uniQure Announces 2018 Financial Results and Highlights Recent
Company Progress
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 2018 and
highlighted recent progress across its business.
"2018 was a year marked by tremendous progress
across all of our programs and gene therapy platform,” stated Matt
Kapusta, chief executive officer of uniQure. “We’re also off to a
very strong start in 2019 having achieved a number of value-driving
milestones across our programs, including the initiation of the
dosing phase of our global HOPE-B pivotal trial of AMT-061 in
hemophilia B and the clearance of our IND for AMT-130 in
Huntington’s disease. Looking ahead in the year, we expect to
complete enrollment in the HOPE-B pivotal trial and report
longer-term follow up from the ongoing Phase IIb study. We
also expect to initiate dosing of a Phase I/II study of AMT-130 and
advance our pipeline programs in hemophilia A, Fabry disease and
Spinocerebellar Ataxia Type 3. We believe achieving these
goals will move us closer to providing potentially transformative
therapies to patients and further demonstrate the importance of our
industry leading technology platform and AAV manufacturing
capabilities.”
Recent Company Progress
— Advancing late-stage
development of AMT-061 for the treatment of hemophilia B
- The Company announced earlier this month that the first patient
has been treated in the global Phase III HOPE-B pivotal study of
AMT-061 in hemophilia B. The Company expects to complete the
enrollment of approximately 50 patients in the trial by
year-end.
- Also this month, the Company presented updated clinical data on
AMT-061 demonstrating increasing and sustained FIX levels of up to
51% of normal after the one-time administration of AMT-061 in the
ongoing Phase IIb study. Mean Factor IX (FIX) activity for the
three patients at 12 weeks increased to 38% of normal, exceeding
threshold FIX levels generally considered sufficient to eliminate
or significantly reduce the risk of bleeding events. None of
the patients received Factor infusions, reported bleeding events or
required immunosuppression over a combined 42 weeks of
observation. The second and third patients had previously
screen-failed and were excluded from another gene therapy study due
to pre-existing neutralizing antibodies to a different AAV vector.
Collectively, these updated data further support AMT-061 as a
potential first- and best-in-class gene therapy for hemophilia
B.
* Epidemiological data indicate that factor activity above 12%
of normal is associated with substantial reduction or elimination
of spontaneous bleeds and factor usage. Den Uijl IE et al
Haemophilia 2011; 17(6):849-53
— Advancing AMT-130 into
clinical development for the treatment of Huntington’s disease
- In January, the Company announced the clearance of its
Investigational New Drug Application (IND) for AMT-130 in
Huntington’s disease. AMT-130 is set to become the first AAV gene
therapy to enter clinical development for Huntington’s disease.
- The Company expects to initiate a dose-escalating, randomized
and controlled Phase I/II clinical study to assess the safety,
tolerability and efficacy of a one-time treatment of AMT-130 in
patients with Huntington’s disease. The Company expects to open
several clinical sites in the United States and begin dosing
patients in the second half of this year, with the potential to
report early safety data on initial patients treated in the study
before the end of the year.
- Multiple preclinical data presentations were featured at a
recent scientific meeting demonstrating the potential of AMT-130 to
restore neuronal function and partial reversal of gliosis as shown
by magnetic resonance spectroscopy (MRS).
- Additional preclinical data were presented on AMT-130
demonstrating restoration of brain cell function and reversal of
neuropathology in a Huntington’s disease mouse model, knock-down of
mutant huntingtin in the cerebral spinal fluid (CSF) of minipigs,
and showing low titers of pre-existing neutralizing antibody (NABs)
in the CSF, irrespective of the levels of NABs in serum, suggesting
the potential of treating a broad patient population with
AMT-130.
— Advancing robust
pipeline of novel gene therapy research programs
- At its Research and Development Day late last year, the Company
unveiled a pipeline expansion of new AAV gene therapy approaches to
liver and central nervous system (CNS) disorders, including AMT-180
for hemophilia A, AMT-190 for Fabry disease and AMT-150 for
Spinocerebellar Ataxia Type 3 (SCA3). The Company expects to
advance the preclinical development of all three programs during
2019, including the initiation of a GLP toxicology study of AMT-180
in the first quarter of 2019.
