uniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Dise...
June 19 2020 - 7:05AM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that the first two patients in the Phase
I/II clinical trial of AMT-130 for the treatment of Huntington’s
disease have been treated. The Phase I/II study is a double-blind,
randomized clinical trial being conducted in the United States,
with now one patient treated with AMT-130, and one patient who
received the imitation surgery.
“For years, uniQure has had an unwavering
commitment to advance this first-in-human AAV gene therapy for
Huntington’s disease into clinical testing, and this moment marks
an important milestone for our company now that we have two AAV
gene therapy candidates in clinical development,” said Matt
Kapusta, chief executive officer of uniQure. “With the first two
patients treated in this trial, we have taken a significant step
forward in advancing AMT-130 closer to our goal of developing a
therapy that inhibits the production of the mutant huntingtin
protein. We are delighted to be working with leading experts in the
field to evaluate this promising candidate.”
The Phase I/II clinical trial of AMT-130 for the
treatment of Huntington’s disease will explore the safety,
tolerability, and efficacy signals in 26 patients with early
manifest Huntington’s disease randomized to treatment with AMT-130
or an imitation (sham) surgery. The five-year, multi-center trial
consists of a blinded 18-month core study period followed by
unblinded long-term follow-up. Patients will receive a single
administration of AMT-130 through MRI-guided, convection-enhanced
stereotactic neurosurgical delivery directly into the striatum
(caudate and putamen). Additional details are available on
www.clinicaltrails.gov (NCT04120493).
The first two patients will be observed for an
initial period of 90 days, followed by a meeting of the Data Safety
Monitoring Board (DSMB). The DSMB will review the data on the first
two patients and make a determination about continued dosing of the
next patients.
AMT-130 is uniQure’s first clinical program
focusing on the central nervous system (CNS) incorporating its
proprietary miQURE™ platform.
“There is an urgent need for disease-modifying
options to treat Huntington’s disease, and we’re excited to have an
investigational gene therapy now available for HD patients,” stated
George Yohrling, chief scientific officer and chief mission officer
at Huntington's Disease Society of America. “Based on the promising
preclinical data presented on AMT-130 over the years, we are
optimistic about its potential to alter the course of this
devastating disease.”
About Huntington’s
DiseaseHuntington’s disease is a rare, inherited
neurodegenerative disorder that leads to motor symptoms including
chorea, and behavioral abnormalities and cognitive decline
resulting in progressive 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 currently approved therapies to delay the onset or to slow
the disease’s progression.
About uniQureuniQure 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 gene therapies to treat patients with hemophilia B,
hemophilia A, Huntington’s disease, Fabry disease, spinocerebellar
ataxia Type 3 and other 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, whether AMT-130 will
prove to be a promising treatment or alter the course of
Huntington’s disease, whether the DSMB will authorize treatment of
additional patients, and whether we will be able to treat 26
patients under the clinical trial. Our actual results could differ
materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with the impact of the ongoing COVID-19 pandemic on our
Company and the wider economy and health care system, our clinical
development activities, clinical results, collaboration
arrangements, 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 April 29, 2020.
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 |
Chiara Russo |
Tom Malone |
Direct: 339-970-7536 |
Direct: 617-306-9137 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: 617-306-9137 |
Mobile: 339-223-8541 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/53dbf6e5-2c8c-447c-abd9-a9d5714f2a8c
https://www.globenewswire.com/NewsRoom/AttachmentNg/6ae019fc-e394-40ff-b1c4-222d71c86145
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