uniQure Announces Enrollment of Next Two Patients in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s...
October 13 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 two additional patient procedures have
been completed in the Phase I/II clinical trial of AMT-130 for the
treatment of Huntington’s disease. The ongoing patient enrollment
follows a meeting last month of the trial’s independent Data Safety
Monitoring Board (DSMB) to review 90-day follow-up data from the
first two patients. The DSMB observed no significant safety
concerns to prevent further dosing. The Phase I/II study is a
double-blinded, randomized and controlled clinical trial being
conducted in the United States. A total of four patients have been
enrolled in the study thus far, including two patients treated with
AMT-130 and two patients who received imitation surgery.
“We are very pleased with the progress being
made to advance this first-in-human AAV gene therapy for
Huntington’s disease,” said Ricardo Dolmetsch, Ph.D., president of
research and development at uniQure. “This is an important
achievement that puts us on our original clinical development
timeline, making up for the modest delay in the study earlier this
year due to COVID-19. In accordance with the study protocol,
patient enrollment is expected to continue after a DSMB meeting to
review 90-day follow-up data on these two new patients and 6-month
data on the first two patients. We expect that this DSMB review
will take place early next year and that patient enrollment in the
10-patient first dose cohort will be completed by mid-2021.”
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 across two dose cohorts. The
multi-center trial consists of a blinded 12-month core study period
followed by unblinded long-term follow-up for 5 years after
administration of AMT-130. 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 Disease
Huntington’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
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, whether patient
enrollment will continue after a DSMB meeting to review follow-up
data, whether the DSMB review will take place early next year, and
whether patient enrollment in the first dose cohort will be
completed by mid-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 July 30, 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. CantorDirect:
339-970-7536m.cantor@uniQure.com |
Chiara RussoDirect:
617-306-9137c.russo@uniQure.com |
Tom MaloneDirect:
339-970-7558t.malone@uniQure.com |
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