATXN3 was observed in the iPSC-derived neurons and astrocytes. Mature miATXN3 molecules were also associated with extracellular vesicles that strongly correlated with the dose and miATXN3 expression, suggesting the potential therapeutic spread of the engineered miATXN3. Additionally, AMT-150 demonstrated ATXN3 knockdown in human neurons and various SCA3 mouse models with subsequent neuropathology improvement.



      • On June 24, 2020, the Company held a conference call in which it announced its plans to aggressively advance and expand its pipeline by accelerating internal research with a focus on CNS disorders and other rare, liver-directed disorders, as well as by evaluating business development opportunities. Additionally, the Company announced plans to invest in technology innovation and continue to further scale manufacturing capabilities. 

                      

    • Appointment of experienced R&D expert to the board

       
      • In June 2020, Leonard E. Post, Ph.D. was appointed to the Company's Board of Directors and will serve as Chair of the Company's Research & Development Committee. Dr. Post has more than 35 years of research and development leadership, serving currently as Chief Scientific Officer of Vivace Therapeutics and its sister company, Virtuoso Therapeutics, both of which are developing oncology therapeutics. From 2010 until 2016, Dr. Post served in various positions at BioMarin (NASDAQ:BMRN), including Chief Scientific Officer, during which time he oversaw the initiation of BioMarin's first gene therapy project for hemophilia A.  Prior to that, Dr. Post served as Chief Scientific Officer of LEAD Therapeutics, Senior Vice President of Research & Development at Onyx Pharmaceuticals and Vice President of Discovery Research at Parke-Davis Pharmaceuticals. He is also currently an advisor to Canaan Partners.



    • Strong cash position to advance the Company's programs



      • As of June 30, 2020, the Company's cash position was $314.3 million, which is expected to fund the Company operations into 2022. This does not include any financial impact associated with the pending collaboration and license agreement with CSL Behring. Assuming the receipt from CSL Behring of the $450 million payment due at the closing, 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)

    • Citi's 15th Annual BioPharma Virtual Conference, September 9-10, 2020

       
    • Wells Fargo Global Healthcare Conference, September 9-10, 2020

       
    • H.C. Wainwright 22nd Annual Global Investment Conference, September 14-15, 2020

       
    • Cantor Fitzgerald Virtual Global Healthcare Conference, September 15-17, 2020

       
    • SVB Leerink CybeRx Series: Rare & Genetics, October 1, 2020

       
    • Jefferies Virtual Gene Therapy/Editing Summit, October 1-2, 2020

       
    • Chardan's Virtual 4th Annual Genetic Medicines Conference, October 5-6, 2020

    Financial Highlights

    Cash Position: As of June 30, 2020, the Company held cash and cash equivalents of $314.3 million, compared to $342.0 million as of March 31, 2020. 

    Revenues: Revenue for the three months ended June 30, 2020 was $1.5 million, compared to $2.5 million during the same period in 2019. 

    R&D Expenses: Research and development expenses were $28.4 million for the three months ended June 30, 2020, compared to $24.2 million during the same period in 2019. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec and AMT-130, increased share-based compensation, the recruitment of personnel to support the development of our product candidates and increased investments in our facilities.

    SG&A Expenses: Selling, general and administrative expenses were $11.5 million for the three months ended June 30, 2020, compared to $7.9 million during the same period in 2019. The change was primarily related to increases in personnel and consulting expenses, professional fees, and share-based compensation expenses.

    Other non-operating items, net: Other expense was $4.3 million for the three months ended June 30, 2020, compared to other expense of $2.1 million during the same period in 2019.

    Net Loss: The net loss for the three months ended June 30, 2020 was $42.6 million, or $0.96 loss per share, compared to $31.4 million, or $0.83 loss per share during the same period in 2019.

    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 about its cash runway, certain statements concerning the transaction between CSL Behring and uniQure, including whether the parties will successfully complete the review under applicable antitrust laws or otherwise close the transaction, whether uniQure will receive more than $2 billion of economics, including upfront cash, milestone, and royalty payments, whether uniQure will expand its pipeline, invest in technology innovation or further scale its manufacturing capabilities, whether uniQure will submit a biologics license application in the United States or a marketing authorization application in the European Union for etranacogene dezaparvovec in 2021 or ever, whether uniQure will present topline data on all patients in its Phase III clinical trial of etranacogene dezaparvovec by the end of 2020 or ever, whether the next two patients will be enrolled in uniQure's Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease in the fourth quarter of 2020 or ever, whether uniQure initiates IND-enabling studies for its Spinocerebellar Ataxia Type 3 (SCA3) program in the third quarter of 2020 or ever, and whether uniQure submits an IND application for SCA3 in 2021 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 2, 2020 and Quarterly Report on Form 10-Q  filed on April 29, 2020. 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



    uniQure N.V.

    UNAUDITED CONSOLIDATED BALANCE SHEETS

         
     June 30,  December 31,  
     2020  2019 
        
     (in thousands, except share and per share amounts) 
    Current assets    
    Cash and cash equivalents$314,265 $377,793 
    Accounts receivable and accrued income from related party 222  947 
    Prepaid expenses 4,082  4,718 
    Other current assets 1,066  748 
    Total current assets 319,635  384,206 
    Non-current assets    
    Property, plant and equipment, net 29,301  28,771 
    Operating lease right-of-use assets 26,139  26,797 
    Intangible assets, net 7,087  5,427 
    Goodwill 496  496 
    Restricted cash 2,683  2,933 
    Total non-current assets  65,706  64,424 
    Total assets$ 385,341 $ 448,630 
    Current liabilities    
    Accounts payable$4,942 $5,681 
    Accrued expenses and other current liabilities 13,250  12,457 
    Current portion of operating lease liabilities 5,495  5,865 
    Current portion of deferred revenue 6,153  7,627 
    Total current liabilities 29,840  31,630 
    Non-current liabilities    
    Long-term debt, net of current portion 35,373  36,062 
    Operating lease liabilities, net of current portion 30,279  31,133 
    Deferred revenue, net of current portion 23,048  23,138 
    Derivative financial instruments related party 832  3,075 
    Other non-current liabilities 464  534 
    Total non-current liabilities 89,996  93,942 
    Total liabilities$ 119,836 $ 125,572 
    Shareholders' equity    
    Total shareholders' equity 265,505  323,058 
    Total liabilities and shareholders' equity$ 385,341 $ 448,630 
         
         

    uniQure N.V.

    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

        
     Three months ended June 30,
      2020   2019 
     (in thousands, except share and per share amounts)
    Total revenues$ 1,535  $ 2,474 
    Operating expenses:   
    Research and development expenses (28,401)  (24,154)
    Selling, general and administrative expenses (11,511)  (7,870)
    Total operating expenses (39,912)  (32,024)
    Other income 669   566 
    Other expense (500)  (347)
    Loss from operations (38,208)  (29,331)
    Non operating items, net (4,343)  (2,068)
    Net loss$ (42,551) $ (31,399)
        
    Basic and diluted net loss per ordinary share$(0.96) $(0.83)
    Weighted average shares used in computing basic and diluted net loss per ordinary share 44,387,463   37,824,928 
        

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  2. Genespire appoints Jörn Aldag as Chairman of the Board of Directors
    Serial entrepreneur brings exceptional experience from a range of leadership roles

    Italy, Milan, 30 July 2020: Genespire, a gene therapy company developing transformative therapies for genetic diseases, today announces the appointment of Jörn Aldag as Chairman of the Board.

    Jörn Aldag brings 23 years of corporate and leadership experience in the life sciences industry from numerous, highly relevant roles, including as the CEO of gene therapy pioneer uniQure N.V. Since 2016 he is the CEO of HOOKIPA Pharma Inc. (NASDAQ:HOOK), a clinical-stage biopharmaceutical company developing a new class of immunotherapeutics targeting infectious diseases and cancer based on its proprietary…

    Genespire appoints Jörn Aldag as Chairman of the Board of Directors

    Serial entrepreneur brings exceptional experience from a range of leadership roles

    Italy, Milan, 30 July 2020: Genespire, a gene therapy company developing transformative therapies for genetic diseases, today announces the appointment of Jörn Aldag as Chairman of the Board.

    Jörn Aldag brings 23 years of corporate and leadership experience in the life sciences industry from numerous, highly relevant roles, including as the CEO of gene therapy pioneer uniQure N.V. Since 2016 he is the CEO of HOOKIPA Pharma Inc. (NASDAQ:HOOK), a clinical-stage biopharmaceutical company developing a new class of immunotherapeutics targeting infectious diseases and cancer based on its proprietary arenavirus platform. During his time at HOOKIPA, he has led the Company through significant growth, including the progression of several programs into clinical development, successfully completing numerous private financings, and a listing on NASDAQ in 2019, raising in total close to $200 million in proceeds. In addition to leading HOOKIPA, Jörn Aldag serves as a board member of Idorsia Pharmaceuticals Ltd, a biopharmaceutical company specialized in the discovery and development of small molecules to provide innovative therapeutic opportunities.

    Prior to joining HOOKIPA, Jörn was CEO of uniQure N.V. (NASDAQ:QURE), where he led the company to obtaining the first regulatory approval of a gene therapy in Europe. Under his leadership, uniQure raised around $200 million in proceeds, and closed a multi-billion-dollar collaboration in cardiovascular gene therapy.

    Previously, Jörn Aldag was President and CEO of Evotec AG, Chairman of the Board of Molecular Partners AG and co-founder of G7 Therapeutics AG, later sold to Heptares Therapeutics.

    Commenting on his new appointment as Chairman of Genespire, Jörn Aldag said: "Gene therapy is coming of age. As Genespire's Chairman I will support Julia Berretta and her team in transforming two exceptional next generation gene therapy platforms into a pipeline of products. Genespire is built around novel gene editing and lenti-viral technologies, which we will use to develop life changing therapies to individuals affected by severe metabolic and immunodeficiency disorders."

    Julia Berretta, Chief Executive Officer of Genespire, commented: "At Genespire we are committed to building an outstanding team. I am delighted to welcome Jörn Aldag to Genespire as Chairman. He brings a wealth of experience from across the life sciences sector which will be invaluable as we grow our team and drive the development of transformative therapeutic solutions for people suffering from genetic diseases."

    Sofinnova Partners' Managing Partner, Graziano Seghezzi, added: "Jörn's stature and authority in the gene therapy domain is well known. He is one of the most accomplished entrepreneurs in the industry and has successfully mentored numerous high-performing biotech CEOs in achieving strategic and operational excellence. His appointment adds to the outstanding caliber of the

    Genespire team and positions the company for continued success."

    In May, Genespire and SR-Tiget announced a strategic alliance for the development of transformative gene therapies for genetic diseases (the full press release can be accessed here).

    Most recently, in June, Genespire and the San Raffaele Telethon Institute for Gene Therapy announced publication in Nature Biotechnology on enhanced gene editing technique in hematopoietic stem cells (the full press release can be accessed here).

                -ends-

    Enquiries:

    Genespire Tel: +39 02 83991300

      
    Consilium Strategic CommunicationsTel: +44 (0) 20 3709 5700
    Amber Fennell / Ashley Tapp

    About Genespire

    Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. Genespire is a spin-out of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. Find out more about us at www.genespire.com.

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  3. KING OF PRUSSIA, Pa., June 24, 2020 /PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ:QURE), a leading gene therapy company. The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.

    One dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels – the blood clotting protein lacking in people with hemophilia B – to a degree that reduces or eliminates the tendency for bleeding for many years. Should AMT-061 be successful, appropriate candidate hemophilia B patients would be able to have a one-time treatment to restore FIX activity to functional levels capable of eliminating the need for frequent and ongoing replacement therapies.

    "Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease," said CSL's CEO and Managing Director Paul Perreault. "With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy."

    Under the agreement with uniQure, upon closing the transaction CSL Behring will have the exclusive global right to commercialize AMT-061. uniQure will receive an upfront cash payment of US$450 million followed by regulatory and commercial sales milestone payments and royalties. Under the terms of the agreement, uniQure will complete the Phase 3 trial and scale up manufacture for early commercial supply under an agreed plan with CSL Behring.  The transaction is subject to customary regulatory clearances before closing.

    "We are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia," stated Matt Kapusta, chief executive officer of uniQure. "We believe that through this arrangement, we are ideally positioned to deliver our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible. The transaction represents a major milestone in the development of etranacogene dezaparvovec and, when closed, we expect that it will provide uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington's disease, as well as to invest further in our leading manufacturing and technology platform."

    In December 2019, uniQure announced that data from its Phase 2b dose-confirmation study of AMT-061 showed that all patients stabilized and sustained FIX activity at functionally high levels one year after a single dose – with increases in FIX activity of up to 50% of normal and a mean of 41%. This exceeds the levels considered sufficient to eliminate or significantly reduce the risk of bleeding events.

    According to CSL Behring's Executive Vice President, Head of Research and Development, and Chief Medical Officer Bill Mezzanotte, "We are exceedingly encouraged by the data we've seen on AMT-061. Not only has the treatment option demonstrated robust clinically meaningful responses in FIX activity, but it has also exhibited excellent safety over multiple years of observation. Expanding our gene therapy portfolio to treat hemophilia B, a disease state well known to CSL Behring, exemplifies how we are strategically aligning our rare and serious disease focus and our targeted therapeutic area focus with our core scientific platforms to transform the lives of patients."

    This acquisition will also enhance CSL Behring's capabilities in its growing gene therapy portfolio. The company is currently developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease and has recently established an alliance with Seattle Children's Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases -- another rare disease area where CSL Behring has leading capabilities.

    Perreault added, "Upon approval, this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option. With the license to AMT-061, we are building on our legacy of delivering lifesaving innovations in hematology where, today, we offer a market leading product for hemophilia B and we are a leader in therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions."

    CSL Behring has put patients first by addressing the world's most serious, complicated and rare diseases for over 100 years. The company is now bringing that same commitment to gene therapy; its mission is to address unmet patient needs and enable patients to get the very most out of life.

    About Etranacogene Dezaparvovec (AMT-061)

    Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with uniQure's AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other 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 26,000 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 to download multimedia:http://www.prnewswire.com/news-releases/csl-behring-agrees-to-acquire-novel-late-stage-gene-therapy-candidate-for-hemophilia-b-patients-from-uniqure-301083302.html

    SOURCE CSL Behring

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  4. ~ CSL Behring Obtains Exclusive Global Rights to Develop and Commercialize
    uniQure's Differentiated Gene Therapy Candidate for Hemophilia B ~

    ~ uniQure Eligible to Receive More Than $2 Billion, Including $450 Million in Upfront Cash, $1.6 Billion in
    Milestone Payments, and Double-Digit Royalties Ranging Up to a Low-Twenties Percentage of Net Sales ~

    ~ Agreement Leverages CSL Behring's Global Hematology Capabilities and Infrastructure
    to Benefit Hemophilia B Patients Worldwide ~ 

    ~ Transaction Expected to Enable uniQure to Strategically Expand and Accelerate Pipeline and Platform ~

    ~ uniQure to Host Conference Call Today, June 24, 2020, at 5:30 p.m. EDT ~

    LEXINGTON, Mass. and AMSTERDAM, June 24, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ…

    ~ CSL Behring Obtains Exclusive Global Rights to Develop and Commercialize

    uniQure's Differentiated Gene Therapy Candidate for Hemophilia B ~

    ~ uniQure Eligible to Receive More Than $2 Billion, Including $450 Million in Upfront Cash, $1.6 Billion in

    Milestone Payments, and Double-Digit Royalties Ranging Up to a Low-Twenties Percentage of Net Sales ~

    ~ Agreement Leverages CSL Behring's Global Hematology Capabilities and Infrastructure

    to Benefit Hemophilia B Patients Worldwide ~ 

    ~ Transaction Expected to Enable uniQure to Strategically Expand and Accelerate Pipeline and Platform ~

    ~ uniQure to Host Conference Call Today, June 24, 2020, at 5:30 p.m. EDT ~

    LEXINGTON, Mass. and AMSTERDAM, June 24, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec, uniQure's investigational gene therapy for patients with 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). Under the terms of the agreement, uniQure will receive a $450 million upfront cash payment and be 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.

