~ Advancing Gene Therapy Programs
in Hemophilia B and Huntington's Disease ~
~ Provides Update on Research Collaboration with Bristol-Myers
Squibb ~
~ Makes New Appointments to Company Leadership and Nominations to
Board of Directors ~
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, Aug. 08, 2017 (GLOBE NEWSWIRE)
-- 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 2017 and highlighted recent progress across its
business.
"We had a highly productive second
quarter and past few weeks in which we announced substantial
progress in advancing our lead program in hemophilia B through
commercial-scale manufacturing and CMC accomplishments, regulatory
preparation and the receipt of broader patent protection around our
insect-cell platform technology," stated Matthew Kapusta, chief
executive officer of uniQure.
"We presented long-term clinical
data in patients with severe hemophilia B demonstrating safety and
durable clinical benefits, as well as the potential to treat nearly
all patients suffering from this life-changing disorder. We
now have full, unencumbered global rights to our hemophilia B
program creating value for shareholders and opening the door to new
potential opportunities, and we are advancing our Huntington's
disease program into an IND-enabling toxicology study in the fall,"
he added. "Furthermore, based on recent encouraging data,
uniQure and Bristol-Myers Squibb intend to advance our S100A1
product candidate into additional preclinical studies, including a
therapeutic heart failure study that we expect to initiate as soon
as possible. We are embarking on a very busy and equally
productive second half of the year, and we look forward to
providing additional company updates."
Second
Quarter 2017 and Recent Company Progress:
· Successful development and
scale up of manufacturing processes for hemophilia B and other gene
therapy programs
-
Developed and optimized a reproducible,
commercial scale, manufacturing process for producing its
hemophilia B gene therapy in accordance with Good Manufacturing
Practices (GMP). This process will be leveraged across each
of the Company's programs, including AMT-130 in Huntington's
disease for which uniQure expects to file an investigational new
drug (IND) application in 2018. A comparability protocol also has
been finalized, and the Company is planning to meet with U.S. and
European regulators in the early fall to further discuss its plans
to begin a pivotal program in hemophilia B in 2018.
· Continued progress on
research collaboration with Bristol-Myers Squibb (BMS) in
congestive heart failure
-
Today, uniQure announced that preliminary data
from a study in large animals demonstrated both DNA delivery and
human S100A1 expression in the myocardium after treatments with
product produced from uniQure's proprietary insect cell,
baculovirus manufacturing process. Based on this finding and
others, BMS and uniQure intend to advance the product candidate
into further preclinical studies, with a goal of initiating a
preclinical therapeutic heart failure study as soon as
possible.
· Presentation of updated,
long-term clinical data from ongoing Phase I/II trial of AMT-060 in
patients with severe hemophilia B
· Presentation of new
clinical data at ISTH demonstrating the potential to expand the
applicability of AAV5-based gene therapy to nearly all patients
suffering from hemophilia B
-
New clinical data demonstrated efficacy of AAV5
gene therapy in the presence of pre-existing neutralizing
antibodies (NABs). All three patients with detected anti-AAV5 NABs
according to the highly sensitive luciferase-based (LUC) NAB assay,
presented increases in FIX expression. To date, 18 patients
across two clinical studies have received intravenous, systemic
administration of AAV5-based gene therapies without any observed
T-cell activation.
- These data suggest AAV5 may have a superior
immunogenicity and safety profile compared to other AAV
vectors.
· Expansion of intellectual
property portfolio with newly issued patent providing broad
protection of insect cell-based AAV manufacturing
-
Patent broadly covers an important component of
insect cell-based AAV manufacturing technology. The newly issued
Hermens '627 patent significantly expands uniQure's leading
intellectual property portfolio related to large-scale, highly
reproducible manufacturing of AAV in insect cells. The technology
covered in the Hermens '627 patent family, which is currently
widely applied in insect cell-based AAV manufacturing,
significantly increases the value of uniQure's patent portfolio and
strengthens the company's leadership in the production of AAV-based
gene therapies.
· Reacquired development and
commercial rights to hemophilia B program from Chiesi
-
uniQure now has full, unencumbered global rights
to its hemophilia B gene therapy program with clinical
proof-of-concept and is positioned to accelerate clinical
development, maximize shareholder value and take advantage of new,
strategic opportunities related to the program.
· Presentation of new
preclinical data at the American Society of Clinical and Gene
Therapy (ASGCT)
-
Data demonstrated successful and effective
transduction of AAV5 in non-human primates with pre-existing
anti-AAV5 neutralizing antibodies (NABs). At all observed levels,
pre-existing neutralizing antibodies for AAV5 did not impact the
transduction effectiveness of the AAV5 vector. This suggests a much
broader potential population of eligible patients than previously
expected for AAV5-based gene therapies, including uniQure's
investigational gene therapy in patients with severe hemophilia
B.
- Data demonstrated successful readministration of
gene therapy in non-human primates and successful transduction of
AAV5 in the presence of pre-existing NABs. Effective repeated
hepatic gene delivery with uniQure's AAV5 vector was demonstrated
after a proprietary immunoadsorption procedure in non-human
primates (NHPs).
· Key management
appointments and Board of Director nominations further expanding
the Company's capabilities across manufacturing, research and
clinical development
-
Sander van Deventer, M.D., Ph.D., appointed as
Chief Scientific Officer; Scott McMillan, Ph.D., appointed as Chief
Operating Officer; and Steven L. Zelenkofske, D.O., appointed as
Chief Medical Officer.
- Christian Klemt promoted to Chief Accounting
Officer.
