uniQure Provides Year-End 2017 Corporate Update and Outlines Near-Term Objectives to Further Drive Shareholder Value
January 08 2018 - 7:00AM
~ Pivotal Trial in Hemophilia B on Track to Begin
in Q3 2018, with Initial AMT-061 Clinical Data by Year End ~
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
StatementsThis 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
79e.mulder@uniQure.com
For Media:
Tom MaloneDirect:
339-970-7558Mobile: 339-223-8541t.malone@uniQure.com
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