8-K

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported):  October 2, 2017

uniQure N.V.

(Exact Name of Registrant as Specified in Charter)

The Netherlands	001-36294	N/A
(State or Other Jurisdiction of Incorporation)	(Commission File Number)	(IRS Employer Identification No.)

Paasheuvelweg 25a, 1105 BP Amsterdam, The Netherlands	N/A
(Address of Principal Executive Offices)	(Zip Code)

Registrant’s telephone number, including area code: +31-20-566-7394

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

oWritten communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

oSoliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

oPre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

oPre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR240.13e-4(c))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company x

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. x

Item 8.01. Other Events

Press Release of uniQure N.V. dated October 2, 2017 announcing company presentations at upcoming October conferences

Press Release of uniQure N.V. dated October 6, 2017 announcing orphan drug designation for AMT-130 in Huntington’s disease

Press Release of uniQure N.V. dated October 18, 2017 announcing the presentation of new preclinical data on AMT-130 in Huntington’s Disease at the ESGCT 25th Anniversary Congress in Berlin

Item 9.01Financial Statements and Exhibits

(d)	Exhibits
99.1	Press Release of uniQure N.V. dated October 2, 2017 announcing company presentations at upcoming October conferences
99.2	Press Release of uniQure N.V. dated October 6, 2017 announcing orphan drug designation for AMT-130 in Huntington’s disease
99.3	Press Release of uniQure N.V. dated October 18, 2017 announcing the presentation of new preclinical data on AMT-130 in Huntington’s Disease at the ESGCT 25th Anniversary Congress in Berlin

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.

	UNIQURE N.V.
Date: October 18, 2017	By:	/S/ MATTHEW KAPUSTA
		Matthew Kapusta
		Chief Executive Officer

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EX-99.1

Exhibit 99.1

uniQure Announces Company Presentations at Upcoming October Conferences

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 02, 2017 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ:QURE) a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced company presentations at the following conferences taking place in October:

Alliance for Regenerative Medicine (ARM) 2017 Cell & Gene Meeting on the Mesa, October 4 - 6 2017, at the Estancia La Jolla Hotel & Spa in La Jolla, California.

·Jonathan Garen, chief business officer, will present a corporate overview on Wednesday, October 4th, 2017 at 3:30 p.m. PDT. The live webcast can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least two weeks following the live event.

European Society of Gene & Cell Therapy (EGSCT) 25th Anniversary Congress, October 17 - 20, 2017, at the BCC Berlin in Berlin, Germany.

·Members of the uniQure research & development team will be presenting new data focused on the progress and optimization of uniQure’s lead programs in gene therapy:

·Title: AAV5-miHTT gene therapy demonstrates sustained huntingtin lowering and functional improvement in Huntington disease mouse models 
Session Title: 1b - Ocular and Central Nervous System Gene and Cell Therapy I 
Date and Time: Wednesday, October 18th, 2017, from 8:30 - 10:40 am CET

·Title: Engineered AAV capsids are successfully produced through the innovative approach of combining directed evolution and the BEVS expression system. 
Poster and Date: P347 - Wednesday, October 18th, 2017.

·Title: Development and characterization of MFP-inducible GeneSwitch system for AAV5 gene therapy of chronic diseases in the liver  
Poster and Date: P160 - Thursday, October 19th, 2017

4th Animal Models of Neurodegenerative Disease Conference, October 22 - 24, 2017, at Chateau Liblice, Czech Republic.

·Members of the uniQure research & development team will be presenting new data focused on progress in technologies and development of animal models in gene therapy for Huntington’s disease:

·Title: Establishing proof-of-concept of gene therapy for Huntington’s disease.
Date: Tuesday, October 24th, 2017, morning session.

·Title: AAV-mediated delivery in large animals. 
Date: Monday, October 23rd, 2017, afternoon session.

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 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
m.cantor@uniQure.com	e.mulder@uniQure.com	t.malone@uniQure.com

EX-99.2

Exhibit 99.2

uniQure Announces FDA Orphan Drug Designation for

AMT-130 in Huntington’s disease

~ First Investigational Gene Therapy in Huntington’s Disease to Receive Designation ~

Lexington, MA and Amsterdam, the Netherlands, October 6, 2017 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-130, its proprietary gene therapy candidate for Huntington’s disease, has received Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). There are currently no approved medical treatments aimed at addressing the underlying cause of Huntington’s disease, and AMT-130 has the potential to play a role in this area of high unmet medical need.

