uniQure Announces FDA Breakthrough Therapy Designation for AMT-060 in Hemophilia B
(Thomson Reuters ONE) -
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 30, 2017 (GLOBE NEWSWIRE)
-- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced
that AMT-060, its proprietary, investigational gene therapy in patients with
severe hemophilia B, has received Breakthrough Therapy designation by the U.S.
Food and Drug Administration (FDA). This designation is based on results from
the ongoing, dose-ranging Phase 1-2 study that show sustained increases in
Factor IX (FIX), reductions in FIX replacement usage and a near cessation of
spontaneous bleeding in patients with severe disease at up to 12 months follow-
up.
"We are very pleased the FDA has designated AMT-060 a Breakthrough Therapy for
patients with hemophilia B," stated Matthew Kapusta, chief executive officer of
uniQure. "The FDA's decision to prioritize and expedite the review of AMT-060
is an important milestone for uniQure and we are committed to working closely
with the FDA to rapidly advance our hemophilia B program into late-stage
development."
Phase 1-2 Data
Updated clinical data from the ongoing, two-cohort Phase 1-2 trial of AMT-060
were recently presented at the 58(th) 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 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. As of the data cutoff date for the ASH
presentation, only one unconfirmed spontaneous bleed was reported during an
aggregate of 94 weeks follow-up 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, constant and clinically meaningful levels of FIX activity for up to 52
weeks post treatment, resulting in 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 to
the AAV5 capsid.
No patients across either cohort have developed inhibitory antibodies
against FIX and no patients screened in the study tested positive for anti-AAV5
antibodies.
About Breakthrough Therapy Designation
The 2012 Food and Drug Administration Safety and Innovation Act (FDASIA)
established the Breakthrough Therapy designation to expedite the development and
review of new drugs with preliminary clinical evidence demonstrating that they
may offer a substantial improvement over available therapies for patients with
serious or life-threatening diseases. The Breakthrough Therapy designation is a
distinct status from both accelerated approval and priority review, which can
also be granted to the same drug if relevant criteria are met. According to FDA
data for its fiscal year 2016, the Center for Biologics Research and Review
received a total of 23 requests for Breakthrough Therapy designation with only
4 designations granted - or 17% of all requests.
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 implementation and effects of the Company's new
strategic and organizational changes, the development of our 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 2015 Annual Report on Form 20-F filed with the Securities and Exchange
Commission on April 4, 2016. 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(at)uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)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
Datum: 30.01.2017 - 13:00 Uhr
Sprache: Deutsch
News-ID 520681
Anzahl Zeichen: 8536
contact information:
Town:
Amsterdam
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 203 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"uniQure Announces FDA Breakthrough Therapy Designation for AMT-060 in Hemophilia B"
steht unter der journalistisch-redaktionellen Verantwortung von
uniQure N.V. (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