    The collaboration leverages CSL Behring's strong global reach and commercial infrastructure in hematology to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

    "We are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia," stated Matt Kapusta, chief executive officer of uniQure. "We believe that through this arrangement, we are ideally positioned to deliver globally our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible."  

    "The transaction represents a major milestone in the development of etranacogene dezaparvovec and, when closed, we expect that it will provide uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington's disease, and to invest further in our leading gene therapy manufacturing and technology platform to support pipeline growth," he added.

    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 2019.

    "Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease," said CSL's CEO and Managing Director Paul Perreault. "With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy. Upon approval, we believe this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option."

    Under the terms of the agreement, 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. 

    The closing of the transaction is contingent on completion of review under antitrust laws in the United States, Australia and the United Kingdom.  

    Accelerate Build-out of Innovative Gene Therapy Pipeline and Platform

    uniQure expects that the agreement will provide additional capital to significantly accelerate and expand its pipeline of innovative gene therapies, including advancing the Phase I/II study of AMT-130 in Huntington's disease, initiating IND-enabling studies of AMT-150 in spinocerebellar ataxia type 3, selecting a lead candidate in Fabry disease and progressing other current and new candidates for central nervous system disorders and rare liver-directed diseases. Regarding AMT-130, uniQure recently announced the successful completion of the first two patient procedures in the Phase I/II study and anticipates announcing early safety data in the second half of 2020 and initial efficacy data in 2021.

    uniQure plans to continue to leverage its leading gene therapy platform, including the Company's deep expertise with AAV5, to develop potentially best-in-class gene therapies. AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including uniQure trials conducted in hemophilia B and other indications. No patient treated in clinical trials with uniQure's AAV5 gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment.  uniQure also may seek to in-license or acquire additional product candidates that align with its research and development strategy.  

    In addition, uniQure plans to further strengthen its proprietary gene therapy platform by expanding its manufacturing capacity to support a broad pipeline, including product candidates for diseases with larger prevalence, as well as investing further in new technologies to improve the efficacy, safety and applicability of its gene therapies to patients.

    As part of uniQure's effort to focus on those gene therapy programs that have the greatest potential to improve patients' lives and generate long-term value for shareholders, uniQure plans to de-prioritize its research program of AMT-180 for patients with hemophilia A.    

    Moelis & Company acted as a financial advisor to uniQure in this transaction.

    Conference Call Today at 5:30 p.m. EDT

    uniQure will host a conference call today, June 24, 2020, at 5:30 p.m. Eastern Daylight Time. The conference call may be accessed by dialing (877) 870-9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode for the call is 9499239. Please specify to the operator that you would like to join the "uniQure Conference Call." The conference call will be webcast live under the investor relations section of uniQure's website at www.uniQure.com and will be archived there following the call for 90 days.

    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 the parties will successfully complete the review under applicable antitrust laws or otherwise close the transaction, 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, whether the parties to the agreement will establish a new standard of care for patients with hemophilia B, whether uniQure will be able to accelerate or expand its pipeline of innovative gene therapies or its technology platform, including advancing the Phase I/II study of AMT-130 in Huntington's disease, initiating IND-enabling studies of AMT-150 in spinocerebellar ataxia type 3, selecting a lead product candidate for Fabry disease, or progressing current or additional candidates for central nervous system disorders and other genetic diseases, whether uniQure will announce early safety data from its Phase I/II study of AMT-130 in the second half of 2020 and initial efficacy data in 2021 or ever, whether uniQure will develop best-in-class gene therapies, whether uniQure will in-license or acquire additional product candidates, whether uniQure will expand its manufacturing capacity to support a broad pipeline, such as product candidates for diseases with larger prevalence, and whether uniQure will obtain enabling technologies that improve the efficacy or safety of its gene therapies. 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 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 Annual Report on Form 10-Q filed on April 29, 2020. 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

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  5. LEXINGTON, Mass. and AMSTERDAM, June 19, 2020 (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 first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease have been treated. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery.

    "For years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for Huntington's disease into clinical testing, and this moment marks an important milestone for our…

    LEXINGTON, Mass. and AMSTERDAM, June 19, 2020 (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 first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease have been treated. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery.

    "For years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for Huntington's disease into clinical testing, and this moment marks an important milestone for our company now that we have two AAV gene therapy candidates in clinical development," said Matt Kapusta, chief executive officer of uniQure. "With the first two patients treated in this trial, we have taken a significant step forward in advancing AMT-130 closer to our goal of developing a therapy that inhibits the production of the mutant huntingtin protein. We are delighted to be working with leading experts in the field to evaluate this promising candidate."

    The Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington's disease randomized to treatment with AMT-130 or an imitation (sham) surgery. The five-year, multi-center trial consists of a blinded 18-month core study period followed by unblinded long-term follow-up. Patients 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.clinicaltrails.gov (NCT04120493).

    The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.

    AMT-130 is uniQure's first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE™ platform.

    "There is an urgent need for disease-modifying options to treat Huntington's disease, and we're excited to have an investigational gene therapy now available for HD patients," stated George Yohrling, chief scientific officer and chief mission officer at Huntington's Disease Society of America. "Based on the promising preclinical data presented on AMT-130 over the years, we are optimistic about its potential to alter the course of this devastating disease."

    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, hemophilia A, 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 AMT-130 will prove to be a promising treatment or alter the course of Huntington's disease, whether the DSMB will authorize treatment of additional patients, and whether we will be able to treat 26 patients under the clinical trial. 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, 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 April 29, 2020. 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
       

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/53dbf6e5-2c8c-447c-abd9-a9d5714f2a8c

    https://www.globenewswire.com/NewsRoom/AttachmentNg/6ae019fc-e394-40ff-b1c4-222d71c86145

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  6. ~ Biopharma Executive Brings 35 Years of Research and Development Leadership ~

    LEXINGTON, Mass. and AMSTERDAM, June 17, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Leonard E. Post, Ph.D. to its Board of Directors. Dr. Post, who will also chair the Company's Research & Development Committee, has extensive biotechnology industry experience including previous global R&D leadership roles at BioMarin and Onyx Pharmaceuticals.

    "It is with great pleasure that we nominate Len to the uniQure board," stated Matthew Kapusta, chief executive officer of uniQure.  "His considerable biotechnology experience…

    ~ Biopharma Executive Brings 35 Years of Research and Development Leadership ~

    LEXINGTON, Mass. and AMSTERDAM, June 17, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Leonard E. Post, Ph.D. to its Board of Directors. Dr. Post, who will also chair the Company's Research & Development Committee, has extensive biotechnology industry experience including previous global R&D leadership roles at BioMarin and Onyx Pharmaceuticals.

    "It is with great pleasure that we nominate Len to the uniQure board," stated Matthew Kapusta, chief executive officer of uniQure.  "His considerable biotechnology experience, including his important work in gene therapy, are valuable assets for uniQure as we work to advance our pipeline candidates and to prepare to file for marketing authorization of etranacogene dezaparvovec in hemophilia B."

    Since 2016, Dr. Post has served as Chief Scientific Officer of Vivace Therapeutics and its sister company, Virtuoso Therapeutics, both of which are developing oncology therapeutics. From 2010 until 2016, Dr. Post served in various positions at BioMarin (NASDAQ:BMRN), including Chief Scientific Officer, during which time he oversaw the initiation of BioMarin's first gene therapy project for hemophilia A.  Prior to that, Dr. Post served as Chief Scientific Officer of LEAD Therapeutics, Senior Vice President of Research & Development at Onyx Pharmaceuticals and Vice President of Discovery Research at Parke-Davis Pharmaceuticals. He is also currently an advisor to Canaan Partners.

    Dr. Post is a virologist by training and did early work on engineering of herpes simplex virus as a postdoctoral fellow.  He has a Bachelor of Science degree in Chemistry from the University of Michigan, and a Doctorate degree in Biochemistry from the University of Wisconsin. 

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:  FOR MEDIA:
       
    Maria E. CantorChiara E. RussoTom Malone
    Direct: 339-970-7536Direct: 617-306-9137 Direct: 339-970-7558
    Mobile:  617-680-9452Mobile: 617-306-9137Mobile: 339-223-8541
       

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/0364d19c-b363-46bb-9277-8e349f7661fd

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  7. ~ Virtual Meeting Only by Live Audio Webcast – No Physical Meeting Location ~

    LEXINGTON, Mass. and AMSTERDAM, June 15, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE) announced today a change to the location and format of its 2020 Annual General Meeting of Shareholders (the "2020 Annual Meeting"). In light of the continuing coronavirus (COVID-19) pandemic and public health concerns, the location and format of the 2020 Annual Meeting will be conducted exclusively by remote communications, via the Internet.

    The meeting will continue to be held on Wednesday, June 17, 2020, at 8:30 a.m. Eastern Standard Time/ 2:30 p.m. Central European Summer Time. Shareholders will not be able to attend the 2020 Annual Meeting in person and will not…

    ~ Virtual Meeting Only by Live Audio Webcast – No Physical Meeting Location ~

    LEXINGTON, Mass. and AMSTERDAM, June 15, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE) announced today a change to the location and format of its 2020 Annual General Meeting of Shareholders (the "2020 Annual Meeting"). In light of the continuing coronavirus (COVID-19) pandemic and public health concerns, the location and format of the 2020 Annual Meeting will be conducted exclusively by remote communications, via the Internet.

    The meeting will continue to be held on Wednesday, June 17, 2020, at 8:30 a.m. Eastern Standard Time/ 2:30 p.m. Central European Summer Time. Shareholders will not be able to attend the 2020 Annual Meeting in person and will not be able to vote their shares in real time, but shareholders of record as of the close of business on May 20, 2020, will be able to participate in the 2020 Annual Meeting through the virtual meeting platform.

    To attend the 2020 Annual Meeting, shareholders may visit either the Investors and Newsroom section of the uniQure corporate website, or go directly to www.meetingcenter.io/287482672 and enter the control number included on the notice, proxy card or voting instruction form previously distributed. Those without a control number may attend the 2020 Annual Meeting as guests by logging in to the same virtual meeting platform and following the instructions on the website for guest access. Unlike shareholders with control numbers, guests will not be able to ask questions at the 2020 Annual Meeting.

    A replay of the webcast will be available via a link on the uniQure website in the Investors & Newsroom section under Corporate Governance.

    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 and partnered gene therapies to treat patients with hemophilia, Huntington's disease and other severe genetic 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
    Mobile:  617-680-9452Mobile: 617-306-9137Mobile:339-223-8541

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  8. ~ Substantial Presence with 22 Data Presentations Highlighting Breadth of Research, Technology and Manufacturing Platform ~

    ~ New Non-Human Primate Data Demonstrate Feasibility of Intracisternal
    Administration of AMT-150 for SCA3 ~

    ~ New Data Show a Single Administration of AAV5-hFIX in Newborn Mice
    Led to Long-Term, Stable hFIX Expression ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 14, 2020 (GLOBE NEWSWIRE) --  uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of Hemophilia A. The data…

    ~ Substantial Presence with 22 Data Presentations Highlighting Breadth of Research, Technology and Manufacturing Platform ~

    ~ New Non-Human Primate Data Demonstrate Feasibility of Intracisternal
    Administration of AMT-150 for SCA3 ~

    ~ New Data Show a Single Administration of AAV5-hFIX in Newborn Mice
    Led to Long-Term, Stable hFIX Expression ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 14, 2020 (GLOBE NEWSWIRE) --  uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of Hemophilia A. The data are featured in presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.  uniQure is delivering a total of 22 data presentations at the meeting, which is taking place virtually from May 12 to May 15.

    "Our collective presence at ASGCT showcases the breadth of uniQure's gene therapy expertise and leadership, including our research capabilities and excellence in developing and manufacturing novel gene therapies," stated Matt Kapusta, chief executive officer at uniQure. "We are happy to share new preclinical data on our gene therapy candidates, as well as our innovations in technology and manufacturing."

    The uniQure presentations in Spinocerebellar Ataxia type 3 (SCA3) show a continuation of strong proof-of-concept data in mice and other preclinical models, as well as encouraging new data in non-human primates (NHPs). Additionally, new data related to AAV biology show that a single administration of AAV5-hFIX in newborn mice led to stable hFIX expression up to 18 months after dosing.

    AMT-150 for Spinocerebellar Ataxia type 3 (SCA3)

    SCA3, also known as Machado-Joseph disease, is caused by a CAG-repeat expansion in the ATXN3 gene that results in an abnormal form of the protein ataxin-3. People with SCA3 experience brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There is currently no treatment available that slows the progressive course of this lethal disease. AMT-150 is a one-time, intracisternally-administered, AAV gene therapy incorporating the Company's proprietary miQURE™ silencing technology that is designed to halt ataxia in early manifest SCA3 patients.  AMT-150 is currently in pre-investigational new drug enabling studies. 

    In an in vivo preclinical study featured in an oral presentation, six non-human primates (NHP) received a one-time injection of AMT-150 via the cisterna magna to assess expression and distribution. Samples taken after 8 weeks showed widespread transduction of the brain and spinal cord, with the highest genome copies found in the posterior fossa and cortical regions. ATXN3-microRNA was expressed in all brain regions and was correlated with the number of vector genome copies. Using in-situ hybridization, researchers confirmed the presence throughout the brain of high quantities of mature miATXN3 microRNA molecules.

    In other preclinical studies featured in separate poster presentations, researchers evaluated AMT-150 in SCA3 mouse models, as well as human induced pluripotent stem cell (iPSC)-derived neurons and astrocytes, to investigate potential off-target effects of AAV5-miATXN3. The iPSC-derived cell cultures, which were derived from two SCA3 patients, represent the most disease-relevant cell type for therapeutic targeting of AMT-150. A clear dose-dependent expression of miATXN3 was observed in the iPSC-derived neurons and astrocytes transduced with AMT-150. Mature miATXN3 molecules were also associated with extracellular vesicles that strongly correlated with the dose and miATXN3 expression, suggesting the potential therapeutic spread of the engineered miATXN3. Additionally, AMT-150 clearly demonstrated ATXN3 knockdown in human neurons and various SCA3 mouse models with subsequent neuropathology improvement.

    AMT-190 for Fabry Disease

    Fabry disease is an X-linked genetic disorder resulting from a deficiency of α-galactosidase A (α-gal or GLA). The current standard of care for Fabry disease is bi-weekly infusions of enzyme replacement therapy, a treatment that has limited effectiveness in many patients due to poor cross-correction, which hampers clearance of substrates in the target organs, in particular the kidney and the heart. In addition, a significant number of patients develop antibodies to the enzyme, α-gal or GLA. AMT-190 provides expression of a proprietary, exclusively licensed modified NAGA (ModNAGA), which shows a high structural resemblance to α-gal and has GLA activity. ModNAGA may have several advantages over current therapies, including higher stability in plasma, circumvention of GLA-inhibitors and better uptake in target organs.

    In vivo studies in wild-type (WT) NHPs were conducted to assess expression of ModNAGA upon AAV-injection. These studies demonstrated that a single administration of AMT-190 resulted in modNAGA expression in the liver and significant increases of GLA activity levels in the NHP plasma.

    Earlier in vivo studies in GLA knock-out (GLA-KO) and WT mice demonstrated that AMT-190 resulted in up to ten-fold higher GLA activity in plasma, compared to the relevant control group, and successful lowering of Gb3 and LysoGb3 in plasma and target organs of GLA-KO mice. The data presented also show that a single administration of AMT-190 in GLA-KO mice continued to elevate GLA-activity levels in the liver and plasma and reduce GLA-substrates in plasma and target organs up to 30-weeks post-injection.

    AMT-180 for Hemophilia A

    Hemophilia A is an X-linked bleeding disorder resulting from a deficiency in coagulation Factor VIII that serves as a cofactor for Factor IX in the activation of the coagulation cascade. About 30 percent of the hemophilia A patient population develops inhibitors to Factor VIII over the course of the disease. AMT-180 comprises a recombinant AAV5 vector incorporating a proprietary modified Factor IX gene.

