uniQure Provides Year-End 2017 Corporate Update and Outlines Near-Term Objectives to Further Drive Shareholder Value
January 08 2018 - 7:00AM
YASTEST
~ Pivotal Trial
in Hemophilia B on Track to Begin in Q3 2018, with Initial AMT-061
Clinical Data by Year End ~
~ AMT-130
Expected to be First AAV-Based Gene Therapy for Huntington's
Disease to Enter Clinic ~
~ Strong Cash
Position Sufficient to Fund Company into 2020 ~
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, Jan. 08, 2018 (GLOBE NEWSWIRE) -- uniQure N.V.
(NASDAQ:QURE), a leader in human gene therapy, today announced its
near-term objectives following a transformational year of
achievement and corporate execution. Key business accomplishments
during 2017 included uniQure's alignment with regulatory
authorities to advance AMT-061 into late-stage clinical development
in hemophilia B, progress with its research pipeline including
AMT-130 in Huntington's disease, further enhancement of the
Company's manufacturing capabilities, and the strengthening of its
financial position.
"We ended 2017 with significant
momentum across all of our programs and the cash to fund operations
into 2020," stated Matthew Kapusta, chief executive officer of
uniQure. "Now that we have established robust,
commercial-scale manufacturing in our Lexington facility, we are
highly focused on the near-term initiation of our pivotal study of
AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in
Huntington's disease."
"We also are intensifying efforts
to further expand our gene therapy product pipeline. In
addition to AMT-126, our BMS-partnered gene therapy candidate for
congestive heart failure, we have a number of promising early-stage
projects focused on liver-directed and CNS disorders. We
expect multiple new INDs to be filed over the next two years,
further leveraging our industry-leading manufacturing and
technology platform."
uniQure management is meeting with
investors and analysts this week in conjunction with the
36th Annual J.P. Morgan Healthcare Conference taking place in
San Francisco. An updated corporate presentation has been posted to
the investor page of the Company's
website, www.uniqure.com.
Key Near-Term Objectives
- Advance AMT-061
into pivotal study for hemophilia B - The Company intends
to initiate a global, pivotal program for AMT-061 in hemophilia B
during the third quarter of 2018. Top-line data from the
initial patients are expected to be announced before the end of
2018. The Chemistry, Manufacturing and Controls (CMC) comparability
protocol, which has been agreed upon with the FDA, is nearly
complete and the data continues to support comparability between
AMT-061 and AMT-060. An amendment to the existing Investigational
New Drug (IND) application is expected to be submitted to the U.S.
Food and Drug Administration (FDA) in the first quarter of
2018.
- Advance AMT-130
into Phase I/II study for Huntington's disease - The
Company plans to complete its Good Laboratory Practices (GLP)
toxicology study of AMT-130 in mid-2018 and file an IND application
with the FDA in the second half of 2018. The Company expects
to begin clinical development of AMT-130 soon after IND acceptance,
with the goal of it being the first AAV-based gene therapy to begin
human clinical evaluation.
- Advance AMT-126 towards IND
filing for congestive heart
failure - In early 2018, the Company and Bristol-Myers
Squibb (BMS) expect to initiate a preclinical heart function study
of AMT-126 in a diseased minipig model. Assuming positive results
from this study, the Company expects BMS will initiate a GLP
toxicology study to support the submission of an IND application
and the initiation of a Phase I/II clinical
study.
- Further expand
product pipeline - In 2018, the Company expects to
introduce new product candidates with a focus on liver-directed and
central nervous system (CNS) disorders, areas in which the Company
has established strong technology and manufacturing
capabilities.
- Execute multiple
manufacturing campaigns - During 2018, the Company will
conduct large-scale manufacturing campaigns in its state-of-the-art
manufacturing facility based in Lexington, MA for each of its key
R&D programs. The Company expects to manufacture and
release current Good Manufacturing Processes (cGMP) compliant
material to be used in the AMT-061 pivotal program, the AMT-130
Phase I/II study and the AMT-126 GLP toxicology
study.
Financial Guidance
As of December 31, 2017, the
Company had approximately $159 million in cash and cash
equivalents, and expects cash on hand will be sufficient to fund
operations into 2020.
Major Accomplishments Throughout
2017
Hemophilia
B - AMT-061
- Achieved alignment with the FDA and
European Medicines Agency (EMA) on expedited clinical and
regulatory pathway for AMT-061, a potential best-in-class gene
therapy combining the Factor IX (FIX)-Padua gene with AAV5
- Demonstrated 6- to 7-fold greater
FIX activity in non-human primates with AMT-061 compared to
AMT-060
- Initiated commercial-scale, cGMP
production of AMT-061 clinical batches
- Acquired patent family covering the
use of FIX-Padua in gene therapy
- Achieved Breakthrough and PRIME
designations from FDA and EMA
Huntington's Disease -
AMT-130
- Initiated and completed dosing of an
IND-enabling, GLP safety study
- Successfully transferred
manufacturing of AMT-130 to Lexington facility and initiated
commercial-scale, cGMP production
- Presented preclinical data in one of
the largest diseased animal models showing widespread distribution
of AMT-130 in the brain and more than 50% knockdown of mutant
huntingtin protein
- Achieved U.S. Orphan Drug
Designation
Congestive Heart Failure -
AMT-126 (partnered with Bristol-Myers Squibb)
- Successfully demonstrated superior
DNA delivery and S100A1 protein expression in a large animal model
after administration of AMT-126
- Successfully transferred
manufacturing of AMT-126 to Lexington facility and initiated cGMP
production
Manufacturing Capabilities
- Successfully produced multiple
AAV5-based, gene therapy products at commercial-scale and under
cGMP conditions using the same fundamental manufacturing process,
controls and methods
- Key patents issued covering aspects
of the manufacturing process critical to large-scale production of
AAV-based gene therapies using an insect cell-based, baculovirus
expression system
Research on AAV5
- Presented clinical data
demonstrating favorable immunogenicity profile of AAV5 and low
prevalence of preexisting neutralizing antibodies
- Presented data demonstrating
successful re-administration of AAV5 in non-human primates
Corporate Developments
- Appointed new members of the
leadership team and board of directors
- Re-acquired global rights to
hemophilia B program
- Completed follow-on offering raising
$91 million in gross proceeds
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.
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, the development of our
gene therapy product candidates, the transition to our AMT-061
product candidate, 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, and the scope of protection provided by our patent
portfolio. Our actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with our and our
collaborators' clinical development activities, collaboration
arrangements, corporate reorganizations and strategic shifts,
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
Quarterly Report on Form 10-Q filed on November 1, 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
For
Investors:
Maria E.
Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Eva M.
Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
For Media:
Tom
Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@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
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