- Madhavan Balachandran and Jeremy P. Springhorn,
Ph.D., nominated to the Board of Directors for approval at the
Extraordinary General Meeting of Shareholders (EGM) on September
14, 2017. Mr. Balachandran and Dr. Springhorn are seasoned industry
executives who served at Amgen Inc., and Alexion Pharmaceuticals
Inc., respectively.
- As previously disclosed, Will Lewis and Sander
van Deventer will resign from the Board of Directors effective at
the Company's EGM.
Upcoming
Anticipated Milestones
Financial
Highlights
Cash
Position: As of June 30, 2017, the Company held cash
and cash equivalents of $104.1 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. 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: Revenues for the three months ended
June 30, 2017 were $4.9 million compared to $4.5 million for the
same period in 2016. Collaboration revenues for the second quarter
of 2017 were $4.2 million, compared to $3.2 million for the
comparable period in 2016.
R&D
Expenses: Research and development expenses for the
three months ended June 30, 2017 were $16.9 million compared to
$19.2 million for the same period in 2016. The decrease was
primarily related to lower costs in relation to the manufacturing
of drug substance to supply our programs.
SG&A
Expenses: Selling, general and administrative
expenses for the three months ended June 30, 2017 were $5.4 million
compared to $7.8 million for the same period in 2016. The second
quarter 2016 includes one-off costs of $1.9 million related to the
Extera arbitration and $0.9 million of costs associated with the
Glybera global registry and Phase IV study, partially offset by
higher share-based compensation expense during the second quarter
of 2017.
Other
Expense: Other expense for the three months ended
June 30, 2017 were $2.6 million, compared to zero for the same
period in 2016. The second quarter 2017 includes $1.7 million of
one-off costs related to the withdrawal of Glybera in 2017 and $0.9
million of costs associated with the exit from our previous
Amsterdam facilities.
Net
Loss: The net loss for the second quarter of 2017 was
$21.3 million, or $0.83 per share, compared to $21.1 million, or
$0.84 per share, for the second 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, 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, and the scope of protection provided by our
patent portfolio. 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 Quarterly Report on Form 10-Q filed on May 9, 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 |
|
|
June 30, |
|
December 31, |
2017 |
|
2016 |
|
in
thousands, except share and per share amounts |
Current
assets |
|
|
|
Cash and cash equivalents |
$ |
104,087 |
|
$ |
132,496 |
Accounts receivables and
accrued income |
|
5,817 |
|
|
9,180 |
Prepaid assets and other
current assets |
|
1,521 |
|
|
2,270 |
Total
current assets |
|
111,425 |
|
|
143,946 |
Non-current assets |
|
|
|
Property, plant and equipment,
net |
|
35,410 |
|
|
35,702 |
Intangible assets and
goodwill |
|
9,251 |
|
|
8,789 |
Other non-current assets |
|
1,879 |
|
|
1,828 |
Total
non-current assets |
|
46,540 |
|
|
46,319 |
Total
assets |
$ |
157,965 |
|
$ |
190,265 |
Current
liabilities |
|
|
|
Accounts payable |
$ |
3,458 |
|
$ |
5,524 |
Accrued expenses and other
current liabilities |
|
9,440 |
|
|
9,766 |
Current portion of long-term
debt |
|
4,319 |
|
|
605 |
Current portion of deferred
rent |
|
710 |
|
|
684 |
Current portion of deferred
revenue |
|
5,203 |
|
|
6,142 |
Total
current liabilities |
|
23,130 |
|
|
22,721 |
Non-current liabilities |
|
|
|
Long-term debt, net of current
portion |
|
16,153 |
|
|
19,631 |
Deferred rent, net of current
portion |
|
8,494 |
|
|
6,781 |
Deferred revenue, net of
current portion |
|
78,728 |
|
|
75,612 |
Contingent consideration |
|
2,415 |
|
|
1,838 |
Other non-current
liabilities |
|
1,759 |
|
|
51 |
Total
non-current liabilities |
|
107,549 |
|
|
103,913 |
Total
liabilities |
|
130,679 |
|
|
126,634 |
Total
shareholders' equity |
|
27,286 |
|
|
63,631 |
Total
liabilities and shareholders' equity |
$ |
157,965 |
|
$ |
190,265 |
|
|
|
|
uniQure
N.V. |
|
|
|
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS |
|
|
|
|
Three months ended June
30, |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
in
thousands, except share and per share amounts |
|
Total
revenues |
$ |
4,942 |
|
|
$ |
4,451 |
|
|
Operating expenses: |
|
|
|
|
Research and development
expenses |
|
(16,866 |
) |
|
|
(19,221 |
) |
|
Selling, general and
administrative expenses |
|
(5,410 |
) |
|
|
(7,834 |
) |
|
Total
operating expenses |
|
(22,276 |
) |
|
|
(27,055 |
) |
|
Other income |
|
266 |
|
|
|
475 |
|
|
Other expense |
|
(2,640 |
) |
|
|
- |
|
|
Loss
from operations |
|
(19,708 |
) |
|
|
(22,129 |
) |
|
Non operating items, net |
|
(1,561 |
) |
|
|
716 |
|
|
Loss
before income tax expense |
|
(21,269 |
) |
|
|
(21,413 |
) |
|
Income tax benefit /
(expense) |
|
- |
|
|
|
333 |
|
|
Net
loss |
$ |
(21,269 |
) |
|
$ |
(21,080 |
) |
|
|
|
|
|
|
Basic and diluted net loss per
common share |
$ |
(0.83 |
) |
|
$ |
(0.84 |
) |
|
Weighted average shares used
in computing basic and diluted net loss per common share |
|
25,560,348 |
|
|
|
25,077,350 |
|
|
uniQure Contacts:
FOR INVESTORS:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
FOR MEDIA:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@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
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