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uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study With FIX-Padua Variant in 2018

~ AMT-060 with the FIX-Padua Modification (AMT-061) Demonstrates Substantial Increase in FIX Activity in Non-human Primates ~

~ Plans to Initiate Pivotal Study with Enhanced AMT-061 in 2018 ~

~ Achieves Alignment with FDA on Streamlined Clinical and Regulatory Strategy for AMT-061, Which Will be Included Under Existing Breakthrough Therapy Designation ~

~ Acquires a Patent Family Covering FIX-Padua in Hemophilia B ~

~ Conference Call Scheduled for Today at 8:30 a.m. ET ~

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 19, 2017 (GLOBE NEWSWIRE) --  uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that following multi-disciplinary meetings with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the company plans to expeditiously advance AMT-061, which combines an AAV5 vector with the FIX-Padua mutant, into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B.

AMT-061 and AMT-060, the latter of which has been tested in 10 patients in an ongoing Phase I/II clinical trial, are identical in structure apart from two nucleotide substitutions in the coding sequence for FIX.  The gene variant, referred to as FIX-Padua, expresses a protein with a single amino acid substitution that has been reported in multiple preclinical and nonclinical studies to provide an approximate 8 to 9-fold increase in FIX activity compared to the wild-type FIX protein. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and proprietary insect cell-based manufacturing platform.

"Our mission in hemophilia B has always been to develop the safest and most effective gene therapy with the broadest application to patients. We believe AMT-061 moves us closer to this goal, as it has the potential to provide optimized clinical and tolerability benefits to nearly all severe and moderately severe patients with hemophilia B," stated Matthew Kapusta, chief executive officer of uniQure.  "We are delighted to have received constructive guidance from both the FDA and EMA, which we believe allows us to expeditiously advance AMT-061 into a pivotal study next year, as previously planned. In anticipation of this, we have begun GMP production of AMT-061 in our Lexington facility and preparations for the pivotal study are underway."

"I believe AMT-061 has the potential to be an important gene therapy for patients suffering 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. "Based on the data generated to date, AMT-061 may be the first gene therapy to provide durable, curative benefits to nearly all patients with hemophilia B, without the complications associated with capsid-related immune responses.  I very much look forward to serving as an investigator in this exciting Phase III program."

Clinical and Regulatory Pathway for AMT-061

AMT-061 Nonclinical Data Demonstrate Tolerability and Substantial Increases in FIX Activity

AMT-061 Continues to Leverage AAV5's Favorable Tolerability and Immunogenicity Results

Commercial-scale, GMP Manufacturing of AMT-061 Clinical Material Underway

Exclusive Patent Covers the Use of Padua in Gene Therapy for Hemophilia B

Conference Call Information

uniQure will host a conference call today, October 19, 2017 at 8:30 a.m. ET to discuss this announcement.  To access the live call by phone, dial (877) 280-2296 (United States) or +44 (0)20 3427 1900 (international); the conference ID is 2516119.  The call may also be accessed through the Investors section of the Company's website at www.uniQure.com.  Following the live webcast, a replay of the call will be available at the same location through November 2, 2017.

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 development of our gene therapy product candidates, 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 August 8, 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




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Source: uniQure N.V. via Globenewswire