    Data from multiple in vivo studies in rodents and NHPs demonstrated that a single administration of AMT-180 was well-tolerated and without increased coagulation activation markers. The preclinical studies were used to predict suitable clinical doses by assessing Factor VIII-independent clotting activity and correlating this to FIX-FIAV protein levels. The studies demonstrated that the dose calculation model in NHPs predicted dose-dependent increases in FVIII-independent activity.  

    A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing

    In addition to updates on our preclinical programs, an oral presentation provides important new data on AAV biology, showing that AAV5/FIX expression was maintained long-term, up to 18 months after dosing, in mice that were treated very early in life. The total number of genomic transgene copies was remarkably constant during the lives of the mice. FIX expression was maintained during the 18 months after dosing, despite the cells in the liver having turned over several times. The original volume of liver that was transduced is smaller in younger mice, but the mice livers did not lose the initial transduction/expression during cell division over time.

    "The question of whether re-treatment will be needed is an important issue in gene therapy," stated Sander van Deventer. "These data show that there was almost no loss of the transduced cells following administration of AAV5-hFIX during the life span of the mouse. This suggests that the AAV episomes were transferred to daughter cells during cell division – a very important finding in terms of AAV biology and an encouraging sign for the longevity of gene therapy in humans." 

    An overview of the data presented at ASGCT can be found on the Investor section of uniQure's corporate 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 and partnered gene therapies to treat patients with hemophilia, Huntington's disease and other severe genetic 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 AMT-150 will be effective as a one-time, intrathecally-administered, AAV gene therapy or will halt ataxia in early manifest SCA3 patients, whether ModNAGA may have one or more advantages over current therapies, including higher stability in plasma, circumvention of GLA-inhibitors and better uptake in target organs, whether the AMT-180 doses predicted by our preclinical studies will be effective in clinical studies in hemophilia A patients, and whether gene therapies will demonstrate longevity for humans in liver or other treatments. 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, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in our Quarterly Report on Form 10-Q filed on April 29, 2020. 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:                                                

    Maria E. Cantor                                                       
    Direct: 339-970-7536                                          
    Mobile: 617-680-9452                                            
                     

    FOR MEDIA:

    Tom Malone                
    Direct: 339-970-7558
    Mobile:339-223-8541
                           

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  9. ~ Completed Target Patient Dosing in Phase III HOPE-B Trial of Etranacogene Dezaparvovec in Hemophilia B ~

    ~ Announced 22 Presentations at Upcoming ASGCT Virtual Annual Meeting ~

    LEXINGTON, Mass. and AMSTERDAM, April 29, 2020 (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 2020 and highlighted recent progress across its business.

    "Despite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of 2020 in a position of strength and remain well capitalized," stated Matt Kapusta, chief executive officer of uniQure. "I am very proud of the continued…

    ~ Completed Target Patient Dosing in Phase III HOPE-B Trial of Etranacogene Dezaparvovec in Hemophilia B ~

    ~ Announced 22 Presentations at Upcoming ASGCT Virtual Annual Meeting ~

    LEXINGTON, Mass. and AMSTERDAM, April 29, 2020 (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 2020 and highlighted recent progress across its business.

    "Despite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of 2020 in a position of strength and remain well capitalized," stated Matt Kapusta, chief executive officer of uniQure. "I am very proud of the continued dedication of our employees in both the United States and the Netherlands, and thank them for the outstanding work they continue to do in driving forward our clinical programs, research pipeline, and manufacturing and technology operations.  We are well positioned for an important second half of the year when we expect to announce new clinical data and advancements from across the business."

    Recent Company Progress

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

    • In the first quarter, the Company announced the achievement of targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. A total of 54 patients now have received the one-time dose of etranacogene dezaparvovec.
       
    • The Company remains on track to provide 26-weeks of Factor IX data for all 54 patients in the HOPE-B trial before the end of this year and to file an application for marketing authorization in 2021.
       
    • The manufacturing process validation for etranacogene dezaparvovec is ongoing, and the Company believes it is able to produce material at commercial scale.

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

    • In March 2020, the Company announced that the first two patients in the Phase I/II clinical trial of AMT-130 in Huntington's disease have been enrolled after successfully meeting all screening and eligibility criteria. Due to the impact of the coronavirus pandemic, the two procedures that were scheduled to occur in late March have been temporarily postponed.
       
    • The Company continues its work to resume treatment in the Phase I/II trial as soon as it is clinically appropriate and will provide further updates on the program at that time.

    Advancing research pipeline of gene therapy candidates and the Company's proprietary technologies

    • Earlier this month, the Company announced its significant scientific presence at the upcoming American Society of Gene and Cell Therapy Virtual Annual Meeting, to be held May 12-15, 2020. In total, 22 data presentations will be delivered by the Company's researchers and scientists, including five oral presentations.

    • The data presentations will highlight the Company's industry-leading research and technology capabilities and feature new preclinical findings on the Company's gene therapy candidates for hemophilia A, spinocerebellar ataxia type 3 and Fabry disease.

    Strong cash position to advance the Company's programs

    • As of March 31, 2020, the Company held cash and cash equivalents of $342.0 million, which is expected to be sufficient to fund the Company's operations into 2022.

    Upcoming Investor Events

    SunTrust Robinson Humphrey (STRH) Virtual Life Sciences Summit, May 5 – 6, 2020.

    American Society of Gene and Cell Therapy Virtual Annual Meeting, May 12 -15, 2020.

    Huntington's Disease Regulatory Science Consortium Virtual Annual Meeting, May 18 – 19, 2020.

    Goldman Sachs Virtual Annual Healthcare Conference, June 9 – 11, 2020.

    Credit Suisse 2020 Summer Biotech Conference, June 22 -24, 2020.

    Financial Highlights

    Cash Position: As of March 31, 2020, the Company held cash and cash equivalents of $342.0 million, compared to $377.8 million as of December 31, 2019. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.

    Revenues: Revenue for the three months ended March 31, 2020 was $0.1 million, compared to $1.1 million for the same period 2019.  The decrease in 2020 reflects the reduction of activities following the end of the initial Research Term in May 2019 of our collaboration with Bristol-Myers Squibb ("BMS").

    R&D Expenses: Research and development expenses were $26.0 million for the three months ended March 31, 2020, compared to $20.5 million for the same period 2019. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec, the Phase I/II trial of AMT-130, increased share-based compensation and the additional recruitment of personnel to support the development of product candidates.

    SG&A Expenses: Selling, general and administrative expenses were $9.1 million for the three months ended March 31, 2020, compared to $8.1 million for the same period 2019. The change was primarily related to increases in personnel and contractor related expenses.

    Other income, net: Other income was $6.5 million for the three months ended March 31, 2020, compared to other expense of $0.3 million for the same period 2019, primarily reflecting foreign currency gains and a gain related to the change in fair value of the BMS warrants.

    Net Loss: The net loss for the three months ended March 31, 2020 and March 31, 2019, was $28.0 million, or $0.63 loss per share and $27.8 million, or $0.74 loss per share, respectively.

    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, hemophilia A, 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, our ability to provide top-line data or any further clinical updates and data with respect to our pivotal Phase III trial of AMT-061 before the end of 2020 or ever, our ability to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, our ability to resume treatment or to dose the first two patients in the AMT-130 Phase I/II clinical trial, our ability to fund operations into 2022, and our ability to announce new clinical data and advancements from across the business later in 2020. 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, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in our Quarterly Report on Form 10-Q filed on April 29, 2020. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541


    uniQure N.V.

    UNAUDITED CONSOLIDATED BALANCE SHEETS

             
             
             
      March 31,   December 31,  
    2020    2019  
      (in thousands, except share and per share amounts)  
    Current assets        
    Cash and cash equivalents $ 342,029   $ 377,793  
    Accounts receivable and accrued income from related party   258     947  
    Prepaid expenses   5,738     4,718  
    Other current assets   1,576     748  
    Total current assets   349,601     384,206  
    Non-current assets        
    Property, plant and equipment, net of accumulated depreciation of $29.8 million as of March 31, 2020 and $28.6 million as of December 31, 2019 respectively   27,736     28,771  
    Operating lease right-of-use assets   26,288     26,797  
    Intangible assets, net   7,211     5,427  
    Goodwill   486     496  
    Restricted cash   2,921     2,933  
    Total non-current assets   64,642     64,424  
    Total assets $ 414,243   $ 448,630  
    Current liabilities        
    Accounts payable $ 4,989   $ 5,681  
    Accrued expenses and other current liabilities   9,507     12,457  
    Current portion of operating lease liabilities   5,900     5,865  
    Current portion of deferred revenue   6,732     7,627  
    Total current liabilities   27,128     31,630  
    Non-current liabilities        
    Long-term debt   36,209     36,062  
    Operating lease liabilities, net of current portion   30,518     31,133  
    Deferred revenue, net of current portion   23,713     23,138  
    Derivative financial instruments related party   1,004     3,075  
    Other non-current liabilities   524     534  
    Total non-current liabilities   91,968     93,942  
    Total liabilities $ 119,096   $ 125,572  
    Total shareholders' equity   295,147     323,058  
    Total liabilities and shareholders' equity $ 414,243   $ 448,630  
             

    uniQure N.V.

    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

             
             
      Three months ended March 31,  
       2020    2019  
      (in thousands, except share and per share amounts)  
    Total revenues $ 104   $ 1,136  
    Operating expenses:        
    Research and development expenses   (26,013)     (20,537)  
    Selling, general and administrative expenses   (9,072)     (8,067)  
    Total operating expenses   (35,085)     (28,604)  
    Other income   857     313  
    Other expense   (339)     (349)  
    Loss from operations   (34,463)     (27,504)  
    Non operating items, net   6,464     (268)  
    Net loss $ (27,999)   $ (27,772)  
             
    Basic and diluted net loss per ordinary share $ (0.63)   $ (0.74)  
    Weighted average shares used in computing basic and diluted net loss per ordinary share   44,279,456     37,676,172  
             

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  10. LEXINGTON, Mass. and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.

    "uniQure's significant scientific presence at this year's ASGCT Annual Meeting demonstrates the robustness of our research capabilities and our excellence in developing novel technologies and commercial-scale gene therapy manufacturing," stated Sander van Deventer, executive vice president of research and product development at uniQure. "We…

    LEXINGTON, Mass. and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.

    "uniQure's significant scientific presence at this year's ASGCT Annual Meeting demonstrates the robustness of our research capabilities and our excellence in developing novel technologies and commercial-scale gene therapy manufacturing," stated Sander van Deventer, executive vice president of research and product development at uniQure. "We are very pleased to present new preclinical data on our gene therapy candidates for hemophilia A, spinocerebellar ataxia type 3, Fabry disease and data on our advances in technology and manufacturing."

    Specific details on uniQure's virtual oral presentations at ASGCT include:

    Title: One-Time Intrathecal Administration of AAV5-miATXN3 in Non-Human Primates
    Date and Time: Wednesday May 13, 4:15 p.m. EDT/ 10:15 p.m. CET

    Title: A Novel NAGA Variant Designed to be Non-immunogenic In Humans and Provide Broad Cross-Correction in Fabry Disease
    Date and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

    Title: A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing
    Date and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

    Title: Characterizing Next-Generation Baculovirus Transduction Processes - A Quality by Design-based Approach for AAV Manufacturing
    Date and Time: Friday May 15, 10:45 a.m. EDT/ 4:45 p.m. CET

    Title: Clearance of Vector DNA From Bodily Fluids in Patients with Severe or Moderate-Severe Hemophilia B Following Systemic Administration of AAV5-hFIX and AAV5-hFIX Padua
    Date and Time: Friday May 15, 11:15 a.m. EDT/ 5:15 p.m. CET

    The following presentations were approved for poster presentation:

    • Lipid Nanoparticle Pre-Treatment Improves rAAV Diffusion in the Primate Liver and Enables an Increase of Therapeutic Transgene Expression
       
    • Prevalence and Avidity Assessment of Pre-existing Neutralizing Antibodies (NABs) Against Adeno-Associated Virus (AAV) Vector Serotypes 2, 5 And 8 Analyzed in The Serum Of 300 Healthy Donors
       
    • AAV Biology Imaging Platform: Uncovering the Cellular Mechanisms Behind the AAV5-Based Vector Delivery System
       
    • Development and Optimization of a qPCR Method to Assess Biodistribution of an AAV5 Vector In NHP And Mouse Studies
       
    • Translatable Biomarkers in Gene Therapy for Huntington Disease: Innovative Approaches and Learnings from Pre-Clinic to the Clinic
       
    • Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Transcriptional Dysregulation, MRS Signal, and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntington's Disease
       
    • Secreted Therapeutics: Monitoring Durability of microRNA-based Gene Therapies in Huntington's Disease
       
    • Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miRNA Gene Therapy
       
    • Human Dose Prediction of a Novel Factor IX Variant Gene Therapy Candidate (AMT-180) Mediating Clotting Independently of Factor VIII
       
    • Assessment of miQURE™ Efficacy and Safety in SCA3 Neurons
       
    • Assessment of the Novel AAV-Based miQURE™ Gene Therapy in SCA3 Animal Models
       
    • Development of an AAV5-Based Gene Therapy for Dyslipidemia
       
    • Generation and Optimization of Insect Based Stable AAV Production Cell-Line
       
    • Generation of a DuoBac Expression System for Robust and High Quality AAV Production
       
    • From Rocking Motion Bioreactors to Stirred Tank Bioreactors the Journey to Scalable AAV Gene Therapy
       
    • Production of Recombinant AAV Vectors in Chemically Defined Media
       
    • Overcoming Challenges for Developing AAV Purification Process for Large-Scale GMP Manufacturing

    The abstracts were published today at the ASGCT Annual Meeting 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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:    
         
    FOR INVESTORS   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541

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  11. LEXINGTON, Mass. and AMSTERDAM, April 17, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the nomination of Leonard E. Post, Ph.D. to stand for election to its Board of Directors. Dr. Post, who will also chair the Company's Research & Development Committee, has extensive biotechnology industry experience including previous global R&D leadership roles at BioMarin and Onyx Pharmaceuticals.

    "It is with great pleasure that we nominate Len to the uniQure board," stated Matthew Kapusta, chief executive officer of uniQure.  "His considerable biotechnology experience, including his important work in gene therapy, are valuable…

    LEXINGTON, Mass. and AMSTERDAM, April 17, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the nomination of Leonard E. Post, Ph.D. to stand for election to its Board of Directors. Dr. Post, who will also chair the Company's Research & Development Committee, has extensive biotechnology industry experience including previous global R&D leadership roles at BioMarin and Onyx Pharmaceuticals.

    "It is with great pleasure that we nominate Len to the uniQure board," stated Matthew Kapusta, chief executive officer of uniQure.  "His considerable biotechnology experience, including his important work in gene therapy, are valuable assets for uniQure as we work to advance our pipeline candidates and to prepare to file for marketing authorization of etranacogene dezaparvovec in hemophilia B."

    Since 2016, Dr. Post has served as Chief Scientific Officer of Vivace Therapeutics and its sister company, Virtuoso Therapeutics, both of which are developing oncology therapeutics. From 2010 until 2016, Dr. Post served in various positions at BioMarin (NASDAQ:BMRN), including Chief Scientific Officer, during which time he oversaw the initiation of BioMarin's first gene therapy project for hemophilia A.  Prior to that, Dr. Post served as Chief Scientific Officer of LEAD Therapeutics, Senior Vice President of Research & Development at Onyx Pharmaceuticals and Vice President of Discovery Research at Parke-Davis Pharmaceuticals. He is also currently an advisor to Canaan Partners.

    Dr. Post is a virologist by training and did early work on engineering of herpes simplex virus as a postdoctoral fellow.  He has a Bachelor of Science degree in Chemistry from the University of Michigan, and a Doctorate degree in Biochemistry from the University of Wisconsin.  Dr. Post's appointment to the Board is subject to approval by the uniQure shareholders at its Annual General Meeting scheduled for June 17, 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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:    
         
    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541
     

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/3b0eca26-91f3-4206-a721-8ab6669d3ae0

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  12. LEXINGTON, Mass. and AMSTERDAM, March 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming investor events in April:

    • Stifel CNS Day Virtual Conference, Wednesday April 1, 2020.

