uniQure Announces Dosing of First Patient in High-Dose Cohort in Phase I/II Hemophilia B Clinical Trial
March 14 2016 - 6:00AM
uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today
announced that the Company has treated the first patient in the
second cohort of its ongoing AMT-060-01 Phase I/II trial in
hemophilia B patients. The primary objective of the second cohort
is to assess the safety of the systemic administration of a higher
dose of uniQure's AMT-060 gene therapy in hemophilia B patients
that present with a severe or moderately-severe disease phenotype.
Secondary objectives of the trial include evaluation of Factor IX
(FIX) activity levels as well as evaluation of annualized bleeding
rates and recombinant FIX usage.
The Company announced preliminary top-line results of the
low-dose cohort on January 7th, 2016, showing that AMT-060 was
generally well tolerated. Additionally, the first two patients that
had completed at least 12 weeks of follow-up showed promising
increases in FIX expression levels of 5.5% and 4.5% of normal as of
the December 16, 2015 cutoff date, and in four of the five patients
dosed the need for continuous, prophylactic rFIX treatment was
eliminated as of January 6th, 2016. Including the patients screened
and treated in the low-dose cohort, a total of eight patients
screened to date for the trial have tested negative for
pre-existing anti-AAV5 antibodies. uniQure intends to present
an updated analysis of all patients in the low-dose
cohort at a scientific conference in the second quarter of
2016.
"Our goal for the second cohort is to evaluate AMT-060's safety
profile at a higher dose and to test our hypothesis that with this
dose we could achieve further increases in FIX expression levels,"
said Deya Corzo, Senior Vice President and Therapeutic Area Head,
Liver/Metabolism. "The early data we announced in January indicate
that our proprietary AAV5 viral vector can achieve a clinically
relevant level of expression of the FIX transgene. Our data thereby
reproduced previously published long-term FIX expression levels
seen with a dose comparable to that used in our low-dose cohort
with the same gene cassette but using a different
vector."
"The successful dosing of the first patient in the high-dose
cohort marks another significant step towards bringing this gene
therapy to patients," said Dan Soland, CEO of uniQure. "With the
greenlight from the Data Monitoring Committee after the first
cohort, we are excited to be able to move the program forward. We
strongly believe that the product profile of AMT-060 has the
potential to improve the quality of life of hemophilia B
patients."
The AMT-060-01 Phase I/II study is a 5-year, open-label,
uncontrolled, dose-ascending trial that includes two cohorts, with
the low-dose cohort using a treatment of 5x1012 gc/kg and the
high-dose cohort using 2x1013 gc/kg. 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. It is
administered, without immunosuppressant therapy, through the
peripheral vein in a single treatment session for approximately 30
minutes. All patients are screened for pre-existing AAV5 antibodies
before treatment and Data Monitoring Committee (DMC) reviews are
conducted after each of the first 2 patients in the second
cohort.
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.
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 course of
development of our program hemophilia B. 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:
Eva Mulder
Direct: +31 20 240 6103
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
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