LEXINGTON, Mass. and AMSTERDAM, the Netherlands,
March 15, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a
leading gene therapy company developing transformative therapies
for patients with severe medical needs, today announces its
financial results for the year 2016 and provides an update on
company progress.
"In 2016, we made significant progress across all
of our core programs and implemented strategic changes that have
strengthened our organization and enhanced focus on our key
priorities," stated Matthew Kapusta, chief executive officer of
uniQure. "We enter 2017 with a solid financial position and strong
clinical data to advance our hemophilia B program into a Phase III
study, as well as to progress our gene therapy candidates in
Huntington's disease and congestive heart failure towards IND
filings. We believe the achievement of these objectives will
deliver meaningful value to shareholders."
Recent Highlights from
2016
-
Promising Updated
Phase I/II Data Presented on AMT-060 at ASH
- Updated results on AMT-060, including up
to 52 weeks of follow-up on the first patient cohort receiving
5x1012 gc/kg
and up to 26 weeks of follow-up on the second patient cohort
receiving a higher dose of 2x1013 gc/kg,
were presented at the 58th American
Society of Hemophilia Annual Meeting.
- Patients in the first dose cohort that discontinued prophylactic
FIX infusions experienced an 85 percent reduction in total FIX
usage and a marked reduction in frequency of spontaneous bleeding
after treatment with AMT-060, including a complete cessation of
spontaneous bleeding during the last 14 weeks of observation.
The five patients in the second dose cohort experienced a near
cessation of spontaneous bleeds with only one spontaneous bleed
reported in 96 weeks of cumulative observation.
- At both doses, AMT-060 appears to be safe and well-tolerated with
no loss of FIX activity, no activation of T-cell response and no
development of inhibitors for any of the 10 patients in the
study. None of the 10 patients in the study tested positive
for anti-AAV5 antibodies.
-
FDA Breakthrough
Therapy Designation Granted for AMT-060 in Hemophilia B
- In January 2017, the U.S. Food and Drug
Administration (FDA), granted Breakthrough Therapy Designation for
AMT-060 in Hemophilia B patients. The designation is based on
results from the ongoing Phase I/II study in patients with severe
disease at up to 12 months follow-up. According to FDA data for its
fiscal year 2016, the Center for Biologics Research and Review
received a total of 23 requests for Breakthrough Therapy
designation with only four designations granted, or 17% of all
requests.
-
Initiated Regulatory
Discussions for Phase III Program in Hemophilia B
- Regulatory discussions have now begun as
the Company conducted its end-of-Phase II meeting with the FDA
earlier this quarter. The meeting was positive, with the FDA
acknowledging the clinical benefit of AMT-060 and expressing no
safety concerns based on the Phase I/II data. The Company
anticipates initiating discussions with the European Medicines
Association later this year. Plans to advance the hemophilia
B program into late-stage clinical development are ongoing, with a
pivotal trial expected to begin in 2018.
-
Preclinical Data
Published on AMT-130 in Huntington's Disease
- uniQure is conducting ongoing preclinical
studies of AMT-130, its wholly-owned AAV5-based gene therapy
product candidate for Huntington's disease. Preclinical data
published in a peer-reviewed journal showed sustained and strong
wild-type HTT protein silencing in humanized control mice,
including knock-down efficiency up to 80% using optimized miHTT
scaffolds. Data from additional preclinical studies will be
presented during the year at various academic meetings.
uniQure continues to advance AMT-130 towards filing an
Investigational New Drug (IND) application to begin clinical
studies in 2018.
-
Research Collaboration
in Congestive Heart Failure Ongoing with Multiple Preclinical
Studies
- Steady progress has been made with
collaborator Bristol-Myers Squibb (BMS) in transferring S100A to an
insect-cell preparation, manufacturing material for nonclinical and
preclinical studies and conducting 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.
-
Completion of a
Company-wide Strategic Review
- uniQure announced the completion of a
company-wide strategic review prioritizing programs in hemophilia
B, Huntington's disease and cardiovascular disease, consolidating
its GMP-manufacturing operations into its Lexington, MA location,
streamlining its organization and strengthening its financial
position.
-
Key Management and
Board Appointments
- uniQure strengthened its senior management
team with the appointment of Paul Firuta as Chief Commercial
Officer, Alex Kuta as Senior Vice President of Regulatory Affairs,
Maria Cantor as Senior Vice President, Investor Relations &
Communications, Jonathan Garen as Chief Business Officer and Maiken
Keson-Brookes as Senior Vice President and General Counsel, all of
whom are based in the U.S.