- The Company also introduced its proprietary miQURE™ gene
silencing platform that has applications across multiple
indications, including Huntington’s disease and SCA3.
Upcoming Events
- Participate in a fireside chat at the Leerink Partners 8th
Annual Global Healthcare Conference on March 1, 2019.
- Present at the Cowen 39th Annual Health Care Conference on
March 12, 2019.
- Present at the Barclays Global Healthcare Conference on March
13, 2019.
- Present preclinical data on the Company’s pipeline and
technology platform at the American Society for Gene and Cell
Therapy (ASGCT) Annual Meeting, April 29- May 2, 2019.
- Present preclinical data on AMT-150 for the treatment of
Spinocerebellar Ataxia Type 3 (SCA3) at the 2019 American Academy
of Neurology (AAN) Annual Meeting in May 4-10, 2019.
Financial Highlights
Cash Position: As of December
31, 2018, the Company held cash and cash equivalents of $234.9
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 2018 was
$11.3 million, compared to $13.1 million for 2017 and $ 25.1
million in 2016. The decrease in collaboration revenue was
primarily due to the termination of the Chiesi co-development
agreement in July 2017, as well as nonrecurring revenue recognized
in the prior year period associated with the production of research
supplies in support of the Company’s collaboration agreement with
Bristol Myers-Squibb.
R&D Expenses: Research and
development expenses were $74.8 million for the year ended 2018,
compared to $72.2 million 2017 and $ 72.5 million in 2016. The
increase was primarily related to costs incurred preparing for the
initiation of the AMT-061 pivotal study as well as costs related to
the GLP toxicology study completed in 2018 for AMT-130. Research
and development expenses for the year ended December 31, 2018
include a $5.4 million noncash impairment loss on an in-process
research asset acquired in the 2014 acquisition of the InoCard
business, as well as $3.8 million of noncash income from the full
release of contingent consideration previously recorded in relation
to that acquisition.
SG&A Expenses: Selling,
general and administrative expenses were $25.3 million for 2018,
compared to $24.6 million for 2017 and $26.0 million for 2016. In
2016, the Company incurred nonrecurring costs related to its
conversion from a foreign private issuer to a U.S. domestic
filer.
Other income, net: Other income
was $0.6 million for 2018, compared to $12.4 million for 2017 and $
1.5 million for 2016. In 2017, the Company recorded other income of
$13.8 million related to the July 2017 termination of the
collaboration with Chiesi.
Net Loss: The net loss for the
full years 2018, 2017 and 2016 was $83.3 million, or $2.34 per
share, $79.3 million, or $2.94 per share, and $73.4 million, or
$2.93 per share, respectively.
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
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 achievement of any
of our planned near term or other milestones, our ability to
provide further clinical updates on the Phase IIb study in 2019 or
at any time, our ability to complete enrollment in our pivotal
Phase III trial of AMT-061, the risk of cessation, delay or lack of
success of any of our ongoing or planned clinical studies,
our ability to initiate dosing of a Phase I/II study of
AMT-130 in the second half of 2019 or ever, our ability to open
several clinical sites for the Phase I/II study in the United
States, our ability to report early safety data on initial patients
treated in the Phase I/II study before the end of the year, our
ability to advance our pipeline programs in hemophilia A, Fabry
disease and Spinocerebellar Ataxia Type 3, our ability to move
closer to providing potentially transformative therapies to
patients and further demonstrate the importance of our industry
