QURE uniQure N.V.

29.42
+0.06  (+0%)
Previous Close 29.36
Open 29.17
52 Week Low 25.8
52 Week High 52.19
Market Cap $1,355,478,634
Shares 46,073,373
Float 45,794,257
Enterprise Value $766,324,550
Volume 191,796
Av. Daily Volume 389,687
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Upcoming Catalysts

Drug Stage Catalyst Date
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B
Phase 3
Phase 3
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AMT-130
Huntington's disease
Phase 1/2
Phase 1/2
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Latest News

  1. ~ Expanded gene therapy pipeline with four new research programs and announced acquisition
    of Corlieve Therapeutics ~

    ~ Presented positive 52-week data from HOPE-B pivotal study demonstrating sustained increases in
    Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal and significant reductions in bleeding events ~

    ~ Completed its global commercialization and license transaction with CSL Behring
    for hemophilia B gene therapy program ~

    ~ Initiated patient enrollment in second dose cohort of Phase I/II clinical trial of AMT-130 for Huntington's
    disease, with initiation of a European Phase Ib/II clinical trial expected in second half of 2021 ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 26, 2021 (GLOBE NEWSWIRE…

    ~ Expanded gene therapy pipeline with four new research programs and announced acquisition

    of Corlieve Therapeutics ~

    ~ Presented positive 52-week data from HOPE-B pivotal study demonstrating sustained increases in

    Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal and significant reductions in bleeding events ~

    ~ Completed its global commercialization and license transaction with CSL Behring

    for hemophilia B gene therapy program ~

    ~ Initiated patient enrollment in second dose cohort of Phase I/II clinical trial of AMT-130 for Huntington's

    disease, with initiation of a European Phase Ib/II clinical trial expected in second half of 2021 ~

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 26, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2021 and highlighted recent progress across its business.

    "We had a highly productive second quarter of this year marked by several notable achievements, including the closing on our global commercial licensing agreement with CSL Behring in hemophilia B and the recent presentation of 52-week data on all patients in our HOPE-B pivotal study showing durable and sustained increases in Factor IX levels within the non-hemophiliac range1, as well as significant reductions in both bleeding events and usage of replacement therapy after a one-time administration of etranacogene dezaparvovec," stated Matt Kapusta, chief executive officer at uniQure. "Additionally, we continued to make strong progress in our U.S. Phase I/II study of AMT-130 in Huntington's disease, including the completion of patient enrollment in the first dose cohort and the initiation of dosing in the second cohort."

    "We also recently hosted a Research and Development Day where we disclosed four new promising research programs that complemented our expanding focus in neurological disease and larger patient populations, including the acquisition of Corlieve Therapeutics and its gene therapy program targeting temporal lobe epilepsy that builds on our expanding focus in serious neurological diseases," he added. "These investments deliver on our commitment to leverage our strong cash position to invest in new research programs and enabling technologies with the potential to drive significant value. In the second half of 2021, we expect to continue our progress across all programs, including availability of 78-week data from the HOPE-B study, initiating patient enrollment for our open-label European study of AMT-130 in Huntington's disease, and announcing initial data on the first four randomized Huntington's disease patients in the ongoing U.S. Phase I/II study."

    1 Srivastava et al, Haemophilia. 2020;26(Suppl 6):1–158.

    Recent Company Progress

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



      • In June 2021, the Company announced initial 52-week post-administration Factor IX data and annualized bleeding rates (ABR) in the HOPE-B pivotal trial of etranacogene dezaparvovec in severe to moderately severe hemophilia B patients. Sustained increase in FIX levels was observed with a mean FIX activity of 41.5% of normal compared to 39.0% at 26-weeks post administration. During the 52-week period after dosing, patients reported an 80% reduction in total bleeding events requiring treatment, an 85% reduction in spontaneous bleeding events requiring treatment, and a 96% reduction in annualized usage of FIX replacement therapy.



      • The Company expects all patients in the HOPE-B clinical trial to complete their 78-week follow-up visits by the end of the third quarter of 2021, and expects the BLA will be submitted in the first quarter of 2022.



