~ Patient Enrollment Underway in Global Phase III
HOPE-B Pivotal Study of AMT-061
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today reported its financial results for the second quarter
of 2018 and highlighted recent progress across its business.
“We have made tremendous progress over the past
several months advancing our gene therapy programs in hemophilia B
and Huntington’s disease, as well as in expanding our intellectual
property portfolio and executing on our manufacturing plan,” said
Matt Kapusta, chief executive officer of uniQure. “Our Phase III
pivotal trial of AMT-061 is actively enrolling patients, and we
have consented and successfully screened the first of three
patients in our Phase IIb dose-confirmation study.”
“The initiation of our registrational study
provides the opportunity for uniQure to be first to market with a
one-time treatment that has the potential to transform the standard
of care for patients with hemophilia B,” Mr. Kapusta added. “We
continue to work toward what we expect will be an equally
productive second half of 2018 with the achievement of multiple
value-creating catalysts across the company, including top-line
data from our dose-confirmation study, the submission of an
Investigational New Drug application for AMT-130 in Huntington’s
disease, and hosting a Research and Development Day that will
feature expansion of our early-stage pipeline.”
Recent Company Progress
- Advancing AMT-061 into late-stage clinical development for the
treatment of hemophilia B
- The first patient in the Phase IIb dose-confirmation study of
AMT-061 in hemophilia B has consented and successfully screened
into the study and is expected to be treated shortly. The Phase IIb
study is an open-label, single-arm, single-dose trial being
conducted in multiple centers in the U.S. Three patients will
receive a single intravenous (IV) infusion of 2x1013 vc/kg and be
evaluated for a period of approximately six to eight weeks to
assess Factor IX (FIX) activity and confirm the dose for the global
Phase III HOPE-B study. Top-line data are expected to be made
available by the end of this year.
- Patient enrollment has commenced for the global Phase III
HOPE-B pivotal study. This registrational study includes a
six-month lead-in phase to collect baseline data as patients will
serve as their own control. The Company expects to begin dosing
patients with AMT-061 in the first quarter of 2019.
- Clinical supplies for the AMT-061 Phase IIb dose-confirmation
study have been successfully shipped to the site in anticipation of
the first patient treatment. Additionally, the production of
clinical batches is underway in the Company’s Lexington, MA
manufacturing facility for the Phase III HOPE- B study.
- The U.S. Patent and Trademark Office granted Patent Number
9,982,248 which broadly covers methods of treating bleeding
disorders including hemophilia B, using AAV gene therapy with
nucleic acid encoding the hyperactive Factor IX (FIX) Padua
variant. This is the second U.S. patent to issue in a patent family
that uniQure acquired from the inventor, Professor
Paolo Simioni, a renowned hemophilia expert at the University
of Padua, Italy. The ‘248 patent was prosecuted by uniQure
and granted after consideration of the prior art.
- Advancing AMT-130 for the treatment of Huntington’s disease
into a Phase I/II clinical study
- The six-month in-life portion of a GLP-safety and toxicology
study on AMT-130 in non-human primates has been completed and all
study reports are expected to be finalized in the third quarter of
2018. Data from this study will be used in support of an
Investigational New Drug (IND) application for AMT-130 which is
expected to be submitted to the U.S. Food and Drug Administration
(FDA) later this year. Following acceptance of this IND, the
Company expects to advance AMT-130 with the goal of being the first
AAV-gene therapy to enter the clinic in Huntington’s disease
patients.
- In June 2018, previously presented preclinical data on AMT-130
were published in the journal, Molecular Therapy. Researchers
investigated the feasibility, efficacy and safety of AMT-130 in a
Huntington’s disease transgenic minipig model. At both three and
six months after treatment, widespread, dose-dependent distribution
of the vector was observed throughout the minipig brain that
correlated strongly with human mutant huntingtin (mHTT) lowering.
Specifically, six months after treatment at the high dose,
researchers observed a mean mHTT lowering of 85.3% in the putamen,
75.0% in the caudate nucleus, 73.7% in the thalamus and 52.0% in
the cortex.
