uniQure Receives European Medicines Agency Priority Medicines (PRIME) Designation for AMT-060 in Hemophilia B
April 25 2017 - 7:05AM
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 25, 2017
(GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene
therapy company advancing transformative therapies for patients
with severe medical needs, today announced that AMT-060, its
proprietary, investigational gene therapy in patients with
severe hemophilia B, has received PRIME designation by the European
Medicines Agency (EMA). This designation is based on results from
the ongoing, dose-ranging Phase 1-2 study that show a near
cessation of spontaneous bleeding in patients with severe disease
at up to 12 months follow-up, clinically significant and sustained
increases in Factor IX (FIX) and substantial reductions in FIX
replacement usage.
"We are very pleased to have
AMT-060 for hemophilia B accepted into the PRIME program," stated
Matthew Kapusta, chief executive officer of uniQure. "Similar to
the Breakthrough Therapy designation that AMT-060 received from the
U.S. Food and Drug Administration earlier this year, we look
forward to this enhanced collaboration with the EMA to advance the
clinical development of this potentially transformative
therapy for hemophilia B patients."
Phase 1-2 Data
Updated clinical data from the
ongoing, two-cohort Phase 1-2 trial of AMT-060 were presented last
December at the 58th American
Society of Hematology (ASH) Annual Meeting. The data included
up to 52 weeks of follow-up from the low-dose cohort and up to 31
weeks of follow-up from the second dose cohort.
Data from the second-dose
cohort show a dose response with substantial improvement in disease
state in all five patients, including the discontinuation of
precautionary FIX infusions in all four patients that previously
required chronic replacement therapy. To date, only one
spontaneous bleed was reported after discontinuation of
prophylactic FIX replacement therapy.
All five patients in
the low-dose cohort, whose bleedings were previously
uncontrolled despite being managed with prophylactic
therapy, continue to maintain robust, constant and clinically
meaningful levels of FIX activity for up to 52 weeks post
treatment, with a complete cessation of spontaneous bleedings in
the last 14 weeks of observation.
AMT-060 continues to be
well-tolerated, and there have been no severe adverse events. Three
out of the total of 10 patients (two in the second-dose cohort and
one previously reported from the low-dose cohort) experienced mild,
asymptomatic elevations of alanine aminotransferase (ALT) and
received a tapering course of corticosteroids per protocol.
Importantly, the temporary elevations in ALT were not
associated with any loss of endogenous FIX activity or T-cell
response.
No patients across either cohort
have developed inhibitory antibodies against FIX, or
demonstrated sustained AAV5 capsid-specific T-cell activation.
About PRIME Designation
The PRIME program was launched by
the EMA in March 2016, and the designation is designed to aid and
expedite the regulatory process for investigational medicines that
may offer a major therapeutic advantage over existing treatments,
or benefit patients without treatment options. To be accepted, an
investigational medicine must show the potential to benefit
patients with unmet medical needs based on early clinical data.
Medicines accepted into the PRIME program are considered
priority medicines within the European Union (EU).
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,
statements regarding the winding down of our Glybera program, the
development of our other gene therapy product candidates, 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. Our actual results
could differ materially from those anticipated in these
forward-looking statements for many reasons, including, without
limitation, risks associated with corporate reorganizations and
strategic shifts, 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 2016 Annual
Report on Form 10-K filed on March 15, 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
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Source: uniQure N.V. via Globenewswire
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