uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease
April 08 2019 - 7:00AM
uniQure Receives FDA Fast Track Designation for AMT-130 Gene
Therapy for the Treatment of Huntington’s Disease
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 Company’s
gene therapy candidate for the treatment of Huntington’s 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 Huntington’s 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
Huntington’s 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 Huntington’s disease, and are on
track to treat the first patient in the second half of 2019.”
The FDA's 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 Huntington’s
DiseaseHuntington’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. 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 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 and partnered gene therapies to treat patients with
hemophilia, Huntington’s disease and other severe genetic diseases.
www.uniQure.com
uniQure Forward-Looking
StatementsThis 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, 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 uniQure’s 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: |
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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 |
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