- Appointed proven leaders to clinical and medical organization,
including Robert Gut, M.D., Ph.D. as Chief Medical Officer
- Robert Gut, M.D., Ph.D. has been appointed Chief Medical
Officer effective August 20, 2018 and will be responsible for
leading all clinical development, medical affairs and related
functions. Dr. Gut will be based in Lexington, MA and report
directly to Mr. Kapusta. He succeeds Steven Zelenkofske, D.O., who
will leave the Company due to personal family reasons that preclude
him from relocating to the Boston area and require him to be closer
to his home in Philadelphia, PA. Dr. Zelenkofske has agreed to
continue to serve as a consultant to the Company through the end of
the third quarter of 2018 to assist with the transition.Dr. Gut has
20 years of experience in the biopharmaceutical industry leading
clinical development, regulatory and medical affairs activities in
hematology and other therapeutic areas. Dr. Gut most recently
served as Chief Medical Officer of Versartis, Inc. until its merger
with Aravive Biologics in June 2018. For the majority of his
career, Dr. Gut served as Vice President, Clinical Development
& Medical Affairs at Novo Nordisk Inc., where he headed the
company's U.S. Biopharm Medical organization with leading products
in hemophilia, including NovoSeven® (Factor VIIa), NovoEight®
(Factor VIII), Tretten® (Factor XIII) and Factor IX.
Dr. Gut's contributions helped achieve nine different FDA
approvals and the successful launches of those products. “I’m
extremely pleased to join the uniQure management team at such an
exciting time and help advance its potentially transformative gene
therapy programs, including AMT-061 in hemophilia B and AMT-130 in
Huntington’s disease,” stated Dr. Gut. “In my career, I have
been keenly focused on developing and delivering innovative
medicine to patients, including those with hemophilia. I very much
look forward to leveraging my experiences and relationships with
the hemophilia community to progress AMT-061 through the HOPE-B
pivotal study.”Dr. Gut has served as a non-executive member of the
uniQure Board of Directors since June 2018, and the Company plans
that he will continue to serve on the Board as an executive
director.
- Alison Long, M.B.B.Ch., Ph.D., has been appointed vice
president, clinical development, hemophilia. She is an
accomplished medical professional with both patient management
experience and extensive biopharmaceutical R&D experience
spanning multiple therapeutic areas, including hematology. Dr. Long
most recently served as vice president, head of clinical research
at Aegerion Pharmaceuticals where she led both the clinical
operations and clinical development programs. Prior to joining
Aegerion, she had been medical director of hematology clinical
development at Biogen, where she delivered strategic medical
leadership for their hemophilia clinical programs.
- Dr. Long joins Joseph Higgins, M.D., who joined uniQure earlier
this year as vice president of clinical development, Huntington's
disease. Dr. Higgins most recently served as the medical director
of neurology for Quest Diagnostics and the
CLIA laboratory director for Athena Diagnostics where he
managed all neurological diagnostic testing at Quest. He
will serve as the lead for the Company’s Huntington's disease
clinical program.
Near-Term Company
Milestones
The company expects to achieve the following
milestones in the second half of 2018:
- Complete the dosing of approximately three patients in the
dose-confirmation study of AMT-061 and announce top-line data.
- Complete the GLP-safety and toxicology study of AMT-130 in
Huntington’s disease and submit the IND.
- Host a Research & Development Day in New York City to
feature expansion of the Company’s early-stage research pipeline
and progress on current pipeline candidates.
- Complete the heart function study of AMT-126 in a diseased
minipig model of congestive heart failure.
Financial Highlights
The company strengthened its financial position
through completion of an underwritten public offering in May 2018.
The gross proceeds from the offering, before deducting
underwriting discounts and commissions and estimated offering
expenses payable by uniQure, were $147.5 million.
Cash Position: As of June 30,
2018, the Company held cash and cash equivalents of $259.2 million,
compared to $159.4 million as of December 31, 2017. The
Company currently expects cash and cash equivalents will be
sufficient to fund operations into 2021.
Revenues: Revenue for the three
months ended June 30, 2018 was $3.1 million, compared to $4.9
million for the comparable period in 2017. Collaboration revenue
for the three months ended June 30, 2018 was $0.9 million, compared
to $4.2 million for the comparable period in 2017. The decrease in
collaboration revenue was primarily due to the termination of the
Chiesi co-development agreement in July 2017.
R&D Expenses: Research and
development expenses were $18.5 million for the three months ended
June 30, 2018, compared to $16.9 million for the comparable period
in 2017. The increase was primarily related to costs incurred
preparing for the initiation of the AMT-061 pivotal study and
continued IND-enabling nonclinical studies of AMT-130.
SG&A Expenses: Selling,
general and administrative expenses were $5.9 million for the three
months ended June 30, 2018, compared to $5.4 million for the
comparable period in 2017.
Other income, net: Other
income, net was an income of $0.1 million for the three months
ended June 30, 2018, compared to an expense of $2.4 million for the
comparable period in 2017.
Net Loss: The net loss was
$20.6 million, or $0.57 per share, for the three months ended June
30, 2018, compared to $21.3 million, or $0.83 per share, for the
comparable period in 2017.