leading technology platform and AAV manufacturing capabilities,
and/or the development and regulatory approval of our product
candidates in the United States or in Europe. 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, clinical results, 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 February 28, 2019. 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: |
|
FOR MEDIA: |
Maria E. Cantor |
Eva M. Mulder |
Tom Malone |
Direct: 339-970-7536 |
Direct: +31 20 240 6103 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: +31 6 52 33 15 79 |
Mobile: 339-223-8541 |
m.cantor@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
|
|
|
|
|
December 31, |
|
December 31, |
|
2018 |
|
2017 |
|
|
|
|
|
|
in thousands |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
234,898 |
|
$ |
159,371 |
|
Accounts receivable and accrued income from related party |
|
233 |
|
|
1,586 |
|
Prepaid expenses |
|
1,116 |
|
|
1,139 |
|
Other current assets |
|
329 |
|
|
687 |
|
Total current assets |
|
236,576 |
|
|
162,783 |
|
Non-current assets |
|
|
|
|
Property, plant and equipment, net |
|
29,179 |
|
|
34,281 |
|
Intangible assets, net |
|
5,201 |
|
|
9,570 |
|
Goodwill |
|
506 |
|
|
530 |
|
Restricted cash |
|
2,444 |
|
|
2,480 |
|
Total non-current
assets |
|
37,330 |
|
|
46,861 |
|
Total assets |
$ |
273,906 |
|
$ |
209,644 |
|
Current liabilities |
|
|
|
|
Accounts payable |
$ |
3,792 |
|
$ |
2,908 |
|
Accrued expenses and other current liabilities |
|
8,232 |
|
|
8,838 |
|
Current portion of long-term debt |
|
- |
|
|
1,050 |
|
Current portion of deferred rent |
|
311 |
|
|
737 |
|
Current portion of deferred revenue |
|
7,634 |
|
|
4,613 |
|
Current portion of contingent consideration |
|
- |
|
|
1,084 |
|
Total current liabilities |
|
19,969 |
|
|
19,230 |
|
Non-current liabilities |
|
|
|
|
Long-term debt, net of current portion |
|
35,471 |
|
|
19,741 |
|
Deferred rent, net of current portion |
|
8,761 |
|
|
9,114 |
|
Deferred revenue, net of current portion |
|
28,861 |
|
|
67,408 |
|
Contingent consideration, net of current portion |
|
- |
|
|
2,880 |
|
Derivative financial instruments related party |
|
803 |
|
|
1,298 |
|
Other non-current liabilities |
|
435 |
|
|
614 |
|
Total non-current liabilities |
|
74,331 |
|
|
101,055 |
|
Total liabilities |
|
94,300 |
|
|
120,285 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
179,606 |
|
|
89,359 |
|
Total liabilities and shareholders' equity |
$ |
273,906 |
|
$ |
209,644 |
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
|
|
|
Years ended December
31, |
|
|
2018 |
|
2017 |
|
2016 |
|
|
|
|
|
|
|
|
|
in thousands, except share and per share
amounts |
|
Total revenues |
$ |
11,284 |
|
|
$ |
13,107 |
|
|
$ |
25,098 |
|
|
Operating expenses: |
|
|
|
|
|
|
Research and development expenses |
|
(74,809 |
) |
|
|
(72,172 |
) |
|
|
(72,510 |
) |
|
Selling, general and administrative expenses |
|
(25,305 |
) |
|
|
(24,635 |
) |
|
|
(25,999 |
) |
|
Total operating expenses |
|
(100,114 |
) |
|
|
(96,807 |
) |
|
|
(98,509 |
) |
|
Other income |
|
2,146 |
|
|
|
15,430 |
|
|
|
1,465 |
|
|
Other expense |
|
(1,548 |
) |
|
|
(3,073 |
) |
|
|
- |
|
|
Loss from operations |
|
(88,232 |
) |
|
|
(71,343 |
) |
|
|
(71,946 |
) |
|
Non operating items, net |
|
5,159 |
|
|
|
(8,116 |
) |
|
|
(283 |
) |
|
Loss before income tax
expense |
|
(83,073 |
) |
|
|
(79,459 |
) |
|
|
(72,229 |
) |
|
Income tax (expense) / benefit |
|
(231 |
) |
|
|
199 |
|
|
|
(1,145 |
) |
|
Net loss |
$ |
(83,304 |
) |
|
$ |
(79,260 |
) |
|
$ |
(73,374 |
) |
|
|
|
|
|
|
|
|
Basic and diluted net loss per ordinary share |
$ |
(2.34 |
) |
|
$ |
(2.94 |
) |
|
$ |
(2.93 |
) |
|
Weighted average shares used in computing basic and diluted net
loss per ordinary share |
|
35,639,745 |
|
|
|
26,984,183 |
|
|
|
25,036,465 |
|
|
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