uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe unmet
medical needs, today announced it has achieved the targeted dosing
of patients in the HOPE-B pivotal trial of etranacogene
dezaparvovec (AMT-061), an investigational AAV5-based gene therapy
incorporating the patent-protected FIX-Padua variant for the
treatment of patients with severe and moderately severe hemophilia
B. The targeted number of patients to be dosed per the clinical
trial protocol was 50. In total, 54 patients have received
the one-time dose of etranacogene dezaparvovec.
“I am very proud of the entire uniQure team, our
investigators, study coordinators and the hemophilia patient
communities who helped make this major milestone possible,” stated
Matt Kapusta, chief executive officer of uniQure. “With target
patient dosing now finished, we are closely monitoring the trial
and working within guidance provided by the FDA regarding COVID-19
to minimize any risk or disruption in patient follow-up visits. We
continue to expect top-line data from the Phase III trial before
the end of this year, which we believe will support a BLA
submission in 2021.”
The pivotal Phase III HOPE-B trial follows the
Company’s ongoing Phase IIb trial of etranacogene dezaparvovec, in
which we have previously reported that, after 52 weeks of follow-up
data, all three patients had stabilized and sustained FIX activity
at therapeutic levels after the one-time administration of
etranacogene dezaparvovec. Additionally, in an ongoing Phase I/II
trial of AMT-060, the Company’s first-generation gene therapy for
the treatment of hemophilia B, all 10 patients continued to show
sustained and stable increases in FIX activity and long-term
clinical benefit, including improved disease phenotype and
substantial reductions in spontaneous bleeds at up to 4 years of
observation.
Update on the Phase I/II Clinical Trial of AMT-130 in
Huntington’s Disease
The first two patients in the Company’s Phase I/II clinical
trial of AMT-130 in Huntington’s disease have been enrolled after
successfully meeting all screening and eligibility criteria. These
patients were scheduled to have their procedures on March 24 and 25
at the Ohio State University. Due to the expanding impact of the
COVID-19 coronavirus pandemic, these procedures have been
temporarily postponed. The decision to postpone treatment in the
trial follows the COVID-19-related State of Emergency declarations
in the United States, where the trial is taking place.
“We are following all federal and local regulations including
the FDA guidance on clinical trial conduct during the COVID-19
pandemic and we are working very closely with trial
investigators to ensure that patient safety remains our top
priority,” stated Robert Gut, M.D., Ph.D., chief medical officer at
uniQure. “Despite this unexpected postponement, we are encouraged
by the progress that we have made with this trial since the
beginning of the year, and the fact that we have patients who are
highly motivated to participate. We will continue our work to
resume treatment in the Phase I/II trial as soon as it is
clinically appropriate.”
uniQure’s Response to
COVID-19
The Company is continuing its operations under
guidance from national, state and local authorities in the
Netherlands and in the United States, including the US Food and
Drug Administration (FDA) and Centers for Disease Control and
Prevention (CDC). In this regard, uniQure is informed by the
direction and flexibility provided by the FDA in its March 18, 2020
Guidance entitled “FDA Guidance on Conduct of Clinical
Trials of Medical Products during COVID-19 Pandemic”.
Ongoing clinical research activities are being closely monitored to
minimize any risk, disruption or delay in either patient dosing or
follow-up visits.
uniQure is focused on ensuring the health and
wellbeing of its global workforce. As of March 13, the Company
mandated a work-from-home policy for all non-essential employees.
As a biopharma research and development company, uniQure is deemed
to provide Essential Services under the “stay at home” advisory
that was issued by the Governor of Massachusetts on March 23, 2020
and is maintaining its mission-critical activities that include
commercial-scale manufacturing operations.
About the Pivotal Phase III HOPE-B
Trial
The pivotal Phase III HOPE-B trial is a
multinational, open-label, single-arm study to evaluate the safety
and efficacy of etranacogene dezaparvovec in 50 patients. Adult
hemophilia B patients classified as severe or moderately severe are
enrolled in a six-month observational period during which time they
will continue to use their current standard of care to establish a
baseline control. After the six-month lead-in period, patients will
receive a single intravenous administration of etranacogene
dezaparvovec at the 2x1013 gc/kg dose. Dosing of patients in the
HOPE-B pivotal trial was initiated in January 2019.
The trial’s primary endpoint is the assessment
of Factor IX activity 26 weeks after dosing. Secondary
endpoints include annualized bleeding rate (ABR) and usage of
Factor IX replacement therapy over a 52-week time frame, as well as
other efficacy and safety aspects. Post-treatment, patients will be
followed for 5 years.
Patients enrolled in the HOPE-B pivotal trial
will be tested for the presence of pre-existing neutralizing
antibodies to AAV5 but will not be excluded from the trial based on
their titers. Previous studies performed by uniQure suggest that
AAV5-based gene therapies may be viable treatments for at least 97%
of patients.
About Etranacogene Dezaparvovec
(AMT-061)
Etranacogene dezaparvovec, also known as
AMT-061, consists of an AAV5 viral vector carrying a gene cassette
with the patent-protected Padua variant of Factor IX (FIX-Padua).
AAV5-based gene therapies have been demonstrated to be safe and
well tolerated in many clinical trials, including four uniQure
trials conducted in 25 patients in hemophilia B and other
indications. No patient treated in clinical trials with the
Company’s AAV5-based gene therapies has experienced any cytotoxic
T-cell-mediated immune response to the capsid. Additionally,
preclinical and clinical data show that AAV5-based gene therapies
may be clinically effective in patients with pre-existing
antibodies to AAV5, thereby potentially increasing patient
eligibility for treatment compared to other gene therapy product
candidates. Etranacogene dezaparvovec has been granted Breakthrough
Therapy Designation by the United States Food and Drug
Administration and access to Priority Medicines (PRIME) regulatory
initiative by the European Medicines Agency.
About AMT-130
AMT-130 is our novel gene therapy candidate for
the treatment of Huntington’s disease, a severe genetic
neurodegenerative disorder causing loss of muscle coordination,
behavioral abnormalities and cognitive decline, often resulting in
complete physical and mental deterioration over a 12 to 15-year
period. AMT-130 utilizes our miQURE™ proprietary, gene-silencing
platform and incorporates an AAV vector carrying a microRNA
specifically designed to silence the huntingtin gene and the
potentially highly toxic exon 1 protein fragment. AMT-130 has
received orphan drug and fast track designations from the FDA and
Orphan Medicinal Product Designation from the EMA.
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, 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 we will obtain top-line data from the HOPE-B Phase III
trial before the end of this year or ever, whether such data will
support a BLA submission in 2021, whether we will be able to resume
treatment in the Phase I/II trial as soon as it is clinically
appropriate, and our expectation to continue our operations during
the COVID-19 pandemic. 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 March 2,
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 |
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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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