ODD in the United States provides a special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S. The ODD programs offer product market exclusivity for up to seven years in the U.S. and ten years in the European Union following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications.

“Attaining orphan designation recognizes the potential that AMT-130 holds in delivering meaningful therapeutic benefit to patients suffering from this devastating disease,” said Matthew Kapusta, chief executive officer of uniQure. “It supports our ongoing development in Huntington’s as we seek to bring the first gene therapy approach to this disease into the clinic next year.”

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA which silences the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein.  Using AAV vectors to deliver micro-RNAs directly into the brain represents a highly innovative 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 over a 12- to 15-year period of time. The disease is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene coding for protein called huntingtin. Despite the clear etiology, there are no therapies available to treat the disease, delay onset or slow progression of a patient’s decline.

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:		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
m.cantor@uniQure.com	e.mulder@uniQure.com	t.malone@uniQure.com

EX-99.3

Exhibit 99.3

uniQure Presents New Preclinical Data on AMT-130 in Huntington’s Disease at the ESGCT 25th Anniversary Congress in Berlin

— One-time Administration of AMT-130 Demonstrates Survival Benefit and Functional Improvement of Huntington’s Disease Symptoms in Preclinical Study —

— IND-enabling Toxicology Study Initiated —

Lexington, MA and Amsterdam, the Netherlands, October 18, 2017 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented new preclinical data on AMT-130, its proprietary gene therapy candidate for the treatment of Huntington’s disease (HD), at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress in Berlin, Germany.

Data from the study demonstrate that following administration of AMT-130 in Huntington’s disease mouse models, significant improvements in both motor-coordination and survival were observed, as well as a dose-dependent, sustained reduction in huntingtin protein. AMT-130 comprises an AAV5 vector carrying a DNA cassette encoding an engineered micro RNA (miHTT) that silences the human huntingtin protein.  The study on functional improvement and sustained huntingtin lowering was performed by members of uniQure’s research department in collaboration with Charles River Discovery Research Services, Finland.

“We are confident that the combination of suppression of neuronal dysfunction, improvement of Huntington’s disease symptoms, extended survival and long-term huntingtin lowering observed in these studies, could translate into patient benefit and improve their quality of life,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “We have now begun our investigational new drug-enabling toxicology studies in rodents and non-human primates that will support an IND application for AMT-130 next year.”

Preclinical Data Findings

This study builds on previous data generated at uniQure, demonstrating a long-term significant suppression of mutant huntingtin protein, the cause of Huntington’s disease, after a single administration of AMT-130 in the Q175 mouse model of Huntington’s disease.

The current study was conducted in the rapidly progressing R6/2 mouse model of Huntington’s disease, which is characterized by early onset of motor symptoms and a much reduced life-span. A single administration of AMT-130 into the striatum was followed by a significant improvement of motor symptoms including improved coordination on the rotarod (a rotating cylinder to test coordination, physical condition and motor planning) as well as a significantly increased median survival from 120 to 149 days, compared with the control group (p = 0.0270). The data also demonstrate a significant reduction in expression of mutant huntingtin protein.

The functional improvements observed in these preclinical studies helped support the Orphan Drug Designation granted to AMT-130 by the U.S. Food and Drug Administration earlier this month and reinforce that huntingtin-lowering gene therapy for Huntington’s disease could modify the course of this devastating 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 over a 12- to 15-year period of time. The disease is caused by an autosomal dominant

mutation, a cytosine-adenine-guanine (CAG) expansion, in the first exon of the huntingtin gene leading to a non-functional, aggregation prone mutated protein. Despite the clear etiology, there are no therapies available to treat the disease, delay onset, or slow progression of a patient’s decline.

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:	For Media:
Eva M. Mulder	Tom Malone
Direct: +31 20 240 6103	Direct: 339-970-7558
Mobile: +31 6 52 33 15 79	Mobile: 339-223-8541
e.mulder@uniQure.com	t.malone@uniQure.com