      • David Cooper, M.D., vice president of clinical research CNS, will participate in the panel discussion, "A Deep Dive into Disease Modifying Strategies in Huntington's Disease", on Wednesday April 1st at 10:00 a.m. EDT. 

      • Matt Kapusta, chief executive officer at uniQure, will participate in the panel discussion, "The Promise and Challenges Associated with Gene Therapy in CNS" on Wednesday April…

    LEXINGTON, Mass. and AMSTERDAM, March 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming investor events in April:

    • Stifel CNS Day Virtual Conference, Wednesday April 1, 2020.

      • David Cooper, M.D., vice president of clinical research CNS, will participate in the panel discussion, "A Deep Dive into Disease Modifying Strategies in Huntington's Disease", on Wednesday April 1st at 10:00 a.m. EDT. 

      • Matt Kapusta, chief executive officer at uniQure, will participate in the panel discussion, "The Promise and Challenges Associated with Gene Therapy in CNS" on Wednesday April 1st at 10:45 a.m. EDT.
       
    • SunTrust Corporate Access Call Series: After the Dust Settles, Friday April 3, 2020. 

      • Matt Kapusta, chief executive officer, and Maria Cantor, senior vice president investor relations and corporate communications, will participate in SunTrust's conference call series, "After the Dust Settles" on Friday April 3rd, at 11:00 a.m. EDT.
       
    • 2020 Virtual Biotech Corporate Access Day hosted by Wells Fargo, April 7&9, 2020.

      • Members of uniQure's management team will be available for virtual investor meetings throughout the day on Tuesday, April 7th.

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:
     
    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder  Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct:  339-970-7558

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  13. LEXINGTON, Mass. and AMSTERDAM, March 26, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. The targeted number of patients to be dosed per the clinical trial protocol was 50.  In total, 54 patients have received the one-time dose of etranacogene dezaparvovec.

    "I am very proud of the entire uniQure team, our investigators, study…

    LEXINGTON, Mass. and AMSTERDAM, March 26, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. The targeted number of patients to be dosed per the clinical trial protocol was 50.  In total, 54 patients have received the one-time dose of etranacogene dezaparvovec.

    "I am very proud of the entire uniQure team, our investigators, study coordinators and the hemophilia patient communities who helped make this major milestone possible," stated Matt Kapusta, chief executive officer of uniQure. "With target patient dosing now finished, we are closely monitoring the trial and working within guidance provided by the FDA regarding COVID-19 to minimize any risk or disruption in patient follow-up visits. We continue to expect top-line data from the Phase III trial before the end of this year, which we believe will support a BLA submission in 2021."

    The pivotal Phase III HOPE-B trial follows the Company's ongoing Phase IIb trial of etranacogene dezaparvovec, in which we have previously reported that, after 52 weeks of follow-up data, all three patients had stabilized and sustained FIX activity at therapeutic levels after the one-time administration of etranacogene dezaparvovec. Additionally, in an ongoing Phase I/II trial of AMT-060, the Company's first-generation gene therapy for the treatment of hemophilia B, all 10 patients continued to show sustained and stable increases in FIX activity and long-term clinical benefit, including improved disease phenotype and substantial reductions in spontaneous bleeds at up to 4 years of observation.

    Update on the Phase I/II Clinical Trial of AMT-130 in Huntington's Disease

    The first two patients in the Company's Phase I/II clinical trial of AMT-130 in Huntington's disease have been enrolled after successfully meeting all screening and eligibility criteria. These patients were scheduled to have their procedures on March 24 and 25 at the Ohio State University. Due to the expanding impact of the COVID-19 coronavirus pandemic, these procedures have been temporarily postponed. The decision to postpone treatment in the trial follows the COVID-19-related State of Emergency declarations in the United States, where the trial is taking place.

    "We are following all federal and local regulations including the FDA guidance on clinical trial conduct during the COVID-19 pandemic  and we are working very closely with trial investigators to ensure that patient safety remains our top priority," stated Robert Gut, M.D., Ph.D., chief medical officer at uniQure. "Despite this unexpected postponement, we are encouraged by the progress that we have made with this trial since the beginning of the year, and the fact that we have patients who are highly motivated to participate.  We will continue our work to resume treatment in the Phase I/II trial as soon as it is clinically appropriate."

    uniQure's Response to COVID-19

    The Company is continuing its operations under guidance from national, state and local authorities in the Netherlands and in the United States, including the US Food and Drug Administration (FDA) and Centers for Disease Control and Prevention (CDC). In this regard, uniQure is informed by the direction and flexibility provided by the FDA in its March 18, 2020 Guidance entitled "FDA Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic". Ongoing clinical research activities are being closely monitored to minimize any risk, disruption or delay in either patient dosing or follow-up visits.

    uniQure is focused on ensuring the health and wellbeing of its global workforce. As of March 13, the Company mandated a work-from-home policy for all non-essential employees. As a biopharma research and development company, uniQure is deemed to provide Essential Services under the "stay at home" advisory that was issued by the Governor of Massachusetts on March 23, 2020 and is maintaining its mission-critical activities that include commercial-scale manufacturing operations. 

    About the Pivotal Phase III HOPE-B Trial

    The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec in 50 patients. Adult hemophilia B patients classified as severe or moderately severe are enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will receive a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the HOPE-B pivotal trial was initiated in January 2019.

    The trial's primary endpoint is the assessment of Factor IX activity 26 weeks after dosing.  Secondary endpoints include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

    Patients enrolled in the HOPE-B pivotal trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5-based gene therapies may be viable treatments for at least 97% of patients.

    About Etranacogene Dezaparvovec (AMT-061)

    Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company's AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.

    About AMT-130

    AMT-130 is our novel gene therapy candidate for the treatment of Huntington's disease, a severe genetic neurodegenerative disorder causing loss of muscle coordination, behavioral abnormalities and cognitive decline, often resulting in complete physical and mental deterioration over a 12 to 15-year period. AMT-130 utilizes our miQURE™ proprietary, gene-silencing platform and incorporates an AAV vector carrying a microRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment. AMT-130 has received orphan drug and fast track designations from the FDA and Orphan Medicinal Product Designation from the EMA.

    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, hemophilia A, 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 obtain top-line data from the HOPE-B Phase III trial before the end of this year or ever, whether such data will support a BLA submission in 2021, whether we will be able to resume treatment in the Phase I/II trial as soon as it is clinically appropriate, and our expectation to continue our operations during the COVID-19 pandemic. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 Annual Report on Form 10-K filed on March 2, 2020. 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    Eva M. Mulder   Tom Malone
    Direct: 339-970-7536   Direct: +31 20 240 6103   Direct: 339-970-7558
    Mobile:  617-680-9452   Mobile: +31 6 52 33 15 79   Mobile: 339-223-8541
             

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  14. LEXINGTON, Mass. and AMSTERDAM, March 03, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in March:

    • Cowen 40th Annual Health Care Conference, March 2 – 4, Boston, MA.

      Matt Kapusta, chief executive officer at uniQure, will be presenting a corporate update today, March 3, at 9:20 a.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event. Members of uniQure's management team also will participate…

    LEXINGTON, Mass. and AMSTERDAM, March 03, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in March:

    • Cowen 40th Annual Health Care Conference, March 2 – 4, Boston, MA.

      Matt Kapusta, chief executive officer at uniQure, will be presenting a corporate update today, March 3, at 9:20 a.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event. Members of uniQure's management team also will participate in one-on-one investor meetings throughout the day.
       
    • Gene Therapy for Blood Disorders Conference, March 3 – 5, Boston, MA.

      • Sander van Deventer, M.D., Ph.D., executive vice president, research & product development at uniQure, will present "Evaluating Immunogenic Responses in Systemic Administration: Determining the ‘Right' Dose" on Wednesday, March 4, at 3:45 p.m. ET.

      • Eileen Sawyer, Ph.D., vice president, global medical affairs of uniQure, will participate in a panel discussion: "Assessing the Commercial Realities of Launching Gene Therapies in the Blood Disorder Space", on Wednesday, March 4, at 9:15 a.m. ET.
       
    • BioCapital Europe by LSP, March 12, Amsterdam, the Netherlands.

      • A corporate update will be presented by Christian Klemt, chief accounting officer of uniQure, on Thursday, March 12, at 9:30 a.m. CET.
       
    • 2020 Summit Meeting on in vivo Gene Therapy and Editing Presented by Chardan, March 23 – 24, Miami, FL.

      • Sander van Deventer will present on the Company's clinical development for hemophilia B on Monday, March 23, at 2:20 p.m. ET, and participate in a panel discussion on AAV gene therapy March 23 at 3:45 p.m. ET.
       
    • 4th Annual Gene Therapy for Rare Disorders 2020 Summit, March 30 – April 2, Boston, MA.

      Alex Kuta, Ph.D., executive vice president operations at uniQure, will present "Extracting Meaningful Data from Clinical Trials to Present to Regulatory Agencies" on Tuesday, March 31, at 2:30 p.m. ET.
       
    • Clinical Biomarkers & World CDx Europe 2020 Conference, March 31 – April 1, London, U.K.

      • Astrid Valles-Sanchez, Ph.D., senior scientist at uniQure will be presenting "Translatable Biomarkers in Gene Therapy for Huntington Disease: Innovative Approaches & Learnings from Pre-Clinic to the Clinic" on Tuesday, March 31, at 3:30 p.m. GMT. 

      • In addition, she will participate in the panel discussion "Precision Medicine Beyond Oncology" on March 31, at 4:30 p.m. GMT. 

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct:  339-970-7558

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  15. ~ Gen-X Will Work with uniQure During an Exclusivity Period to Identify Novel Promoters for uniQure's Product Portfolio ~

    ~ uniQure Receives Option to Acquire Gen-X Business for Use in Gene Therapy ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, March 02, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, and Gen-X, a spin-off company from the Netherlands Cancer Institute, today announced that they have signed an exclusive research collaboration and license agreement with Gen-X, with an option for uniQure to acquire Gen-X, to identify novel synthetic promoters for use in gene therapy products.

    Gen-X performs functional annotation…

    ~ Gen-X Will Work with uniQure During an Exclusivity Period to Identify Novel Promoters for uniQure's Product Portfolio ~

    ~ uniQure Receives Option to Acquire Gen-X Business for Use in Gene Therapy ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, March 02, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, and Gen-X, a spin-off company from the Netherlands Cancer Institute, today announced that they have signed an exclusive research collaboration and license agreement with Gen-X, with an option for uniQure to acquire Gen-X, to identify novel synthetic promoters for use in gene therapy products.

    Gen-X performs functional annotation of the non-coding genome using its proprietary Survey of Regulatory Elements (SuRE) technology. This technology measures the potential of hundreds of millions of DNA elements for their ability to serve as promoters or enhancers, driving the gene expression with a range of unique properties for gene therapy applications. The SuRE technology is unique among other promoter-identification technologies in finding non-coding promoter elements throughout the genome at remote sites where activity is regulated.

    "Novel promoters are an important area of innovation in gene therapy with the potential to enable valuable functionality and improve treatment options," stated Jonathan Garen, chief business officer of uniQure.  "With the unique SuRE methodology developed by Gen-X, we will now leverage our modular technology platform to potentially identify new promoters and enhancers that drive gene expression in the right cell-type and under the right conditions. We are excited about the collaboration, providing us with the potential of applying this state-of-the-art technology and increasing the expression and effectiveness of our next generation gene therapy product candidates."

    During the collaboration, Gen-X will work exclusively with uniQure to develop promoters for the uniQure research pipeline. Through the SuRE technology, the Gen-X promoter library is designed to identify novel promoters to regulate gene activity and, if needed, conditionally control protein production. The parties will also explore further development of AAV vector libraries containing fragments of the non-coding human genome. Active promoter elements can be directly identified following administration of these libraries in vivo.

    "We are very proud to enter into a partnership with uniQure, giving us the opportunity to demonstrate the value of the Gen-X platform in a gene therapy setting," stated Joris van Arensbergen, Gen-X founder and CEO. "We believe that combining our platform with uniQure's leading expertise in AAV gene therapy will accelerate the development of advanced gene therapy candidates."

    Until the expiry of the research term of the collaboration, uniQure has an option to acquire the issued and outstanding shares of Gen-X for a pre-agreed price. Financial terms on the research collaboration and license agreement were not disclosed.     

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    About Gen-X
    Gen-X is a biotechnology company located in Amsterdam, the Netherlands performing functional annotation of the non-coding genome on an unprecedented scale using its SuRE technology. The company provides screening services to gene therapy companies for the identification of DNA elements that enable controlled transgene expression.

    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 generation of new technology or products, and the payment of associated future milestones and royalties. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 2, 2020. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541
         

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  16. ~ Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
    in Hemophilia B ~

    ~ First Patient Procedure in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease
    Expected to Occur Around the End of the First Quarter of 2020 ~

    ~ Strong Balance Sheet Expected to Fund Operations into 2022 ~

    LEXINGTON, Mass. and AMSTERDAM, March 02, 2020 (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 2019 and highlighted recent progress across its business.

    "The past year was marked by strong execution in our pivotal, Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia…

    ~ Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
    in Hemophilia B ~

    ~ First Patient Procedure in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease
    Expected to Occur Around the End of the First Quarter of 2020 ~

    ~ Strong Balance Sheet Expected to Fund Operations into 2022 ~

    LEXINGTON, Mass. and AMSTERDAM, March 02, 2020 (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 2019 and highlighted recent progress across its business.

    "The past year was marked by strong execution in our pivotal, Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia B for which we achieved full enrollment and expect to announce top-line data before the end of this year," stated Matt Kapusta, chief executive officer of uniQure. "We believe etranacogene dezaparvovec has a potential best-in-class profile as a one-time treatment for patients with hemophilia B, and our primary goal is to advance this program for regulatory review and potential approval."

    "We have also successfully screened patients in the randomized Phase I/II clinical trial of AMT-130 in Huntington's disease and expect the first procedure to occur around the end of this first quarter of 2020," he added. "In addition, we have continued to advance our research pipeline of innovative AAV gene therapy candidates and further build on our proprietary, commercial-scale manufacturing capabilities. We expect this year to be a transformational one for uniQure in which we announce new clinical data and advance our internally discovered investigational gene therapies."

    Recent Company Progress

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

      • Nearly all of the eligible patients in the pivotal, Phase III HOPE-B study of etranacogene dezaparvovec have received their one-time administration of etranacogene dezaparvovec. Approximately 60 patients were enrolled in the study, which is being conducted in approximately 40 sites in the United States and Europe. The Company is on track to provide top-line data on all patients in the HOPE-B study before the end of this year, and to submit a Biologics License Application (BLA) for marketing authorization in 2021.

      • Late last year at the annual meeting of the American Society of Hematology, the Company presented one-year follow-up data on the three patients in the Phase IIb study of etranacogene dezaparvovec. Mean FIX activity for the three patients at 52 weeks after administration was 41% of normal.  No patient in the study had reported any bleeding events, and all patients in the study have remained free of prophylaxis after receiving etranacogene dezaparvovec.

      • The Company is producing etranacogene dezaparvovec at commercial scale for product launch and plans to conduct manufacturing process validation throughout the year.

    • Advancing AMT-130 into clinical development for the treatment of Huntington's disease
      
      • The Company and the U.S. Food and Drug Administration have agreed to broaden the eligibility criteria for the Phase I/II study of AMT-130 in patients with Huntington's disease, and numerous patients are actively screening across multiple sites. Initial patients have successfully passed the screening protocol, and the first surgical procedure is expected to take place around the end of the first quarter of 2020. The Company expects to provide a safety update after the study's Data Safety Monitoring Board convenes later in the year. Important imaging and biomarker data from this first-in-human AAV-gene therapy trial in Huntington's disease are expected in 2021.

    • Advancing research pipeline of gene therapy candidates towards the clinic

      • The Company is advancing multiple preclinical and nonclinical studies of AMT-180 for the treatment of hemophilia A and expects to submit an Investigational New Drug (IND) application this year.  AMT-180 incorporates the Company's proprietary modified Factor IX transgene that has been demonstrated in preclinical studies to convey Factor VIII-independent activity and circumvent inhibitors to Factor VIII.