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

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



      • In May 2021, the Company announced the completion of patient enrollment of the first dose cohort of ten patients in its ongoing Phase I/II study of AMT-130 in Huntington's disease. The U.S. study is a multi-center, double-blinded, randomized and controlled trial to assess the safety and efficacy of AMT-130. The Data Safety Monitoring Board recommended proceeding with patient enrollment in the second dose cohort after reviewing nine-month safety data from the first two enrolled patients, six-month safety data from the next two enrolled patients, and the 30-day safety data from the remaining six patients.



      • In June 2021, the Company announced the first two patient procedures were completed in the second dose cohort of Phase I/II clinical trial. Enrollment of the next two patient procedures will take place after a planned DSMB meeting expected to be held later in the third quarter of 2021.



      • Initial data from the first four enrolled patients in this first-in-human AAV-gene therapy trial are anticipated by year-end 2021. The Company also remains on track to initiate an open label, Phase Ib/II clinical study of AMT-130 in Europe with enrollment beginning in the second half of 2021.

    • Expanding the pipeline and strengthening the platform



      • In June, the Company announced the planned acquisition of Corlieve Therapeutics, a pre-clinical gene therapy company based in France. Corlieve's lead program, now designated as AMT-260, employs miRNA silencing technology to suppress aberrantly expressed kainite receptors in the hippocampus of patients with temporal lobe epilepsy (TLE). TLE represents a large unmet clinical need affecting approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery. The acquisition is expected to close in the third quarter of 2021.



      • In June, the Company hosted a virtual Research & Development Day to highlight new investments in its expanding gene therapy pipeline focusing on central nervous system and rare, liver-directed disorders, as well as new advancements in platform technology and manufacturing. Three new, internally developed pre-clinical programs were announced in Parkinson's disease (AMT-210), amyotrophic lateral sclerosis (ALS) (AMT-160), and autosomal dominant Alzheimer's disease (AMT-240). Additional highlights from the event included updates on the clinical development of AMT-130 in Huntington's disease and advancements in the ongoing preclinical development of AMT-150 for spinocerebellar ataxia 3 and AMT-190 for Fabry disease.



      • In February 2021, the Company began an expansion of the Amsterdam site to include a cleanroom capable of manufacturing cGMP materials at a 500-liter scale and additional laboratories to support the expansions of research and development activities.

    • Expansion of the leadership team



      • In May 2021, Pierre Caloz joined the leadership team as Chief Operating Officer overseeing all manufacturing operations, global CMC development and innovation, supply chain and facilities. Mr. Caloz joins the Company with nearly 20 years of global operations experience in the biopharma industry, including CSL Behring, Merck-Serono, Abgenix and Amgen.



      • On June 15, 2021, Christian Klemt was appointed as Chief Financial Officer. Mr. Klemt was the Company's Chief Accounting Officer from August 2017 to June 2021, and he will continue to serve as general manager of the Amsterdam site.

    • Strong cash position to advance the Company's programs



      • As of June 30, 2021, the Company's cash position was $677.3 million. The Company expects cash and cash equivalents will be sufficient to fund operations into the first half of 2024, excluding any of the $1.6 billion in future milestones or royalties on net sales the Company may receive under the CSL Behring Agreement. The receipt of approximately $300 million in milestone payments relates to regulatory submissions, first commercial sales and certain development milestones would be expected to extend the cash runway by approximately 18 months.

    Upcoming Investor Events (each to be conducted virtually)

    • Citi's Annual Biopharma Virtual Conference, September 8-10, 2021
    • Wells Fargo Virtual Healthcare Conference, September 9-10, 2021
    • SVB Leerink CybeRx Series Event, September 21-23, 2021
    • Cantor Fitzgerald Virtual Global Healthcare Conference, September 27-29, 2021
    • Chardan's 5th Annual Genetic Medicines Conference, October 4-5, 2021

    Financial Highlights

    Cash Position: As of June 30, 2021, the Company held cash and cash equivalents of $677.3 million, compared to $244.9 million as of December 31, 2020. Upon the CSL Behring Agreement becoming fully effective on May 6, 2021, the Company received $462.4 million of payments. Additionally, in January 2021, the Company and Hercules entered into an amended debt facility agreement increasing the aggregate principle to up to $135.0 million, of which the Company drew down an additional $35.0 million for a total of $70.0 million outstanding under the facility as of June 30, 2021. In March 2021, the Company entered into an Open Market Sale Agreement with SVB Leerink LLC providing for the sale of up to $200.0 million of its ordinary shares from time to time in ‘at-the-market' offerings under a shelf registration statement. Pursuant to this agreement, as of April 30, 2021, the Company had sold 921,730 ordinary shares for gross proceeds of approximately $29.6 million.

