~ Pivotal Trial in Hemophilia B on Track to Begin in Q3 2018, with Initial AMT-061 Clinical Data by Year End ~

~ AMT-130 Expected to be First AAV-Based Gene Therapy for Huntington’s Disease to Enter Clinic ~

~ Strong Cash Position Sufficient to Fund Company into 2020 ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 08, 2018 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced its near-term objectives following a transformational year of achievement and corporate execution. Key business accomplishments during 2017 included uniQure’s alignment with regulatory authorities to advance AMT-061 into late-stage clinical development in hemophilia B, progress with its research pipeline including AMT-130 in Huntington’s disease, further enhancement of the Company’s manufacturing capabilities, and the strengthening of its financial position. 

“We ended 2017 with significant momentum across all of our programs and the cash to fund operations into 2020,” stated Matthew Kapusta, chief executive officer of uniQure.  “Now that we have established robust, commercial-scale manufacturing in our Lexington facility, we are highly focused on the near-term initiation of our pivotal study of AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in Huntington’s disease.”  

“We also are intensifying efforts to further expand our gene therapy product pipeline.  In addition to AMT-126, our BMS-partnered gene therapy candidate for congestive heart failure, we have a number of promising early-stage projects focused on liver-directed and CNS disorders.  We expect multiple new INDs to be filed over the next two years, further leveraging our industry-leading manufacturing and technology platform.” 

uniQure management is meeting with investors and analysts this week in conjunction with the 36th Annual J.P. Morgan Healthcare Conference taking place in San Francisco. An updated corporate presentation has been posted to the investor page of the Company’s website, www.uniqure.com

Key Near-Term Objectives

  • Advance AMT-061 into pivotal study for hemophilia B – The Company intends to initiate a global, pivotal program for AMT-061 in hemophilia B during the third quarter of 2018.  Top-line data from the initial patients are expected to be announced before the end of 2018. The Chemistry, Manufacturing and Controls (CMC) comparability protocol, which has been agreed upon with the FDA, is nearly complete and the data continues to support comparability between AMT-061 and AMT-060. An amendment to the existing Investigational New Drug (IND) application is expected to be submitted to the U.S. Food and Drug Administration (FDA) in the first quarter of 2018. 
     
  • Advance AMT-130 into Phase I/II study for Huntington’s disease – The Company plans to complete its Good Laboratory Practices (GLP) toxicology study of AMT-130 in mid-2018 and file an IND application with the FDA in the second half of 2018.  The Company expects to begin clinical development of AMT-130 soon after IND acceptance, with the goal of it being the first AAV-based gene therapy to begin human clinical evaluation.     
     
  • Advance AMT-126 towards IND filing for congestive heart failure – In early 2018, the Company and Bristol-Myers Squibb (BMS) expect to initiate a preclinical heart function study of AMT-126 in a diseased minipig model. Assuming positive results from this study, the Company expects BMS will initiate a GLP toxicology study to support the submission of an IND application and the initiation of a Phase I/II clinical study. 
     
  • Further expand product pipeline – In 2018, the Company expects to introduce new product candidates with a focus on liver-directed and central nervous system (CNS) disorders, areas in which the Company has established strong technology and manufacturing capabilities.  
        
  • Execute multiple manufacturing campaigns – During 2018, the Company will conduct large-scale manufacturing campaigns in its state-of-the-art manufacturing facility based in Lexington, MA for each of its key R&D programs.  The Company expects to manufacture and release current Good Manufacturing Processes (cGMP) compliant material to be used in the AMT-061 pivotal program, the AMT-130 Phase I/II study and the AMT-126 GLP toxicology study.  

Financial Guidance

As of December 31, 2017, the Company had approximately $159 million in cash and cash equivalents, and expects cash on hand will be sufficient to fund operations into 2020.

Major Accomplishments Throughout 2017

Hemophilia B – AMT-061

  • Achieved alignment with the FDA and European Medicines Agency (EMA) on expedited clinical and regulatory pathway for AMT-061, a potential best-in-class gene therapy combining the Factor IX (FIX)-Padua gene with AAV5
  • Demonstrated 6- to 7-fold greater FIX activity in non-human primates with AMT-061 compared to AMT-060
  • Initiated commercial-scale, cGMP production of AMT-061 clinical batches
  • Acquired patent family covering the use of FIX-Padua in gene therapy
  • Achieved Breakthrough and PRIME designations from FDA and EMA

Huntington’s Disease – AMT-130

  • Initiated and completed dosing of an IND-enabling, GLP safety study
  • Successfully transferred manufacturing of AMT-130 to Lexington facility and initiated commercial-scale, cGMP production
  • Presented preclinical data in one of the largest diseased animal models showing widespread distribution of AMT-130 in the brain and more than 50% knockdown of mutant huntingtin protein
  • Achieved U.S. Orphan Drug Designation

Congestive Heart Failure – AMT-126 (partnered with Bristol-Myers Squibb)

  • Successfully demonstrated superior DNA delivery and S100A1 protein expression in a large animal model after administration of AMT-126
  • Successfully transferred manufacturing of AMT-126 to Lexington facility and initiated cGMP production  

Manufacturing Capabilities

  • Successfully produced multiple AAV5-based, gene therapy products at commercial-scale and under cGMP conditions using the same fundamental manufacturing process, controls and methods 
  • Key patents issued covering aspects of the manufacturing process critical to large-scale production of AAV-based gene therapies using an insect cell-based, baculovirus expression system

Research on AAV5

  • Presented clinical data demonstrating favorable immunogenicity profile of AAV5 and low prevalence of preexisting neutralizing antibodies
  • Presented data demonstrating successful re-administration of AAV5 in non-human primates

Corporate Developments

  • Appointed new members of the leadership team and board of directors
  • Re-acquired global rights to hemophilia B program
  • Completed follow-on offering raising $91 million in gross proceeds

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. 

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 development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates, and the scope of protection provided by our patent portfolio. 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 November 1, 2017. 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    
m.cantor@uniQure.com

Eva M. Mulder             
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com  

For Media:

Tom Malone
Direct:  339-970-7558
Mobile:  339-223-8541
t.malone@uniQure.com