      • In addition, the Company expects to initiate IND-enabling studies for Spinocerebellar Ataxia Type 3 in 2020, supporting the submission of an IND application planned for 2021. SCA3 patients experience brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There is currently no treatment available that slows the progressive course of this lethal disease. AMT-150 is a one-time administered AAV gene therapy incorporating the Company's proprietary miQURE™ silencing technology, similar to the technology used in the Company's AMT-130 program for the treatment for Huntington's disease, and is designed to halt ataxia in early manifest SCA3 patients.

    • Strong cash position to advance the Company's programs

      • As of December 31, 2019, the Company's cash position was $377.8 million, which is expected to fund the Company operations into 2022.

    Upcoming Investor Events

    • 40th Annual Cowen & Co. Healthcare Conference, March 2 – 4, 2020, Boston, MA.
       
    • BioCapital Europe 2020 by LSP, March 12, Amsterdam, the Netherlands.
       
    • Gene Therapy Summit by Chardan, March 23 – 24, 2020, Miami, FL.
       
    • Stifel CNS Day, April 1, 2020, Boston, MA
       
    • Wells Fargo Corporate Access Day, April 7, 2020, Boston, MA.
       
    • H.C. Wainwright Global Life Sciences Conference, April 19 – 21, 2020, London, UK.
       
    • Kempen Life Sciences Conference, April 21 – 22, 2020, Amsterdam, the Netherlands.

    Financial Highlights

    Cash Position: As of December 31, 2019, the Company held cash and cash equivalents of $377.8 million, compared to $234.9 million as of December 31, 2018. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.

    Revenues: Revenue for the year ended December 31, 2019 was $7.3 million, compared to $11.3 million for the year ended December 31, 2018.  The decrease in 2019 reflects the termination of the S100A1 program for the treatment of congestive heart failure under our collaboration with Bristol-Myers Squibb.

    R&D Expenses: Research and development expenses were $94.7 million for the year ended December 31, 2019, compared to $74.8 million for the year ended December 31, 2018. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec, the recently initiated Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site.

    SG&A Expenses: Selling, general and administrative expenses were $33.5 million for the year ended December 31, 2019, compared to $25.3 million for the year ended December 31, 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.

    Other income, net: Other expense was $0.1 million for 2019, compared to other income of $0.6 million for 2018.

    Net Loss: The net loss for the full years 2019 and 2018 was $124.2 million, or $3.11 per share and $83.3 million, or $2.34 per share, respectively.

    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, hemophilia A, 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 ability to provide top-line data or any further clinical updates and data with respect to our pivotal Phase III study of AMT-061 before the end of 2020 or ever, the ability to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, the risk of cessation, delay or lack of success of any ongoing or planned clinical studies, the ability to dose the first sentinel patient in the AMT-130 Phase I/II clinical study by around the end of the first quarter of 2020, or ever, the ability to fund operations into 2022, whether AMT-061 proves to have a best-in-class profile as a one-time treatment for patients with hemophilia, whether 2020 will be a transformational year for uniQure in which new clinical data is announced or investigational gene therapies are advanced, whether we can provide a safety update for AMT-130 this year, whether the Data Safety Monitoring Board for AMT-130 will convene later in 2021, whether important imaging and biomarker data from the AMT-130 trial will be obtained in 2021, or ever, whether IND-enabling studies for Spinocerebellar Ataxia Type 3 (SCA3) are initiated in 2020, and whether an IND application for SCA3 will be filed in 2021, 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 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 Annual Report on Form 10-K filed on March 2, 2020. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541
     


    uniQure N.V.
     
    UNAUDITED CONSOLIDATED BALANCE SHEETS
                 
      December 31,   December 31,  
    2019   2018  
      (in thousands, except share and per share amounts)  
    Current assets            
    Cash and cash equivalents $  377,793   $  234,898  
    Accounts receivable and accrued income from related party   947     233  
    Prepaid expenses   4,718     1,116  
    Other current assets   748     329  
    Total current assets   384,206     236,576  
    Non-current assets            
    Property, plant and equipment, net   28,771     29,179  
    Operating lease right-of-use assets   26,797     -  
    Intangible assets, net   5,427     5,201  
    Goodwill   496     506  
    Restricted cash   2,933     2,444  
    Total non-current assets   64,424     37,330  
    Total assets $  448,630   $  273,906  
    Current liabilities            
    Accounts payable $  5,681   $  3,792  
    Accrued expenses and other current liabilities   12,457     8,232  
    Current portion of operating lease liabilities   5,865     -  
    Current portion of deferred rent   -     311  
    Current portion of deferred revenue   7,627     7,634  
    Total current liabilities   31,630     19,969  
    Non-current liabilities            
    Long-term debt   36,062     35,471  
    Operating lease liabilities, net of current portion   31,133     -  
    Deferred rent, net of current portion   -     8,761  
    Deferred revenue, net of current portion   23,138     28,861  
    Derivative financial instruments related party   3,075     803  
    Other non-current liabilities   534     435  
    Total non-current liabilities   93,942     74,331  
    Total liabilities   125,572     94,300  
    Total shareholders' equity   323,058     179,606  
    Total liabilities and shareholders' equity $  448,630   $  273,906  
                 

     

    uniQure N.V.
     
    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
                 
      Years ended December 31,    
        2019       2018       2017    
                             
      (in thousands, except share and per share amounts)  
    Total revenues   7,281       11,284       13,107    
    Operating expenses:            
    Research and development expenses   (94,737 )     (74,809 )     (72,172 )  
    Selling, general and administrative expenses   (33,544 )     (25,305 )     (24,635 )  
    Total operating expenses   (128,281 )     (100,114 )     (96,807 )  
    Other income   1,888       2,146       15,430    
    Other expense   (2,028 )     (1,548 )     (3,073 )  
    Loss from operations   (121,140 )     (88,232 )     (71,343 )  
    Non operating items, net   (3,061 )     5,159       (8,116 )  
    Loss before income tax expense   (124,201 )     (83,073 )     (79,459 )  
    Income tax expense / (benefit)   -       (231 )     199    
    Net loss $ (124,201 )   $ (83,304 )   $ (79,260 )  
                 
    Basic and diluted net loss per ordinary share $ (3.11 )   $ (2.34 )   $ (2.94 )  
    Weighted average shares used in computing basic and diluted net loss per ordinary share   39,999,450       35,639,745       26,984,183    
                 

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  17. ~ Up to Two Years of Follow-up in Large Transgenic Huntington's Disease Model Demonstrates Stable mHTT Protein Lowering ~

     ~ Novel Preclinical Data Demonstrates Successful Lowering of Pathogenic Exon 1 Fragment ~

    ~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntington's Disease Gene-Therapy Studies ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of Huntington's disease (HD), at the 15th Annual CHDI Huntington's…

    ~ Up to Two Years of Follow-up in Large Transgenic Huntington's Disease Model Demonstrates Stable mHTT Protein Lowering ~

     ~ Novel Preclinical Data Demonstrates Successful Lowering of Pathogenic Exon 1 Fragment ~

    ~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntington's Disease Gene-Therapy Studies ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of Huntington's disease (HD), at the 15th Annual CHDI Huntington's disease Therapeutics Conference in Palm Springs, California.

    "Our data presentations at CHDI illustrate the increasing potential of AMT-130 to target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species and show meaningful activity using the presence of extracellular vesicles as a potential biomarker," stated Sander van Deventer, M.D., Ph.D., executive vice president, research & product development of uniQure. "In addition, we highlight the use of magnetic resonance spectroscopy as a potentially important imaging biomarker to measure the restoration of target tissue. Collectively, these findings represent a robust package of new preclinical data to better inform how researchers and clinicians pursue a much-needed treatment for this devastating disease."

    Four scientific abstracts submitted by uniQure researchers were accepted for presentation at the conference, of which one is an oral presentation to be featured today. Important findings across several preclinical studies presented at the conference include the following:

    Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-clinical Studies

    • In identifying translatable biomarkers for gene therapy studies in Huntington's disease, transgenic Huntington's disease minipigs 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 two 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 ongoing study that now includes up to two years of follow-up, demonstrated stable mHTT protein lowering of up to 30 percent in the cerebrospinal fluid (CSF) of minipigs after a single administration of AMT-130.

    Secreted Therapeutics: Monitoring Durability of microRNA-based Gene Therapies in Huntington's disease

    • Neurons derived from the induced pluripotent stem cells (iPSC) of Huntington's disease patients were used to study the presence of extracellular therapeutic microRNA targeting human huntingtin (miHTT) after transduction with AMT-130. Extracellular vesicles were identified as carriers of RNA species, including microRNAs, and are therefore potential biomarkers for diagnosis and pharmacokinetics. Results from this new study demonstrated that therapeutic miHTT molecules are secreted in a dose-dependent manner from AMT-130-treated neuronal cells, providing the potential of a promising translational marker to monitor long-term expression of AMT-130 gene therapy in the brain.
       
    • To support these results, non-human primates received a single administration of AMT-130 resulting in widespread distribution of therapeutic miHTT molecules in both the striatum and cortex, as well as the presence of therapeutic miHTT molecules in the CSF up to 6 months after intrastriatal injection of AMT-130.

    Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miHTT Gene Therapy

    • In a study using the Q175FDN mouse model, the target engagement of aberrantly spliced exon 1 HTT transcript (exon 1 HTT) was investigated. These new preclinical data demonstrated that the mice treated with AMT-130 experienced a dose-dependent lowering of exon 1 HTT mRNA in both the striatum and cortex, as well as the full-length HTT mRNA. The lowering was shown to be well correlated with the detected levels of vector DNA and mature miHTT molecules in the mice treated with AMT-130.

    Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Neuronal Function, MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntington's disease

    • Magnetic resonance spectroscopy (MRS) data in the Q175FDN mouse model of Huntington's disease demonstrate that levels of N-acetyl aspartate (NAA), a neuronal integrity marker, were restored in mice treated with the high dose of AMT-130, supporting the use of imaging biomarkers in gene therapy studies for Huntington's disease. Importantly, effects of mHTT silencing in the Q175FDN model on MRS were complemented by additional data on striatal gene expression profiles, demonstrating a trend towards normalization with AMT-130 treatment. These new data build on earlier MRS and MRI data suggesting improvement in brain cell function, a reversal in Huntington's disease neuropathology and a partial reversal of volume loss in the hippocampus, a key brain region involved in memory.

    The uniQure data presentations featured at CHDI are available on the investor page of the Company's website, www.uniQure.com

    About Huntington's Disease
    Huntington's disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete 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 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, hemophilia A, 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 AMT-130 will target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species or show meaningful activity using the presence of extracellular vesicles as a potential biomarker, and whether magnetic resonance spectroscopy will be an important imaging biomarker to measure the restoration of target tissue. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 Quarterly Report on Form 10-Q filed on October 28, 2019. 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  Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541

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  18. LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that seven presentations, of which one is an oral presentation, will be delivered at the upcoming congress of the European Association for Haemophilia and Allied Disorders (EAHAD), held in The Hague, the Netherlands, February 5 – 7, 2020.

    Specific details on uniQure's presentations at EAHAD include:  

    • Title: One Year Data from A Phase 2b Trial Of AMT-061 (AAV5-Padua hFIX Variant), An Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B (ORAL10)
      Oral Session Title: Session 6: SLAM…

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that seven presentations, of which one is an oral presentation, will be delivered at the upcoming congress of the European Association for Haemophilia and Allied Disorders (EAHAD), held in The Hague, the Netherlands, February 5 – 7, 2020.

    Specific details on uniQure's presentations at EAHAD include:  

    • Title: One Year Data from A Phase 2b Trial Of AMT-061 (AAV5-Padua hFIX Variant), An Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B (ORAL10)
      Oral Session Title: Session 6: SLAM
      Date and Time: Friday February 7, 8:30 – 10:00 a.m. CET

    • Title: Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up To 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B (Poster 100)
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET

    • Title: Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX Or AAV5-hFIX Padua In Patients with Severe or Moderate-Severe Hemophilia B (Poster 89)
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET
             
    • Title: Prevalence and Affinity/Avidity Assessment of Pre-Existing NABS Against AAV1, 2, 5 And 8 Analyzed In the Serum Of 300 Healthy Donors (Poster 54)
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET

    • Title: Seroprevalence Of Pre-Existing NABS Against AAV1, 2, 5, 6 And 8 in the South African Hemophilia B Patient Population (Poster 116)
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET

    • Title: Evaluation of A Blood Coagulation Factor IX Variant That Functions Independently of Factor Viii as An Alternative Treatment of Hemophilia A
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET

    • Title: Gene Therapy for People with Haemophilia B (PWHB): Development of A Cost-Effectiveness Model Framework (Poster 154)
      Date and Time: Wednesday February 5, 6:30 – 7:30 p.m. CET

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor  Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541
       

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  19. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 31, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in February:

    • 13th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD), February 5 – 7, The Hague, the Netherlands.

      • uniQure will deliver 7 presentations on the development of its gene therapy candidates etranacogene dezaparvovec for hemophilia B, AMT-180 for hemophilia A, and its technology platform.
      • Further details on the company's presentations will follow closer to conference. 

    • 16th Annual WORLD Symposium, February

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 31, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in February:

    • 13th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD), February 5 – 7, The Hague, the Netherlands.

      • uniQure will deliver 7 presentations on the development of its gene therapy candidates etranacogene dezaparvovec for hemophilia B, AMT-180 for hemophilia A, and its technology platform.
      • Further details on the company's presentations will follow closer to conference. 

    • 16th Annual WORLD Symposium, February 10 – 13, Orlando, FL.

      • Ying Poi Liu, Ph.D., senior scientist at uniQure, will present on the development of an AAV5-based gene therapy for Fabry disease, on Tuesday, February 11, from 4:30 – 6:30 p.m. ET.
       
    • CHDI 15th Annual Huntington's Therapeutics Conference, February 22 – 25, Palm Springs, CA.

      • uniQure will deliver 5 presentations on the development of AMT-130, the company's AAV-gene therapy candidate for the treatment of Huntington's disease.
      • Further details on the company's presentations will follow closer to the conference.
       
    • 64th Annual Meeting of the Society of Thrombosis and Haemostasis Research – GTH 2020, February 18 – 21, Bremen, Germany.

      • Professor Wolfgang Miesbach, M.D., Ph.D., of the University Hospital Frankfurt in Germany, will present 12-month follow-up data on the company's gene therapy candidate etranacogene dezaparvovec in hemophilia B, as well as four-year follow-up data on AMT-060, also in patients with hemophilia B.

    • 9th Annual SVB Leerink Global Healthcare Conference, February 25 – 27, New York City.

      • Matt Kapusta, chief executive officer at uniQure, will participate in a fireside chat with research analyst Joseph Schwarz, on Tuesday, February 25, from 2:30 – 2:55 p.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
      • Members of uniQure's management team will participate in one-on-one investor meetings throughout the day on Tuesday, February 25.

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558

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  20. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 19, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced two publications of preclinical data from its gene therapy candidate AMT-130 in Huntington's disease, in the journals Nucleic Acids Research and Molecular Therapy – Methods & Clinical Development.

    The first publication in Nucleic Acids Research examines the tolerability and efficacy of non-selective huntingtin protein (HTT) lowering using an AAV5 encoded micro-RNA targeting human HTT (AAV5-miHTT) in the humanized Hu128/21 mouse model of Huntington's disease. The Hu128/21 model represents a genetically accurate…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 19, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced two publications of preclinical data from its gene therapy candidate AMT-130 in Huntington's disease, in the journals Nucleic Acids Research and Molecular Therapy – Methods & Clinical Development.

    The first publication in Nucleic Acids Research examines the tolerability and efficacy of non-selective huntingtin protein (HTT) lowering using an AAV5 encoded micro-RNA targeting human HTT (AAV5-miHTT) in the humanized Hu128/21 mouse model of Huntington's disease. The Hu128/21 model represents a genetically accurate model of Huntington's disease, as it expresses both the mutant and wild type full-length human HTT transgene. The animals received bilateral intrastriatal infusions by convection-enhanced delivery of either the control dose, or three ascending doses at the onset of the disease and were evaluated for 7 months, when sacrifice occurred.