- Following its Annual General Meeting, the Company completed
corporate governance changes in transitioning from a two-tier
Supervisory Board and Management Board to a single Board of
Directors structure with executive and non-executive members. In
addition, the Company appointed Jack Kaye, a seasoned financial
executive with more than 40 years of diversified experience, to its
Board of Directors.
Matthew Kapusta was appointed by the Board of Directors as Chief
Executive Officer (CEO). Mr. Kapusta had previously served as
interim CEO and as Chief Financial Officer of the Company.
Mr. Kapusta will continue to serve as an executive member of the
uniQure Board of Directors.
Financial Highlights
Cash Position: As of
December 31, 2016, the Company held cash and cash equivalents of
$132.5 million, compared with $221.6 million as of December 31,
2015. 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 state-of-the-art manufacturing facility in Lexington,
Massachusetts and the build-out of its new research facility in
Amsterdam, the Netherlands. 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. As a result of these initiatives,
the Company expects its cash on hand will be sufficient to fund
operations into 2019.
Revenues: Revenue for
2016 was $25.1 million, compared with $10.6 million in 2015 and
$6.1 million in 2014. The increases are driven by research activity
associated with S100A1 for heart failure, which are fully
reimbursed by BMS in accordance with the Company's collaboration
agreement.
R&D Expenses:
Research and development expenses were $72.5 million in 2016,
compared with $59.1 million in 2015 and $43.8 million in 2014. The
increase is related to the continuation of uniQure's Phase I/II
clinical study of AMT-060 in hemophilia B, the intensification of
the Company's activities to support the research of S100A1, the
continued progression of uniQure's preclinical candidate for
Huntington's disease, increased activity in the Company's U.S.
facility and ongoing studies associated with the Company's
collaboration with 4D Molecular Therapeutics to develop
next-generation vector serotypes.
SG&A Expenses:
Selling, general and administrative expenses were $26.0 million in
2016, compared with $23.4 million in 2015 and $17.1 million in
2014.
Other income, generated from research and
development subsidies, was $1.5 million in 2016 compared with $0.8
million in 2015 and $1.0 million in 2014.
Net Loss: The net loss
for the fourth quarter of 2016 was $14.7 million, or $0.58 per
share, compared with $16.9 million, or $0.69 per share, for the
fourth quarter of 2015. The net loss for the full years 2016,
2015 and 2014 was $73.4 million, or $2.93 per share, $82.1 million,
or $3.72 per share, and $49.8 million, or $2.91 per share,
respectively.
Form S-3 registration:
In March 2015, the Company filed a "shelf registration statement"
on Form F-3 with the Securities and Exchange Commission. Now that
the Company reports as a domestic issuer, the F-3 registration
statement is no longer effective, and therefore the Company today
will file a new shelf registration statement on Form S-3.
This will provide the Company with important financial flexibility
for advancing its programs in the future. The Company has no
immediate plans for any financing transaction.
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
reorganizations and strategic shifts, 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
2015 Annual Report on Form 20-F filed with the Securities and
Exchange Commission on April 4, 2016 and its 2016 Annual Report on
Form 10-K filed on or about the date hereof. 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 CONSOLIDATED BALANCE
SHEETS |
|
|
|
|
|
|
|
|
|
December
31, |
|
December
31, |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
in
thousands, except share and per share amounts |
|
Current assets |
|
|
|
|
|
Cash
