uniQure Announces Positive Recommendation from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the T...
February 08 2021 - 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 independent Data Safety Monitoring
Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130
for the treatment of Huntington’s disease has met and reviewed the
six-month safety data from the first two enrolled patients and the
90-day safety data from the next two enrolled patients in the
study. No significant safety concerns were noted to prevent further
dosing, and the final six patients in the first cohort are now
cleared for enrollment. uniQure expects to achieve full patient
enrollment in cohort one by mid-year 2021. The Phase I/II study is
a double-blind, randomized clinical trial being conducted in the
United States. To date, two patients have been treated with
AMT-130, and two patients received the imitation surgery.
“We are encouraged with the positive
recommendation to proceed with patient enrollment from this second
DSMB meeting,” said Ricardo Dolmetsch, president of research and
development at uniQure. “We will now focus on enrolling the last
six additional patients in the first cohort and look forward to
sharing initial biomarker and imaging data towards the end of the
year.”
About the Phase I/II Clinical Trial of
AMT-130
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 total patients with early
manifest Huntington’s disease split into a 10 patient, low-dose
cohort followed by a 16 patient, higher-dose cohort randomized to
treatment with AMT-130 or an imitation (sham) surgery. The
five-year, multi-center trial consists of a blinded 12-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.clinicaltrials.gov (NCT04120493).
AMT-130 is uniQure’s first clinical program
focusing on the central nervous system (CNS) incorporating its
proprietary miQURE™ platform.
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 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 gene therapies to treat patients with hemophilia B,
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 we will be able
to enroll the next six patients in the clinical trial by mid-year
2021 and whether we will be able to announce initial biomarker and
imaging data by the end of 2021. 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 periodic securities filings, including its Annual Report
on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q
filed on October 27, 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: |
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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 |
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