    Revenues: Revenue for the three months ended June 30, 2021, were $463.9 million, compared to $1.5 million during the same period in 2020. The increase is a result of $462.4 million of license revenue recognized from the CSL Behring Agreement.

    Cost of Contract Revenues: Cost of contract revenues for the three months ended June 30, 2021, was $23.2 million compared to nil for the same period in 2020. Costs incurred in 2021 are associated with the license revenue recognized under the CSL Behring Agreement.

    R&D Expenses: Research and development expenses were $32.8 million for the three months ended June 30, 2021, compared to $28.4 million during the same period in 2020. The change was primarily related to recruitment of personnel to support the development of product candidates, advancing the clinical developments of Huntington's disease, and increased activities associated with preclinical product candidates.

    SG&A Expenses: Selling, general and administrative expenses were $17.3 million for the three months ended June 30, 2021, compared to $11.5 million during the same period in 2020. The change was primarily related to costs incurred for financial advisory fees in relation to the licensing transaction with CSL Behring, increases in personnel and consulting expenses to support growth, and increased share-based compensation.

    Other non-operating items, net: Other income, net was $4.7 million for the three months ended June 30, 2021, compared to other expense, net of $4.3 million during the same period in 2020. The increase in income is primarily due to foreign currency gains in the current period compared to a net foreign currency loss in the same period in 2020.

    Net Loss: The net income for the three months ended June 30, 2021, was $399.5 million, or $8.68 basic net income per share and $8.51 diluted net income per share, compared to a loss of $42.6 million, or $0.96 basic and diluted loss per share during the same periods in 2020.

    About uniQure

    uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Forward-Looking Statements

    This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, uniQure's expectations its plans to announce data from the HOPE-B pivotal trial, patient enrollment in the second cohort of AMT-130, the initiation of an open-label European study of AMT-130, the announcement of data on the first four patients in the Phase I/II study of AMT-130, the submission of the regulatory filing for marketing approval of etranacogene dezaparvovec, the potential financial compensation we may be paid pursuant to our agreement with CSL Behring, or other updates on the research pipeline, and our plans to advance or expand our pipeline, accelerate research, identify business development opportunities, invest in technology, or scale our manufacturing capabilities. uniQure's actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's periodic securities filings, including its Annual Report on Form 10-K filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and uniQure assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

    uniQure Contacts:

    FOR INVESTORS: FOR MEDIA:
       
    Maria E. 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,  
    2021 2020 
     (in thousands, except share and per share amounts) 
    Current assets    
    Cash and cash equivalents$677,330 $244,932 
    Accounts receivables 2,705  6,618 
    Prepaid expenses 10,861  4,337 
    Other current assets and receivables 3,328  3,024 
    Total current assets 694,224  258,911 
    Non-current assets    
    Property, plant and equipment, net 38,371  32,328 
    Operating lease right-of-use assets 26,782  26,086 
    Intangible assets, net 2,637  3,361 
    Goodwill 525  542 
    Deferred tax assets 15,965  16,419 
    Other non-current assets 5,760  2,748 
    Total non-current assets  90,040  81,484 
    Total assets$ 784,264 $ 340,395 
    Current liabilities    
    Accounts payable$16,817 $3,772 
    Accrued expenses and other current liabilities 23,526  18,038 
    Current portion of operating lease liabilities 5,752  5,524 
    Total current liabilities 46,095  27,334 
    Non-current liabilities    
    Long-term debt 70,780  35,617 
    Operating lease liabilities, net of current portion 30,534  30,403 
    Other non-current liabilities 3,247  3,136 
    Total non-current liabilities 104,561  69,156 
    Total liabilities$ 150,656 $ 96,490 
    Shareholders' equity    
    Total shareholders' equity 633,608  243,905 
    Total liabilities and shareholders' equity$ 784,264 $ 340,395 



    uniQure N.V.

    UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

          
      Three months ended June 30, 
       2021   2020  
      (in thousands, except share and per share amounts) 
     Total revenues$ 463,868  $ 1,535  
     Operating expenses:    
     Cost of contract revenues (23,178)  -  
     Research and development expenses (33,401)  (28,401) 
     Selling, general and administrative expenses (16,645)  (11,511) 
     Total operating expenses (73,224)  (39,912) 
     Other income 7,590   669  
     Other expense (226)  (500) 
     Loss from operations 398,008   (38,208) 
     Non-operating items, net 4,718   (4,343) 
     Loss before income tax expense  402,726   (42,551) 
     Income tax expense (3,258)  -  
     Net loss$ 399,468  $ (42,551) 
     Earnings per ordinary share - basic    
     Basic net income/(loss) per ordinary share$8.68  $(0.96) 
     Earnings per ordinary share - diluted    
     Diluted net income/(loss) per ordinary share$8.51  $(0.96) 
     Weighted average shares - basic 46,037,900   44,387,463  
     Weighted average shares - diluted 46,929,870   44,387,463  
          


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  2. LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that eight data presentations, of which three are oral presentations, will be delivered at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress being held July 17-21, 2021.

    Specific details on uniQure's presentations at ISTH include:

    • Title: Five Year Data Confirms Stable FIX Expression and Sustained Reductions in Bleeding and Factor IX Use Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B (Abstract OC 26.3)
      Oral Session Title: Hemostatic Agents and…

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that eight data presentations, of which three are oral presentations, will be delivered at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress being held July 17-21, 2021.

    Specific details on uniQure's presentations at ISTH include:

    • Title: Five Year Data Confirms Stable FIX Expression and Sustained Reductions in Bleeding and Factor IX Use Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B (Abstract OC 26.3)

      Oral Session Title: Hemostatic Agents and Hemostatic Efficacy

      Date and Time: Monday, July 19, 11:00 – 12:00 p.m. EDT
    • Title: Clinical Outcomes in Adults with Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial (Abstract OC 67.3)

      Oral Session Title: Safety and Efficacy in Gene Therapy for Hemophilia

      Date and Time: Wednesday, July 21, 10:00 – 11:00 a.m. EDT
    • Title: Liver Safety Case Report from the Phase 3 HOPE-B Gene Therapy Trial in Adults with Hemophilia B (Abstract OC 67.4)

      Oral Session Title: Safety and Efficacy in Gene Therapy for Hemophilia

      Date and Time: Wednesday, July 21, 10:00 – 11:00 a.m. EDT
    • Title: Etranacogene dezaparvovec (AAV5-Padua hFIX variant, AMT-061), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: 2.5 Year data from a Phase 2b Trial (Live Poster, Abstract LPB0020)

      Poster Session Title: Oligonucleotide Therapeutics

      Live ePoster Date and Time: Sunday, July 18, 3:00 – 4:00 p.m. EDT
    • Title: 52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderate-Severe Hemophilia B: Data from the Phase 3 HOPE-B Gene Therapy Trial (Abstract PB0653)

      Date and Time: All ePosters will be made available on Saturday, July 17



    • Title: Management of Infusion Reactions: Lessons from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec in Adults with Hemophilia B (Abstract PB0659)

      Date and Time: All ePosters will be made available on Saturday, July 17
    • Title: Real-world Outcomes in People with Severe Hemophilia B Receiving FIX Prophylaxis across Europe: A CHESS II Analysis (Abstract PB0463)

      Date and Time: All ePosters will be made available on Saturday, July 17

    • Title: Preferences of People with Hemophilia A and B for Treatments Including Gene Therapies in the US: A Discrete Choice Experiment (Abstract PB0658)

      Date and Time: All ePosters will be made available on Saturday, July 17

    uniQure Vice President of Clinical Development David Cooper, M.D., also will give an invited presentation to the ISTH SSC (Scientific and Standardization Committee) subcommittee entitled "Factor VIII, Factor IX and Rare Coagulation Disorders." His pre-recorded presentation, Rationale for Adeno-associated Virus (AAV) Mediated Gene Therapy in Patients with Pre-existing anti-AAV5 antibodies, will take place on Saturday, July 17 from 9:24 to 9:30 a.m.