    A dose-dependent suppression of full length HTT (both wild-type and mutant) was observed 7 months after a single injection of AAV5-miHTT, with reductions of 92% in the striatum and 64% in the frontal cortex. AAV5 also showed broad distribution at all time points evaluated and was clearly observed in the striatum, the hippocampus and the cortex. These data are consistent with prior studies and further demonstrate that AAV5 undergoes anterograde and retrograde axonal transport following infusion, resulting in broad transduction of the structures affected by Huntington's disease. AAV5-miHTT resulted in both cognitive and psychiatric improvements, as well as the prevention of neuronal degeneration, indicating the potential therapeutic benefit of non-selective HTT lowering. The authors concluded that the Hu128/21 model is not ideal for evaluating therapeutic changes in motor performance as a result of inherently increasing obesity in the mice that likely contributed to progressive motor deficits. The authors did cite improved motor coordination in an R6/2 mouse model that better demonstrates the characteristic weight loss associated with Huntington's disease.

    Tolerability was evaluated using measures of gliosis, brain morphology, body weight and survival. Results demonstrated that non-selective knock down of HTT was safe and well tolerated in the humanized mouse model, even in the absence of background mouse wild-type HTT. Additionally, there were no lateral ventricle size changes observed at any dose tested.

    "The impact on many features of Huntington's disease with the AMT-130 gene therapy approach are encouraging and support its continued assessment in patients with this debilitating disease," stated Michael R. Hayden, Killiam Professor of Medical Genetics at the CMMT and the University of British Columbia, and co-author of the study.

    These findings were further supported by a recently published study in Molecular Therapy - Methods & Clinical Development, on neuronal and astrocytic cell cultures derived from induced pluripotent stem cells (iPSCs) from two Huntington's disease patients with different CAG-repeat lengths.

    Results from this study demonstrated significant non-selective reduction of up to 68% of human HTT in Huntington's disease patient-derived iPSC neurons and astrocytes, with no observed toxicity or off-target effects in gene expression and regulation.

    "These results are very encouraging in the context of AMT-130 as a potential gene therapy for Huntington's disease, where durable and potent suppression of huntingtin protein is needed in the striatum and cortex," stated Sander van Deventer, M.D., Ph.D., EVP research and product development. "Moreover, these studies examine for the first time the tolerability and efficacy of non-selective human HTT lowering and demonstrate the approach is well-tolerated, even in the absence of background wild-type huntingtin protein. The data provide further support for the potential safety and efficacy of AMT-130 and we remain excited to advance our recently initiated Phase I/II clinical trial of AMT-130 in patients with Huntington's disease."

    The publication, "Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease", is available online in the journal Nucleic Acids Research.

    The publication, "AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes", is available online in journal Molecular Therapy - Methods & Clinical Development.

    About AMT-130
    AMT-130 is a gene therapy product candidate consisting of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington's disease.

    About Huntington's Disease
    Huntington's disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete 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 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, hemophilia A, 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 AMT-130 may prove to be clinically safe or efficacious or become a successful treatment for patients suffering from Huntington's disease. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 Quarterly Report on Form 10-Q filed on October 28, 2019. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541
     

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  21. ~ Sustained FIX Activity at Therapeutic Levels1 Up to 50% of Normal at One Year After Administration of Etranacogene Dezaparvovec in Phase IIb Study ~

    ~ Zero Bleeds and No Requirement for Immunosuppression in One Year Following Dosing with Etranacogene Dezaparvovec ~

    ~ Long-term Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 4 Years of Follow-Up ~

    ~ Investor Webcast on Monday, December 9, 2019 at 7:00 a.m. EST ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 08, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated clinical data on the three patients treated in uniQure's…

    ~ Sustained FIX Activity at Therapeutic Levels1 Up to 50% of Normal at One Year After Administration of Etranacogene Dezaparvovec in Phase IIb Study ~

    ~ Zero Bleeds and No Requirement for Immunosuppression in One Year Following Dosing with Etranacogene Dezaparvovec ~

    ~ Long-term Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 4 Years of Follow-Up ~

    ~ Investor Webcast on Monday, December 9, 2019 at 7:00 a.m. EST ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 08, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated clinical data on the three patients treated in uniQure's ongoing Phase IIb study of etranacogene dezaparvovec, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In addition, uniQure presented up to 4 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data are being presented this weekend in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH), taking place in Orlando, Florida.

    At Least One Year of Stable, Therapeutic Levels of FIX Activity in Patients Treated with Etranacogene Dezaparvovec

    The Phase IIb study of etranacogene dezaparvovec is an open-label, single-dose, single-arm, multi-center trial being conducted in the United States. Three patients with severe hemophilia (endogenous Factor IX (FIX) activity less than or equal to one percent) were enrolled in the study and received a single intravenous infusion of 2x1013 gc/kg. Prior to the administration of etranacogene dezaparvovec, all three patients showed low levels of pre-existing neutralizing antibodies to AAV5 but were not excluded from the trial on that basis. The patients in the Phase IIb study were followed for 52 weeks to assess FIX activity, bleeding rates and usage of FIX replacement therapy, and will be monitored for five years to evaluate the safety of etranacogene dezaparvovec.

    Featured in a poster presentation at ASH, the 52 weeks of follow-up data show that all three patients have stabilized and sustained FIX activity at therapeutic levels after the one-time administration of etranacogene dezaparvovec. Mean FIX activity for the three patients at 52 weeks after administration was 41% of normal, with the first patient achieving FIX activity of 50% of normal, the second patient achieving FIX activity of 31% of normal and the third patient achieving FIX activity of 41% of normal. The second and third patients had previously screen-failed and were excluded from another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector. Reported FIX activity was measured using an activated partial thromboplastin time (aPTT) assay performed at a central laboratory.

    At one year after dosing, no patient in the study has reported any bleeding events. All patients have remained free of prophylaxis after receiving etranacogene dezaparvovec. One patient, who had earlier in the study undergone hip surgery due to a pre-existing condition, experienced back pain and treated himself with a single infusion of factor replacement, which was later determined by the patient and the investigator to be unrelated to a bleed. No other FIX infusions were reported by any patient.

    "These updated data show that a single administration of etranacogene dezaparvovec has been well-tolerated now out 52 weeks and has increased FIX activity into the therapeutic range for people living with hemophilia B," stated Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan and a principal investigator in the HOPE-B clinical trial. "These data show a full year of meaningful clinical benefit for all three patients in the study, including durable levels of FIX activity with no bleeds, no requirement for infusions of FIX replacement therapy outside of surgery, and no need for immunosuppression."

    "We are very pleased with these latest results, and continue to believe that etranacogene dezaparvovec has the potential to be the first- and best-in-class gene therapy for patients with hemophilia B," stated Robert Gut, M.D., Ph.D., chief medical officer of uniQure. "We remain focused on dosing all patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and expect to announce top-line data on our primary endpoint of Factor IX activity by the end of 2020."

    Stable FIX Expression and Durable Reductions in Bleeding and FIX Consumption for up to 4 Years Following AMT-060 Gene Therapy

    In the ongoing Phase I/II study of AMT-060, all 10 patients continue to show long-term clinical benefit, including sustained increases in FIX activity, reduced usage of FIX replacement therapy, and decreased bleeding frequency. At up to 4 years of follow-up, AMT-060 continues to be well-tolerated, with no new serious adverse events and no development of inhibitors since the last reported data.

    All five patients in the second dose cohort of 2x1013 gc/kg (the dose being studied in the ongoing Phase III HOPE-B study of etranacogene dezaparvovec) continue to be free of routine FIX replacement therapy at 3.5 years after treatment. Based on the six months of data collected during the fourth year of follow-up, the mean annualized bleeding rate was zero compared to an average of 4 bleeds during the year prior to treatment, representing a 100% reduction. During this same period, the usage of FIX replacement therapy also declined 100% compared to the year prior to treatment. Mean FIX activity over 3.5 years was 7.5%.  

    AMT-060 is uniQure's first-generation gene therapy, consisting of an AAV5 vector carrying a gene cassette with the wild-type FIX gene. uniQure expects that data from this Phase I/II trial of AMT-060 will be part of the regulatory submission for marketing approval of etranacogene dezaparvovec.

    "The Phase I/II study of AMT-060 continues to demonstrate notable long-term tolerability," stated Professor Wolfgang Miesbach, M.D., Ph.D., of the University Hospital Frankfurt in Germany. "We have now demonstrated evidence of durable clinical benefits, including sustained FIX activity, improved disease phenotype and substantial reductions in spontaneous bleeds for up to 4 years following administration. These data have the potential to be very meaningful for hemophilia B patients."

    About Etranacogene Dezaparvovec

    Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure holds multiple issued patents in the United States and Canada broadly covering methods of treating bleeding disorders, including hemophilia B, using AAV gene therapy with the FIX-Padua variant.

    AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the uniQure's AAV5 gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

    About the Pivotal Phase III HOPE-B Study

    The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Sixty-two adult hemophilia B patients classified as severe or moderately severe were enrolled in a six-month observational period, during which time they continued to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients receive a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the HOPE-B pivotal trial was initiated in January 2019.

    The study's primary endpoint is the assessment of Factor IX activity 26 weeks after dosing.  Secondary endpoints include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

    Patients enrolled in the HOPE-B pivotal trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5-based gene therapies may be viable treatments for at least 97% of patients.

    Investor/Analyst Breakfast and Webcast Information

    uniQure will host an investor breakfast and webcast on Monday, December 9, 2019 at 7:00 a.m. EST featuring Dr. Steven Pipe, principal investigator of the HOPE-B clinical trial, to review the updated data on etranacogene dezaparvovec. To access the call by phone, dial 1-866-966-1396 (United States) or +44 20 719 280 00 (international); the conference ID is 41 64 967.

    The webcast may also be accessed through the Investors section of the uniQure's website at http://uniqure.com/investors-newsroom/overview.php. Following the live webcast, a replay of the call will be available for two weeks. 

    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 and partnered gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and cardiovascular 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 will be the first-in-class or best-in-class gene therapy for patients with hemophilia B, whether uniQure will dose all patients in its ongoing HOPE-B pivotal trial, whether uniQure will announce top-line data on the primary endpoint of Factor IX activity in its HOPE-B pivotal trial by the end of 2020 or ever, and whether any clinical data associated with etranacogene dezaparvovec or AMT-060 will be meaningful for hemophilia B patients. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 Quarterly Report on Form 10-Q filed on October 28, 2019. 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
    Eva M. Mulder 
    Direct: +31 20 240 6103
    Mobile: +31 6 52 33 15 79
    Tom Malone
    Direct: 339-970-7558
    Mobile: 339-223-8541


    ____________
    1 Epidemiological data indicate that factor activity above 12% of normal is associated with substantial reduction or elimination of spontaneous bleeds and factor usage. Den Uijl IE et al Haemophilia 2011; 17(6):849-53

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  22. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 02, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in December:

    • Piper Jaffray Healthcare Conference, December 3 – 5, New York City

      ° Matt Kapusta, chief executive officer, will participate in a fireside chat on Tuesday, December 3, at 8:30 a.m. ET.

      ° Members of uniQure's management team will also participate in one-on-one investor meetings throughout the day on Tuesday, December 3.
    • Evercore ISI 2nd Annual HealthCONx Conference, December 3 – 5, Boston, MA

      ° Matt Kapusta will participate in a fireside at…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 02, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in December:

    • Piper Jaffray Healthcare Conference, December 3 – 5, New York City

      ° Matt Kapusta, chief executive officer, will participate in a fireside chat on Tuesday, December 3, at 8:30 a.m. ET.

      ° Members of uniQure's management team will also participate in one-on-one investor meetings throughout the day on Tuesday, December 3.
    • Evercore ISI 2nd Annual HealthCONx Conference, December 3 – 5, Boston, MA

      ° Matt Kapusta will participate in a fireside at 9:30 a.m. ET on Wednesday, December 4

      ° Members of uniQure's management team will also participate in one-on-one investor meetings throughout the day on Wednesday, December 4.

    • CBI's Rare Disease Clinical Development & Access Summit 2019, December 3 – 4, Arlington, VA

      ° Nick Li, senior director of HEOR, will be joining a panel discussion on "Optimize Reimbursement and Access Through Proactive Planning in Clinical Development" on Wednesday, December 4 at 2 p.m. ET. 
    • American Society of Hematology (ASH) Annual Meeting, December 7 – 10, Orlando, Florida

      ° uniQure management will host an investor & analyst breakfast featuring Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, who will review the one-year follow-up data on etranacogene dezaparvovec presented at ASH.

      ° The investor & analyst breakfast will take place on Monday, December 9, from 7:00 a.m. to 8:00 a.m. ET, at the Hilton Orlando, Florida. The meeting will be webcast live along with slides and can be accessed by visiting the investor relations section of the Company's website at www.uniQure.com.
    • Gene Therapy for Neurological Disorders, December 10 – 12, Boston, MA

      ° Sander van Deventer, EVP of research & product development, will present "Novel AAV-delivered Gene Silencing Technologies Targeting the CNS: Imaging of miRNAs & Therapeutic Efficacy in Huntington's Disease & SCA3" on Thursday, December 12 at 9:45 a.m. ET.

    • Lega Italiana Ricerca Huntington, December 14, Rome, Italy

      ° Pavlina Konstantinova, VP of new therapeutic target development, will be presenting "Development of a microRNA-based Gene Therapy for Huntington's Disease" on Saturday, December 14 at 2:30 p.m. CET.

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:

    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558

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  23. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 06, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that six data presentations will be delivered at the 61st American Society of Hematology (ASH) Annual Meeting, taking place in Orlando, Florida.

    "We are looking forward to our strong presence at ASH next month," stated Matt Kapusta, chief executive officer at uniQure. "The breadth of data to be presented at ASH this year represents the continued development of uniQure's industry-leading gene therapy platform and expertise in the hemophilia field." 

    Investor and Analyst Breakfast and Webcast

    uniQure management…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 06, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that six data presentations will be delivered at the 61st American Society of Hematology (ASH) Annual Meeting, taking place in Orlando, Florida.

    "We are looking forward to our strong presence at ASH next month," stated Matt Kapusta, chief executive officer at uniQure. "The breadth of data to be presented at ASH this year represents the continued development of uniQure's industry-leading gene therapy platform and expertise in the hemophilia field." 

    Investor and Analyst Breakfast and Webcast

    uniQure management will host an investor & analyst breakfast featuring Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, (AMT-061), who will review the 12-month follow-up data on etranacogene dezaparvovec (AMT-061) presented at ASH. The investor & analyst breakfast will take place on Monday, December 9, from 7:00 a.m. to 8:00 a.m. ET, at the Hilton Orlando, Florida.

    The meeting will be webcast live along with slides and can be accessed by visiting the investor relations section of the Company's website at www.uniQure.com

    To request attendance at the meeting, please RSVP to by November 30, 2019, as space is limited.

    Specific details on uniQure's presentations at ASH include:

    Title: One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B
    Session Name: 801. Gene Therapy and Transfer: Poster II 
    Date: Sunday, December 8, 2019
    Presentation Time: 6:00 - 8:00 p.m. ET
    Location: Orange County Convention Center, Hall B
       
    Title: Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B
    Session Name: 801. Gene Therapy and Transfer: Poster I
    Date: Saturday, December 7, 2019
    Presentation Time: 5:30 - 7:30 p.m. ET
    Location:  Orange County Convention Center, Hall B
       
    Title: Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population
    Session Name:  801. Gene Therapy and Transfer: Poster II
    Date: Sunday, December 8, 2019
    Presentation Time: 6:00 - 8:00 p.m. ET
     Location: Orange County Convention Center, Hall B
       
    Title: Evaluation of a Blood Coagulation Factor IX Variant that Functions Independently of Factor VIII as an Alternative Treatment of Hemophilia A
    Session Name: 321. Blood Coagulation and Fibrinolytic Factors: Poster I
    Date: Saturday, December 7, 2019
    Presentation Time: 5:30 - 7:30 p.m. ET
    Location: Orange County Convention Center, Hall B
       
    Title: Prophylactic Treatment in People with Severe Hemophilia B in the US: An Analysis of Real-World Healthcare System Costs and Clinical Outcomes
    Session Name: 901. Health Services Research—Non-Malignant Conditions: Poster I
    Date: Saturday, December 7, 2019
    Presentation Time: 5:30 - 7:30 p.m. ET
    Location: Orange County Convention Center, Hall B
       
    Title: Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B
    Session Name: 801. Gene Therapy and Transfer: Poster I
    Date: Saturday, December 7, 2019
    Presentation Time: 5:30 - 7:30 p.m. ET
    Location: Orange County Convention Center, Hall B

    The conference abstracts were made available today and can be accessed through this link: ASH abstracts.

