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): April 8, 2019
uniQure N.V.
(Exact Name of Registrant as Specified in Charter)
The Netherlands |
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001-36294 |
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(State or Other
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(Commission
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(IRS Employer
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Paasheuvelweg 25a,
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Registrants 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):
o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-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 o |
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. o
Item 8.01. Other Events
On April 8, 2019, the Company issued a press release, a copy of which is attached as Exhibit 99.1 and is incorporated herein by reference, announcing that the Company received Fast Trask Designation from the U.S. Food and Drug Administration for AMT-130 in Huntingtons disease.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits
99.1 |
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Press release dated April 8, 2019, announcing that the Company has received FDA Fast Track Designation for AMT-130 gene therapy for the treatment of Huntingtons disease. |
EXHIBIT INDEX
Exhibit
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Desciption |
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99.1 |
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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.
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UNIQURE N.V. |
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Date: April 9, 2019 |
By: |
/S/ MATTHEW KAPUSTA |
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Matthew Kapusta |
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Chief Executive Officer and interim Chief Financial Officer |
uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntingtons Disease
~ On Track to Treat First Patient in Phase I/II Study of AMT-130 in 2H19 ~
Lexington, Mass. and Amsterdam, the Netherlands, April 8, 2019 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. Food and Drug Administration (FDA) has granted Fast Track designation for AMT-130, the Companys gene therapy candidate for the treatment of Huntingtons disease. AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that non-selectively lowers or knocks-down human huntingtin protein in Huntingtons disease patients. AMT-130 also is unique in that it targets the highly toxic exon1 protein fragment that is even more toxic than the mutant huntingtin protein.
Achieving Fast Track Designation from the FDA underscores the high unmet medical need for patients suffering from Huntingtons disease, for which there are currently no approved, disease-modifying treatments, stated Matt Kapusta, chief executive officer of uniQure. We are nearing the initiation of a Phase I/II study of AMT-130, the first one-time administered AAV gene therapy to enter clinical testing for Huntingtons disease, and are on track to treat the first patient in the second half of 2019.
The FDAs Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. A therapy granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA) or New Drug Application (NDA).
About Huntingtons Disease
Huntingtons 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 Huntingtons disease, there are no therapies to delay the onset or to slow the diseases 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 and partnered gene therapies to treat patients with hemophilia, Huntingtons 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 managements 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 initiate dosing of a Phase I/II study of AMT-130 in the second half of 2019 or ever, and our ability to open clinical sites for the Phase I/II study in the United States. 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 uniQures Annual Report on Form 10-K filed on February 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: |
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FOR MEDIA: |
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Maria E. Cantor |
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Eva M. Mulder |
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Tom Malone |
Direct: 339-970-7536 |
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Direct: +31 20 240 6103 |
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Direct: 339-970-7558 |
Mobile: 617-680-9452 |
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Mobile: +31 6 52 33 15 79 |
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Mobile:339-223-8541 |
m.cantor@uniQure.com |
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e.mulder@uniQure.com |
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t.malone@uniQure.com |