YASTEST
- Clinical Benefit Maintained
in All Patients, with Durable Increases in FIX Clotting
Activity at Up to Two Years of Follow-Up -
- Second Dose Cohort
Demonstrated 89% Reduction in Spontaneous Bleeds, with No Confirmed
Bleeds Reported in Last 12 Months -
- AAV5-Based Gene Therapy
Continues to Show Favorable Safety Profile, with No Immune
Responses or Loss of FIX Activity in Any Patient -
- Company Preparing to
Initiate Pivotal Study with the FIX-Padua Modification (AMT-061) in
2018 -
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, Dec. 11, 2017 (GLOBE NEWSWIRE) -- uniQure
N.V. (NASDAQ:QURE), a leading gene therapy company advancing
transformative therapies for patients with severe medical needs,
today announced updated results from its ongoing, dose-ranging
Phase I/II trial of AMT-060, its investigational gene
therapy in patients with severe hemophilia B. The data
includes up to two years of follow-up from the low-dose cohort and
up to 18 months of follow-up from the second, higher-dose
cohort.
The AAV5-based AMT-060 remains
safe and well-tolerated with up to two years of follow-up, with no
new serious adverse events and no development of inhibitors. No
patient in the study has had any loss of Factor IX (FIX) activity
or capsid-specific, T-cell-mediated immune response.
Eighteen-month follow-up data from
the second-dose cohort continue to show stable FIX activity with
substantial improvement in disease state in all five patients,
including the discontinuation of routine prophylactic FIX infusions
in all patients that previously required chronic replacement
therapy. The annualized spontaneous bleeding rate for the second
dose cohort declined 89% to a mean of 0.3 bleeds after gene
transfer. In the last year of follow-up, no patient in the second
cohort has reported any spontaneous bleeds.
These clinical data were presented
this morning in an oral presentation at the 58th American Society
of Hematology (ASH) Annual Meeting taking place in Atlanta,
Georgia.
"We continue to observe a
therapeutic benefit from AMT-060 that is clearly superior to
patients' previous prophylactic FIX replacement therapy
regimen, with stable elevations in Factor IX levels and a cessation
of spontaneous bleeds," stated Professor Frank W.G. Leebeek, M.D.
Ph.D. of the Erasmus University Medical Center in Rotterdam, the
Netherlands.
"Most importantly, the AAV5-based
AMT-060 remains safe and well-tolerated, with no loss of FIX
activity, no activation of T-cell response and no development of
inhibitors for any of the 10 patients in the study, up to two years
after treatment. The safety profile observed in this study
continues to suggest that the AAV5 vector offers long-term safety,
efficacy and the potential for broad application in hemophilia B
patients."
uniQure announced in October that,
following meetings with the FDA and EMA, it plans to initiate a
pivotal study in 2018 with AMT-061, which combines an AAV5 vector
with the FIX-Padua mutant. AMT-061 and AMT-060 are identical in
structure apart from two nucleotide substitutions in the coding
sequence for FIX. The gene variant, referred to as FIX-Padua,
has been reported in multiple preclinical and nonclinical studies
to provide an approximate 8 to 9-fold increase in FIX clotting
activity compared to the wild-type FIX gene. All other critical
quality attributes of AMT-061 are expected to be comparable to
those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and
proprietary insect cell-based manufacturing platform.
"These data give us continued
confidence that our AAV5-based gene therapies offer multi-year
durability, superior safety and broad applicability as a
result of a favorable immunogenicity profile compared to other AAV
vectors," stated Matthew Kapusta, chief executive officer of
uniQure. "We believe AMT-061 has the potential to provide
curative benefits to nearly all hemophilia B patients, without the
complications associated with capsid-related immune responses.
Preparations for the pivotal study are underway and the
manufacturing of AMT-061 for clinical use has been initiated."
Phase 1/2 Trial Overview
The AMT-060 gene therapy consists of a codon-optimized wild type
FIX gene cassette, the LP1 liver promoter and an AAV5 viral
vector manufactured by uniQure using its proprietary insect
cell-based technology platform.
-
The Phase I/II, open-label, multi-center study
includes 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 5x1012gc/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.
Data Update from Phase I/II
Clinical Trial of AMT-060 in Hemophilia B Patients
Data as of
October 26, 2017:
About
Hemophilia B
Hemophilia B is a serious and rare inherited disease in males
characterized by insufficient blood clotting. The condition can
lead to repeated and sometimes life-threatening episodes of
external and internal bleeding following accidental trauma or
medical interventions. Severe hemophilia is characterized by
recurrent episodes of spontaneous joint bleeds, that cause
long-term damage to the joints resulting in disabling arthropathy.
Bleeds may be fatal if they occur in the brain. The deficient blood
clotting results from the lack of functional human Factor IX, or
hFIX. Treatment of hemophilia B today consists of prophylactic or
on-demand protein replacement therapy, in which one to three times
weekly intravenous administrations of plasma-derived or recombinant
hFIX are required to prevent bleeding and once daily infusions in
case bleeding occurs. Hemophilia B occurs in approximately 1 out of
30,000 live births.
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, Huntington's disease and cardiovascular
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, the development of our
gene therapy product candidates, the transition to our AMT-061
product candidate, the success of our collaborations and the risk
of cessation, delay or lack of success of any of our ongoing or
planned clinical studies and/or development of our product
candidates, and the scope of protection provided by our patent
portfolio. 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, 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 November 1, 2017. 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:
Maria E.
Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniqure.com
Tom
Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
Eva M.
Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
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