    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, hemophilia A, 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 ability of etranacogene dezaparvovec to be a first-in-class or best-in-class gene therapy for patients with hemophilia B, the ability to add up to six additional patients before the end of September 2019, and whether top-line data from the Phase III trial can be shared next 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 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 Quarterly Report on Form 10-Q filed on July 29, 2019. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541

     

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  24. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 05, 2019 (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 U.S. Patent and Trademark Office has issued U.S. Patent Number 10,465,180. This patent covers adeno-associated virus vectors comprising a nucleic acid encoding FIX-Padua, promoter sequences, transcription termination and control elements. This patent is uniQure's third U.S patent, along with the previously issued U.S. Patent Number 9,982,248 and U.S. Patent Number 9,249,405, in a patent family that uniQure acquired in 2017 from the inventor, Professor Paolo Simioni, a renowned hemophilia expert at the…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 05, 2019 (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 U.S. Patent and Trademark Office has issued U.S. Patent Number 10,465,180. This patent covers adeno-associated virus vectors comprising a nucleic acid encoding FIX-Padua, promoter sequences, transcription termination and control elements. This patent is uniQure's third U.S patent, along with the previously issued U.S. Patent Number 9,982,248 and U.S. Patent Number 9,249,405, in a patent family that uniQure acquired in 2017 from the inventor, Professor Paolo Simioni, a renowned hemophilia expert at the University of Padua, Italy. 

    "uniQure continues to build a significant intellectual property portfolio, including its FIX-Padua patents," said David Cerveny, chief legal officer at uniQure. "These latest claims broaden the scope of uniQure's FIX-Padua patent protection as they relate to any AAV comprising a nucleic acid encoding FIX-Padua together with promoter sequences, transcription termination and control elements. We believe this patent strengthens our intellectual property portfolio and adds further protection to the use of the FIX-Padua in AAV gene therapy to treat hemophilia B."

    The Factor IX Padua variant is a Factor IX protein carrying a leucine at the R338 position, often called the "FIX-Padua" or "Padua mutant." Previously-issued claims focus on the use of AAV gene therapy with FIX-Padua to treat hemophilia B and other bleeding disorders, as well as compositions of FIX-Padua nucleic acids and polypeptides (proteins), as well as their therapeutic uses. 

    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 and partnered gene therapies to treat patients with hemophilia, Huntington's disease and cardiovascular 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 scope or strength of protection provided by our patent portfolio and our ability to have additional patents issue from our current patent applications. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators' clinical development activities, 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 Quarterly Report on Form 10-Q filed on April 30, 2018. 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:

    Maria E. Cantor  Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct:  339-970-7558
    Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:  339-223-8541

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  25. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 01, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in November:

    • Huntington Study Group 2019: Navigating Huntington's Disease, November 7 – 9, Sacramento, California.

      ° 
      Eileen Sawyer, VP of Global Medical Affairs, will give an oral presentation on the study design of our Phase I/II clinical trial of AMT-130 for Huntington's disease on November 7, and will be participating in the "Research Roundup" roundtable on November 8, at 5:30 p.m. PT.

    • Credit Suisse 28th Annual Healthcare Conference, November 11 –

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 01, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in November:

    • Huntington Study Group 2019: Navigating Huntington's Disease, November 7 – 9, Sacramento, California.

      ° 
      Eileen Sawyer, VP of Global Medical Affairs, will give an oral presentation on the study design of our Phase I/II clinical trial of AMT-130 for Huntington's disease on November 7, and will be participating in the "Research Roundup" roundtable on November 8, at 5:30 p.m. PT.

    • Credit Suisse 28th Annual Healthcare Conference, November 11 – 13, Scottsdale, Arizona.

      ° 
      Members of uniQure's management team will attend and participate in one-on-one investor meetings throughout the day on Tuesday, November 12.
    • 10th Annual World Orphan Drug Congress, November 11 – 14, Barcelona, Spain.

      ° 
      Sander van Deventer, EVP Research & Product Development will participate in the panel discussion "Executive Insights: What technologies need to be unlocked? Discussing all the scientific aspects that are impacting the reality of gene therapy" on Wednesday November 13 at 9:55 a.m. CET.

      ° In addition, he will participate in the panel discussion "European Investment Landscape for Cell & Gene Therapy" on Thursday November 14, at 2:40 p.m. CET.
       
    • Barclays Gene Editing & Gene Therapy Summit, November 13, New York City.

      ° 
      Matt Kapusta, chief executive officer will present a corporate update on Wednesday November 13, at 10:15 a.m. ET. Members of uniQure's management team will participate in one-on-one investor meetings throughout the day on Wednesday, November 13.
       
    • World Federation of Hemophilia Global Forum, November 13 – 14, Montreal, Canada

      ° 
      Eileen Sawyer will give an oral presentation on "Interim Results from a Phase 2b trial of Etranacogene Dezaparvovec (AMT-061: AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B", on Thursday November 14, at 9:00 a.m. ET.
       
    • Stifel 2019 Healthcare Conference, November 19 – 20, New York City.

      ° 
      Matt Kapusta will present a corporate update on Tuesday November 19 at 9:30 a.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • 29th Annual Network for European CNS Transplantation and Restoration (NECTAR) Meeting, November 28 – 29, Cardiff, United Kingdom.

      ° 
      Pavlina Konstantinova, VP new therapeutic target development at uniQure, will be presenting "Development of a microRNA-based Gene Therapy for Huntington's Disease ", on Thursday, November 28 at 10:30 a.m. GMT.

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:   
         
    FOR INVESTORS:  FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558

    Primary Logo

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  26. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 31, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced the publication of 26-week interim data from the ongoing Phase IIb clinical trial of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B, in the journal, Blood Advances.

    With a combined follow-up of 78 weeks of observation on the three adult male participants with severe hemophilia B, the mean Factor IX (FIX) activity was 47 percent of normal (range of 33 percent to 57 percent of normal) following a single administration of etranacogene dezaparvovec (AMT-061). Consistent with the observed…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 31, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced the publication of 26-week interim data from the ongoing Phase IIb clinical trial of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B, in the journal, Blood Advances.

    With a combined follow-up of 78 weeks of observation on the three adult male participants with severe hemophilia B, the mean Factor IX (FIX) activity was 47 percent of normal (range of 33 percent to 57 percent of normal) following a single administration of etranacogene dezaparvovec (AMT-061). Consistent with the observed Factor IX activity, the treatment was associated with a complete cessation of bleeds up to the 26-week interim endpoint, and no patient in the study required any infusions of FIX replacement therapy during this time.

    In the year prior to administration of etranacogene dezaparvovec, all three participants received prophylactic FIX replacement, as well as additional doses of FIX to treat bleeding events. Medical records showed that the 3 participants experienced a total of 9 bleeds in the year prior to treatment despite routine FIX prophylaxis. 

    Etranacogene dezaparvovec was generally well tolerated, with no clinically significant elevations of liver enzymes or inflammatory markers observed, and no use of corticosteroids related to treatment required.

    "In individuals with severe and moderately severe hemophilia B, etranacogene dezaparvovec resulted in clinically-relevant increases in FIX activity, cessation of bleeds and abrogated the need for FIX replacement, despite the presence of pre-existing anti-AAV5 neutralizing antibodies detected by the highly sensitive luciferase assay," stated Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan and an investigator in the Phase IIb clinical trial. "While these observations are limited to three participants, the consistency of results among participants supports the ongoing pivotal Phase III HOPE B trial which will further assess the safety and efficacy of etranacogene dezaparvovec. If these results are confirmed by the Phase III trial, physicians will be provided with increased confidence that reliable clinical outcomes can be achieved following gene transfer."

    "This publication supports the potential of etranacogene dezaparvovec to substantially improve the quality of life for hemophilia B patients through a one-time administration that results in sustained Factor IX activity and may result in a cessation of bleeding episodes," stated Robert Gut, M.D., Ph.D., chief medical officer at uniQure and co-author of the paper. "We are pleased to have these interim safety and efficacy data of etranacogene dezaparvovec published in a peer-reviewed journal and look forward to sharing top-line data from the pivotal Phase III HOPE-B study in 2020."

    The Company has completed patient enrollment in the HOPE-B pivotal trial and expects top-line data by the end of next year.

    "Etranacogene dezaparvovec (AMT-061 Phase 2b): Normal/Near Normal FIX Activity and Bleed Cessation in Hemophilia B," is available on-line in the journal, Blood Advances

    About Etranacogene Dezaparvovec (AMT-061)

    Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company's AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

    About the Pivotal Phase III HOPE-B Study

    The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Adult hemophilia B patients classified as severe or moderately severe are enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will receive a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the HOPE-B pivotal trial was initiated in January 2019.

    The study's primary endpoint is the assessment of Factor IX activity 26 weeks after dosing. Secondary endpoints include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

    Patients enrolled in the HOPE-B pivotal trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5-based gene therapies may be viable treatments for at least 97% of patients.

    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, hemophilia A, 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 ability of etranacogene dezaparvovec to substantially improve the quality of life for hemophilia B patients through a one-time administration or to result in sustained Factor IX activity or to result in a cessation of bleeding episodes, whether the Phase IIb or Phase III trial data or any data will provide an increased confidence that reliable clinical outcomes can be achieved following gene transfer, and whether top-line data from the Phase III trial can be shared in 2020 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 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 Quarterly Report on Form 10-Q filed on July 29, 2019. 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
     
    Eva M. Mulder
    Direct: +31 20 240 6103
    Mobile: +31 6 52 33 15 79 
     
    Tom Malone
    Direct: 339-970-7558
    Mobile: 339-223-8541
     

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  27. ~ Announced Completion of Full Patient Enrollment in HOPE-B Pivotal Study of Etranacogene Dezaparvovec ~

    ~ Achieved Site Initiation and Began Patient Screening for Phase I/II Clinical Study of AMT-130 in Huntington's Disease ~

    ~Strong Balance Sheet with September 30, 2019 Cash and Cash Equivalents of $403 Million Expected to Fund Operations into Mid-2022 ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 28, 2019 (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 third quarter of 2019 and highlighted recent progress across its business.

    "We achieved a major company milestone for 2019 with…

    ~ Announced Completion of Full Patient Enrollment in HOPE-B Pivotal Study of Etranacogene Dezaparvovec ~

    ~ Achieved Site Initiation and Began Patient Screening for Phase I/II Clinical Study of AMT-130 in Huntington's Disease ~

    ~Strong Balance Sheet with September 30, 2019 Cash and Cash Equivalents of $403 Million Expected to Fund Operations into Mid-2022 ~

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 28, 2019 (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 third quarter of 2019 and highlighted recent progress across its business.

    "We achieved a major company milestone for 2019 with the earlier than expected completion of full patient enrollment in the HOPE-B pivotal study of etranacogene dezaparvovec in hemophilia B, as well as the initiation of clinical sites and patient screening for the Phase I/II clinical study of AMT-130 in Huntington's disease," stated Matt Kapusta, chief executive officer of uniQure. "We also strengthened our balance sheet with new capital that we expect will fund operations into mid-2022 and allows us to deliver on key corporate goals across the business.  We look forward to continuing our momentum through the remainder of the year and sharing our progress during what we believe will be a very productive 2020."

    Recent Company Progress

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

      • In early September 2019, the Company announced the achievement of patient enrollment in its HOPE-B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B. Due to the high level of interest in the study from both patients and study investigators, the study was over-enrolled with 6 patients, providing a cumulative enrollment of 62 patients across 39 sites in the United States and Europe.  

      • With the achieved enrollment of the HOPE-B pivotal study, the Company expects to provide top-line Factor IX (FIX) activity data on all patients before the end of 2020, and to submit a Biologics License Application (BLA) for marketing authorization of etranacogene dezaparvovec in 2021.

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

      • The Company continues to make important progress with its dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington's disease. Several clinical sites are now open for enrollment and patient screening is underway. The Company expects to enroll the first sentinel patient later this year or in early 2020.

    • Advancing research pipeline of gene therapy candidates towards the clinic

      • Numerous preclinical and nonclinical studies of AMT-180 for the treatment of hemophilia A are underway to support the submission of a clinical trial application next year. AMT-180 incorporates a proprietary, exclusively licensed, modified Factor IX gene that has been demonstrated in preclinical studies to convey Factor VIII-independent activity and circumvent inhibitors to Factor VIII.

      • The Company continues to progress preclinical studies of AMT-150 for the treatment of spinocerebellar ataxia type 3. uniQure recently selected its lead AMT-150 candidate and expects to initiate IND-enabling studies in the first half of 2020. In May 2019, the Company presented data from a preclinical study demonstrating that a single administration of AMT-150 in the cerebrospinal fluid resulted in strong AAV transduction and significant mutant ataxin-3 lowering of up to 65%. 

      • The Company is currently conducting preclinical studies of AMT-190 for the treatment of Fabry disease and expects to select a lead candidate in 2020. AMT-190 incorporates a proprietary, exclusively licensed, modified NAGA (ModNAGA) variant that may have several advantages over other therapies for Fabry disease, including higher stability in blood, circumvention of inhibitors, better biodistribution in the target organs, secondary toxic metabolite reduction and improved cross-correction of neighboring cells.

    • Successful follow-on offering funding operations into mid-2022

      • In September 2019, the Company successfully completed the public follow-on offering of 5.6 million ordinary shares at a public offering price of $46.00 per ordinary share, raising approximately $242.8 million in net proceeds.

      • As of September 30, 2019, the Company held $403.2 million in cash and cash equivalents, positioning the Company to complete the clinical development of etranacogene dezaparvovec in hemophilia B, begin preparations for commercial launch, to fund the Phase I/II clinical study of AMT-130 in Huntington's disease as well as the further development of our other preclinical product candidates.

    Upcoming Investor and Industry Events

    • World Orphan Drug Congress, November 11 – 14, 2019, Washington, D.C.
       
    • Credit Suisse Healthcare Conference, November 12 – 13, 2019, Phoenix, AZ.
       
    • Barclays Gene Therapy Summit, November 13, 2019, New York City, NY.
       
    • 2019 International Ataxia Research Conference, November 13 – 16, 2019, Washington, D.C.
       
    • Stifel Global Healthcare Conference, November 19 – 20, 2019, New York City, NY.
       
    • 29th Annual NECTAR Meeting, November 28 -29, 2019, Cardiff, UK.
       
    • Piper Jaffray, 31st Annual Healthcare Conference, December 3, 2019, New York City, NY.
       
    • Evercore ISI 2nd Annual HealthCONx Conference, December 4, 2019, Boston, MA.

    Financial Highlights

    Cash Position: As of September 30, 2019, the Company held cash and cash equivalents of $403.2 million, compared to $184.1 million as of June 30, 2019. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.

    Revenues: Revenue for the three months ended September 30, 2019 was $1.0 million, compared to $3.1 million during the same period 2018. The decrease reflects the October 2018 termination of the development of S100A1 under our collaboration with Bristol-Myers Squibb.

    R&D Expenses: Research and development expenses were $23.6 million for the three months ended September 30, 2019, compared to $20.5 million during the same period 2018. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec, the planned Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site.

    SG&A Expenses: Selling, general and administrative expenses were $8.9 million for three months ended September 30, 2019, compared to $5.9 million during the same period 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.

