uniQure Announces First Quarter 2017 Financial Results and Highlights Recent Company Progress
May 09 2017 - 7:00AM
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company
developing transformative therapies for patients with severe
medical needs, today reported its financial results for the first
quarter of 2017 and highlighted recent progress in advancing its
pipeline.
“In early 2017, we continued to make progress
across our strategic priorities, including our gene therapy
programs in hemophilia B, Huntington’s disease and congestive heart
failure,” stated Matthew Kapusta, chief executive officer of
uniQure. “Over the past few months, AMT-060 received Breakthrough
Therapy and PRIME designation based on the promising data from our
ongoing Phase I/II study, and we look forward to presenting updated
data from the study in July at ISTH. We also initiated
discussions with the FDA regarding planning for a pivotal study,
and expect additional interactions with regulators in 2017 as we
prepare for late-stage development.”
“In addition, we recently presented promising,
new preclinical data in Huntington’s disease from a large animal
model, and remain on track to initiate an IND-enabling toxicology
study in the second half of 2017,” added Mr. Kapusta. “We
also continue to advance, in collaboration with BMS, our
preclinical studies of S100A1 in heart failure, as well as other
early stage product candidates targeting CNS and liver-directed
diseases, and we look forward to providing an update on our
progress this year.”
First Quarter 2017 and Recent
Significant Corporate Highlights
- Received European Medicines Agency (EMA) Priority Medicines
(PRIME) Designation for AMT-060, its proprietary investigational
gene therapy in patients with severe hemophilia B.- This
designation follows the Breakthrough Therapy designation for
AMT-060 received earlier this year from the U.S. Food and Drug
Administration (FDA) and is based on results from the
ongoing, dose-ranging Phase I/II study that show a near cessation
of spontaneous bleeding in patients with severe disease at up to 12
months follow-up, clinically significant and sustained increases in
Factor IX (FIX) and substantial reductions in FIX
replacement usage.
- Received positive feedback on progressing its gene therapy
program in hemophilia B from the FDA following an end-of-Phase II
meeting. - The FDA acknowledged the early clinical benefit observed
with AMT-060 and expressed no safety concerns based on the Phase
I/II data that were presented at a major medical conference late
last year. The Company expects to have a similar end-of-Phase
II meeting with the EMA later this quarter. - The Company is
completing the technology transfer from its Amsterdam site to its
state-of-the-art manufacturing facility in Lexington, MA.
Comprehensive planning to advance the hemophilia B program into
late-stage clinical development is ongoing, with a pivotal study
expected to begin in 2018.
- Presented new preclinical data on AMT-130 in Huntington's
Disease at CHDI's 12th Annual Huntington's Disease Therapeutics
Conference. - Data from the study demonstrate for the first time
efficacy in one of the largest HD animal models available for
testing, including a strong dose-dependent reduction of mutant
huntingtin protein and widespread vector distribution in the brain.
- Expression of mutant HTT mRNA was significantly reduced in all
regions of the brain transduced by AMT-130 by 50% to 80%, as well
in the cortex by up to 40%, compared with control. Researchers also
observed a dose-dependent reduction in mutant huntingtin protein
levels of more than 50% in the brain, as well as similar trends in
cerebral spinal fluid. - IND-enabling toxicology study is on
track to begin in the second half of 2017.
- Announced that it will not seek marketing authorization renewal
for Glybera® in Europe. - The current marketing authorization for
Glybera will expire on October 25, 2017. The Company’s
decision involved a thoughtful and careful evaluation of patient
needs and the clinical use of the therapy, and is not related to
any risk-benefit concern. Glybera's usage has been extremely
limited and it is not expected that patient demand would increase
materially in future years. - uniQure expects to reduce future
expenses related to the product by approximately $2 million
annually, beginning in 2018 and net of any payments to Chiesi, its
commercial partner for Glybera.
- Published data demonstrating wide distribution of AAV5 in the
central nervous system (CNS). - The publication in the journal Gene
Therapy highlighted data demonstrating widespread transduction in
the CNS following direct injection of uniQure's AAV5 vector in a
large animal model. The method of injection used was found to
result in controlled and accurate administration with no adverse
events observed in the non-human primates. The study will help
guide clinical development of uniQure’s gene therapy product
candidate in Huntington's disease, as well as other product
candidates targeting CNS disorders that are being evaluated by the
Company.