    About uniQure

    uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in hemophilia B and Huntington's disease and preclinical candidates in Fabry disease, spinocerebellar ataxia Type 3, temporal lobe epilepsy, Alzheimer's, Parkinson's, and ALS. www.uniQure.com  

    uniQure Contacts:

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


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  3. ~ Expands uniQure's Pipeline of Innovative Gene Therapies to Treat Neurological Disorders ~

    ~ Strengthens uniQure's Global Leadership in the Development of Gene Therapies that Employ miRNA Silencing Technology ~

    ~ Large Opportunity Targeting an Estimated 1.3 million TLE Patients in the U.S. and Europe with Approximately 800,000 Drug-Resistant Patients ~

    ~ Preclinical Proof-of-Concept Data from Disease Model and from Resected Hippocampi from Refractory TLE Patients Demonstrate Clear Suppression of Chronic Spontaneous Epileptic Seizures ~

    LEXINGTON, Mass. and AMSTERDAM and PARIS, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical…

    ~ Expands uniQure's Pipeline of Innovative Gene Therapies to Treat Neurological Disorders ~

    ~ Strengthens uniQure's Global Leadership in the Development of Gene Therapies that Employ miRNA Silencing Technology ~

    ~ Large Opportunity Targeting an Estimated 1.3 million TLE Patients in the U.S. and Europe with Approximately 800,000 Drug-Resistant Patients ~

    ~ Preclinical Proof-of-Concept Data from Disease Model and from Resected Hippocampi from Refractory TLE Patients Demonstrate Clear Suppression of Chronic Spontaneous Epileptic Seizures ~

    LEXINGTON, Mass. and AMSTERDAM and PARIS, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced it has entered into a definitive agreement to acquire Corlieve Therapeutics and its lead program, which will be known as AMT-260, to treat temporal lobe epilepsy, the most common form of focal epilepsy.

    Corlieve's lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (TLE). TLE affects approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.

    AMT-260 was originally developed by Corlieve in collaboration with Christophe Mulle, Ph.D., CNRS Research Director, at the Interdisciplinary Institute of Neurosciences, CNRS, University of Bordeaux, Valerie Crepel, Ph.D., Inserm Research Director at the Institut de Neurobiologie de la Méditerranée, INSERM, Aix-Marseille University, and REGENXBIO Inc. Drs. Mulle and Crepel will continue their collaborations with uniQure.

    "The acquisition of Corlieve provides an extraordinary opportunity to transform the lives of hundreds of thousands of patients around the world suffering from epilepsy and aligns with our vision of pursuing unmet medical needs for disorders that impact large populations and can be addressed with gene therapies directed to the CNS and liver," stated Matt Kapusta, Chief Executive Officer of uniQure. "The groundbreaking work of the Corlieve team, in collaboration with Drs. Mulle and Crepel, has led to compelling preclinical results in temporal lobe epilepsy that we believe can strategically leverage uniQure's leading position in developing and delivering gene therapies that employ miRNA silencing technology. We look forward to welcoming the Corlieve team into the uniQure family as we join forces to advance this important and potentially transformative therapy into clinical studies."

    "I am very proud of what Corlieve has achieved in such a short amount of time. With the dedication and focus of our team and our collaboration partners, we have taken a promising therapeutic approach discovered by our scientific founders and created a potential transformative therapeutic opportunity for patients with refractory TLE," said Richard Porter, Ph.D., founder and Chief Executive Officer of Corlieve. "As leaders in the field for miRNA gene therapy for neurological conditions, uniQure is the ideal long-term partner for us, and we look forward to working together to advance our program rapidly to the clinic for the benefit of the patients we serve."

    Upon the closing of the transaction, Dr. Porter will assume the role at uniQure of General Manager of the Corlieve subsidiary.

    "As a founding investor, we are delighted to see that the translational work conducted by Corlieve is recognized by uniQure, further reinforcing our commitment to company creation based on partnering with the industry," said Vanessa Malier, Managing Partner at Kurma Partners and Chairman of Corlieve.

    Transaction Details

    Under the terms of the agreement, uniQure will pay €46.3 million in an upfront payment of cash to acquire Corlieve. 

    Corlieve shareholders are eligible to receive the following additional payments of which up to 25% will be payable in uniQure ordinary shares at uniQure's election: up to €43.7 million in development milestones through Phase I/II and €160 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union.  

    Corlieve has an established license and collaboration agreement with REGENXBIO that includes an exclusive license to AAV9 for the specific genetic target of AMT-260. Under the license and collaboration agreement, REGENXBIO received equity in Corlieve and is eligible to receive milestone payments and royalties on net sales of AMT-260.