    Net Loss: The net loss for the three months ended September 30, 2019 was $23.6 million, or $0.58 per share, compared to $22.0 million, or $0.59 per share during the same period 2018.

    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, hemophilia A, 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, our ability to provide further clinical updates and data with respect to  our pivotal Phase III study of etranacogene dezaparvovec before the end of 2020 or ever, the ability to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies, our ability to enroll the first sentinel patient in our AMT-130 Phase I/II clinical study in early 2020 or ever, and our ability to fund our operations into mid-2020. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with 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 October 28, 2019. 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 
    Eva M. Mulder 
    Direct: +31 20 240 6103
    Mobile: +31 6 52 33 15 79
     
    Tom Malone
    Direct: 339-970-7558
    Mobile:339-223-8541



    uniQure N.V.

    UNAUDITED CONSOLIDATED BALANCE SHEETS

      September 30,   December 31,  
    2019   2018  
           
      (in thousands, except share and per share amounts)  
    Current assets        
    Cash and cash equivalents $ 403,163   $ 234,898  
    Accounts receivable and accrued income from related party   101     233  
    Prepaid expenses   3,820     1,116  
    Other current assets   558     329  
    Total current assets   407,642     236,576  
    Non-current assets        
    Property, plant and equipment, net of accumulated depreciation of $26.9 million as of September 30, 2019, and $22.9 million as of December 31, 2018, respectively.   28,278     29,179  
    Operating lease right-of-use assets   26,852     -  
    Intangible assets, net   5,424     5,201  
    Goodwill   482     506  
    Restricted cash   2,914     2,444  
    Total non-current assets   63,950     37,330  
    Total assets $ 471,592   $ 273,906  
    Current liabilities        
    Accounts payable $ 3,782   $ 3,792  
    Accrued expenses and other current liabilities   12,702     8,232  
    Current portion of operating lease liabilities   4,436     -  
    Current portion of deferred rent   -     311  
    Current portion of deferred revenue   7,527     7,634  
    Total current liabilities   28,447     19,969  
    Non-current liabilities        
    Long-term debt   35,928     35,471  
    Operating lease liabilities, net of current portion   31,242     -  
    Deferred rent, net of current portion   -     8,761  
    Deferred revenue, net of current portion   23,882     28,861  
    Derivative financial instruments related party   1,354     803  
    Other non-current liabilities   507     435  
    Total non-current liabilities   92,913     74,331  
    Total liabilities   121,360   $ 94,300  
    Total shareholders' equity   350,232     179,606  
    Total liabilities and shareholders' equity $ 471,592   $ 273,906  
       


    uniQure N.V.

    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

      Three months ended September 30,
                   
                   
        2019       2018  
       
      in thousands, except share and per share amounts
    Total revenues $ 1,046     $ 3,148  
    Operating expenses:      
    Research and development expenses   (23,554 )     (20,541 )
    Selling, general and administrative expenses   (8,929 )     (5,898 )
    Total operating expenses   (32,483 )     (26,439 )
    Other income   453       557  
    Other expense   (342 )     (490 )
    Loss from operations   (31,326 )     (23,224 )
    Non operating items, net   7,722       1,157  
    Loss before income tax expense   (23,604 )     (22,067 )
    Income tax expense   -       32  
    Net loss $ (23,604 )   $ (22,035 )
           
    Basic and diluted net loss per ordinary share $ (0.58 )   $ (0.59 )
    Weighted average shares used in computing basic and diluted net loss per ordinary share   40,738,938       37,247,193  
           

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  28. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 27, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in October:

    • 2019 Cantor Global Healthcare Conference, October 2 – 4, New York City.

      °  
      Matt Kapusta, chief executive officer at uniQure, will present a corporate update on Wednesday, October 2, at 9:30 a.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • 2019 Cell & Gene Meeting

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 27, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in October:

    • 2019 Cantor Global Healthcare Conference, October 2 – 4, New York City.

      °  
      Matt Kapusta, chief executive officer at uniQure, will present a corporate update on Wednesday, October 2, at 9:30 a.m. ET. The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • 2019 Cell & Gene Meeting on the Mesa, October 2 – 4, Carlsbad, California.

      °  
      Jonathan Garen, chief business officer at uniQure, will participate in a panel discussion "Cell and Gene Therapy for Neurological Product Indications" on Wednesday October 2, at 4:00 p.m. PT.
      °  Mr. Garen also will present a corporate update on Wednesday, October 2, at 2:45 p.m. PT.
       
    • National Hemophilia Foundation 71st Bleeding Disorders Conference, October 3 – 5, Anaheim, California.

      °  
      uniQure will deliver the following presentations during the conference:

          •  Title: No Evidence of Germline Transmission of Vector DNA Following Intravenous Administration of AAV5-hFIX to Male Mice
              
      Abstract number: CRA21
              Presentation date: Friday, October 4
              Presentation time: 5:15 – 6:15 p.m. PT

          •  Title: AMT-061 (AAV5-Padua hFIX variant) an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Follow-up up to 9 Months in a Phase 2b trial
              Abstract number: QOL29
              Presentation date: Friday, October 4
              Presentation time: 5:15 – 6:15 p.m. PT

          •  Title: An Evaluation of Health Utility and Quality-of-Life in Hemophilia: A Systematic Literature Review
              Presentation date: Friday, October 4
              Presentation time: 5:15 – 6:15 p.m. PT
    • European Huntington Association 2019 Conference, October 4 – 6, Bucharest, Romania.

      °  
      Melvin Evers, Ph.D., associate director research at uniQure, will be presenting on the Company's gene therapy candidate AMT-130 for Huntington's disease on Sunday October 6, at 10:30 a.m. CET.
      °  Dr. Evers will also participate in a panel discussion at the conclusion of the presentation session "Drug Trials in Huntington's Disease: What is Happening Right Now?" at 11:20 a.m. CET.
       
    • Chardan 3rd Annual Genetic Medicines Conference, October 7 – 8, New York City

      °  
      Matt Kapusta will participate in a fireside chat with research analyst Gbola Amusa on Tuesday, October 8, at 11:00 a.m. ET. The live webcast of the fireside chat can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • European Society for Gene and Cell Therapy 2019 Collaborative Congress, October 22 – 25, Barcelona, Spain.

      °  
      uniQure will deliver the following presentations during the conference:

            Title: A Novel AAV-Based miQURE Gene Therapy for Spinocerebellar Ataxia Type 3
              
      Abstract number: P247
              Presentation date: Wednesday, October 23
              Presentation time: 1:00 – 2:30 p.m. CET

            Title: Stem Cell Derived Brain Organoids, a Promising Model to Study Adeno Associated Viruses for CNS Gene Therapy
              
      Abstract number: P381
              Presentation date: Wednesday, October 23
              Presentation time: 1:00 – 2:30 p.m. CET
              
          •  Title: Prevalence and Affinity/Avidity Assessment of Pre-Existing NABs Against AAV2, 5 and 8 Analyzed in the Serum of 300 Healthy Donors
              
      Abstract number: P336
              Presentation date: Thursday, October 24
              Presentation time: 1:00 – 2:30 p.m. CET

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:

    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor  Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct:  339-970-7558

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  29. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 05, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs today announced the pricing of its underwritten public offering of 4,891,305 of its ordinary shares at a public offering price of $46.00 per share. The gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and estimated offering expenses payable by uniQure, are expected to be approximately $225 million. All ordinary shares to be sold in the offering are being sold by uniQure. In addition, uniQure has granted to the underwriters a 30-day option to purchase up to 733,695 additional…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 05, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs today announced the pricing of its underwritten public offering of 4,891,305 of its ordinary shares at a public offering price of $46.00 per share. The gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and estimated offering expenses payable by uniQure, are expected to be approximately $225 million. All ordinary shares to be sold in the offering are being sold by uniQure. In addition, uniQure has granted to the underwriters a 30-day option to purchase up to 733,695 additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is expected to close on or about September 10, 2019, subject to the satisfaction of customary closing conditions.

    Goldman Sachs & Co. LLC and SVB Leerink are acting as joint book-running managers for the offering. Stifel is also acting as a book-running manager for the offering. Cantor Fitzgerald & Co. and SunTrust Robinson Humphrey are acting as co-lead managers and H.C. Wainwright & Co. is acting as the co-manager for the offering.

    The securities described above are being offered by uniQure pursuant to its automatically effective shelf registration statement on Form S-3 (File No. 333-225636) filed with the Securities Exchange Commission (the "SEC") on June 14, 2018.  A preliminary prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC and a final prospectus supplement and the accompanying prospectus relating to this offering will be filed with the SEC.  The preliminary prospectus supplement and accompanying prospectus is available, and the final prospectus supplement and accompanying prospectus will be available, for free on the SEC's website at http://www.sec.gov.  When available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained from Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 212-902-9316 or by emailing , SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525 ext. 6132 or by email at , and Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, CA 94104, by telephone at (415) 364-2720 or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities, in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement.

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.

    Forward-Looking Statement

    This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, statements regarding our expectations of market conditions, the satisfaction of customary closing conditions related to the public offering, and other statements including the words "may," "will," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and the completion of the public offering on the anticipated terms, or at all, continued interest in our rare disease portfolio, the ability to develop our product candidates and technologies, the impact of changes in the financial markets and global economic conditions, and other risks as are set forth in uniQure's Quarterly Report on Form 10-Q filed with the SEC on July 29, 2019. uniQure undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    uniQure Contacts

    For Investors:                                                                                          For Media:

    Maria Cantor                                 Eva M. Mulder                                   Tom Malone
    Direct : +339-970-7536                  Direct: +31 20 240 6103                     Direct: 339-970-7758
    Mobile: 617-680-9452                    Mobile: +31 6 52 33 15 79                  Mobile: 339-223-8541
                                          

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  30. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 03, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September.  Etranacogene dezaparvovec…

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Sept. 03, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September.  Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.

    "We are extremely pleased to reach this important milestone in our ongoing development of etranacogene dezaparvovec, which we believe has the potential to be the first and best-in-class gene therapy for patients with hemophilia B," stated Matt Kapusta, chief executive officer of uniQure. "We appreciate the tremendous support from the hemophilia patient community in achieving this important goal ahead of schedule and look forward to sharing top-line data from the Phase III trial, which we expect to do next year."

    The pivotal Phase III HOPE-B study builds on the success of the interim 36-week results of the Company's ongoing Phase IIb study of etranacogene dezaparvovec, which demonstrated that a single administration of the investigational gene therapy resulted in sustained increases in Factor IX (FIX) levels up to 54% of normal, and a mean FIX level of 45% of normal. During that time, no patient reported any bleeding events or required any infusion of FIX replacement therapy for bleeds or experienced any material loss of FIX activity. Additionally, an ongoing Phase I/II study of AMT-060, the Company's first-generation gene therapy for the treatment of hemophilia B, demonstrated that all 10 patients continue to show sustained and stable increases in FIX activity and long-term clinical benefits at up to 3.5 years of observation.

    "We are very excited and proud to have achieved the targeted patient enrollment in the HOPE-B study in just a little more than one year from study initiation," added Robert Gut, M.D., Ph.D., chief medical officer at uniQure.  "This multi-center, multinational trial involves 39 clinical sites across 9 countries, and highlights the outstanding effort and passion of our clinical operations, clinical development, medical affairs and project management teams.  We would like to thank all study participants, advisors, primary investigators and the whole study staff in the United States and Europe for their great contribution and support."

    About the Pivotal Phase III HOPE-B Study

    The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Adult hemophilia B patients classified as severe or moderately severe are enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients will receive a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the HOPE-B pivotal trial was initiated in January 2019.

    The study's primary endpoint is the assessment of Factor IX activity 26 weeks after dosing.  Secondary endpoints include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

    Patients enrolled in the HOPE-B pivotal trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers. Previous studies performed by uniQure suggest that AAV5-based gene therapies may be viable treatments for at least 97% of patients.

    About Etranacogene Dezaparvovec (AMT-061)

    Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company's AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

    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, hemophilia A, 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 ability of etranacogene dezaparvovec to be a first-in-class or best-in-class gene therapy for patients with hemophilia B, the ability to add up to six additional patients before the end of September 2019, and whether top-line data from the Phase III trial can be shared next 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 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 Quarterly Report on Form 10-Q filed on July 29, 2019. 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 Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
    Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541

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  31. LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Aug. 30, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in September:

    • Citi 14th Annual Biotech Conference, September 4 – 5, Boston, MA.

       Matt Kapusta, chief executive officer at uniQure, and  Sander van Deventer, M.D., Ph.D., executive vice president research and product development, will attend and participate in investor meetings at the conference on Wednesday September 4.
      • Mr. Kapusta also will participate in the panel discussion "Peering Into the Gene Therapy Crystal Ball - What Does the Future Look

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Aug. 30, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in September:

    • Citi 14th Annual Biotech Conference, September 4 – 5, Boston, MA.

       Matt Kapusta, chief executive officer at uniQure, and  Sander van Deventer, M.D., Ph.D., executive vice president research and product development, will attend and participate in investor meetings at the conference on Wednesday September 4.
      • Mr. Kapusta also will participate in the panel discussion "Peering Into the Gene Therapy Crystal Ball - What Does the Future Look Like?" that same day at 10:15 a.m. ET. The live webcast of the panel discussion can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • Wells Fargo 2019 Healthcare Conference, September 4 – 5, Boston, MA. 

      • Matt Kapusta and Sander van Deventer will attend and participate in investor meetings at the conference on Thursday, September 5.
      • Mr. Kapusta also will present a corporate update on Thursday, September 5, at 10:20 a.m. ET.   The live webcast of the corporate update can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • Morgan Stanley 17th Annual Global Healthcare Conference, September 9 – 11, New York City.

      • Matt Kapusta will host investor meetings at the conference on Monday, September 9, and also participate in a fireside chat at 8:10 a.m. ET. The live webcast of the panel discussion can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
       
    • National Hemophilia Foundation (NHF) 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia, September 13 – 14, Washington, DC.

       On Friday, September 13 at 9:15 a.m. ET, Eileen Sawyer, Ph.D., vice president of medical affairs, will present "Recent Progress in the Development Program of AMT-061 for Persons with Severe or Moderately Severe Hemophilia B."

      • At 2:00 p.m. ET on Friday, September 13, Sander van Deventer will present "Overcoming pre-existing immunity".
       
    • The 5th Animal Models of Neurodegenerative Diseases, September 15 – 18, Chateau Liblice, CZ.

      • On Monday September 16, Melvin Evers, Ph.D., associate director research at uniQure, will  present "The development of microRNA-based gene therapy for Huntington's disease."

      • 
      Astrid Valles-Sanchez, Ph.D., senior scientist at uniQure, will  present "Translational efficacy measures of huntingtin lowering in small and large animal models of Huntington's disease" on Tuesday September 17.
       
    • International Congress of Parkinson's Disease and Movement Disorders – MDS 2019, September 22 -26, 2019, Nice, FR. 

      • uniQure will deliver the following presentations on the development of AMT-130 for Huntington's disease during the conference:


      • Title: MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates
        Abstract number: 18
        Presentation Date: Monday, September 23
        Presentation time: 1:45 – 3:15 p.m. CET

      • Title: Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington's Disease Patients
        Abstract number: 14
        Presentation Date: Monday, September 23
        Presentation time: 1:45 – 3:15 p.m. CET

      • Title: Sustained Mutant Huntingtin Lowering in the Brain and Cerebrospinal Fluid of Huntington's Disease Minipigs Mediated by AAV5-miHTT Gene Therapy
        Abstract number: 53
        Presentation Date: Monday, September 23
        Presentation time: 1:45 – 3:15 p.m. CET

      • Title: Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntington's Disease
        Abstract number: 25
        Presentation date: Monday, September 23
        Presentation time: 1:45 – 3:15 p.m. CET                               

    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, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Contacts:     
         
    FOR INVESTORS:   FOR MEDIA:
         
    Maria E. Cantor Eva M. Mulder Tom Malone
    Direct: 339-970-7536 Direct: +31 20 240 6103 Direct:  339-970-7558

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