YASTEST
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, Nov. 01, 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 third
quarter of 2017 and highlighted recent progress across its
business.
"In recent months, we have made
tremendous progress advancing our gene therapy candidates in
hemophilia B and Huntington's disease, and continue to build
momentum as we near the end of the year," stated Matthew Kapusta,
chief executive officer of uniQure. "Based on recent meetings with
the FDA and EMA, we now have what we believe is a clear pathway to
initiate a pivotal study in 2018 with AMT-061, an AAV5-Padua gene
therapy. We believe AMT-061 has the potential to provide
consistent, long-term benefits to nearly all patients with
hemophilia B, without the complications often associated with
immune responses to the capsid. Preparations for the pivotal
study are underway and the manufacturing of AMT-061 for clinical
use has been initiated."
"Regarding AMT-130 in Huntington's
disease, we received Orphan Drug Designation from the FDA and
presented new preclinical data demonstrating functional and
survival benefit," added Mr. Kapusta. "We are also pleased to
announce that we recently completed dosing of a toxicology study
that will support an IND filing in 2018. For the remainder of
this year, we remain focused on the execution across our programs
and look forward to sharing updates on our progress in the near
future."
Third
Quarter 2017 and Recent Company Progress:
· Achieved
significant progress with hemophilia B gene therapy program by
introducing the Padua transgene
-
Based on recent meetings with the U.S. Food and
Drug Administration (FDA) and the European Medicines Agency (EMA),
the Company plans to initiate a pivotal study in 2018 with AMT-061,
an AAV5-Padua FIX gene therapy.
- The FDA agreed that AMT-061 will fall under the
existing Breakthrough Therapy Designation and Investigational New
Drug (IND) for AMT-060, and the EMA also agreed that AMT-061 will
fall under the current PRIME designation. The Company also achieved
general agreement with the FDA and EMA on the AMT-061 comparability
plan and proposed pivotal trial design.
- A Good Laboratory Practices (GLP), nonclinical
study of AMT-061 performed in non-human primates demonstrated a
strong correlation between dose and human FIX (hFIX) expression
levels, as well as the biological activity of the expressed hFIX
protein. AMT-061 demonstrated substantial increases in hFIX
clotting activity compared to AMT-060, and was well-tolerated with
no evidence of any significant toxicological findings.
- uniQure has initiated production of multiple
clinical-grade batches of AMT-061 in its state-of-the-art
Lexington, MA manufacturing facility. Production is at commercial
scale and utilizing current Good Manufacturing Practices.
Data reviewed to date support comparability between AMT-061 and
AMT-060.
· Acquired exclusive patent family covering the use of Padua
in gene therapy for hemophilia B
· Continued progress on AMT-130 gene therapy in
Huntington's disease
-
Preclinical data presented at the European
Society of Gene and Cell Therapy (ESGCT) 25thAnniversary
Congress in Berlin, Germany demonstrated substantial improvement in
motor symptoms and median survival following a single
administration of AMT-130 (AAV5-miHTT) gene therapy in a mouse
model with highly aggressive disease.
- The Company has completed the dosing of non-human
primates in a GLP toxicology study of AMT-130. This study
will support the filing of an IND in 2018, with the goal of being
the first one-time administered gene therapy to enter a clinical
study for Huntington's disease.
- Orphan Drug Designation (ODD) from the FDA was
granted to AMT-130 in October 2017.
· Added talent to leadership team and Board of
Directors
· Completed follow-on offering
-
On October 27, the Company completed an
underwritten public offering of 5,000,000 of its ordinary shares at
a public offering price of $18.25 per share. The gross proceeds
from the offering, before deducting the underwriting discounts and
commissions and estimated offering expenses, were approximately
$91,250,000. uniQure has granted to the underwriters a 30-day
option to purchase up to 750,000 additional ordinary shares at the
public offering price, less underwriting discounts and
commissions.
Upcoming
Anticipated Milestones
Financial
Highlights
Cash
Position: As of September 30, 2017, the Company held
cash and cash equivalents of $88.9 million, compared to $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.
Revenues: Revenues for the three months ended
September 30, 2017 were $2.3 million compared to $7.2 million for
the same period in 2016. Collaboration revenues for the third
quarter of 2017 were $1.1 million, compared to $6.0 million for the
comparable period in 2016. The decrease in collaboration
revenue was primarily due to the termination of the Chiesi
co-development agreement in July 2017, as well as nonrecurring
revenue recognized in the prior year period associated with the
production of AMT-126 product supplies.
R&D
Expenses: Research and development expenses for the
three months ended September 30, 2017 were $20.1 million compared
to $16.6 million for the same period in 2016. The increase
primarily results from non-cash changes in fair value of the
contingent consideration related to the Company's 2014 acquisition
of InoCard.