    The transaction has been approved by the Boards of both companies and does not require uniQure shareholder approval. The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l'Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier. Currently, the transaction is anticipated to be completed early in the third quarter of 2021.

    SVB Leerink LLC is acting as sole financial advisor and Morgan Lewis is acting as legal advisor to uniQure. McDermott Will & Emery is acting as legal advisor to Corlieve.

    About Corlieve Therapeutics

    Corlieve Therapeutics is a biotechnology company focused on bringing novel therapeutic options to patients with severe neurological disorders. The lead project is targeting aberrantly expressed kainate receptors in the hippocampus of patients with refractory TLE using a gene therapy approach. Corlieve was founded by Kurma Partners 4Q2019 on the basis of a partnership with REGENXBIO Inc., SATT Aquitaine Science Transfert, and Inserm Transfert. Corlieve is supported by its investors and partners Kurma Partners, Eurazeo, Pureos Bioventures, SATT Aquitaine Science Transfert, Inserm Transfert, Inserm CNRS, and REGENXBIO. For more information, please visit www.corlieve.com.

    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 uniQure or Corlieve will advance the AMT-260 program to the clinic rapidly or at all, and whether the transaction is completed early in the third quarter of 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 risks associated with the integration of Corlieve and the execution of the AMT-260 development efforts, regulatory review of the 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 Quarterly Report on Form 10-Q filed on May 10, 2021. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

    Corlieve Contacts:

    Amy Conrad

    Juniper Point

    858-366-3243



    Or

    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

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  4. ~ Sustained increases in Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal in full study population one year following a single administration of etranacogene dezaparvovec ~

    ~ Held pre-BLA submission meeting with FDA and aligned on primary endpoint analysis ~   

    LEXINGTON, Mass. and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe…

    ~ Sustained increases in Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal in full study population one year following a single administration of etranacogene dezaparvovec ~

    ~ Held pre-BLA submission meeting with FDA and aligned on primary endpoint analysis ~   

    LEXINGTON, Mass. and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date.

    Data from the HOPE-B pivotal study showed that participants continued to demonstrate durable, sustained increases in Factor IX (FIX) activity at 52-weeks post-infusion with a mean FIX activity of 41.5 percent of normal, as measured by a one-stage APTT-based clotting assay, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up. There continued to be no clinically significant correlation between pre-existing neutralizing antibodies to AAV5 (NAbs) and FIX activity in patients with NAb titers up to 678.2, a range expected to include more than 93 percent of the general population.

    During the 52-week period, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80 percent from a prospectively collected 3.39 at baseline to 0.68 bleeding episodes per year (p-value <0.0001). The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85 percent from a prospectively collected 1.16 at baseline to 0.18 bleeds per year during the 52-week period (p-value <0.0001).    

    Usage of FIX replacement therapy (IU/year and infusions/year) in all patients declined 96 percent during the 52-week period, with 52 of 54 patients (96 percent) successfully discontinuing their prophylactic infusions. As previously announced, of the two non-responders, one patient only received a partial dose (less than 10 percent of the dosage) due to an infusion reaction and a second patient had an unusually high pre-existing NAb titer of 3,212, which is expected in less than 1 percent of the general population.

    Etranacogene dezaparvovec continues to be generally well-tolerated with no treatment-related serious adverse events. No inhibitors to FIX have been reported and no consistent relationship between safety and pre-existing NAb titers has been observed.

    "We continue to be very encouraged by the data generated from the HOPE-B pivotal study of etranacogene dezaparvovec, which have been accepted for presentation at the annual International Society on Thrombosis and Haemostasis congress taking place next month," stated Ricardo Dolmetch, Ph.D., president of research and development at uniQure. "The 52-week data show mean FIX activity in the normal range and increase our confidence in the potential durability and long-term benefits of etranacogene dezaparvovec, bringing us one step closer to our goal of delivering this groundbreaking therapy to fulfill an unmet medical need for patients living with hemophilia B."

