--Meaningful Factor IX Expression
Validates Successful Transduction of the Liver Using uniQure's
Proprietary AAV5 Vector--
--Four of Five Patients Have Fully
Discontinued Prophylactic Recombinant Factor IX
Therapy--
--Conference Call to Discuss Data
Scheduled for 8:30 am EST Today, January 7--
uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today
announced preliminary topline results from the low-dose cohort of
an ongoing Phase I/II clinical trial being conducted in adult
hemophilia B patients treated with uniQure's novel AAV5/FIX gene
therapy, AMT-060. All five patients in the low-dose cohort had
Factor IX (FIX) phenotypic features of severe or moderately-severe
hemophilia including documented Factor IX (FIX) levels less than
1-2% and required chronic treatment with prophylactic recombinant
FIX (rFIX) therapy at the time of enrollment.
The first two patients out of five in the low dose cohort have
completed at least 20 and 12 weeks of follow up and had
central-lab-confirmed FIX expression levels of 5.5% and 4.5% of
normal, respectively at the cutoff date of December 16th, 2015. The
three additional patients have been dosed, but had not achieved the
full 12 weeks of follow-up at the cutoff date. However, as of
January 6, 2016, four of the five patients, including the first two
patients enrolled in the study, have met a secondary objective in
the trial by fully discontinuing prophylactic rFIX. The 12 week
follow-up, post AMT-060 administration, marks the period in which
investigators in the trial attempt discontinuation of prophylactic
rFIX, based on FIX expression levels. The first patient in the
low-dose cohort experienced a mild, transient and asymptomatic
elevation of transaminase levels at around 10 weeks post
treatment. This patient received a short, 8-week course of
tapering prednisolone doses with rapid return of transaminase
levels to baseline values. No elevated transaminase levels have
been observed in the other four patients thus far, with all
patients being on therapy for at least 10 weeks as of January 6,
2016.
AMT-060 consists of a codon-optimized wild type FIX gene and the
LP1 liver promoter together with the AAV5 viral vector,
manufactured using uniQure's proprietary insect cell based
manufacturing technology. AMT-060 is administered, without
immunosuppressant therapy, through the peripheral vein in one
treatment session for approximately 30 minutes. The study includes
two cohorts, with the low-dose cohort using 5x1012 gc/kg and the
high-dose cohort using 2x1013 gc/kg. Thus far, there have been no
patient screening failures due to pre-existing
neutralizing antibodies against AAV5 and no patients have
developed inhibitory FIX antibodies.
These early data from the low-dose cohort suggest that AMT-060
is generally well-tolerated and capable of successfully transducing
the liver resulting in clinically meaningful FIX expression
levels. This current trial uses a starting dose of AAV5/FIX
gene therapy that is similar to the highest dose of the same FIX
gene cassette evaluated in a study conducted by Prof. Amit Nathwani
and the St. Jude Children's Hospital using an AAV8 serotype vector,
and uniQure's preliminary data are comparable with the endogenous
FIX expression levels achieved in the St. Jude study. The results
of the St. Jude study, which were published in the New England
Journal of Medicine in 2011 and 2014, demonstrated that a durable
mean FIX expression of 5.1% of normal (range 2.9% to 7.2%) can be
achieved with this gene cassette and result in meaningful long-term
clinical benefits for patients. In the St. Jude study, four of
six patients treated at the high dose had transient elevations of
transaminase levels, managed with a tapering prednisolone regimen.
The FIX gene cassette used in the St. Jude study is exclusively
licensed by uniQure.
"Thus far, the overall tolerability and FIX expression profile
in the low-dose cohort is encouraging for patients with hemophilia
B and support the continuation of the study," commented Professor
Frank W.G. Leebeek, M.D. Ph.D. of the Erasmus Medical Center in
Rotterdam, an investigator in the study. "Previous studies have
demonstrated that maintaining durable FIX expression around 3% to
5% of normal may have a significant clinical benefit as measured by
significant reduction in consumption of units of FIX concentrate
and lower risk of spontaneous bleeding episodes."
uniQure intends to present a more complete analysis of these
data from this low-dose cohort at a scientific conference in the
second quarter of 2016. Subject to the Data Monitoring
Committee's approval, the Company also anticipates initiating
enrollment of the high-dose cohort this quarter.
"These preliminary topline results support our hypothesis that
AAV5/FIX can deliver clinically meaningful expression levels of FIX
for patients with hemophilia B," commented Dan Soland, Chief
Executive Officer of uniQure. "So far, our AAV5-based gene
therapies have been systemically administered to 13 adult patients
across two clinical studies in two different disease states, and
via direct central nervous system administration in four children
in a third study, providing us with a strong safety dataset on the
AAV5 vector and our proprietary insect cell based manufacturing
technology."
"Today, we are the only AAV gene therapy company in the world
with both proprietary, commercial-scale manufacturing capabilities
and encouraging clinical data across multiple diseases," continued
Mr. Soland. "These preliminary results further support our
modular platform approach and leadership in gene therapy."
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. The episodes can cause long-term
damage, for example to the joints, and can 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 frequent intravenous administrations
of plasma-derived or recombinant hFIX are required to stop or
prevent bleeding. Hemophilia B occurs in approximately 1 out
of 30,000 live births.
Conference Call and Webcast
uniQure will discuss the data in a webcast conference call at
8:30 am EST on Thursday, January 7, 2016. The webcast and the
accompanying slides, which can be downloaded as a PDF file, can be
located on uniQure's website at
http://www.uniqure.com/news/calendar-of-events/. The conference
call can be accessed by dialling one of the numbers listed below
five minutes prior to the start of the call and providing the
confirmation code: 1640435.
London, United
Kingdom: |
+44 (0) 20 3427 1906 |
New York, United States of
America: |
+1 212 444 0412 |
Berlin, Germany: |
+49 (0) 30 3001 90534 |
Amsterdam,
Netherlands: |
+31 (0) 20 716 8296 |
Paris, France: |
+33 (0) 1 76 77 22 31 |
Milan, Italy: |
+39 02 3859 1420 |
Brussels,
Belgium: |
+32 (0) 2 402 3092 |
Montreal, Canada: |
+1 514 841 2154 |
To ensure a timely connection to the webcast, it is recommended
that users register at least 15 minutes prior to the scheduled
start time. The webcast replay will be available for at least 72
hours following the call.
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 CNS, liver/metabolic and cardiovascular diseases.
www.uniQure.com
uniQure Forward-Looking Statement
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, statements regarding the future
development of AMT-060. Our actual results could differ materially
from those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with
collaboration arrangements, our and our collaborators' clinical
development activities, 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 2014 Annual Report on Form 20-F filed
with the Securities and Exchange Commission on April 7, 2015. 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.
CONTACT: uniQure:
Direct: +31 20 240 6110
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or
+1 781 235 3060
gschweitzer@macbiocom.com
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