uniQure Receives European Medicines Agency Priority Medicines (PRIME) Designation for AMT-060 in Hemophilia B
April 25 2017 - 7:05AM
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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