~ 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 ~
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
- Clinical data presented at the International Society on
Thrombosis and Hemostasis (ISTH) annual congress on 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.
- All 10 patients in the study demonstrated improvements in their
disease state as measured by reduced FIX replacement therapy and
bleeding frequency. Across both dose cohorts, cumulative annualized
FIX consumption decreased by 79%, and in the second-dose cohort, no
spontaneous bleeds were reported in the last six months of
follow-up - with a reduction in the annualized spontaneous bleed
rate of 84% compared to the one-year period prior to gene
transfer.
• 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
- Regulatory meetings with U.S. Food and Drug Administration and
European Medicines Agency regarding late-stage clinical development
in hemophilia B
- Completion of comparability testing in hemophilia B and
technology transfer from Amsterdam to Lexington facility
- Initiation of IND-enabling safety toxicology study of AMT-130
in Huntington’s disease
- Initiation of preclinical therapeutic heart study for
S100A1 gene therapy targeting congestive heart failure
- Presentation of non-human primate data in Huntington’s
disease
- Ongoing longer-term follow up and durability data on
AMT-060
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
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 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
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