uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease
October 06 2017 - 7:00AM
YASTEST
~ First Investigational Gene Therapy in
Huntington's Disease to Receive Designation ~
LEXINGTON, Mass. and AMSTERDAM, The Netherlands,
Oct. 06, 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 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:
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
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Mar 2024 to Apr 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Apr 2023 to Apr 2024