    Regulatory Update

    The Company and its partner, CSL Behring, have had recent communications with U.S. Food and Drug Administration (FDA), including a pre-biologics licensing application (BLA) submission meeting held on June 4, 2021. The FDA confirmed that the primary evidence of durability of effect to inform regulatory decision-making will come from patients followed for at least a 52-week period beginning when etranacogene dezaparvovec-derived FIX levels have achieved steady state, rather than when etranacogene dezaparvovec is administered. This feedback was based upon review of statistical analysis plans, as no clinical data was provided or discussed. All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec. As a result, uniQure will now conduct as the sole primary endpoint a non-inferiority analysis of annualized bleeding rates (ABR) at 78 weeks after the administration (approximately 52-weeks after steady-state is achieved). The Company expects all patients to complete their 78-week follow-up visits by the end of the third quarter of 2021, and the Company and CSL Behring expect to submit the BLA in first quarter of 2022.   

    About the HOPE-B Pivotal Clinical 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. Fifty-four adult hemophilia B patients classified as severe or moderately severe (defined as less than or equal to 2% of normal FIX activity) and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline ABR. After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Patients were not excluded from the trial based on their pre-existing NAbs to AAV5. Forty-three percent of patients in the study had pre-existing NAbs to AAV5 up to a maximum observed pre-dosing titer of over 3,200.  

    About 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). Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicine (PRIME) regulatory initiative by the European Medicines Agency. uniQure and CSL Behring entered into a commercialization and license agreement providing CSL Behring exclusive global commercialization rights to etranacogene dezaparvovec. The collaboration combines uniQure's differentiated gene therapy candidate in hemophilia B and CSL Behring's strong global reach and commercial infrastructure in hematology in an effort to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

    AAV5-based gene therapies have been demonstrated to be safe and well tolerated in a multitude of clinical trials, including being well-tolerated to date in six uniQure trials conducted in nearly 90 patients in hemophilia B and other indications. No patient treated in clinical trials with the uniQure's AAV5 gene therapies has experienced any confirmed cytotoxic T-cell-mediated immune response to the capsid. Additionally, pre-clinical 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 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 patients in the Hope-B pivotal trial will complete their 78-week follow-up visits by the end of the third quarter of 2021, whether CSL Behring will submit a BLA for etranacogene dezaparvovec in the first quarter of 2022, whether the clinical data proves to be meaningful for the long-term outlook for hemophilia gene therapy or etranacogene dezaparvovec, whether etranacogene dezaparvovec has the potential to provide well-tolerated, long-term clinical benefits, and whether AAV5-based gene therapies can provide clinical benefit to patients with pre-existing neutralizing antibodies. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our and our collaborators' clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's periodic securities filings, including is Annual Report on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q filed on May 10, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

    uniQure Contacts

    FOR INVESTORS: FOR MEDIA:
       
    Maria E. 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, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first two patient procedures have been completed in the higher dose, second cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. The initiation of patient enrollment in this 16-patient cohort follows a meeting last month of the trial's independent Data Safety Monitoring Board (DSMB) that reviewed safety data for the fully enrolled first cohort of ten patients. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States…

    LEXINGTON, Mass. and AMSTERDAM, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first two patient procedures have been completed in the higher dose, second cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease. The initiation of patient enrollment in this 16-patient cohort follows a meeting last month of the trial's independent Data Safety Monitoring Board (DSMB) that reviewed safety data for the fully enrolled first cohort of ten patients. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, seven patients now have been treated with AMT-130 across both trial cohorts, and five patients have received the imitation surgery.

    "We are very pleased with the progress that we continue to make in this Phase 1-2 clinical trial and that we are now administering AMT-130 at the higher dose," said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. "We look forward to ongoing patient enrollment in the second cohort and to initiating clinical development of AMT-130 in Europe in the second half of this year. We also remain on track to share initial imaging and biomarker data from the U.S. clinical trial before the end of the year."

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

    The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease will explore the safety, tolerability, and efficacy signals in 26 total patients with early manifest Huntington's disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose cohort randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. 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.clinicaltrials.gov (NCT04120493).

    The European, open-label Phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntington's disease across two dose cohorts. Together with the U.S. study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible. 

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

    About Huntington's Disease

    Huntington's disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington's disease, there are no currently approved therapies to delay the onset or to slow the disease's progression.

    About uniQure

    uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

    uniQure Forward-Looking Statements

    This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether we achieve further patient enrollment in the second cohort, whether we initiate dosing in our European open-label Phase Ib/II in the second half of 2021 or ever, and whether we share initial imaging and biomarker data towards the end of the year or ever. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's periodic securities filings, including its Annual Report on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q filed on May 10, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

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


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