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
liver/metabolic, central nervous system 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 the completion of our
Phase IIb study, the release of top-line clinical data, the
opportunity to be first to market with a Hemophilia B product, the
filing of an IND or other regulatory filings or their acceptance by
regulatory authorities, initiating a Huntington’s Disease program
or other program, trial or study that is the first in the clinic,
completion of our GLP safety and toxicology study, our heart
function study or other studies, the achievement of any of our
planned near term or other milestones, 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. 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
Annual Report on Form 10-K filed on April 30th, 2018. 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: |
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FOR
INVESTORS: |
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|
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FOR
MEDIA: |
Maria
E. Cantor |
|
Eva
M. Mulder |
|
Tom
Malone |
Direct:
339-970-7536 |
|
Direct: +31
20 240 6103 |
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Direct:
339-970-7558 |
Mobile:
617-680-9452 |
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Mobile: +31 6
52 33 15 79 |
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Mobile:
339-223-8541 |
m.cantor@uniQure.com |
|
e.mulder@uniQure.com |
|
t.malone@uniQure.com |
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uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
June 30, |
|
December 31, |
|
2018 |
|
|
2017 |
|
in thousands, except share and per share
amounts |
Current assets |
|
|
|
Cash and
cash equivalents |
$ |
259,180 |
|
$ |
159,371 |
Accounts
receivables and accrued income |
|
1,037 |
|
|
1,586 |
Prepaid
assets and other current assets |
|
2,489 |
|
|
1,826 |
Total current assets |
|
262,706 |
|
|
162,783 |
Non-current assets |
|
|
|
Property, plant and equipment, net |
|
32,126 |
|
|
34,281 |
Intangible assets and goodwill |
|
11,249 |
|
|
10,100 |
Restricted cash |
|
2,458 |
|
|
2,480 |
Total non-current
assets |
|
45,833 |
|
|
46,861 |
Total assets |
$ |
308,539 |
|
$ |
209,644 |
Current liabilities |
|
|
|
Accounts
payable |
$ |
3,866 |
|
$ |
2,908 |
Accrued
expenses and other current liabilities |
|
8,920 |
|
|
8,838 |
Current
portion of long-term debt |
|
8,028 |
|
|
1,050 |
Current
portion of deferred rent |
|
1,082 |
|
|
737 |
Current
portion of deferred revenue |
|
8,463 |
|
|
4,613 |
Current
portion of contingent consideration |
|
1,081 |
|
|
1,084 |
Total current liabilities |
|
31,440 |
|
|
19,230 |
Non-current liabilities |
|
|
|
Long-term debt, net of current portion |
|
12,840 |
|
|
19,741 |
Deferred
rent, net of current portion |
|
8,464 |
|
|
9,114 |
Deferred
revenue, net of current portion |
|
32,853 |
|
|
67,408 |
Contingent consideration, net of current portion |
|
2,704 |
|
|
2,880 |
Derivative financial instruments related party |
|
1,309 |
|
|
1,298 |
Other
non-current liabilities |
|
513 |
|
|
614 |
Total non-current liabilities |
|
58,683 |
|
|
101,055 |
Total liabilities |
|
90,123 |
|
|
120,285 |
Total shareholders' equity |
|
218,416 |
|
|
89,359 |
Total liabilities and shareholders' equity |
$ |
308,539 |
|
$ |
209,644 |
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS
OF OPERATIONS
|
Three months ended
June 30, |
|
|
|
2018 |
|
|
|
2017 |
|
|
|
in thousands, except share and per share
amounts |
|
Total revenues |
$ |
3,050 |
|
|
$ |
4,942 |
|
|
Operating expenses: |
|
|
|
|
Research
and development expenses |
|
(18,493 |
) |
|
|
(16,866 |
) |
|
Selling,
general and administrative expenses |
|
(5,896 |
) |
|
|
(5,410 |
) |
|
Total operating expenses |
|
(24,389 |
) |
|
|
(22,276 |
) |
|
Other
income |
|
565 |
|
|
|
266 |
|
|
Other
expense |
|
(429 |
) |
|
|
(2,640 |
) |
|
Loss from operations |
|
(21,203 |
) |
|
|
(19,708 |
) |
|
Non
operating items, net |
|
972 |
|
|
|
(1,561 |
) |
|
Loss before income tax expense |
|
(20,231 |
) |
|
|
(21,269 |
) |
|
Income
tax expense |
|
(361 |
) |
|
|
- |
|
|
Net loss |
$ |
(20,592 |
) |
|
$ |
(21,269 |
) |
|
|
|
|
|
|
Basic
and diluted net loss per common share |
$ |
(0.57 |
) |
|
$ |
(0.83 |
) |
|
Weighted
average shares used in computing basic and diluted net loss per
common share |
|
36,205,061 |
|
|
|
25,560,348 |
|
|
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