SG&A
Expenses: Selling, general and administrative
expenses for the three months ended September 30, 2017 were $5.6
million compared to $5.1 million for the same period in 2016. The
increase was primarily related to larger share-based compensation
expenses in the current year period.
Other
income: Other income for the three months ended
September 30, 2017 were $14.4 million compared to $0.3 million for
the same period in 2016. The current year period includes the full
amortization of the outstanding deferred revenue of $13.8 million
following the termination of the Company's collaboration with
Chiesi in July 2017.
Net
Loss: The net loss for the third quarter of 2017 was
$10.2 million, or $0.40 per share, compared to $15.3 million, or
$0.61 per share, for the third 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
transition to our AMT-061 product candidate, 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
August 8, 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
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
uniQure N.V. |
|
UNAUDITED CONSOLIDATED BALANCE SHEETS |
|
|
September 30, |
|
December 31, |
2017 |
|
2016 |
|
in
thousands, except share and per share amounts |
Current
assets |
|
|
|
Cash and cash equivalents |
$ |
88,934 |
|
$ |
132,496 |
Accounts receivables and
accrued income |
|
1,945 |
|
|
9,180 |
Prepaid assets and other
current assets |
|
1,436 |
|
|
2,270 |
Total
current assets |
|
92,315 |
|
|
143,946 |
Non-current assets |
|
|
|
Property, plant and equipment,
net |
|
34,653 |
|
|
35,702 |
Intangible assets and
goodwill |
|
9,549 |
|
|
8,789 |
Other non-current assets |
|
2,469 |
|
|
1,828 |
Total
non-current assets |
|
46,671 |
|
|
46,319 |
Total
assets |
$ |
138,986 |
|
$ |
190,265 |
Current
liabilities |
|
|
|
Accounts payable |
$ |
2,987 |
|
$ |
5,524 |
Accrued expenses and other
current liabilities |
|
10,165 |
|
|
9,766 |
Current portion of long-term
debt |
|
6,232 |
|
|
605 |
Current portion of deferred
rent |
|
724 |
|
|
684 |
Current portion of deferred
revenue |
|
4,249 |
|
|
6,142 |
Current portion of contingent
consideration |
|
1,017 |
|
|
- |
Total
current liabilities |
|
25,374 |
|
|
22,721 |
Non-current liabilities |
|
|
|
Long-term debt, net of current
portion |
|
14,353 |
|
|
19,631 |
Deferred rent, net of current
portion |
|
8,829 |
|
|
6,781 |
Deferred revenue, net of
current portion |
|
67,863 |
|
|
75,612 |
Contingent consideration, net
of current portion |
|
2,593 |
|
|
1,838 |
Other non-current
liabilities |
|
367 |
|
|
51 |
Total
non-current liabilities |
|
94,005 |
|
|
103,913 |
Total
liabilities |
|
119,379 |
|
|
126,634 |
Total
shareholders' equity |
|
19,607 |
|
|
63,631 |
Total
liabilities and shareholders' equity |
$ |
138,986 |
|
$ |
190,265 |
uniQure N.V. |
|
|
|
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS |
|
|
|
|
Three months ended September
30, |
|
|
2017 |
|
2016 |
|
|
in
thousands, except share and per share amounts |
|
Total
revenues |
$ |
2,260 |
|
|
$ |
7,221 |
|
|
Operating expenses: |
|
|
|
|
Research and development
expenses |
|
(20,103 |
) |
|
|
(16,604 |
) |
|
Selling, general and
administrative expenses |
|
(5,584 |
) |
|
|
(5,113 |
) |
|
Total
operating expenses |
|
(25,687 |
) |
|
|
(21,717 |
) |
|
Other income |
|
14,413 |
|
|
|
336 |
|
|
Other expense |
|
(261 |
) |
|
|
- |
|
|
Loss
from operations |
|
(9,275 |
) |
|
|
(14,160 |
) |
|
Non operating items, net |
|
(1,248 |
) |
|
|
(935 |
) |
|
Loss
before income tax expense |
|
(10,523 |
) |
|
|
(15,095 |
) |
|
Income tax benefit /
(expense) |
|
278 |
|
|
|
(177 |
) |
|
Net
loss |
$ |
(10,245 |
) |
|
$ |
(15,272 |
) |
|
|
|
|
|
|
Basic and diluted net loss per
common share |
$ |
(0.40 |
) |
|
$ |
(0.61 |
) |
|
Weighted average shares used
in computing basic and diluted net loss per common share |
|
25,632,642 |
|
|
|
25,142,660 |
|
|
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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