- Advanced research collaboration focused on cardiovascular
diseases with multiple ongoing preclinical studies. -
Continued progress is being made with collaborator Bristol-Myers
Squibb (BMS) in multiple dose-ranging analyses and comparability
studies in healthy and diseased pigs. This ongoing work is in
support of an IND application expected to be filed by BMS in
2018.
Upcoming Events in Second-Quarter and
Mid-2017
- American Society of Gene and Cell Therapy (ASGCT)
meeting, May 10-13, 2017: - Five abstracts have been
accepted for presentation at ASGCT. Members of uniQure’s
research and development team, together with external
collaborators, will present data focused on progress in
re-administration protocols, gene therapy delivery systems and
optimized vector distribution technologies in haemophilia B and
Huntington’s disease. - An investor and analyst breakfast meeting
and webcast featuring senior leaders of its research and
development team will be held on Friday, May 12, 2017 at 7:00 a.m.
EDT. To attend, please RSVP to Investors@uniQure.com as space
is limited. The live webcast can be accessed through the link
displayed in the Investor section of the uniQure website at:
http://uniqure.com/investors-newsroom/events-presentations.php.
- American Biomanufacturing Summit, May 23-24,
2017:- uniQure will present “Developing a Scalable
Disposable Manufacturing Facility for Producing Gene Therapies” on
Tuesday, May 23, 2017. The Company’s Lexington-based facility
is one of the largest, most versatile gene therapy manufacturing
facilities in the world, with state-of-the-art commercial-scale
production capabilities that utilize 100% single-use
components.
- International Society of Thrombosis and Hemostasis
(ISTH) meeting, July 8-13, 2017:- uniQure expects to
present long-term data and additional analysis from the ongoing,
dose-ranging Phase I/II study of AMT-060 in patients with severe
hemophilia B. The presentation is expected to include up to
eighteen months of follow-up from the study’s low-dose cohort and
up to twelve months of follow-up from the study’s second,
higher-dose cohort.
Financial Highlights
Cash Position: As of
March 31, 2017, the Company held cash and cash equivalents of
$120.3 million, compared with $132.5 million as of December 31,
2016. The decrease in cash was primarily related to the
advancement of its clinical and preclinical gene therapy targets,
general corporate activities and capital expenditures related to
its facilities in Lexington, MA and Amsterdam. The Company
intends to significantly reduce capital expenditures in 2017 and
2018 and realize operational cost savings from the strategic
restructuring initiated in November 2016 and the withdrawal of
Glybera in October 2017. As a result of these initiatives,
the Company expects its cash on hand will be sufficient to fund
operations into 2019.
Revenues: Revenue for the
first quarter of 2017 was $3.3 million compared with $4.3 million
in the first quarter of 2016. The reduction in the current year
period was driven by a decrease in collaboration revenue compared
to the prior year period. The reduction was primarily due to
the timing of various reimbursable activities, including the
production of preclinical material for the Company’s S100A1 gene
therapy that occurred in the prior year period.
R&D Expenses:
Research and development expenses for the first quarter of 2017
were $17.0 million compared with $16.7 million in the first quarter
of 2016. The increase was primarily related to higher
operating expenses and depreciation related to our Amsterdam
facilities.
SG&A Expenses:
Selling, general and administrative expenses for the first quarter
of 2017 were $6.4 million compared with $7.3 million in the first
quarter of 2016. The decrease was primarily related to
one-time costs related to our CEO transition incurred in the prior
year period.
Net Loss: The net loss
for the first quarter of 2017 was $20.3 million, or $0.80 per
share, compared with $22.3 million, or $0.90 per share, for the
first quarter of 2016.