and cash equivalents |
|
$ |
132,496 |
|
|
$ |
221,626 |
|
|
Accounts receivable and accrued income |
|
|
3,680 |
|
|
|
- |
|
|
Accounts receivable and accrued income from related parties |
|
|
5,500 |
|
|
|
4,129 |
|
|
Inventories |
|
|
- |
|
|
|
474 |
|
|
Prepaid expenses |
|
|
996 |
|
|
|
690 |
|
|
Other
current assets |
|
|
1,274 |
|
|
|
1,194 |
|
|
Total current assets |
|
|
143,946 |
|
|
|
228,113 |
|
|
Non-current assets |
|
|
|
|
|
Property, plant and equipment, net |
|
|
35,702 |
|
|
|
26,011 |
|
|
Intangible assets, net |
|
|
8,324 |
|
|
|
6,815 |
|
|
Goodwill |
|
|
465 |
|
|
|
481 |
|
|
Other
non-current assets |
|
|
1,828 |
|
|
|
1,243 |
|
|
Total non-current
assets |
|
|
46,319 |
|
|
|
34,550 |
|
|
Total assets |
|
$ |
190,265 |
|
|
$ |
262,663 |
|
|
|
|
|
|
|
|
Current liabilities |
|
|
|
|
|
Accounts payable |
|
$ |
5,524 |
|
|
$ |
4,059 |
|
|
Accrued expenses and other current liabilities |
|
|
9,766 |
|
|
|
9,863 |
|
|
Current portion of long-term debt |
|
|
605 |
|
|
|
5,579 |
|
|
Current portion of deferred rent |
|
|
684 |
|
|
|
630 |
|
|
Current portion of deferred revenue |
|
|
6,142 |
|
|
|
6,778 |
|
|
Total current liabilities |
|
|
22,721 |
|
|
|
26,909 |
|
|
Non-current liabilities |
|
|
|
|
|
Long-term debt, net of current portion |
|
|
19,631 |
|
|
|
14,631 |
|
|
Deferred rent, net of current portion |
|
|
6,781 |
|
|
|
6,247 |
|
|
Deferred revenue, net of current portion |
|
|
75,612 |
|
|
|
83,445 |
|
|
Contingent consideration |
|
|
1,838 |
|
|
|
2,926 |
|
|
Other
non-current liabilities |
|
|
51 |
|
|
|
578 |
|
|
Total non-current liabilities |
|
|
103,913 |
|
|
|
107,827 |
|
|
Total liabilities |
|
|
126,634 |
|
|
|
134,736 |
|
|
Commitments and contingencies |
|
|
|
|
|
Shareholders' equity |
|
|
|
|
|
Ordinary shares, €0.05 par value: 60,000,000 shares authorized at
December 31, 2016 and 2015 and 25,257,420 and 24,327,944 shares
issued and outstanding at December 31, 2016 and 2015,
respectively. |
|
|
1,593 |
|
|
|
1,542 |
|
|
Additional paid-in-capital |
|
|
464,653 |
|
|
|
455,897 |
|
|
Accumulated other comprehensive loss |
|
|
(6,557 |
) |
|
|
(6,828 |
) |
|
Accumulated deficit |
|
|
(396,058 |
) |
|
|
(322,684 |
) |
|
Total shareholders' equity |
|
|
63,631 |
|
|
|
127,927 |
|
|
Total liabilities and shareholders'
equity |
|
$ |
190,265 |
|
|
$ |
262,663 |
|
|
uniQure
N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS AND COMPREHENSIVE LOSS |
|
|
|
|
|
|
|
|
|
|
|
Years ended December
31, |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2014 |
|
|
|
|
in
thousands, except share and per share amounts |
|
Total revenues |
|
$ |
25,098 |
|
|
|
10,578 |
|
|
|
6,141 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development expenses |
|
|
(72,510 |
) |
|
|
(59,125 |
) |
|
|
(43,772 |
) |
|
Selling, general and administrative expenses |
|
|
(25,999 |
) |
|
|
(23,383 |
) |
|
|
(17,073 |
) |
|
Total operating expenses |
|
|
(98,509 |
) |
|
|
(82,508 |
) |
|
|
(60,845 |
) |
|
Other
income |
|
|
1,465 |
|
|
|
779 |
|
|
|
1,022 |
|
|
Loss from operations |
|
|
(71,946 |
) |
|
|
(71,151 |
) |
|
|
(53,682 |
) |
|
Non
operating items, net |
|
|
(283 |
) |
|
|
(12,111 |
) |
|
|
3,370 |
|
|
Loss before income tax benefit /
(expense) |
|
|
(72,229 |
) |
|
|
(83,262 |
) |
|
|
(50,312 |
) |
|
Income
tax benefit / (expense) |
|
|
(1,145 |
) |
|
|
1,179 |
|
|
|
535 |
|
|
Net loss |
|
$ |
(73,374 |
) |
|
$ |
(82,083 |
) |
|
$ |
(49,777 |
) |
|
|
|
|
|
|
|
|
|
Basic
and diluted net loss per common share |
|
$ |
(2.93 |
) |
|
$ |
(3.72 |
) |
|
$ |
(2.91 |
) |
|
Weighted average shares used in computing basic and diluted net
loss per common share |
|
|
25,036,465 |
|
|
|
22,082,345 |
|
|
|
17,121,328 |
|
|
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
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Mar 2024 to Apr 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Apr 2023 to Apr 2024