uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced updated clinical data on the three patients
treated in the Company’s ongoing Phase IIb study of AMT-061, an
investigational AAV5-based gene therapy containing a
patent-protected FIX-Padua variant for the treatment of patients
with severe and moderately severe hemophilia B. In addition, the
Company presented up to 3.5 years of follow-up data on the 10
patients in the Phase I/II trial of AMT-060, its first-generation
gene therapy for the treatment of hemophilia B. These clinical data
were presented on Saturday, July 6 in oral presentations at the
27th Congress of the International Society on Thrombosis and
Haemostasis (ISTH), taking place in Melbourne, Australia.
AMT-061 (AAV5-Padua hFIX variant) an
Enhanced Vector for Gene Transfer in Adults with Severe or
Moderate-Severe Hemophilia B: Follow-Up Out to 36
Weeks
The Phase IIb study of AMT-061 is an open-label,
single-dose, single-arm, multi-center trial being conducted in the
United States. Three patients with severe hemophilia (endogenous
Factor IX (FIX) activity less than or equal to one percent) were
enrolled in the study and received a single intravenous infusion of
2x1013 vc/kg. Prior to the administration of AMT-061, all three
patients showed low levels of pre-existing neutralizing antibodies
to AAV5 but were not excluded from the trial on that basis.
Patients in the Phase IIb study will be followed for 52 weeks to
assess FIX activity, bleeding rates and usage of FIX replacement
therapy, and will be monitored for five years to evaluate the
safety of AMT-061.
Featured in an oral presentation at ISTH, the 36
weeks of follow-up data show that all three patients have sustained
increases in FIX levels after the one-time administration of
AMT-061, with two of the three patients maintaining FIX activity in
the normal range. Mean FIX activity for the three patients at 36
weeks after administration was 45% of normal, with the first
patient achieving FIX activity of 54% of normal, the second patient
achieving FIX activity of 30% of normal and the third patient
achieving FIX activity of 51% of normal. The second and third
patients had previously screen-failed and were excluded from
another gene therapy study due to pre-existing neutralizing
antibodies to a different AAV vector. Reported FIX activity was
measured using an activated partial thromboplastin time (aPTT)
assay performed at a central laboratory.
No patient in the study has experienced a
material loss of FIX activity, reported any bleeding events or
required any infusions of FIX replacement therapy for bleeds. One
patient underwent hip surgery due to a pre-existing condition and
was treated perioperatively with short-acting factor replacement.
This was reported by the investigator as a serious adverse event
unrelated to AMT-061.
“These updated data continue to show that a
single administration of AMT-061 is well-tolerated and has the
potential to increase FIX activity into the normal range for people
living with hemophilia B,” stated Matt Kapusta, chief executive
officer of uniQure. “We continue to be very pleased with these
results, which as of this data report show durable increases in FIX
activity with no bleeds and no requirement for infusions of FIX
replacement therapy or immunosuppression. We believe AMT-061 has
the potential to be a first- and best-in-class gene therapy for
patients with hemophilia B, and we remain focused on our goal of
completing enrollment in our ongoing HOPE-B pivotal trial by the
end of this year.”
Stable Expression of FIX and Maintained
Reductions in Bleeding and FIX Consumption Following AMT-060 Gene
Therapy with up to 3.5 Years of Follow-Up
In the ongoing Phase I/II study of AMT-060, all
10 patients continue to show long-term meaningful clinical impact,
including sustained increases in FIX activity and improvements in
their disease state as measured by reduced usage of FIX replacement
therapy and decreased bleeding frequency. At up to 3.5 years of
follow-up, AMT-060 continues to be safe and well-tolerated, with no
new serious adverse events and no development of inhibitors since
the last reported data.
All five patients in the second dose cohort of
2x1013 gc/kg continue to be free of routine FIX replacement therapy
at up to 3 years after treatment. During the last 12 months of
observation, the mean annualized bleeding rate was 0.7 bleeds,
representing an 83% reduction to the year prior to treatment.