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, 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
restructuring, 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 2016 Annual
Report on Form 10-K filed with the Securities and Exchange
Commission on March 15, 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 N.V.UNAUDITED
CONDENSED CONSOLIDATED BALANCE SHEETS |
|
March 31, |
|
December 31, |
|
2017 |
|
|
|
2016 |
|
|
in thousands, except share and per share
amounts |
Current assets |
|
|
|
Cash and
cash equivalents |
$ |
120,271 |
|
|
$ |
132,496 |
|
Accounts
receivable and accrued income |
|
1,233 |
|
|
|
3,680 |
|
Accounts
receivable and accrued income from related party |
|
517 |
|
|
|
5,500 |
|
Prepaid
expenses |
|
1,129 |
|
|
|
996 |
|
Other
current assets |
|
579 |
|
|
|
1,274 |
|
Total current assets |
|
123,729 |
|
|
|
143,946 |
|
Non-current assets |
|
|
|
Property, plant and equipment, net |
|
35,241 |
|
|
|
35,702 |
|
Intangible assets, net |
|
8,357 |
|
|
|
8,324 |
|
Goodwill |
|
471 |
|
|
|
465 |
|
Other
non-current assets |
|
1,837 |
|
|
|
1,828 |
|
Total non-current
assets |
|
45,906 |
|
|
|
46,319 |
|
Total assets |
$ |
169,635 |
|
|
$ |
190,265 |
|
Current liabilities |
|
|
|
Accounts
payable |
$ |
3,954 |
|
|
$ |
5,524 |
|
Accrued
expenses and other current liabilities |
|
7,154 |
|
|
|
9,766 |
|
Current
portion of long-term debt |
|
2,443 |
|
|
|
605 |
|
Current
portion of deferred rent |
|
697 |
|
|
|
684 |
|
Current
portion of deferred revenue |
|
6,225 |
|
|
|
6,142 |
|
Total current liabilities |
|
20,473 |
|
|
|
22,721 |
|
Non-current liabilities |
|
|
|
Long-term debt, net of current portion |
|
17,920 |
|
|
|
19,631 |
|
Deferred
rent, net of current portion |
|
8,090 |
|
|
|
6,781 |
|
Deferred
revenue, net of current portion |
|
75,404 |
|
|
|
75,612 |
|
Contingent consideration |
|
1,989 |
|
|
|
1,838 |
|
Other
non-current liabilities |
|
32 |
|
|
|
51 |
|
Total non-current liabilities |
|
103,435 |
|
|
|
103,913 |
|
Total liabilities |
|
123,908 |
|
|
|
126,634 |
|
Commitments and contingencies (see note 13) |
|
|
|
Shareholders' equity |
|
|
|
Ordinary
shares, €0.05 par value: 60,000,000 shares authorized at March 31,
2017, and December 31, 2016, and 25,475,894 and 25,257,420 shares
issued and outstanding at March 31, 2017, and December 31, 2016,
respectively. |
|
1,604 |
|
|
|
1,593 |
|
Additional paid-in-capital |
|
466,688 |
|
|
|
464,653 |
|
Accumulated other comprehensive loss |
|
(6,235 |
) |
|
|
(6,557 |
) |
Accumulated deficit |
|
(416,330 |
) |
|
|
(396,058 |
) |
Total shareholders' equity |
|
45,727 |
|
|
|
63,631 |
|
Total liabilities and shareholders' equity |
$ |
169,635 |
|
|
$ |
190,265 |
|
uniQure N.V.UNAUDITED
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
in thousands, except share and per share
amounts |
|
|
Total revenues |
$ |
3,321 |
|
|
|
4,295 |
|
|
|
Operating expenses: |
|
|
|
|
|
Research
and development expenses |
|
(16,994 |
) |
|
|
(16,706 |
) |
|
|
Selling,
general and administrative expenses |
|
(6,358 |
) |
|
|
(7,298 |
) |
|
|
Total operating expenses |
|
(23,352 |
) |
|
|
(24,004 |
) |
|
|
Other
income |
|
316 |
|
|
|
445 |
|
|
|
Loss from operations |
|
(19,715 |
) |
|
|
(19,264 |
) |
|
|
Non
operating items, net |
|
(557 |
) |
|
|
(2,478 |
) |
|
|
Loss before income tax benefit / (expense) |
|
(20,272 |
) |
|
|
(21,742 |
) |
|
|
Income
tax benefit / (expense) |
|
- |
|
|
|
(557 |
) |
|
|
Net loss |
$ |
(20,272 |
) |
|
$ |
(22,299 |
) |
|
|
|
|
|
|
|
|
Basic
and diluted net loss per common share |
$ |
(0.80 |
) |
|
$ |
(0.90 |
) |
|
|
Weighted
average shares used in computing basic and diluted net loss per
common share |
|
25,443,609 |
|
|
|
24,696,643 |
|
|
|
|
|
|
|
|
|
uniQure Contacts:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
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