LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, May 09, 2017 (GLOBE NEWSWIRE)
-- 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 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, 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