During this same period, the usage of FIX replacement therapy
declined 96% compared to the year prior to treatment. Steady-state
mean yearly FIX activity at 3 years was 7.9%, as compared to 7.1%
in the first year and 8.4% in the second year.
AMT-060 is uniQure’s first-generation gene
therapy, consisting of an AAV5 vector carrying a gene cassette with
the wild-type FIX gene. Data from this Phase I/II trial of AMT-060
will be part of the regulatory submission for marketing approval of
AMT-061.
“The Phase I/II study of AMT-060 continues to
demonstrate the impressive long-term safety and tolerability of
uniQure’s gene therapy platform in hemophilia B,” stated Professor
Frank W.G. Leebeek, M.D., Ph.D. of the Erasmus University Medical
Center in Rotterdam, the Netherlands. “We have now demonstrated
evidence of durable clinical benefits, including sustained FIX
activity, improved disease phenotype and substantial reductions in
spontaneous bleeds for up to 3.5 years after administration. These
data are very encouraging for uniQure’s hemophilia B program.”
About AMT-061
AMT-061 consists of an AAV5 viral vector
carrying a gene cassette with the patent-protected Padua variant of
Factor IX (FIX-Padua). uniQure holds multiple issued patents in the
United States and Canada broadly covering methods of treating
bleeding disorders, including hemophilia B, using AAV gene therapy
with the FIX-Padua variant. Additional patents are pending in the
European Union.
AAV5-based gene therapies have been demonstrated
to be safe and well tolerated in a multitude of 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 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.
About the Pivotal Phase III HOPE-B
Study
The pivotal Phase III HOPE-B trial is a
multinational, multi-center, open-label, single-arm study to
evaluate the safety and efficacy of AMT-061. Approximately 55 adult
hemophilia B patients classified as severe or moderately severe
will be 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
AMT-061 at the 2x1013 gc/kg dose confirmed in the Phase IIb trial.
Dosing of patients in the HOPE-B pivotal trial is now underway.
The primary endpoint of the study will be based
on the FIX activity level achieved following the administration of
AMT-061, and the secondary endpoints will measure annualized FIX
replacement therapy usage, annualized bleed rates and safety.
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 gene therapies may be viable treatments for at least 97% of
patients.
About the Phase I/II study of
AMT-060
The Phase I/II study is an open-label,
multi-center study including 10 patients each receiving a one-time,
30-minute, intravenous administration of AMT-060, without the
prophylactic use of corticosteroids. The study includes two dose
cohorts of five patients each, with the first cohort receiving
5x1012 gc/kg and the second cohort receiving 2x1013 gc/kg. Nine
patients in the trial were classified as having severe (<1% FIX
activity) hemophilia. One patient in the low-dose cohort had a
moderate/severe (1.5% FIX activity) phenotype.
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 B, hemophilia A,
Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3
and cardiovascular 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, the completion of our
Phase IIb study, the ability of AMT-061 to provide all people
living with hemophilia B access to a one-time treatment capable of
normalizing FIX activity or eliminating the need for replacement
therapy or reducing or eliminating the risk of immune responses
that require immunosuppression or that may lead to a loss of
efficacy, the ability of AMT-061 to be a first-in-class or
best-in-class gene therapy for patients with hemophilia B, the
ability of AMT-061 to provide durable clinical benefits, including
sustained FIX activity, improved disease phenotype or substantial
reductions in spontaneous bleeds, our ability to complete
enrollment in our ongoing HOPE-B pivotal trial by the end of this
year, our ability to achieve any of our other planned near term or
other milestones, the risk of cessation, delay or lack of success
of any of our ongoing or planned clinical studies, and/or the
development and regulatory approval of our product candidates. 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 Quarterly Report on Form 10-Q filed on April 29, 2019.
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: |
FOR
MEDIA: |
|
|
|
Maria E.
Cantor |
Eva M.
Mulder |
Tom
Malone |
Direct: 339-970-7536 |
Direct: +31 20 240 6103 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: +31 6 52 33 15 79 |
Mobile: 339-223-8541 |
m.cantor@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
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